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COVID-19 interrupted global poliovirus surveillance and immunization
Most (86%) of these outbreaks were caused by cVDPV2 (circulating VDPV type 2 poliovirus, which originated with the vaccine), and most occurred in Africa, according to a new study of vaccine-derived poliovirus outbreaks between Jan. 2020 and June 2021 published in the CDC’s Morbidity and Mortality Weekly Report.
The Global Polio Eradication Initiative (GPEI) was launched in 1988 and used live attenuated oral poliovirus vaccine (OPV). Since then, cases of wild poliovirus have declined more than 99.99%.
The cVDPV2 likely originated among children born in areas with poor vaccine coverage. Jay Wenger, MD, director, Polio, at the Bill and Melinda Gates Foundation, told this news organization that “the inactivated vaccines that we give in most developed countries now are good in that they provide humoral immunity, the antibodies in the bloodstream. They don’t necessarily provide mucosal immunity. They don’t make the kid’s gut immune to getting reinfected or actually immune to reproducing the virus if they get it in their gut. So we could still have a situation where everybody was vaccinated with IPV [inactivated poliovirus], but the virus could still be transmitting around because kids’ guts would still be producing the virus and there will still be transmission in your population, probably without much or any paralysis because of the IPV. As soon as that virus hit a population that was not vaccinated, they would get paralyzed.”
Dr. Wenger added, “The ideal vaccine would be an oral vaccine that didn’t mutate back and couldn’t cause these VDPVs.” Scientists developed such a vaccine, approved by the World Health Organization last year under an Emergency Use Authorization. This nOPV2 (novel oral poliovirus type 2) vaccine has been given since March 2021 in areas with the VDPD2 outbreaks. The nOPV2 should allow them to “basically stamp out the outbreaks.”
The world had almost eradicated the disease, with the last cases of polio from wild virus occurring in Nigeria, Afghanistan, and Pakistan as of 2014. Africa was declared free of wild polio in 2020 after it had been eradicated from Nigeria, which accounted for more than half of the world’s cases only a decade earlier. Now cVDPV outbreaks affect 28 African countries, plus Iran, Yemen, Afghanistan, Pakistan, Tajikistan, Malaysia, the Philippines, and Indonesia. And there was also one case in China. Globally, there were 1,335 cases of cVDPV causing paralysis during the reporting period.
The COVID-19 pandemic has had a significant impact on polio, accounting for much of this year’s increase in cases. Dr. Wenger said, “We couldn’t do any campaigns. We pretty much stopped doing outbreak response campaigns in the middle of the year because of COVID.”
The CDC report notes that many of the supplementary immunizations in response to cVDPV2 outbreaks were of “poor quality,” and prolonged delays enabled geographically expanding cVDPV2 transmission.
Steve Wassilak, MD, chief coauthor of the CDC study, told this news organization that, because of COVID, “what we’ve been lacking is a rapid response for the most part. Some of that is due to laboratory delays and shipment because of COVID’s effect on international travel.” He noted, however, that there has been good recovery in surveillance and immunization activities despite COVID. And, he added, eradication “can be done, and many outbreaks have closed even during the [COVID] outbreak.”
Dr. Wassilak said that in Nigeria, “the face of the campaign became national.” In Pakistan, much of the work is done by national and international partners.
Dr. Wenger said that in Nigeria and other challenging areas, “the approach was essentially to make direct contact with the traditional leaders and the religious leaders and the local actors in each of these populations. So, it’s really getting down to the grassroots level.” Infectious disease officials send teams to speak with individuals in the “local, traditional leader system.”
“Just talking to them actually got us a long way and giving them the information that they need. In most cases, I mean, people want to do things to help their kids,” said Dr. Wenger.
For now, the initial plan, per the CDC, is to “initiate prompt and high coverage outbreak responses with available type 2 OPV to interrupt transmission” until a better supply of nOPV2 is available, then switch to IPVs.
Dr. Wenger and Dr. Wassilak report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Most (86%) of these outbreaks were caused by cVDPV2 (circulating VDPV type 2 poliovirus, which originated with the vaccine), and most occurred in Africa, according to a new study of vaccine-derived poliovirus outbreaks between Jan. 2020 and June 2021 published in the CDC’s Morbidity and Mortality Weekly Report.
The Global Polio Eradication Initiative (GPEI) was launched in 1988 and used live attenuated oral poliovirus vaccine (OPV). Since then, cases of wild poliovirus have declined more than 99.99%.
The cVDPV2 likely originated among children born in areas with poor vaccine coverage. Jay Wenger, MD, director, Polio, at the Bill and Melinda Gates Foundation, told this news organization that “the inactivated vaccines that we give in most developed countries now are good in that they provide humoral immunity, the antibodies in the bloodstream. They don’t necessarily provide mucosal immunity. They don’t make the kid’s gut immune to getting reinfected or actually immune to reproducing the virus if they get it in their gut. So we could still have a situation where everybody was vaccinated with IPV [inactivated poliovirus], but the virus could still be transmitting around because kids’ guts would still be producing the virus and there will still be transmission in your population, probably without much or any paralysis because of the IPV. As soon as that virus hit a population that was not vaccinated, they would get paralyzed.”
Dr. Wenger added, “The ideal vaccine would be an oral vaccine that didn’t mutate back and couldn’t cause these VDPVs.” Scientists developed such a vaccine, approved by the World Health Organization last year under an Emergency Use Authorization. This nOPV2 (novel oral poliovirus type 2) vaccine has been given since March 2021 in areas with the VDPD2 outbreaks. The nOPV2 should allow them to “basically stamp out the outbreaks.”
The world had almost eradicated the disease, with the last cases of polio from wild virus occurring in Nigeria, Afghanistan, and Pakistan as of 2014. Africa was declared free of wild polio in 2020 after it had been eradicated from Nigeria, which accounted for more than half of the world’s cases only a decade earlier. Now cVDPV outbreaks affect 28 African countries, plus Iran, Yemen, Afghanistan, Pakistan, Tajikistan, Malaysia, the Philippines, and Indonesia. And there was also one case in China. Globally, there were 1,335 cases of cVDPV causing paralysis during the reporting period.
The COVID-19 pandemic has had a significant impact on polio, accounting for much of this year’s increase in cases. Dr. Wenger said, “We couldn’t do any campaigns. We pretty much stopped doing outbreak response campaigns in the middle of the year because of COVID.”
The CDC report notes that many of the supplementary immunizations in response to cVDPV2 outbreaks were of “poor quality,” and prolonged delays enabled geographically expanding cVDPV2 transmission.
Steve Wassilak, MD, chief coauthor of the CDC study, told this news organization that, because of COVID, “what we’ve been lacking is a rapid response for the most part. Some of that is due to laboratory delays and shipment because of COVID’s effect on international travel.” He noted, however, that there has been good recovery in surveillance and immunization activities despite COVID. And, he added, eradication “can be done, and many outbreaks have closed even during the [COVID] outbreak.”
Dr. Wassilak said that in Nigeria, “the face of the campaign became national.” In Pakistan, much of the work is done by national and international partners.
Dr. Wenger said that in Nigeria and other challenging areas, “the approach was essentially to make direct contact with the traditional leaders and the religious leaders and the local actors in each of these populations. So, it’s really getting down to the grassroots level.” Infectious disease officials send teams to speak with individuals in the “local, traditional leader system.”
“Just talking to them actually got us a long way and giving them the information that they need. In most cases, I mean, people want to do things to help their kids,” said Dr. Wenger.
For now, the initial plan, per the CDC, is to “initiate prompt and high coverage outbreak responses with available type 2 OPV to interrupt transmission” until a better supply of nOPV2 is available, then switch to IPVs.
Dr. Wenger and Dr. Wassilak report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Most (86%) of these outbreaks were caused by cVDPV2 (circulating VDPV type 2 poliovirus, which originated with the vaccine), and most occurred in Africa, according to a new study of vaccine-derived poliovirus outbreaks between Jan. 2020 and June 2021 published in the CDC’s Morbidity and Mortality Weekly Report.
The Global Polio Eradication Initiative (GPEI) was launched in 1988 and used live attenuated oral poliovirus vaccine (OPV). Since then, cases of wild poliovirus have declined more than 99.99%.
The cVDPV2 likely originated among children born in areas with poor vaccine coverage. Jay Wenger, MD, director, Polio, at the Bill and Melinda Gates Foundation, told this news organization that “the inactivated vaccines that we give in most developed countries now are good in that they provide humoral immunity, the antibodies in the bloodstream. They don’t necessarily provide mucosal immunity. They don’t make the kid’s gut immune to getting reinfected or actually immune to reproducing the virus if they get it in their gut. So we could still have a situation where everybody was vaccinated with IPV [inactivated poliovirus], but the virus could still be transmitting around because kids’ guts would still be producing the virus and there will still be transmission in your population, probably without much or any paralysis because of the IPV. As soon as that virus hit a population that was not vaccinated, they would get paralyzed.”
Dr. Wenger added, “The ideal vaccine would be an oral vaccine that didn’t mutate back and couldn’t cause these VDPVs.” Scientists developed such a vaccine, approved by the World Health Organization last year under an Emergency Use Authorization. This nOPV2 (novel oral poliovirus type 2) vaccine has been given since March 2021 in areas with the VDPD2 outbreaks. The nOPV2 should allow them to “basically stamp out the outbreaks.”
The world had almost eradicated the disease, with the last cases of polio from wild virus occurring in Nigeria, Afghanistan, and Pakistan as of 2014. Africa was declared free of wild polio in 2020 after it had been eradicated from Nigeria, which accounted for more than half of the world’s cases only a decade earlier. Now cVDPV outbreaks affect 28 African countries, plus Iran, Yemen, Afghanistan, Pakistan, Tajikistan, Malaysia, the Philippines, and Indonesia. And there was also one case in China. Globally, there were 1,335 cases of cVDPV causing paralysis during the reporting period.
The COVID-19 pandemic has had a significant impact on polio, accounting for much of this year’s increase in cases. Dr. Wenger said, “We couldn’t do any campaigns. We pretty much stopped doing outbreak response campaigns in the middle of the year because of COVID.”
The CDC report notes that many of the supplementary immunizations in response to cVDPV2 outbreaks were of “poor quality,” and prolonged delays enabled geographically expanding cVDPV2 transmission.
Steve Wassilak, MD, chief coauthor of the CDC study, told this news organization that, because of COVID, “what we’ve been lacking is a rapid response for the most part. Some of that is due to laboratory delays and shipment because of COVID’s effect on international travel.” He noted, however, that there has been good recovery in surveillance and immunization activities despite COVID. And, he added, eradication “can be done, and many outbreaks have closed even during the [COVID] outbreak.”
Dr. Wassilak said that in Nigeria, “the face of the campaign became national.” In Pakistan, much of the work is done by national and international partners.
Dr. Wenger said that in Nigeria and other challenging areas, “the approach was essentially to make direct contact with the traditional leaders and the religious leaders and the local actors in each of these populations. So, it’s really getting down to the grassroots level.” Infectious disease officials send teams to speak with individuals in the “local, traditional leader system.”
“Just talking to them actually got us a long way and giving them the information that they need. In most cases, I mean, people want to do things to help their kids,” said Dr. Wenger.
For now, the initial plan, per the CDC, is to “initiate prompt and high coverage outbreak responses with available type 2 OPV to interrupt transmission” until a better supply of nOPV2 is available, then switch to IPVs.
Dr. Wenger and Dr. Wassilak report no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Tofacitinib approved for new ankylosing spondylitis indication
The Food and Drug Administration approved a supplemental new drug application for tofacitinib (Xeljanz, Xeljanz XR) that adds active ankylosing spondylitis in adults to its list of indications, according to a Dec. 14 announcement from manufacturer Pfizer.
The approval makes the drug the first Janus kinase (JAK) inhibitor to be approved for ankylosing spondylitis, joining tofacitinib’s other indications of rheumatoid arthritis, psoriatic arthritis, ulcerative colitis, and polyarticular-course juvenile idiopathic arthritis.
Like other JAK inhibitors that are indicated for immune-mediated inflammatory diseases, tofacitinib’s use for all indications is limited to patients who have had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers.
The agency based its decision on the results of a phase 3, multicenter, randomized, double-blind, placebo-controlled trial in 269 adults with active ankylosing spondylitis that tested tofacitinib 5 mg twice daily.
The study met its primary endpoint showing that at week 16 the percentage of tofacitinib-treated patients who achieved 20% improvement in Assessment in SpondyloArthritis International Society response criteria (ASAS20) was significantly greater than with placebo (56.4% vs. 29.4%; P < .0001). The percentage of responders for ASAS40 criteria was likewise significantly greater with tofacitinib vs. placebo (40.6% vs. 12.5%; P < .0001). Pfizer said that the safety profile of tofacitinib observed in patients with ankylosing spondylitis was consistent with the safety profile observed in patients with either rheumatoid arthritis or psoriatic arthritis.
Pfizer noted in its announcement that the FDA updated the prescribing information this month for tofacitinib (and other JAK inhibitors approved for immune-mediated inflammatory conditions, upadacitinib [Rinvoq] and baricitinib [Olumiant]). This update included a new boxed warning for major adverse cardiovascular events and updated boxed warnings regarding mortality, malignancies, and thrombosis. These changes were made in light of results from the ORAL Surveillance postmarketing study of patients with rheumatoid arthritis aged 50 years and older with at least one cardiovascular risk factor. That study found an association between tofacitinib and increased risk of heart attack or stroke, cancer, blood clots, and death in comparison with patients who took the TNF blockers adalimumab or etanercept.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration approved a supplemental new drug application for tofacitinib (Xeljanz, Xeljanz XR) that adds active ankylosing spondylitis in adults to its list of indications, according to a Dec. 14 announcement from manufacturer Pfizer.
The approval makes the drug the first Janus kinase (JAK) inhibitor to be approved for ankylosing spondylitis, joining tofacitinib’s other indications of rheumatoid arthritis, psoriatic arthritis, ulcerative colitis, and polyarticular-course juvenile idiopathic arthritis.
Like other JAK inhibitors that are indicated for immune-mediated inflammatory diseases, tofacitinib’s use for all indications is limited to patients who have had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers.
The agency based its decision on the results of a phase 3, multicenter, randomized, double-blind, placebo-controlled trial in 269 adults with active ankylosing spondylitis that tested tofacitinib 5 mg twice daily.
The study met its primary endpoint showing that at week 16 the percentage of tofacitinib-treated patients who achieved 20% improvement in Assessment in SpondyloArthritis International Society response criteria (ASAS20) was significantly greater than with placebo (56.4% vs. 29.4%; P < .0001). The percentage of responders for ASAS40 criteria was likewise significantly greater with tofacitinib vs. placebo (40.6% vs. 12.5%; P < .0001). Pfizer said that the safety profile of tofacitinib observed in patients with ankylosing spondylitis was consistent with the safety profile observed in patients with either rheumatoid arthritis or psoriatic arthritis.
Pfizer noted in its announcement that the FDA updated the prescribing information this month for tofacitinib (and other JAK inhibitors approved for immune-mediated inflammatory conditions, upadacitinib [Rinvoq] and baricitinib [Olumiant]). This update included a new boxed warning for major adverse cardiovascular events and updated boxed warnings regarding mortality, malignancies, and thrombosis. These changes were made in light of results from the ORAL Surveillance postmarketing study of patients with rheumatoid arthritis aged 50 years and older with at least one cardiovascular risk factor. That study found an association between tofacitinib and increased risk of heart attack or stroke, cancer, blood clots, and death in comparison with patients who took the TNF blockers adalimumab or etanercept.
A version of this article first appeared on Medscape.com.
The Food and Drug Administration approved a supplemental new drug application for tofacitinib (Xeljanz, Xeljanz XR) that adds active ankylosing spondylitis in adults to its list of indications, according to a Dec. 14 announcement from manufacturer Pfizer.
The approval makes the drug the first Janus kinase (JAK) inhibitor to be approved for ankylosing spondylitis, joining tofacitinib’s other indications of rheumatoid arthritis, psoriatic arthritis, ulcerative colitis, and polyarticular-course juvenile idiopathic arthritis.
Like other JAK inhibitors that are indicated for immune-mediated inflammatory diseases, tofacitinib’s use for all indications is limited to patients who have had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers.
The agency based its decision on the results of a phase 3, multicenter, randomized, double-blind, placebo-controlled trial in 269 adults with active ankylosing spondylitis that tested tofacitinib 5 mg twice daily.
The study met its primary endpoint showing that at week 16 the percentage of tofacitinib-treated patients who achieved 20% improvement in Assessment in SpondyloArthritis International Society response criteria (ASAS20) was significantly greater than with placebo (56.4% vs. 29.4%; P < .0001). The percentage of responders for ASAS40 criteria was likewise significantly greater with tofacitinib vs. placebo (40.6% vs. 12.5%; P < .0001). Pfizer said that the safety profile of tofacitinib observed in patients with ankylosing spondylitis was consistent with the safety profile observed in patients with either rheumatoid arthritis or psoriatic arthritis.
Pfizer noted in its announcement that the FDA updated the prescribing information this month for tofacitinib (and other JAK inhibitors approved for immune-mediated inflammatory conditions, upadacitinib [Rinvoq] and baricitinib [Olumiant]). This update included a new boxed warning for major adverse cardiovascular events and updated boxed warnings regarding mortality, malignancies, and thrombosis. These changes were made in light of results from the ORAL Surveillance postmarketing study of patients with rheumatoid arthritis aged 50 years and older with at least one cardiovascular risk factor. That study found an association between tofacitinib and increased risk of heart attack or stroke, cancer, blood clots, and death in comparison with patients who took the TNF blockers adalimumab or etanercept.
A version of this article first appeared on Medscape.com.
Califf plans work on opioids, accelerated approvals on return to FDA
Robert M. Califf, MD, plans to take a close look at federal policies on opioid prescriptions in his expected second turn as the top U.S. regulator of medical products, as well as keep closer tabs on the performance of drugs cleared with accelerated approvals.
Dr. Califf on Tuesday fielded questions at a Senate hearing about his nomination by President Joe Biden to serve as administrator of the U.S. Food and Drug Administration, a role in which he served in the Obama administration. He also spoke about the need to bolster the nation’s ability to maintain an adequate supply of key medical products, including drugs.
Members of the Senate Health, Education, Labor and Pensions Committee, which is handling Dr. Califf’s nomination, were largely cordial and supportive during the hearing. Sen. Patty Murray (D-Wash.), the committee chair, and the panel’s top Republican, Sen. Richard Burr of North Carolina, addressed Dr. Califf during the hearing as if he would soon serve again as the FDA’s leader. Both were among the senators who voted 89-4 to confirm Dr. Califf in a February 2016 vote.
Dr. Califf “was previously confirmed to lead FDA in an overwhelming bipartisan vote, and I look forward to working with him again to ensure FDA continues to protect families across the country, uphold the gold standard of safety and effectiveness, and put science and data first,” Sen. Murray said.
Less enthusiastic about Dr. Califf was Sen. Bernie Sanders (I-VT), who was among the seven senators who did not vote on Dr. Califf’s nomination in 2016.
Sen. Sanders objected in 2016 to Dr. Califf’s ties to the pharmaceutical industry, and he did so again Tuesday. A noted leader in conducting clinical trials, Dr. Califf has worked with many drugmakers. But at the hearing, Dr. Califf said he concurs with Sen. Sanders on an idea strongly opposed by the pharmaceutical industry.
In response to Sen. Sanders’ question, Dr. Califf said he already is “on record as being in favor of Medicare negotiating with the industry on prices.”
The FDA would not take direct part in negotiations, as this work would be handled by the Centers for Medicare & Medicaid Services. Democrats want to give Medicare some negotiating authority through their sweeping Build Back Better Act.
People in the United States are dismayed over both the cost of prescription drugs and the widespread distribution of prescription painkillers that helped fuel the current opioid epidemic, Sen. Sanders told Dr. Califf. Many people will be concerned about an FDA commissioner who has benefited from close ties to the industry, Sen. Sanders said.
“How are they going to believe that you’re going to be an independent and strong voice against this enormously powerful, special interest?” Sen. Sanders asked.
“I’m totally with you on the concept that the price of pharmaceuticals is way too high in this country,” Dr. Califf said in reply.
Dr. Califf was paid $2.7 million in salary and bonus by Verily Life Sciences, the biomedical research organization operated by Alphabet, parent company of Google, according to his federal financial disclosure. He also reported holding board positions with pharmaceutical companies AmyriAD and Centessa Pharmaceuticals.
Bloomberg Government reported that Dr. Califf has ties to about 16 other research organizations and biotech companies. Bloomberg Government also said that, in his earlier FDA service, Dr. Califf kept a whiteboard in his office that listed all the activities and projects that required his recusal, citing as a source Howard Sklamberg, who was a deputy commissioner under Dr. Califf.
“He was very, very, very careful,” Mr. Sklamberg, who’s now an attorney at Arnold & Porter LLP, told Bloomberg Government.
‘Work to do’ on opioids
Senators looped back repeatedly to the topic of opioids during Dr. Califf’s hearing, reflecting deep concerns about the FDA’s efforts to warn of the risks of prescription painkillers.
There were an estimated 100,306 drug overdose deaths in the United States in the 12 months ending in April, an increase of 28.5% from the 78,056 deaths during the same period the year before, according to the Centers for Disease Control and Prevention.
Dr. Califf said he plans to focus on what information the FDA conveys to the public about the risks of prescription painkillers, including a look at what the labels for these products say.
“I am committed to do a comprehensive review of the status of opioids, early in my tenure,” Dr. Califf said.
Dr. Califf indicated that physicians are still too quick to provide excess doses of these medicines, despite years of efforts to restrain their use. He said he knows relatives who were given 30-day prescriptions for opioids after minor surgery.
“So I know we have work to do,” Dr. Califf said.
Concerns about the FDA’s previous work in managing opioids has led to protests from a few Democratic senators about the prospect of President Biden nominating the acting FDA commissioner, Janet Woodcock, MD, for the permanent post.
At the hearing, Sen. Ben Ray Luján (D-NM) raised the case of the FDA’s approval of the powerful Zohydro painkiller. The agency approved that drug despite an 11-2 vote against it by the FDA’s Anesthetic and Analgesic Drug Products Advisory Committee.
Sen. Luján asked Dr. Califf what he would do if an FDA advisory committee voted “overwhelmingly” against recommending approval of a medicine, as happened in the Zohydro case.
While not mentioned by Sen. Luján in this exchange during the hearing with Dr. Califf, the FDA staff’s rejection of recommendations of advisory committees has been a growing concern among researchers.
The agency last year approved aducanumab (Aduhelm, Biogen), a drug for Alzheimer’s disease, dismissing the advice of its Peripheral and Central Nervous System Drugs Advisory Committee. That decision triggered the resignation of several members of the panel. The FDA staff also earlier rejected the conclusion the majority of members of the same advisory committee offered in 2016 on eteplirsen (Exondys 51, Sarepta), a drug for Duchenne muscular dystrophy.
Dr. Califf told Sen. Luján he had done recent research into how often the FDA staff does not concur with the recommendations of an advisory committee. He said the FDA takes a different course of action in about 25% of cases. In about three-quarters of those cases, the FDA staff opts for a “more stringent” approach regarding allowing the public access to the drug, as opposed to a more generous one as seen in the Zohydro, Aduhelm, and Exondys 51 cases.
Still, Dr. Califf said that when there’s an 11-2 advisory committee vote against recommendation of a product, “the leaders at FDA really need to take a close look” at what’s happening.
Question on accelerated approvals
The FDA’s approval of aducanumab drew attention to a debate already underway about conditional clearances known as accelerated approvals.
The FDA has used this path since the 1990s to speed access to drugs for serious conditions. The trade-off for early access is that the agency sometimes makes the wrong call based on initial findings, and clears a medicine later found not to benefit patients as expected.
The FDA’s cancer division is in the midst of public efforts to address cases where drugmakers have not been able to deliver studies that support accelerated approvals of their oncology drugs. In addition, the Office of Inspector General of the U.S. Department of Health & Human Services announced in August that it is reviewing the FDA’s handling of the accelerated approval process.
At Tuesday’s hearing, Sen. Burr grilled Dr. Califf about how he would respond to calls to change how the FDA handles the accelerated-approval process.
“Can you commit to me and to patients who may rely on cutting-edge treatments that you will not support efforts to narrow this pathway or raise the bar for drugs to be approved under those pathways?” Burr asked Califf.
Dr. Califf responded by saying he was “a fan of accelerated approval – for the right conditions.”
Earlier, in his opening statement, Dr. Califf had said his mother benefited directly from the accelerated approval of new drugs for multiple myeloma. Dr. Califf told Sen. Burr that he had spent “countless hours with patient groups” and understands the need to speed the approval of medicines for serious diseases.
But the FDA also has to make sure it holds up its end of the bargain struck with accelerated approvals. This involves checking on how these medicines work once they are marketed.
“We’re accepting that there’s more uncertainty,” Dr. Califf said. “That means we’ve got to have a better system to evaluate these products as they’re used on the market. And I think there are ways that we can do that now. Technology is making this possible in ways that it just was not possible before.”
Worries about the medical supply chain
Sen. Susan Collins (R-Maine) asked Dr. Califf about the vulnerability of the U.S. medical system to disruptions of the supply chain. She raised concerns about China’s dominance in antibiotic manufacturing as an example. She asked if Congress could do more to encourage domestic manufacturing of medical supplies, such as by offering tax incentives.
Dr. Califf told Sen. Collins he shared her concern about the U.S. manufacturing of ingredients used in both branded and generic drugs. He said he recently has served on a committee of the National Academy of Medicine that is examining supply chain issues.
This committee will soon release a report with specific recommendations, Dr. Califf said.
“We don’t have enough competitive entities in what’s become sort of a commodity business” of drug manufacturing, Dr. Califf said. “So we need a number of steps to make the system more resilient.”
A version of this article first appeared on Medscape.com.
Robert M. Califf, MD, plans to take a close look at federal policies on opioid prescriptions in his expected second turn as the top U.S. regulator of medical products, as well as keep closer tabs on the performance of drugs cleared with accelerated approvals.
Dr. Califf on Tuesday fielded questions at a Senate hearing about his nomination by President Joe Biden to serve as administrator of the U.S. Food and Drug Administration, a role in which he served in the Obama administration. He also spoke about the need to bolster the nation’s ability to maintain an adequate supply of key medical products, including drugs.
Members of the Senate Health, Education, Labor and Pensions Committee, which is handling Dr. Califf’s nomination, were largely cordial and supportive during the hearing. Sen. Patty Murray (D-Wash.), the committee chair, and the panel’s top Republican, Sen. Richard Burr of North Carolina, addressed Dr. Califf during the hearing as if he would soon serve again as the FDA’s leader. Both were among the senators who voted 89-4 to confirm Dr. Califf in a February 2016 vote.
Dr. Califf “was previously confirmed to lead FDA in an overwhelming bipartisan vote, and I look forward to working with him again to ensure FDA continues to protect families across the country, uphold the gold standard of safety and effectiveness, and put science and data first,” Sen. Murray said.
Less enthusiastic about Dr. Califf was Sen. Bernie Sanders (I-VT), who was among the seven senators who did not vote on Dr. Califf’s nomination in 2016.
Sen. Sanders objected in 2016 to Dr. Califf’s ties to the pharmaceutical industry, and he did so again Tuesday. A noted leader in conducting clinical trials, Dr. Califf has worked with many drugmakers. But at the hearing, Dr. Califf said he concurs with Sen. Sanders on an idea strongly opposed by the pharmaceutical industry.
In response to Sen. Sanders’ question, Dr. Califf said he already is “on record as being in favor of Medicare negotiating with the industry on prices.”
The FDA would not take direct part in negotiations, as this work would be handled by the Centers for Medicare & Medicaid Services. Democrats want to give Medicare some negotiating authority through their sweeping Build Back Better Act.
People in the United States are dismayed over both the cost of prescription drugs and the widespread distribution of prescription painkillers that helped fuel the current opioid epidemic, Sen. Sanders told Dr. Califf. Many people will be concerned about an FDA commissioner who has benefited from close ties to the industry, Sen. Sanders said.
“How are they going to believe that you’re going to be an independent and strong voice against this enormously powerful, special interest?” Sen. Sanders asked.
“I’m totally with you on the concept that the price of pharmaceuticals is way too high in this country,” Dr. Califf said in reply.
Dr. Califf was paid $2.7 million in salary and bonus by Verily Life Sciences, the biomedical research organization operated by Alphabet, parent company of Google, according to his federal financial disclosure. He also reported holding board positions with pharmaceutical companies AmyriAD and Centessa Pharmaceuticals.
Bloomberg Government reported that Dr. Califf has ties to about 16 other research organizations and biotech companies. Bloomberg Government also said that, in his earlier FDA service, Dr. Califf kept a whiteboard in his office that listed all the activities and projects that required his recusal, citing as a source Howard Sklamberg, who was a deputy commissioner under Dr. Califf.
“He was very, very, very careful,” Mr. Sklamberg, who’s now an attorney at Arnold & Porter LLP, told Bloomberg Government.
‘Work to do’ on opioids
Senators looped back repeatedly to the topic of opioids during Dr. Califf’s hearing, reflecting deep concerns about the FDA’s efforts to warn of the risks of prescription painkillers.
There were an estimated 100,306 drug overdose deaths in the United States in the 12 months ending in April, an increase of 28.5% from the 78,056 deaths during the same period the year before, according to the Centers for Disease Control and Prevention.
Dr. Califf said he plans to focus on what information the FDA conveys to the public about the risks of prescription painkillers, including a look at what the labels for these products say.
“I am committed to do a comprehensive review of the status of opioids, early in my tenure,” Dr. Califf said.
Dr. Califf indicated that physicians are still too quick to provide excess doses of these medicines, despite years of efforts to restrain their use. He said he knows relatives who were given 30-day prescriptions for opioids after minor surgery.
“So I know we have work to do,” Dr. Califf said.
Concerns about the FDA’s previous work in managing opioids has led to protests from a few Democratic senators about the prospect of President Biden nominating the acting FDA commissioner, Janet Woodcock, MD, for the permanent post.
At the hearing, Sen. Ben Ray Luján (D-NM) raised the case of the FDA’s approval of the powerful Zohydro painkiller. The agency approved that drug despite an 11-2 vote against it by the FDA’s Anesthetic and Analgesic Drug Products Advisory Committee.
Sen. Luján asked Dr. Califf what he would do if an FDA advisory committee voted “overwhelmingly” against recommending approval of a medicine, as happened in the Zohydro case.
While not mentioned by Sen. Luján in this exchange during the hearing with Dr. Califf, the FDA staff’s rejection of recommendations of advisory committees has been a growing concern among researchers.
The agency last year approved aducanumab (Aduhelm, Biogen), a drug for Alzheimer’s disease, dismissing the advice of its Peripheral and Central Nervous System Drugs Advisory Committee. That decision triggered the resignation of several members of the panel. The FDA staff also earlier rejected the conclusion the majority of members of the same advisory committee offered in 2016 on eteplirsen (Exondys 51, Sarepta), a drug for Duchenne muscular dystrophy.
Dr. Califf told Sen. Luján he had done recent research into how often the FDA staff does not concur with the recommendations of an advisory committee. He said the FDA takes a different course of action in about 25% of cases. In about three-quarters of those cases, the FDA staff opts for a “more stringent” approach regarding allowing the public access to the drug, as opposed to a more generous one as seen in the Zohydro, Aduhelm, and Exondys 51 cases.
Still, Dr. Califf said that when there’s an 11-2 advisory committee vote against recommendation of a product, “the leaders at FDA really need to take a close look” at what’s happening.
Question on accelerated approvals
The FDA’s approval of aducanumab drew attention to a debate already underway about conditional clearances known as accelerated approvals.
The FDA has used this path since the 1990s to speed access to drugs for serious conditions. The trade-off for early access is that the agency sometimes makes the wrong call based on initial findings, and clears a medicine later found not to benefit patients as expected.
The FDA’s cancer division is in the midst of public efforts to address cases where drugmakers have not been able to deliver studies that support accelerated approvals of their oncology drugs. In addition, the Office of Inspector General of the U.S. Department of Health & Human Services announced in August that it is reviewing the FDA’s handling of the accelerated approval process.
At Tuesday’s hearing, Sen. Burr grilled Dr. Califf about how he would respond to calls to change how the FDA handles the accelerated-approval process.
“Can you commit to me and to patients who may rely on cutting-edge treatments that you will not support efforts to narrow this pathway or raise the bar for drugs to be approved under those pathways?” Burr asked Califf.
Dr. Califf responded by saying he was “a fan of accelerated approval – for the right conditions.”
Earlier, in his opening statement, Dr. Califf had said his mother benefited directly from the accelerated approval of new drugs for multiple myeloma. Dr. Califf told Sen. Burr that he had spent “countless hours with patient groups” and understands the need to speed the approval of medicines for serious diseases.
But the FDA also has to make sure it holds up its end of the bargain struck with accelerated approvals. This involves checking on how these medicines work once they are marketed.
“We’re accepting that there’s more uncertainty,” Dr. Califf said. “That means we’ve got to have a better system to evaluate these products as they’re used on the market. And I think there are ways that we can do that now. Technology is making this possible in ways that it just was not possible before.”
Worries about the medical supply chain
Sen. Susan Collins (R-Maine) asked Dr. Califf about the vulnerability of the U.S. medical system to disruptions of the supply chain. She raised concerns about China’s dominance in antibiotic manufacturing as an example. She asked if Congress could do more to encourage domestic manufacturing of medical supplies, such as by offering tax incentives.
Dr. Califf told Sen. Collins he shared her concern about the U.S. manufacturing of ingredients used in both branded and generic drugs. He said he recently has served on a committee of the National Academy of Medicine that is examining supply chain issues.
This committee will soon release a report with specific recommendations, Dr. Califf said.
“We don’t have enough competitive entities in what’s become sort of a commodity business” of drug manufacturing, Dr. Califf said. “So we need a number of steps to make the system more resilient.”
A version of this article first appeared on Medscape.com.
Robert M. Califf, MD, plans to take a close look at federal policies on opioid prescriptions in his expected second turn as the top U.S. regulator of medical products, as well as keep closer tabs on the performance of drugs cleared with accelerated approvals.
Dr. Califf on Tuesday fielded questions at a Senate hearing about his nomination by President Joe Biden to serve as administrator of the U.S. Food and Drug Administration, a role in which he served in the Obama administration. He also spoke about the need to bolster the nation’s ability to maintain an adequate supply of key medical products, including drugs.
Members of the Senate Health, Education, Labor and Pensions Committee, which is handling Dr. Califf’s nomination, were largely cordial and supportive during the hearing. Sen. Patty Murray (D-Wash.), the committee chair, and the panel’s top Republican, Sen. Richard Burr of North Carolina, addressed Dr. Califf during the hearing as if he would soon serve again as the FDA’s leader. Both were among the senators who voted 89-4 to confirm Dr. Califf in a February 2016 vote.
Dr. Califf “was previously confirmed to lead FDA in an overwhelming bipartisan vote, and I look forward to working with him again to ensure FDA continues to protect families across the country, uphold the gold standard of safety and effectiveness, and put science and data first,” Sen. Murray said.
Less enthusiastic about Dr. Califf was Sen. Bernie Sanders (I-VT), who was among the seven senators who did not vote on Dr. Califf’s nomination in 2016.
Sen. Sanders objected in 2016 to Dr. Califf’s ties to the pharmaceutical industry, and he did so again Tuesday. A noted leader in conducting clinical trials, Dr. Califf has worked with many drugmakers. But at the hearing, Dr. Califf said he concurs with Sen. Sanders on an idea strongly opposed by the pharmaceutical industry.
In response to Sen. Sanders’ question, Dr. Califf said he already is “on record as being in favor of Medicare negotiating with the industry on prices.”
The FDA would not take direct part in negotiations, as this work would be handled by the Centers for Medicare & Medicaid Services. Democrats want to give Medicare some negotiating authority through their sweeping Build Back Better Act.
People in the United States are dismayed over both the cost of prescription drugs and the widespread distribution of prescription painkillers that helped fuel the current opioid epidemic, Sen. Sanders told Dr. Califf. Many people will be concerned about an FDA commissioner who has benefited from close ties to the industry, Sen. Sanders said.
“How are they going to believe that you’re going to be an independent and strong voice against this enormously powerful, special interest?” Sen. Sanders asked.
“I’m totally with you on the concept that the price of pharmaceuticals is way too high in this country,” Dr. Califf said in reply.
Dr. Califf was paid $2.7 million in salary and bonus by Verily Life Sciences, the biomedical research organization operated by Alphabet, parent company of Google, according to his federal financial disclosure. He also reported holding board positions with pharmaceutical companies AmyriAD and Centessa Pharmaceuticals.
Bloomberg Government reported that Dr. Califf has ties to about 16 other research organizations and biotech companies. Bloomberg Government also said that, in his earlier FDA service, Dr. Califf kept a whiteboard in his office that listed all the activities and projects that required his recusal, citing as a source Howard Sklamberg, who was a deputy commissioner under Dr. Califf.
“He was very, very, very careful,” Mr. Sklamberg, who’s now an attorney at Arnold & Porter LLP, told Bloomberg Government.
‘Work to do’ on opioids
Senators looped back repeatedly to the topic of opioids during Dr. Califf’s hearing, reflecting deep concerns about the FDA’s efforts to warn of the risks of prescription painkillers.
There were an estimated 100,306 drug overdose deaths in the United States in the 12 months ending in April, an increase of 28.5% from the 78,056 deaths during the same period the year before, according to the Centers for Disease Control and Prevention.
Dr. Califf said he plans to focus on what information the FDA conveys to the public about the risks of prescription painkillers, including a look at what the labels for these products say.
“I am committed to do a comprehensive review of the status of opioids, early in my tenure,” Dr. Califf said.
Dr. Califf indicated that physicians are still too quick to provide excess doses of these medicines, despite years of efforts to restrain their use. He said he knows relatives who were given 30-day prescriptions for opioids after minor surgery.
“So I know we have work to do,” Dr. Califf said.
Concerns about the FDA’s previous work in managing opioids has led to protests from a few Democratic senators about the prospect of President Biden nominating the acting FDA commissioner, Janet Woodcock, MD, for the permanent post.
At the hearing, Sen. Ben Ray Luján (D-NM) raised the case of the FDA’s approval of the powerful Zohydro painkiller. The agency approved that drug despite an 11-2 vote against it by the FDA’s Anesthetic and Analgesic Drug Products Advisory Committee.
Sen. Luján asked Dr. Califf what he would do if an FDA advisory committee voted “overwhelmingly” against recommending approval of a medicine, as happened in the Zohydro case.
While not mentioned by Sen. Luján in this exchange during the hearing with Dr. Califf, the FDA staff’s rejection of recommendations of advisory committees has been a growing concern among researchers.
The agency last year approved aducanumab (Aduhelm, Biogen), a drug for Alzheimer’s disease, dismissing the advice of its Peripheral and Central Nervous System Drugs Advisory Committee. That decision triggered the resignation of several members of the panel. The FDA staff also earlier rejected the conclusion the majority of members of the same advisory committee offered in 2016 on eteplirsen (Exondys 51, Sarepta), a drug for Duchenne muscular dystrophy.
Dr. Califf told Sen. Luján he had done recent research into how often the FDA staff does not concur with the recommendations of an advisory committee. He said the FDA takes a different course of action in about 25% of cases. In about three-quarters of those cases, the FDA staff opts for a “more stringent” approach regarding allowing the public access to the drug, as opposed to a more generous one as seen in the Zohydro, Aduhelm, and Exondys 51 cases.
Still, Dr. Califf said that when there’s an 11-2 advisory committee vote against recommendation of a product, “the leaders at FDA really need to take a close look” at what’s happening.
Question on accelerated approvals
The FDA’s approval of aducanumab drew attention to a debate already underway about conditional clearances known as accelerated approvals.
The FDA has used this path since the 1990s to speed access to drugs for serious conditions. The trade-off for early access is that the agency sometimes makes the wrong call based on initial findings, and clears a medicine later found not to benefit patients as expected.
The FDA’s cancer division is in the midst of public efforts to address cases where drugmakers have not been able to deliver studies that support accelerated approvals of their oncology drugs. In addition, the Office of Inspector General of the U.S. Department of Health & Human Services announced in August that it is reviewing the FDA’s handling of the accelerated approval process.
At Tuesday’s hearing, Sen. Burr grilled Dr. Califf about how he would respond to calls to change how the FDA handles the accelerated-approval process.
“Can you commit to me and to patients who may rely on cutting-edge treatments that you will not support efforts to narrow this pathway or raise the bar for drugs to be approved under those pathways?” Burr asked Califf.
Dr. Califf responded by saying he was “a fan of accelerated approval – for the right conditions.”
Earlier, in his opening statement, Dr. Califf had said his mother benefited directly from the accelerated approval of new drugs for multiple myeloma. Dr. Califf told Sen. Burr that he had spent “countless hours with patient groups” and understands the need to speed the approval of medicines for serious diseases.
But the FDA also has to make sure it holds up its end of the bargain struck with accelerated approvals. This involves checking on how these medicines work once they are marketed.
“We’re accepting that there’s more uncertainty,” Dr. Califf said. “That means we’ve got to have a better system to evaluate these products as they’re used on the market. And I think there are ways that we can do that now. Technology is making this possible in ways that it just was not possible before.”
Worries about the medical supply chain
Sen. Susan Collins (R-Maine) asked Dr. Califf about the vulnerability of the U.S. medical system to disruptions of the supply chain. She raised concerns about China’s dominance in antibiotic manufacturing as an example. She asked if Congress could do more to encourage domestic manufacturing of medical supplies, such as by offering tax incentives.
Dr. Califf told Sen. Collins he shared her concern about the U.S. manufacturing of ingredients used in both branded and generic drugs. He said he recently has served on a committee of the National Academy of Medicine that is examining supply chain issues.
This committee will soon release a report with specific recommendations, Dr. Califf said.
“We don’t have enough competitive entities in what’s become sort of a commodity business” of drug manufacturing, Dr. Califf said. “So we need a number of steps to make the system more resilient.”
A version of this article first appeared on Medscape.com.
FDA authorizes Pfizer boosters for 16- and 17-year-olds
, clearing the way for millions of teenagers to get a third dose of vaccine starting 6 months after their second dose.
The FDA said it was basing its emergency authorization of boosters for 16- and 17-year-olds on data from 200 individuals who were 18-55 years of age when they received a booster dose. They are requiring Pfizer to collect data on safety in postauthorization studies.
“The FDA has determined that the benefits of a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine or Comirnaty outweigh the risks of myocarditis and pericarditis in individuals 16 and 17 years of age to provide continued protection against COVID-19 and the associated serious consequences that can occur including hospitalization and death,” the agency said in a news release.
Israel has been giving booster doses of Pfizer’s vaccine to everyone 12 and up since late August. Data from that country show that myocarditis cases continue to be very rare, even in younger age groups, and are mild and temporary.
The authorization comes as the effectiveness of the current vaccines against the new Omicron variant has become a point of intense scientific inquiry.
Early studies suggest that booster doses may be necessary to keep Omicron at bay, at least until new variant-specific vaccines are ready next spring.
Current evidence suggests that the protection of the vaccines is holding up well against severe disease and death, at least with Delta and early iterations of the virus.
How well they will do against Omicron, and how severe Omicron infections may be for different age groups, remain open questions.
On Dec. 8, the World Health Organization urged countries not to wait for all the science to come in, but to act now to contain any potential threat.
The first pieces of evidence on Omicron suggest that it is highly contagious, perhaps even more than Delta, though early reports suggest symptoms caused by this version of the new coronavirus may be less severe than in previous waves. Experts have cautioned that the true severity of Omicron infections isn’t yet known, since the first cases have been detected in younger people, who tend to have milder COVID-19 symptoms than those of adults and seniors.
“Vaccination and getting a booster when eligible, along with other preventive measures like masking and avoiding large crowds and poorly ventilated spaces, remain our most effective methods for fighting COVID-19,” Acting FDA Commissioner Janet Woodcock, MD, said in a news release. “As people gather indoors with family and friends for the holidays, we can’t let up on all the preventive public health measures that we have been taking during the pandemic. With both the Delta and Omicron variants continuing to spread, vaccination remains the best protection against COVID-19.”
In mid-November, the FDA authorized boosters of the Pfizer vaccine for all individuals 18 and older, but the agency held off on expanding the use of boosters for younger age groups, partly because they have the highest risk of myocarditis, a very rare side effect.
Myocarditis cases seem to be temporary, with patients making a full recovery, though they need to be monitored in the hospital. The risk of myocarditis with a COVID-19 infection is many times higher than it is from a vaccine.
There have been little data to support the need for boosters in this age group, because children and teens tend to experience milder COVID-19 disease, though they are still at risk for post–COVID-19 complications such as long COVID and a delayed reaction to the virus called Post Acute Sequelae of SARS-CoV2 Infection among Children, or PAS-C.
All that changed with the arrival of Omicron.
A version of this article first appeared on WebMD.com.
, clearing the way for millions of teenagers to get a third dose of vaccine starting 6 months after their second dose.
The FDA said it was basing its emergency authorization of boosters for 16- and 17-year-olds on data from 200 individuals who were 18-55 years of age when they received a booster dose. They are requiring Pfizer to collect data on safety in postauthorization studies.
“The FDA has determined that the benefits of a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine or Comirnaty outweigh the risks of myocarditis and pericarditis in individuals 16 and 17 years of age to provide continued protection against COVID-19 and the associated serious consequences that can occur including hospitalization and death,” the agency said in a news release.
Israel has been giving booster doses of Pfizer’s vaccine to everyone 12 and up since late August. Data from that country show that myocarditis cases continue to be very rare, even in younger age groups, and are mild and temporary.
The authorization comes as the effectiveness of the current vaccines against the new Omicron variant has become a point of intense scientific inquiry.
Early studies suggest that booster doses may be necessary to keep Omicron at bay, at least until new variant-specific vaccines are ready next spring.
Current evidence suggests that the protection of the vaccines is holding up well against severe disease and death, at least with Delta and early iterations of the virus.
How well they will do against Omicron, and how severe Omicron infections may be for different age groups, remain open questions.
On Dec. 8, the World Health Organization urged countries not to wait for all the science to come in, but to act now to contain any potential threat.
The first pieces of evidence on Omicron suggest that it is highly contagious, perhaps even more than Delta, though early reports suggest symptoms caused by this version of the new coronavirus may be less severe than in previous waves. Experts have cautioned that the true severity of Omicron infections isn’t yet known, since the first cases have been detected in younger people, who tend to have milder COVID-19 symptoms than those of adults and seniors.
“Vaccination and getting a booster when eligible, along with other preventive measures like masking and avoiding large crowds and poorly ventilated spaces, remain our most effective methods for fighting COVID-19,” Acting FDA Commissioner Janet Woodcock, MD, said in a news release. “As people gather indoors with family and friends for the holidays, we can’t let up on all the preventive public health measures that we have been taking during the pandemic. With both the Delta and Omicron variants continuing to spread, vaccination remains the best protection against COVID-19.”
In mid-November, the FDA authorized boosters of the Pfizer vaccine for all individuals 18 and older, but the agency held off on expanding the use of boosters for younger age groups, partly because they have the highest risk of myocarditis, a very rare side effect.
Myocarditis cases seem to be temporary, with patients making a full recovery, though they need to be monitored in the hospital. The risk of myocarditis with a COVID-19 infection is many times higher than it is from a vaccine.
There have been little data to support the need for boosters in this age group, because children and teens tend to experience milder COVID-19 disease, though they are still at risk for post–COVID-19 complications such as long COVID and a delayed reaction to the virus called Post Acute Sequelae of SARS-CoV2 Infection among Children, or PAS-C.
All that changed with the arrival of Omicron.
A version of this article first appeared on WebMD.com.
, clearing the way for millions of teenagers to get a third dose of vaccine starting 6 months after their second dose.
The FDA said it was basing its emergency authorization of boosters for 16- and 17-year-olds on data from 200 individuals who were 18-55 years of age when they received a booster dose. They are requiring Pfizer to collect data on safety in postauthorization studies.
“The FDA has determined that the benefits of a single booster dose of the Pfizer-BioNTech COVID-19 Vaccine or Comirnaty outweigh the risks of myocarditis and pericarditis in individuals 16 and 17 years of age to provide continued protection against COVID-19 and the associated serious consequences that can occur including hospitalization and death,” the agency said in a news release.
Israel has been giving booster doses of Pfizer’s vaccine to everyone 12 and up since late August. Data from that country show that myocarditis cases continue to be very rare, even in younger age groups, and are mild and temporary.
The authorization comes as the effectiveness of the current vaccines against the new Omicron variant has become a point of intense scientific inquiry.
Early studies suggest that booster doses may be necessary to keep Omicron at bay, at least until new variant-specific vaccines are ready next spring.
Current evidence suggests that the protection of the vaccines is holding up well against severe disease and death, at least with Delta and early iterations of the virus.
How well they will do against Omicron, and how severe Omicron infections may be for different age groups, remain open questions.
On Dec. 8, the World Health Organization urged countries not to wait for all the science to come in, but to act now to contain any potential threat.
The first pieces of evidence on Omicron suggest that it is highly contagious, perhaps even more than Delta, though early reports suggest symptoms caused by this version of the new coronavirus may be less severe than in previous waves. Experts have cautioned that the true severity of Omicron infections isn’t yet known, since the first cases have been detected in younger people, who tend to have milder COVID-19 symptoms than those of adults and seniors.
“Vaccination and getting a booster when eligible, along with other preventive measures like masking and avoiding large crowds and poorly ventilated spaces, remain our most effective methods for fighting COVID-19,” Acting FDA Commissioner Janet Woodcock, MD, said in a news release. “As people gather indoors with family and friends for the holidays, we can’t let up on all the preventive public health measures that we have been taking during the pandemic. With both the Delta and Omicron variants continuing to spread, vaccination remains the best protection against COVID-19.”
In mid-November, the FDA authorized boosters of the Pfizer vaccine for all individuals 18 and older, but the agency held off on expanding the use of boosters for younger age groups, partly because they have the highest risk of myocarditis, a very rare side effect.
Myocarditis cases seem to be temporary, with patients making a full recovery, though they need to be monitored in the hospital. The risk of myocarditis with a COVID-19 infection is many times higher than it is from a vaccine.
There have been little data to support the need for boosters in this age group, because children and teens tend to experience milder COVID-19 disease, though they are still at risk for post–COVID-19 complications such as long COVID and a delayed reaction to the virus called Post Acute Sequelae of SARS-CoV2 Infection among Children, or PAS-C.
All that changed with the arrival of Omicron.
A version of this article first appeared on WebMD.com.
New HIV PrEP guidelines call for clinicians to talk to patients about HIV prevention meds
Starting Dec. 8, the Centers for Disease Control and Prevention recommends all clinicians talk to their sexually active adolescent and adult patients about HIV pre-exposure prophylaxis (PrEP) at least once and prescribe the prevention pills to anyone who asks for them, whether or not you understand their need for it.
“PrEP is a part of good primary care,” Demetre Daskalakis, MD, CDC’s director of the division of HIV/AIDS prevention, said in an interview. “Listening to people and what they need, as opposed to assessing what you think they need, is a seismic shift in how PrEP should be offered.”
The expanded recommendation comes as part of the 2021 update to the U.S. Public Health Service’s PrEP prescribing guidelines. It’s the third iteration since the Food and Drug Administration approved the first HIV prevention pill in 2012, and the first to include guidance on how to prescribe and monitor an injectable version of PrEP, which the FDA may approve as early as December 2021.
There are currently two pills, Truvada (emtricitabine/tenofovir disoproxil fumarate, Gilead Sciences and generic) and Descovy (emtricitabine/tenofovir alafenamide, Gilead Sciences). The pills have been found to be up to 99% effective in preventing HIV acquisition. The new injectable cabotegravir appears to be even more effective.
The broadened guidance is part of an effort from the country’s top health officials to expand PrEP prescribing from infectious disease specialists and sexual health clinics to health care professionals, including gynecologists, internal medicine physicians, and family practice clinicians. It appears to be necessary. In 2020, just 25% of the 1.2 million Americans who could benefit from PrEP were taking it, according to CDC data.
But those rates belie stark disparities in PrEP use by race and gender. The vast majority of those using PrEP are White Americans and men. About 66% of White Americans who could benefit from PrEP used it in 2020, and more than a quarter of the men who could benefit used it. By contrast, just 16% of Latinx people who could benefit had a prescription. And fewer than 1 in 10 Black Americans, who make up nearly half of those with indications for PrEP, had a prescription. The same was true for the women who could benefit.
Researchers and data from early PrEP demonstration projects have documented that clinicians are less likely to refer or prescribe the HIV prevention pills to Black people, especially the Black cisgender and transgender women and same-gender-loving men who bear the disproportionate burden of new cases in the United States, as well as fail to prescribe the medication to people who inject drugs.
Normalizing PrEP in primary care
When Courtney Sherman, DNP, APRN, first heard about PrEP in the early 2010s, she joked that her reaction was: “You’re ridiculous. You’re making that up. That’s not real.”
Ms. Sherman is now launching a tele-PrEP program from CAN Community Health, a nonprofit network of community health centers in southern Florida. The tele-PrEP program is meant to serve people in Florida and beyond, to increase access to the pill in areas with few health care professionals, or clinicians unwilling to prescribe it.
“When I go other places, I can’t do what I do for a living without getting some sort of bizarre comment or look,” she said. But the looks don’t just come from family, friends, or her children’s teachers. They come from colleagues, too. “What I’ve learned is that anybody – anybody – can be impacted [by HIV] and the illusion that ‘those people who live over there do things that me and my kind don’t do’ is just garbage.”
That’s the PrEP stigma that the universal PrEP counseling in the guidelines is meant to override, said Dr. Daskalakis. Going forward, he said that informing people about PrEP should be treated as normally as counseling people about smoking.
“You can change the blank: You talk to all adolescents and adults about not smoking,” he said. “This is: ‘Tell adolescents and adults about ways you can prevent HIV, and PrEP is one of them.’ ”
The guidelines also simplify for monitoring lab levels for the current daily pills, checking creatinine clearance levels twice a year in people older than age 50 and once a year in those younger than 50 taking the oral pills. Dr. Daskalakis said that should ease the burden of monitoring PrEP patients for health care professionals with busy caseloads.
It’s a move that drew praise from Shawnika Hull, PhD, assistant professor of health communications at Rutgers University, New Brunswick, N.J.. Dr. Hull’s recent data showed that clinicians who espoused more biased racial views were also less likely to prescribe PrEP to Black women who asked for it.
“Public health practitioners and scientists have been advocating for this as a strategy, as one way to address several ongoing barriers to PrEP specifically but also equity in PrEP,” said Dr. Hull. “This sort of universal provision of information is really an important strategy to try to undo some of the deeply intertwined barriers to uptake.”
‘Don’t grill them’
The updated guidelines keep the number and proportion of Americans who could benefit from PrEP the same: 1.2 million Americans, with nearly half of those Black. And the reasons people would qualify for PrEP remain the same: inconsistent condom use, sharing injection drug equipment, and a STI diagnosis in the last 6 months. There are also 57 jurisdictions, including seven rural states, where dating and having sex carries an increased risk of acquiring HIV because of high rates of untreated HIV in the community.
That’s why the other big change in the update is guidance to prescribe PrEP to whoever asks for it, whether the patient divulges their risk or not. Or as Dr. Daskalakis puts it: “If someone asks for PrEP, don’t grill them.”
There are lots of reasons that someone might ask for PrEP without divulging their risk behaviors, said Dr. Daskalakis, who was an infectious disease doctor in New York back in 2012 (and a member of the FDA committee) when the first pill for PrEP was approved. He said he’s seen this particularly with women who ask about it. Asking for PrEP ends up being an “ice breaker” to discussing the woman’s sexual and injection drug use history, which can then improve the kinds of tests and vaccinations clinicians suggest for her.
“So many women will open the door and say, ‘I want to do this,’ and not necessarily want to go into the details,” he said. “Now, will they go into the details later? Absolutely. That’s how you create trust and connection.”
A mandate and a guideline
Leisha McKinley-Beach, MPH, a member of the U.S. Women and PrEP Working Group, has been urging greater funding and mandates to expand PrEP to women since the first pill was approved. And still, Ms. McKinley-Beach said she recently met a woman who worked for a community group scheduling PrEP appointments for gay men. But the woman didn’t know that she, too, could take it.
The American Academy of Family Physicians recommends health care professionals offer PrEP to those who can benefit. The American College of Obstetricians and Gynecologists have a 2014 committee opinion stating that PrEP “may be a useful tool for women at highest risk of HIV acquisition.”
But the ACOG opinion is not a recommendation, stating that it “should not be construed as dictating an exclusive course of treatment or procedure to be followed.” Ms. McKinley-Beach said she hopes that the new CDC guidelines will prompt ACOG and other professional organizations to issue statements to include PrEP education in all health assessments. A spokesperson for ACOG said that the organization had not seen the new CDC guidelines and had no statement on them, but pointed out that the 2014 committee opinion is one of the “highest level of documents we produce.
“We have failed for nearly a decade to raise awareness that PrEP is also a prevention strategy for women,” Ms. McKinley-Beach said in an interview. “In many ways, we’re still back in 2012 as it relates to women.”
Dr. Hull reported having done previous research funded by Gilead Sciences and having received consulting fees from Gilead Sciences in 2018. Ms. McKinley-Beach reported receiving honoraria from ViiV Healthcare. Ms. Sherman and Dr. Daskalakis disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Starting Dec. 8, the Centers for Disease Control and Prevention recommends all clinicians talk to their sexually active adolescent and adult patients about HIV pre-exposure prophylaxis (PrEP) at least once and prescribe the prevention pills to anyone who asks for them, whether or not you understand their need for it.
“PrEP is a part of good primary care,” Demetre Daskalakis, MD, CDC’s director of the division of HIV/AIDS prevention, said in an interview. “Listening to people and what they need, as opposed to assessing what you think they need, is a seismic shift in how PrEP should be offered.”
The expanded recommendation comes as part of the 2021 update to the U.S. Public Health Service’s PrEP prescribing guidelines. It’s the third iteration since the Food and Drug Administration approved the first HIV prevention pill in 2012, and the first to include guidance on how to prescribe and monitor an injectable version of PrEP, which the FDA may approve as early as December 2021.
There are currently two pills, Truvada (emtricitabine/tenofovir disoproxil fumarate, Gilead Sciences and generic) and Descovy (emtricitabine/tenofovir alafenamide, Gilead Sciences). The pills have been found to be up to 99% effective in preventing HIV acquisition. The new injectable cabotegravir appears to be even more effective.
The broadened guidance is part of an effort from the country’s top health officials to expand PrEP prescribing from infectious disease specialists and sexual health clinics to health care professionals, including gynecologists, internal medicine physicians, and family practice clinicians. It appears to be necessary. In 2020, just 25% of the 1.2 million Americans who could benefit from PrEP were taking it, according to CDC data.
But those rates belie stark disparities in PrEP use by race and gender. The vast majority of those using PrEP are White Americans and men. About 66% of White Americans who could benefit from PrEP used it in 2020, and more than a quarter of the men who could benefit used it. By contrast, just 16% of Latinx people who could benefit had a prescription. And fewer than 1 in 10 Black Americans, who make up nearly half of those with indications for PrEP, had a prescription. The same was true for the women who could benefit.
Researchers and data from early PrEP demonstration projects have documented that clinicians are less likely to refer or prescribe the HIV prevention pills to Black people, especially the Black cisgender and transgender women and same-gender-loving men who bear the disproportionate burden of new cases in the United States, as well as fail to prescribe the medication to people who inject drugs.
Normalizing PrEP in primary care
When Courtney Sherman, DNP, APRN, first heard about PrEP in the early 2010s, she joked that her reaction was: “You’re ridiculous. You’re making that up. That’s not real.”
Ms. Sherman is now launching a tele-PrEP program from CAN Community Health, a nonprofit network of community health centers in southern Florida. The tele-PrEP program is meant to serve people in Florida and beyond, to increase access to the pill in areas with few health care professionals, or clinicians unwilling to prescribe it.
“When I go other places, I can’t do what I do for a living without getting some sort of bizarre comment or look,” she said. But the looks don’t just come from family, friends, or her children’s teachers. They come from colleagues, too. “What I’ve learned is that anybody – anybody – can be impacted [by HIV] and the illusion that ‘those people who live over there do things that me and my kind don’t do’ is just garbage.”
That’s the PrEP stigma that the universal PrEP counseling in the guidelines is meant to override, said Dr. Daskalakis. Going forward, he said that informing people about PrEP should be treated as normally as counseling people about smoking.
“You can change the blank: You talk to all adolescents and adults about not smoking,” he said. “This is: ‘Tell adolescents and adults about ways you can prevent HIV, and PrEP is one of them.’ ”
The guidelines also simplify for monitoring lab levels for the current daily pills, checking creatinine clearance levels twice a year in people older than age 50 and once a year in those younger than 50 taking the oral pills. Dr. Daskalakis said that should ease the burden of monitoring PrEP patients for health care professionals with busy caseloads.
It’s a move that drew praise from Shawnika Hull, PhD, assistant professor of health communications at Rutgers University, New Brunswick, N.J.. Dr. Hull’s recent data showed that clinicians who espoused more biased racial views were also less likely to prescribe PrEP to Black women who asked for it.
“Public health practitioners and scientists have been advocating for this as a strategy, as one way to address several ongoing barriers to PrEP specifically but also equity in PrEP,” said Dr. Hull. “This sort of universal provision of information is really an important strategy to try to undo some of the deeply intertwined barriers to uptake.”
‘Don’t grill them’
The updated guidelines keep the number and proportion of Americans who could benefit from PrEP the same: 1.2 million Americans, with nearly half of those Black. And the reasons people would qualify for PrEP remain the same: inconsistent condom use, sharing injection drug equipment, and a STI diagnosis in the last 6 months. There are also 57 jurisdictions, including seven rural states, where dating and having sex carries an increased risk of acquiring HIV because of high rates of untreated HIV in the community.
That’s why the other big change in the update is guidance to prescribe PrEP to whoever asks for it, whether the patient divulges their risk or not. Or as Dr. Daskalakis puts it: “If someone asks for PrEP, don’t grill them.”
There are lots of reasons that someone might ask for PrEP without divulging their risk behaviors, said Dr. Daskalakis, who was an infectious disease doctor in New York back in 2012 (and a member of the FDA committee) when the first pill for PrEP was approved. He said he’s seen this particularly with women who ask about it. Asking for PrEP ends up being an “ice breaker” to discussing the woman’s sexual and injection drug use history, which can then improve the kinds of tests and vaccinations clinicians suggest for her.
“So many women will open the door and say, ‘I want to do this,’ and not necessarily want to go into the details,” he said. “Now, will they go into the details later? Absolutely. That’s how you create trust and connection.”
A mandate and a guideline
Leisha McKinley-Beach, MPH, a member of the U.S. Women and PrEP Working Group, has been urging greater funding and mandates to expand PrEP to women since the first pill was approved. And still, Ms. McKinley-Beach said she recently met a woman who worked for a community group scheduling PrEP appointments for gay men. But the woman didn’t know that she, too, could take it.
The American Academy of Family Physicians recommends health care professionals offer PrEP to those who can benefit. The American College of Obstetricians and Gynecologists have a 2014 committee opinion stating that PrEP “may be a useful tool for women at highest risk of HIV acquisition.”
But the ACOG opinion is not a recommendation, stating that it “should not be construed as dictating an exclusive course of treatment or procedure to be followed.” Ms. McKinley-Beach said she hopes that the new CDC guidelines will prompt ACOG and other professional organizations to issue statements to include PrEP education in all health assessments. A spokesperson for ACOG said that the organization had not seen the new CDC guidelines and had no statement on them, but pointed out that the 2014 committee opinion is one of the “highest level of documents we produce.
“We have failed for nearly a decade to raise awareness that PrEP is also a prevention strategy for women,” Ms. McKinley-Beach said in an interview. “In many ways, we’re still back in 2012 as it relates to women.”
Dr. Hull reported having done previous research funded by Gilead Sciences and having received consulting fees from Gilead Sciences in 2018. Ms. McKinley-Beach reported receiving honoraria from ViiV Healthcare. Ms. Sherman and Dr. Daskalakis disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Starting Dec. 8, the Centers for Disease Control and Prevention recommends all clinicians talk to their sexually active adolescent and adult patients about HIV pre-exposure prophylaxis (PrEP) at least once and prescribe the prevention pills to anyone who asks for them, whether or not you understand their need for it.
“PrEP is a part of good primary care,” Demetre Daskalakis, MD, CDC’s director of the division of HIV/AIDS prevention, said in an interview. “Listening to people and what they need, as opposed to assessing what you think they need, is a seismic shift in how PrEP should be offered.”
The expanded recommendation comes as part of the 2021 update to the U.S. Public Health Service’s PrEP prescribing guidelines. It’s the third iteration since the Food and Drug Administration approved the first HIV prevention pill in 2012, and the first to include guidance on how to prescribe and monitor an injectable version of PrEP, which the FDA may approve as early as December 2021.
There are currently two pills, Truvada (emtricitabine/tenofovir disoproxil fumarate, Gilead Sciences and generic) and Descovy (emtricitabine/tenofovir alafenamide, Gilead Sciences). The pills have been found to be up to 99% effective in preventing HIV acquisition. The new injectable cabotegravir appears to be even more effective.
The broadened guidance is part of an effort from the country’s top health officials to expand PrEP prescribing from infectious disease specialists and sexual health clinics to health care professionals, including gynecologists, internal medicine physicians, and family practice clinicians. It appears to be necessary. In 2020, just 25% of the 1.2 million Americans who could benefit from PrEP were taking it, according to CDC data.
But those rates belie stark disparities in PrEP use by race and gender. The vast majority of those using PrEP are White Americans and men. About 66% of White Americans who could benefit from PrEP used it in 2020, and more than a quarter of the men who could benefit used it. By contrast, just 16% of Latinx people who could benefit had a prescription. And fewer than 1 in 10 Black Americans, who make up nearly half of those with indications for PrEP, had a prescription. The same was true for the women who could benefit.
Researchers and data from early PrEP demonstration projects have documented that clinicians are less likely to refer or prescribe the HIV prevention pills to Black people, especially the Black cisgender and transgender women and same-gender-loving men who bear the disproportionate burden of new cases in the United States, as well as fail to prescribe the medication to people who inject drugs.
Normalizing PrEP in primary care
When Courtney Sherman, DNP, APRN, first heard about PrEP in the early 2010s, she joked that her reaction was: “You’re ridiculous. You’re making that up. That’s not real.”
Ms. Sherman is now launching a tele-PrEP program from CAN Community Health, a nonprofit network of community health centers in southern Florida. The tele-PrEP program is meant to serve people in Florida and beyond, to increase access to the pill in areas with few health care professionals, or clinicians unwilling to prescribe it.
“When I go other places, I can’t do what I do for a living without getting some sort of bizarre comment or look,” she said. But the looks don’t just come from family, friends, or her children’s teachers. They come from colleagues, too. “What I’ve learned is that anybody – anybody – can be impacted [by HIV] and the illusion that ‘those people who live over there do things that me and my kind don’t do’ is just garbage.”
That’s the PrEP stigma that the universal PrEP counseling in the guidelines is meant to override, said Dr. Daskalakis. Going forward, he said that informing people about PrEP should be treated as normally as counseling people about smoking.
“You can change the blank: You talk to all adolescents and adults about not smoking,” he said. “This is: ‘Tell adolescents and adults about ways you can prevent HIV, and PrEP is one of them.’ ”
The guidelines also simplify for monitoring lab levels for the current daily pills, checking creatinine clearance levels twice a year in people older than age 50 and once a year in those younger than 50 taking the oral pills. Dr. Daskalakis said that should ease the burden of monitoring PrEP patients for health care professionals with busy caseloads.
It’s a move that drew praise from Shawnika Hull, PhD, assistant professor of health communications at Rutgers University, New Brunswick, N.J.. Dr. Hull’s recent data showed that clinicians who espoused more biased racial views were also less likely to prescribe PrEP to Black women who asked for it.
“Public health practitioners and scientists have been advocating for this as a strategy, as one way to address several ongoing barriers to PrEP specifically but also equity in PrEP,” said Dr. Hull. “This sort of universal provision of information is really an important strategy to try to undo some of the deeply intertwined barriers to uptake.”
‘Don’t grill them’
The updated guidelines keep the number and proportion of Americans who could benefit from PrEP the same: 1.2 million Americans, with nearly half of those Black. And the reasons people would qualify for PrEP remain the same: inconsistent condom use, sharing injection drug equipment, and a STI diagnosis in the last 6 months. There are also 57 jurisdictions, including seven rural states, where dating and having sex carries an increased risk of acquiring HIV because of high rates of untreated HIV in the community.
That’s why the other big change in the update is guidance to prescribe PrEP to whoever asks for it, whether the patient divulges their risk or not. Or as Dr. Daskalakis puts it: “If someone asks for PrEP, don’t grill them.”
There are lots of reasons that someone might ask for PrEP without divulging their risk behaviors, said Dr. Daskalakis, who was an infectious disease doctor in New York back in 2012 (and a member of the FDA committee) when the first pill for PrEP was approved. He said he’s seen this particularly with women who ask about it. Asking for PrEP ends up being an “ice breaker” to discussing the woman’s sexual and injection drug use history, which can then improve the kinds of tests and vaccinations clinicians suggest for her.
“So many women will open the door and say, ‘I want to do this,’ and not necessarily want to go into the details,” he said. “Now, will they go into the details later? Absolutely. That’s how you create trust and connection.”
A mandate and a guideline
Leisha McKinley-Beach, MPH, a member of the U.S. Women and PrEP Working Group, has been urging greater funding and mandates to expand PrEP to women since the first pill was approved. And still, Ms. McKinley-Beach said she recently met a woman who worked for a community group scheduling PrEP appointments for gay men. But the woman didn’t know that she, too, could take it.
The American Academy of Family Physicians recommends health care professionals offer PrEP to those who can benefit. The American College of Obstetricians and Gynecologists have a 2014 committee opinion stating that PrEP “may be a useful tool for women at highest risk of HIV acquisition.”
But the ACOG opinion is not a recommendation, stating that it “should not be construed as dictating an exclusive course of treatment or procedure to be followed.” Ms. McKinley-Beach said she hopes that the new CDC guidelines will prompt ACOG and other professional organizations to issue statements to include PrEP education in all health assessments. A spokesperson for ACOG said that the organization had not seen the new CDC guidelines and had no statement on them, but pointed out that the 2014 committee opinion is one of the “highest level of documents we produce.
“We have failed for nearly a decade to raise awareness that PrEP is also a prevention strategy for women,” Ms. McKinley-Beach said in an interview. “In many ways, we’re still back in 2012 as it relates to women.”
Dr. Hull reported having done previous research funded by Gilead Sciences and having received consulting fees from Gilead Sciences in 2018. Ms. McKinley-Beach reported receiving honoraria from ViiV Healthcare. Ms. Sherman and Dr. Daskalakis disclosed no relevant financial relationships.
A version of this article first appeared on Medscape.com.
Record-breaking autism rates reported with new CDC criteria
Childhood autism rates are at the highest level since the Centers for Disease Control and Prevention began tracking the disorder in 2000, new data released Dec. 2 show.
The increase likely reflects improvements in diagnosis and identification of autism spectrum disorder (ASD), not an increase in incidence, study authors with the CDC’s Autism and Developmental Disabilities Monitoring (ADDM) Network told this news organization.
Using a new surveillance methodology, researchers found that 2.3% of 8-year-olds in communities in 11 states across the United States had an autism diagnosis in 2018, up from 1.9% in 2016.
A separate report on early identification in 4-year-olds shows that children born in 2014 were 50% more likely to receive an autism diagnosis or ASD special education classification by 48 months of age than those born in 2010, signaling improved early diagnosis.
Taken together, the data suggest efforts to raise awareness about autism are working, though researchers were quick to say much work remains.
“It was not surprising to me and in fact it was reassuring that the number of children diagnosed with autism is higher and is actually approaching prevalence of autism that has been noted in some national surveys of parents,” Stuart Shapira, MD, PhD, associate director for science in CDC’s National Center on Birth Defects and Developmental Disability, told this news organization.
“It means we’re doing a better job of identifying children, which helps to get them into services earlier so they can achieve their best developmental outcome.”
The studies, published online in Morbidity and Mortality Weekly Report, are the first to use a new ASD surveillance protocol that relies on ASD diagnosis or special education classification and billing codes and eliminates comprehensive records analysis by trained clinician reviewers.
Racial disparities
The updated methodology was less labor intensive and reduced the time it took to produce the report, but it is not without its critics, who claim the new protocol will undercount the number of children with ASD.
Created in 2000 and funded by the CDC, the ADDM Network is the only surveillance program in the United States that tracks the number and characteristics of children with ASD in multiple communities in the U.S.
When ADDM released its first report in 2007 from six states and based on data from the year 2000, ASD prevalence was 6.7 per 1,000 children, or 1 in 150 children.
In the latest report, which includes data from 2018, the autism prevalence rate across 11 states was 23.0 per 1,000 children, or 1 in 44 children.
That rate is closer to reported autism prevalence from the National Survey of Children’s Health and the National Health Interview Survey, both of which rely on parent-reported ASD diagnoses.
in Arizona, Arkansas, California, Georgia, Maryland, Minnesota, Missouri, New Jersey, Tennessee, Utah, and Wisconsin.
Children were counted as having autism if their records included an ASD diagnosis, a special education classification of ASD, or an ASD International Classification of Diseases (ICD) code. A total of 5,058 children met those criteria.
Rates of ASD ranged from a low of 1.7% in Missouri to 3.9% in California and were 4.2 times higher in boys than in girls. Just under half of the children with ASD were evaluated by age 36 months.
Although the overall ASD prevalence was similar among White, Black, Hispanic, and Asian/Pacific Islander children, the report highlighted a number of other racial disparities overall and in individual states.
For example, among those with ASD and data on cognitive ability, 35.2% had an intelligence quotient score of 70 or lower. Black children with ASD were far more likely to have an IQ of 70 or less (49.8%) than Hispanic (33.1%) or White (29.7%) children.
“The persistent disparities in co-occurring intellectual disabilities in children with autism is something that we continue to see and suggests that we need to better understand exactly what’s happening,” Matthew Maenner, PhD, an epidemiologist and autism surveillance team lead with the CDC’s National Center on Birth Defects and Developmental Disabilities, told this news organization.
Another long-standing trend observed again in the new report on prevalence among 8-year-olds is low ASD prevalence among Hispanic children. While the overall estimate showed similar autism rates, a closer review of state-level data reveals a different picture.
“In almost half of the sites, Hispanic children were less likely to be identified as having ASD,” he said. “This gets lost if you look only at the overall estimate.”
New methodology
When ADDM released its first report in 2007, autism diagnosis was widely inadequate in the United States. Relying on only confirmed ASD diagnoses would significantly underestimate the number of children with the disorder, so the CDC added “active case finding” to the protocol.
Trained clinician reviewers analyzed individual notes from medical and educational records for every 8-year-old in ADDM Network sites, looking for evidence of characteristics and behaviors associated with autism. The process was labor- and time-intensive and took up to 4 years to complete.
In 2018, the CDC began investigating ways to speed the process and came up with the strategy used in the latest report. The new protocol was faster, easier, and less expensive. Although he says cost was never the deciding factor, Dr. Maenner acknowledges that had they stuck with the original protocol, they would have been forced to reduce the number of ADDM Network sites.
Dr. Maenner argues that a comparison of the two protocols shows the new method doesn’t compromise accuracy and may actually capture children who lacked the medical or educational records the previous protocol required for a count. But not everyone agrees.
“I thought the point was to be as accurate and complete as possible in doing the surveillance,” Walter Zahorodny, PhD, associate professor of pediatrics at Rutgers University, New Brunswick, N.J., and principal investigator of the New Jersey ADDM Network site, told this news organization. “In states where there’s a high detail of information in records, like New Jersey, it’s going to underestimate the count.”
Dr. Zahorodny says the latest data prove his point. In 2016, under the old methodology, ASD prevalence was 3.1% in the state. In 2018, under the new protocol, prevalence was 2.84%, a decrease of about 20% that Dr. Zahorodny pins squarely on the elimination of ADDM clinical reviewers.
But New Jersey is the only state that participated in both the 2016 and 2018 surveillance periods to report a decrease in ASD prevalence. The other eight states all found autism rates in their states went up.
Sydney Pettygrove, PhD, associate professor of public health and pediatrics at the University of Arizona, Tucson, and a principal investigator for the ADDM site in Arizona, told this news organization that when she first learned the CDC was rolling out a new methodology, she and other investigators were concerned.
“People were really upset. I was really upset,” she said. “I had formed an opinion based on the earlier data that this would not be a good idea.”
In 2000, when ASD surveillance began in Arizona, nearly 30% of children identified by ADDM clinical reviewers as having autism had no mention of the disorder in their records. Today, that percentage is closer to 5%.
“In 2000 it would have been catastrophic to try to estimate the prevalence of autism with the new protocol,” said Dr. Pettygrove. As it turns out, under the new protocol, prevalence rates in Arizona increased from 16.0 per 1,000 children in 2016 to 24.9 in 2018.
Built-in bias eliminated?
In addition to speeding up the process, the new methodology might have other benefits as well. Under the old ADDM surveillance protocol, children who lacked certain medical or educational records did not meet the ASD case definition and weren’t counted.
A 2019 study showed that this disproportionately affected Black and Hispanic children, who had significantly less access to health care professionals than White children.
As a result, “the old methodology had a bias built into it,” Maureen Durkin, PhD, DrPH, coauthor of that study and chair of population health sciences at the University of Wisconsin–Madison and principal investigator for the ADDM site in Wisconsin, told this news organization.
“Clinician reviewers ended up putting these children in the ‘suspected ASD’ category because they couldn’t call it a case under the case definition,” Dr. Durkin said. “There was a fairly large percentage of suspected cases and a disproportionate number of those kids were children of color.”
Although she can’t say for sure, Dr. Durkin said it’s possible the new protocol could eliminate some of that bias.
CDC researchers also attribute the new method to an expanded study of early diagnosis among 4-year-olds. In previous years, only a handful of the ADDM Network sites participating in the 8-year-old surveillance project also studied early diagnosis in 4-year-olds.
This year, all 11 sites took part in the early diagnosis analysis, tripling the number of children included in the analysis. That made it possible to include, for the first time, Asian/Pacific Islander children in this analysis.
In the past, ASD prevalence has trended higher in White children, compared with other racial groups. The new data found that ASD prevalence among 4-year-olds was significantly lower in White children (12.9 per 1,000 children) than in Black, Hispanic, or Asian/Pacific Islander children (16.6, 21.1, and 22.7 per 1,000, respectively). Prevalence in American Indian/Alaska Native children was the lowest among all racial groups (11.5 per 1,000).
It’s the first time researchers have seen this pattern in any ADDM report, Kelly Shaw, PhD, lead author of that study and an epidemiologist with the National Center on Birth Defects and Developmental Disability at the CDC, told this news organization.
These data don’t provide clues about the potential cause of that disparity, Dr. Shaw said. It’s likely an indication of better identification of ASD in those communities, she said, and not a sign of increased incidence of autism among Black, Hispanic, or Asian/Pacific Islander children.
“We don’t have any evidence to suggest or expect that autism would be increasing differentially among groups,” Dr. Shaw said.
The data suggest “we are making some progress but there certainly is still room for improvement,” Dr. Shaw said.
Study authors report no conflicts of interest.
A version of this article first appeared on Medscape.com.
Childhood autism rates are at the highest level since the Centers for Disease Control and Prevention began tracking the disorder in 2000, new data released Dec. 2 show.
The increase likely reflects improvements in diagnosis and identification of autism spectrum disorder (ASD), not an increase in incidence, study authors with the CDC’s Autism and Developmental Disabilities Monitoring (ADDM) Network told this news organization.
Using a new surveillance methodology, researchers found that 2.3% of 8-year-olds in communities in 11 states across the United States had an autism diagnosis in 2018, up from 1.9% in 2016.
A separate report on early identification in 4-year-olds shows that children born in 2014 were 50% more likely to receive an autism diagnosis or ASD special education classification by 48 months of age than those born in 2010, signaling improved early diagnosis.
Taken together, the data suggest efforts to raise awareness about autism are working, though researchers were quick to say much work remains.
“It was not surprising to me and in fact it was reassuring that the number of children diagnosed with autism is higher and is actually approaching prevalence of autism that has been noted in some national surveys of parents,” Stuart Shapira, MD, PhD, associate director for science in CDC’s National Center on Birth Defects and Developmental Disability, told this news organization.
“It means we’re doing a better job of identifying children, which helps to get them into services earlier so they can achieve their best developmental outcome.”
The studies, published online in Morbidity and Mortality Weekly Report, are the first to use a new ASD surveillance protocol that relies on ASD diagnosis or special education classification and billing codes and eliminates comprehensive records analysis by trained clinician reviewers.
Racial disparities
The updated methodology was less labor intensive and reduced the time it took to produce the report, but it is not without its critics, who claim the new protocol will undercount the number of children with ASD.
Created in 2000 and funded by the CDC, the ADDM Network is the only surveillance program in the United States that tracks the number and characteristics of children with ASD in multiple communities in the U.S.
When ADDM released its first report in 2007 from six states and based on data from the year 2000, ASD prevalence was 6.7 per 1,000 children, or 1 in 150 children.
In the latest report, which includes data from 2018, the autism prevalence rate across 11 states was 23.0 per 1,000 children, or 1 in 44 children.
That rate is closer to reported autism prevalence from the National Survey of Children’s Health and the National Health Interview Survey, both of which rely on parent-reported ASD diagnoses.
in Arizona, Arkansas, California, Georgia, Maryland, Minnesota, Missouri, New Jersey, Tennessee, Utah, and Wisconsin.
Children were counted as having autism if their records included an ASD diagnosis, a special education classification of ASD, or an ASD International Classification of Diseases (ICD) code. A total of 5,058 children met those criteria.
Rates of ASD ranged from a low of 1.7% in Missouri to 3.9% in California and were 4.2 times higher in boys than in girls. Just under half of the children with ASD were evaluated by age 36 months.
Although the overall ASD prevalence was similar among White, Black, Hispanic, and Asian/Pacific Islander children, the report highlighted a number of other racial disparities overall and in individual states.
For example, among those with ASD and data on cognitive ability, 35.2% had an intelligence quotient score of 70 or lower. Black children with ASD were far more likely to have an IQ of 70 or less (49.8%) than Hispanic (33.1%) or White (29.7%) children.
“The persistent disparities in co-occurring intellectual disabilities in children with autism is something that we continue to see and suggests that we need to better understand exactly what’s happening,” Matthew Maenner, PhD, an epidemiologist and autism surveillance team lead with the CDC’s National Center on Birth Defects and Developmental Disabilities, told this news organization.
Another long-standing trend observed again in the new report on prevalence among 8-year-olds is low ASD prevalence among Hispanic children. While the overall estimate showed similar autism rates, a closer review of state-level data reveals a different picture.
“In almost half of the sites, Hispanic children were less likely to be identified as having ASD,” he said. “This gets lost if you look only at the overall estimate.”
New methodology
When ADDM released its first report in 2007, autism diagnosis was widely inadequate in the United States. Relying on only confirmed ASD diagnoses would significantly underestimate the number of children with the disorder, so the CDC added “active case finding” to the protocol.
Trained clinician reviewers analyzed individual notes from medical and educational records for every 8-year-old in ADDM Network sites, looking for evidence of characteristics and behaviors associated with autism. The process was labor- and time-intensive and took up to 4 years to complete.
In 2018, the CDC began investigating ways to speed the process and came up with the strategy used in the latest report. The new protocol was faster, easier, and less expensive. Although he says cost was never the deciding factor, Dr. Maenner acknowledges that had they stuck with the original protocol, they would have been forced to reduce the number of ADDM Network sites.
Dr. Maenner argues that a comparison of the two protocols shows the new method doesn’t compromise accuracy and may actually capture children who lacked the medical or educational records the previous protocol required for a count. But not everyone agrees.
“I thought the point was to be as accurate and complete as possible in doing the surveillance,” Walter Zahorodny, PhD, associate professor of pediatrics at Rutgers University, New Brunswick, N.J., and principal investigator of the New Jersey ADDM Network site, told this news organization. “In states where there’s a high detail of information in records, like New Jersey, it’s going to underestimate the count.”
Dr. Zahorodny says the latest data prove his point. In 2016, under the old methodology, ASD prevalence was 3.1% in the state. In 2018, under the new protocol, prevalence was 2.84%, a decrease of about 20% that Dr. Zahorodny pins squarely on the elimination of ADDM clinical reviewers.
But New Jersey is the only state that participated in both the 2016 and 2018 surveillance periods to report a decrease in ASD prevalence. The other eight states all found autism rates in their states went up.
Sydney Pettygrove, PhD, associate professor of public health and pediatrics at the University of Arizona, Tucson, and a principal investigator for the ADDM site in Arizona, told this news organization that when she first learned the CDC was rolling out a new methodology, she and other investigators were concerned.
“People were really upset. I was really upset,” she said. “I had formed an opinion based on the earlier data that this would not be a good idea.”
In 2000, when ASD surveillance began in Arizona, nearly 30% of children identified by ADDM clinical reviewers as having autism had no mention of the disorder in their records. Today, that percentage is closer to 5%.
“In 2000 it would have been catastrophic to try to estimate the prevalence of autism with the new protocol,” said Dr. Pettygrove. As it turns out, under the new protocol, prevalence rates in Arizona increased from 16.0 per 1,000 children in 2016 to 24.9 in 2018.
Built-in bias eliminated?
In addition to speeding up the process, the new methodology might have other benefits as well. Under the old ADDM surveillance protocol, children who lacked certain medical or educational records did not meet the ASD case definition and weren’t counted.
A 2019 study showed that this disproportionately affected Black and Hispanic children, who had significantly less access to health care professionals than White children.
As a result, “the old methodology had a bias built into it,” Maureen Durkin, PhD, DrPH, coauthor of that study and chair of population health sciences at the University of Wisconsin–Madison and principal investigator for the ADDM site in Wisconsin, told this news organization.
“Clinician reviewers ended up putting these children in the ‘suspected ASD’ category because they couldn’t call it a case under the case definition,” Dr. Durkin said. “There was a fairly large percentage of suspected cases and a disproportionate number of those kids were children of color.”
Although she can’t say for sure, Dr. Durkin said it’s possible the new protocol could eliminate some of that bias.
CDC researchers also attribute the new method to an expanded study of early diagnosis among 4-year-olds. In previous years, only a handful of the ADDM Network sites participating in the 8-year-old surveillance project also studied early diagnosis in 4-year-olds.
This year, all 11 sites took part in the early diagnosis analysis, tripling the number of children included in the analysis. That made it possible to include, for the first time, Asian/Pacific Islander children in this analysis.
In the past, ASD prevalence has trended higher in White children, compared with other racial groups. The new data found that ASD prevalence among 4-year-olds was significantly lower in White children (12.9 per 1,000 children) than in Black, Hispanic, or Asian/Pacific Islander children (16.6, 21.1, and 22.7 per 1,000, respectively). Prevalence in American Indian/Alaska Native children was the lowest among all racial groups (11.5 per 1,000).
It’s the first time researchers have seen this pattern in any ADDM report, Kelly Shaw, PhD, lead author of that study and an epidemiologist with the National Center on Birth Defects and Developmental Disability at the CDC, told this news organization.
These data don’t provide clues about the potential cause of that disparity, Dr. Shaw said. It’s likely an indication of better identification of ASD in those communities, she said, and not a sign of increased incidence of autism among Black, Hispanic, or Asian/Pacific Islander children.
“We don’t have any evidence to suggest or expect that autism would be increasing differentially among groups,” Dr. Shaw said.
The data suggest “we are making some progress but there certainly is still room for improvement,” Dr. Shaw said.
Study authors report no conflicts of interest.
A version of this article first appeared on Medscape.com.
Childhood autism rates are at the highest level since the Centers for Disease Control and Prevention began tracking the disorder in 2000, new data released Dec. 2 show.
The increase likely reflects improvements in diagnosis and identification of autism spectrum disorder (ASD), not an increase in incidence, study authors with the CDC’s Autism and Developmental Disabilities Monitoring (ADDM) Network told this news organization.
Using a new surveillance methodology, researchers found that 2.3% of 8-year-olds in communities in 11 states across the United States had an autism diagnosis in 2018, up from 1.9% in 2016.
A separate report on early identification in 4-year-olds shows that children born in 2014 were 50% more likely to receive an autism diagnosis or ASD special education classification by 48 months of age than those born in 2010, signaling improved early diagnosis.
Taken together, the data suggest efforts to raise awareness about autism are working, though researchers were quick to say much work remains.
“It was not surprising to me and in fact it was reassuring that the number of children diagnosed with autism is higher and is actually approaching prevalence of autism that has been noted in some national surveys of parents,” Stuart Shapira, MD, PhD, associate director for science in CDC’s National Center on Birth Defects and Developmental Disability, told this news organization.
“It means we’re doing a better job of identifying children, which helps to get them into services earlier so they can achieve their best developmental outcome.”
The studies, published online in Morbidity and Mortality Weekly Report, are the first to use a new ASD surveillance protocol that relies on ASD diagnosis or special education classification and billing codes and eliminates comprehensive records analysis by trained clinician reviewers.
Racial disparities
The updated methodology was less labor intensive and reduced the time it took to produce the report, but it is not without its critics, who claim the new protocol will undercount the number of children with ASD.
Created in 2000 and funded by the CDC, the ADDM Network is the only surveillance program in the United States that tracks the number and characteristics of children with ASD in multiple communities in the U.S.
When ADDM released its first report in 2007 from six states and based on data from the year 2000, ASD prevalence was 6.7 per 1,000 children, or 1 in 150 children.
In the latest report, which includes data from 2018, the autism prevalence rate across 11 states was 23.0 per 1,000 children, or 1 in 44 children.
That rate is closer to reported autism prevalence from the National Survey of Children’s Health and the National Health Interview Survey, both of which rely on parent-reported ASD diagnoses.
in Arizona, Arkansas, California, Georgia, Maryland, Minnesota, Missouri, New Jersey, Tennessee, Utah, and Wisconsin.
Children were counted as having autism if their records included an ASD diagnosis, a special education classification of ASD, or an ASD International Classification of Diseases (ICD) code. A total of 5,058 children met those criteria.
Rates of ASD ranged from a low of 1.7% in Missouri to 3.9% in California and were 4.2 times higher in boys than in girls. Just under half of the children with ASD were evaluated by age 36 months.
Although the overall ASD prevalence was similar among White, Black, Hispanic, and Asian/Pacific Islander children, the report highlighted a number of other racial disparities overall and in individual states.
For example, among those with ASD and data on cognitive ability, 35.2% had an intelligence quotient score of 70 or lower. Black children with ASD were far more likely to have an IQ of 70 or less (49.8%) than Hispanic (33.1%) or White (29.7%) children.
“The persistent disparities in co-occurring intellectual disabilities in children with autism is something that we continue to see and suggests that we need to better understand exactly what’s happening,” Matthew Maenner, PhD, an epidemiologist and autism surveillance team lead with the CDC’s National Center on Birth Defects and Developmental Disabilities, told this news organization.
Another long-standing trend observed again in the new report on prevalence among 8-year-olds is low ASD prevalence among Hispanic children. While the overall estimate showed similar autism rates, a closer review of state-level data reveals a different picture.
“In almost half of the sites, Hispanic children were less likely to be identified as having ASD,” he said. “This gets lost if you look only at the overall estimate.”
New methodology
When ADDM released its first report in 2007, autism diagnosis was widely inadequate in the United States. Relying on only confirmed ASD diagnoses would significantly underestimate the number of children with the disorder, so the CDC added “active case finding” to the protocol.
Trained clinician reviewers analyzed individual notes from medical and educational records for every 8-year-old in ADDM Network sites, looking for evidence of characteristics and behaviors associated with autism. The process was labor- and time-intensive and took up to 4 years to complete.
In 2018, the CDC began investigating ways to speed the process and came up with the strategy used in the latest report. The new protocol was faster, easier, and less expensive. Although he says cost was never the deciding factor, Dr. Maenner acknowledges that had they stuck with the original protocol, they would have been forced to reduce the number of ADDM Network sites.
Dr. Maenner argues that a comparison of the two protocols shows the new method doesn’t compromise accuracy and may actually capture children who lacked the medical or educational records the previous protocol required for a count. But not everyone agrees.
“I thought the point was to be as accurate and complete as possible in doing the surveillance,” Walter Zahorodny, PhD, associate professor of pediatrics at Rutgers University, New Brunswick, N.J., and principal investigator of the New Jersey ADDM Network site, told this news organization. “In states where there’s a high detail of information in records, like New Jersey, it’s going to underestimate the count.”
Dr. Zahorodny says the latest data prove his point. In 2016, under the old methodology, ASD prevalence was 3.1% in the state. In 2018, under the new protocol, prevalence was 2.84%, a decrease of about 20% that Dr. Zahorodny pins squarely on the elimination of ADDM clinical reviewers.
But New Jersey is the only state that participated in both the 2016 and 2018 surveillance periods to report a decrease in ASD prevalence. The other eight states all found autism rates in their states went up.
Sydney Pettygrove, PhD, associate professor of public health and pediatrics at the University of Arizona, Tucson, and a principal investigator for the ADDM site in Arizona, told this news organization that when she first learned the CDC was rolling out a new methodology, she and other investigators were concerned.
“People were really upset. I was really upset,” she said. “I had formed an opinion based on the earlier data that this would not be a good idea.”
In 2000, when ASD surveillance began in Arizona, nearly 30% of children identified by ADDM clinical reviewers as having autism had no mention of the disorder in their records. Today, that percentage is closer to 5%.
“In 2000 it would have been catastrophic to try to estimate the prevalence of autism with the new protocol,” said Dr. Pettygrove. As it turns out, under the new protocol, prevalence rates in Arizona increased from 16.0 per 1,000 children in 2016 to 24.9 in 2018.
Built-in bias eliminated?
In addition to speeding up the process, the new methodology might have other benefits as well. Under the old ADDM surveillance protocol, children who lacked certain medical or educational records did not meet the ASD case definition and weren’t counted.
A 2019 study showed that this disproportionately affected Black and Hispanic children, who had significantly less access to health care professionals than White children.
As a result, “the old methodology had a bias built into it,” Maureen Durkin, PhD, DrPH, coauthor of that study and chair of population health sciences at the University of Wisconsin–Madison and principal investigator for the ADDM site in Wisconsin, told this news organization.
“Clinician reviewers ended up putting these children in the ‘suspected ASD’ category because they couldn’t call it a case under the case definition,” Dr. Durkin said. “There was a fairly large percentage of suspected cases and a disproportionate number of those kids were children of color.”
Although she can’t say for sure, Dr. Durkin said it’s possible the new protocol could eliminate some of that bias.
CDC researchers also attribute the new method to an expanded study of early diagnosis among 4-year-olds. In previous years, only a handful of the ADDM Network sites participating in the 8-year-old surveillance project also studied early diagnosis in 4-year-olds.
This year, all 11 sites took part in the early diagnosis analysis, tripling the number of children included in the analysis. That made it possible to include, for the first time, Asian/Pacific Islander children in this analysis.
In the past, ASD prevalence has trended higher in White children, compared with other racial groups. The new data found that ASD prevalence among 4-year-olds was significantly lower in White children (12.9 per 1,000 children) than in Black, Hispanic, or Asian/Pacific Islander children (16.6, 21.1, and 22.7 per 1,000, respectively). Prevalence in American Indian/Alaska Native children was the lowest among all racial groups (11.5 per 1,000).
It’s the first time researchers have seen this pattern in any ADDM report, Kelly Shaw, PhD, lead author of that study and an epidemiologist with the National Center on Birth Defects and Developmental Disability at the CDC, told this news organization.
These data don’t provide clues about the potential cause of that disparity, Dr. Shaw said. It’s likely an indication of better identification of ASD in those communities, she said, and not a sign of increased incidence of autism among Black, Hispanic, or Asian/Pacific Islander children.
“We don’t have any evidence to suggest or expect that autism would be increasing differentially among groups,” Dr. Shaw said.
The data suggest “we are making some progress but there certainly is still room for improvement,” Dr. Shaw said.
Study authors report no conflicts of interest.
A version of this article first appeared on Medscape.com.
FDA approves time-saving combo for r/r multiple myeloma
The U.S. Food and Drug Administration who have had one to three prior lines of therapy.
Using the newly approved combination in this setting is a time-saver for patients and clinics, observed an investigator.
“The approval of subcutaneous daratumumab in combination with Kd will help clinicians address unmet patient needs by reducing the administration time from hours to just minutes and reducing the frequency of infusion-related reactions, as compared to the intravenous daratumumab formulation in combination with Kd,” said Ajai Chari, MD, of Mount Sinai Cancer Clinical Trials Office in New York City in a Janssen press statement.
Efficacy data for the new approval come from a single-arm cohort of PLEIADES, a multicohort, open-label trial. The cohort included 66 patients with relapsed or refractory multiple myeloma who had received one or more prior lines of therapy. Patients received daratumumab + hyaluronidase-fihj subcutaneously in combination with carfilzomib and dexamethasone.
The main efficacy outcome measure was overall response rate, which was 84.8%. At a median follow-up of 9.2 months, the median duration of response had not been reached.
The response rate with the new combination, which features a subcutaneous injection, was akin to those with the older combination, which features the more time-consuming IV administration and was FDA approved, according to the company press release.
The most common adverse reactions (≥20%) occurring in patients treated with Darzalex Faspro, Kyprolis, and dexamethasone were upper respiratory tract infections, fatigue, insomnia, hypertension, diarrhea, cough, dyspnea, headache, pyrexia, nausea, and edema peripheral.
A version of this article first appeared on Medscape.com .
The U.S. Food and Drug Administration who have had one to three prior lines of therapy.
Using the newly approved combination in this setting is a time-saver for patients and clinics, observed an investigator.
“The approval of subcutaneous daratumumab in combination with Kd will help clinicians address unmet patient needs by reducing the administration time from hours to just minutes and reducing the frequency of infusion-related reactions, as compared to the intravenous daratumumab formulation in combination with Kd,” said Ajai Chari, MD, of Mount Sinai Cancer Clinical Trials Office in New York City in a Janssen press statement.
Efficacy data for the new approval come from a single-arm cohort of PLEIADES, a multicohort, open-label trial. The cohort included 66 patients with relapsed or refractory multiple myeloma who had received one or more prior lines of therapy. Patients received daratumumab + hyaluronidase-fihj subcutaneously in combination with carfilzomib and dexamethasone.
The main efficacy outcome measure was overall response rate, which was 84.8%. At a median follow-up of 9.2 months, the median duration of response had not been reached.
The response rate with the new combination, which features a subcutaneous injection, was akin to those with the older combination, which features the more time-consuming IV administration and was FDA approved, according to the company press release.
The most common adverse reactions (≥20%) occurring in patients treated with Darzalex Faspro, Kyprolis, and dexamethasone were upper respiratory tract infections, fatigue, insomnia, hypertension, diarrhea, cough, dyspnea, headache, pyrexia, nausea, and edema peripheral.
A version of this article first appeared on Medscape.com .
The U.S. Food and Drug Administration who have had one to three prior lines of therapy.
Using the newly approved combination in this setting is a time-saver for patients and clinics, observed an investigator.
“The approval of subcutaneous daratumumab in combination with Kd will help clinicians address unmet patient needs by reducing the administration time from hours to just minutes and reducing the frequency of infusion-related reactions, as compared to the intravenous daratumumab formulation in combination with Kd,” said Ajai Chari, MD, of Mount Sinai Cancer Clinical Trials Office in New York City in a Janssen press statement.
Efficacy data for the new approval come from a single-arm cohort of PLEIADES, a multicohort, open-label trial. The cohort included 66 patients with relapsed or refractory multiple myeloma who had received one or more prior lines of therapy. Patients received daratumumab + hyaluronidase-fihj subcutaneously in combination with carfilzomib and dexamethasone.
The main efficacy outcome measure was overall response rate, which was 84.8%. At a median follow-up of 9.2 months, the median duration of response had not been reached.
The response rate with the new combination, which features a subcutaneous injection, was akin to those with the older combination, which features the more time-consuming IV administration and was FDA approved, according to the company press release.
The most common adverse reactions (≥20%) occurring in patients treated with Darzalex Faspro, Kyprolis, and dexamethasone were upper respiratory tract infections, fatigue, insomnia, hypertension, diarrhea, cough, dyspnea, headache, pyrexia, nausea, and edema peripheral.
A version of this article first appeared on Medscape.com .
FDA panel backs first pill for COVID-19 by a small margin
, according to a panel of experts that advises the Food and Drug Administration on its regulatory decisions for these types of drugs.
The FDA’s Antimicrobial Drugs Advisory Committee narrowly voted to authorize the drug molnupiravir, voting 13 to 10 to support emergency use, which requires a medication to meet a lower standard of evidence than does full approval.
The FDA is not bound by the committee’s vote but typically follows its advice.
If authorized by the agency, molnupiravir would be the first antiviral agent available as a pill to treat COVID-19. Other therapies to treat the infection are available — monoclonal antibodies and the drug remdesivir — but they are given by infusion.
The United Kingdom has already authorized the use of Merck’s drug.
“This was clearly a difficult decision,” said committee member Michael Green, MD, a pediatric infectious disease expert at the University of Pittsburg School of Medicine.
Green said he voted yes, and that the drug’s ability to prevent deaths in the study weighed heavily on his decision. He said given uncertainties around the drug both the company and FDA should keep a close eye on patients taking the drug going forward.
“Should an alternative oral agent become available that had a better safety profile and equal or better efficacy profile, the agency might reconsider its authorization,” he said.
Others didn’t agree that the drug should be allowed onto the market.
“I voted no,” said Jennifer Le, PharmD, a professor of clinical pharmacy at the University of California. Dr. Le said the modest benefit of the medication didn’t outweigh all the potential safety issues. “I think I just need more efficacy and safety data,” she said.
Initial results from the first half of people enrolled in the clinical trial found the pill cut the risk of hospitalization or death by 50% in patients at higher risk of severe outcomes from COVID-19.
But later results, released just days before the meeting, showed that the drug’s effectiveness had dropped to about 30%.
In the updated analysis, 48 patients out of the 709 who were taking the drug were hospitalized or died within 29 days compared to 68 out of 699 who randomly got the placebo. There was one death in the group that got molnupiravir compared to nine in the placebo group. Nearly all those deaths occurred during the first phase of the study.
On Nov. 30 Merck explained that the drug’s efficacy appeared to fall, in part, because the placebo group had experienced fewer hospitalizations and deaths than expected during the second half of the study, making the drug look less beneficial by comparison.
The company said it wasn’t sure why patients in the placebo group had fared so much better in later trial enrollments.
“The efficacy of this product is not overwhelmingly good,” said committee member David Hardy, MD, an infectious disease expert at Charles Drew University School of Medicine in Los Angeles. “And I think that makes all of us a little uncomfortable about whether this is an advanced therapeutic because it’s an oral medication rather than an intravenous medication,” he said during the panel’s deliberations.
“I think we have to be very careful about how we’re going to allow people to use this,” Dr. Hardy said.
Many who voted for authorization thought use of the drug should be restricted to unvaccinated people who were at high risk of severe COVID-19 outcomes, the same population enrolled in the clinical trial. People in the trial were considered at higher risk if they were over age 60, had cancer, chronic kidney disease, chronic obstructive pulmonary disease, were obese, or had heart disease or diabetes.
There are some significant limitations of the study that may affect how the drug is used. Vaccinated people couldn’t enroll in the study, so it’s not known if the medication would have any benefit for them. Nearly two-thirds of the U.S. population is fully vaccinated. The study found no additional benefit of the medication compared to the placebo in people who had detectable antibodies, presumably from a prior infection.
Animal studies found that the drug — which kills the virus by forcing it to make errors as it copies its genetic material inside cells — could disrupt bone formation. For that reason, the manufacturer and the FDA agreed that it should not be used in anyone younger than age 18.
Animal studies also indicated that the drug could cause birth defects. For that reason, the company said the drug shouldn’t be given to women who are pregnant or breastfeeding and said doctors should make sure women of childbearing age aren’t pregnant before taking the medication.
Some members of the panel felt that pregnant women and their doctors should be given the choice of whether or not to use the drug, given that pregnant women are at high risk for severe COVID-19 outcomes and infused therapies may not be available in all settings.
Other members of the committee said they were uncomfortable authorizing the drug given its potential to mutate the virus.
The drug, which forces the virus to mutate as it copies its RNA, eventually causes the virus to make so many errors in its genetic material that it can no longer make more of itself and the immune system clears it out of the body.
But it takes a few days to work — the drug is designed to be taken for 5 consecutive days -- and studies of the viral loads of patients taking the drug show that through the first 2 days, viral loads remain detectable as these mutations occur.
Studies by the FDA show some of those mutations in the spike protein are the same ones that have helped the virus become more transmissible and escape the protection of vaccines.
So the question is whether someone taking the medication could develop a dangerous mutation and then infect someone else, sparking the spread of a new variant.
Nicholas Kartsonis, MD, a vice president at Merck, said that the company was still analyzing data.
“Even if the probability is very low — 1 in 10,000 or 1 in 100,000 -- that this drug would induce an escape mutant for which the vaccines we have would not cover, that would be catastrophic for the whole world, actually,” said committee member James Hildreth, MD, an immunologist and president of Meharry Medical College, Nashville. “Do you have sufficient data on the likelihood of that happening?” he asked Dr. Kartsonis of Merck.
“So we don’t,” Dr. Kartsonis said.
He said, in theory, the risk of mutation with molnupiravir is the same as seen with the use of vaccines or monoclonal antibody therapies. Dr. Hildreth wasn’t satisfied with that answer.
“With all respect, the mechanism of your drug is to drive [genetic mutations], so it’s not the same as the vaccine. It’s not the same as monoclonal antibodies,” he said.
Dr. Hildreth later said he didn’t feel comfortable voting for authorization given the uncertainties around escape mutants. He voted no.
“It was an easy vote for me,” he said.
A version of this article first appeared on Medscape.com.
, according to a panel of experts that advises the Food and Drug Administration on its regulatory decisions for these types of drugs.
The FDA’s Antimicrobial Drugs Advisory Committee narrowly voted to authorize the drug molnupiravir, voting 13 to 10 to support emergency use, which requires a medication to meet a lower standard of evidence than does full approval.
The FDA is not bound by the committee’s vote but typically follows its advice.
If authorized by the agency, molnupiravir would be the first antiviral agent available as a pill to treat COVID-19. Other therapies to treat the infection are available — monoclonal antibodies and the drug remdesivir — but they are given by infusion.
The United Kingdom has already authorized the use of Merck’s drug.
“This was clearly a difficult decision,” said committee member Michael Green, MD, a pediatric infectious disease expert at the University of Pittsburg School of Medicine.
Green said he voted yes, and that the drug’s ability to prevent deaths in the study weighed heavily on his decision. He said given uncertainties around the drug both the company and FDA should keep a close eye on patients taking the drug going forward.
“Should an alternative oral agent become available that had a better safety profile and equal or better efficacy profile, the agency might reconsider its authorization,” he said.
Others didn’t agree that the drug should be allowed onto the market.
“I voted no,” said Jennifer Le, PharmD, a professor of clinical pharmacy at the University of California. Dr. Le said the modest benefit of the medication didn’t outweigh all the potential safety issues. “I think I just need more efficacy and safety data,” she said.
Initial results from the first half of people enrolled in the clinical trial found the pill cut the risk of hospitalization or death by 50% in patients at higher risk of severe outcomes from COVID-19.
But later results, released just days before the meeting, showed that the drug’s effectiveness had dropped to about 30%.
In the updated analysis, 48 patients out of the 709 who were taking the drug were hospitalized or died within 29 days compared to 68 out of 699 who randomly got the placebo. There was one death in the group that got molnupiravir compared to nine in the placebo group. Nearly all those deaths occurred during the first phase of the study.
On Nov. 30 Merck explained that the drug’s efficacy appeared to fall, in part, because the placebo group had experienced fewer hospitalizations and deaths than expected during the second half of the study, making the drug look less beneficial by comparison.
The company said it wasn’t sure why patients in the placebo group had fared so much better in later trial enrollments.
“The efficacy of this product is not overwhelmingly good,” said committee member David Hardy, MD, an infectious disease expert at Charles Drew University School of Medicine in Los Angeles. “And I think that makes all of us a little uncomfortable about whether this is an advanced therapeutic because it’s an oral medication rather than an intravenous medication,” he said during the panel’s deliberations.
“I think we have to be very careful about how we’re going to allow people to use this,” Dr. Hardy said.
Many who voted for authorization thought use of the drug should be restricted to unvaccinated people who were at high risk of severe COVID-19 outcomes, the same population enrolled in the clinical trial. People in the trial were considered at higher risk if they were over age 60, had cancer, chronic kidney disease, chronic obstructive pulmonary disease, were obese, or had heart disease or diabetes.
There are some significant limitations of the study that may affect how the drug is used. Vaccinated people couldn’t enroll in the study, so it’s not known if the medication would have any benefit for them. Nearly two-thirds of the U.S. population is fully vaccinated. The study found no additional benefit of the medication compared to the placebo in people who had detectable antibodies, presumably from a prior infection.
Animal studies found that the drug — which kills the virus by forcing it to make errors as it copies its genetic material inside cells — could disrupt bone formation. For that reason, the manufacturer and the FDA agreed that it should not be used in anyone younger than age 18.
Animal studies also indicated that the drug could cause birth defects. For that reason, the company said the drug shouldn’t be given to women who are pregnant or breastfeeding and said doctors should make sure women of childbearing age aren’t pregnant before taking the medication.
Some members of the panel felt that pregnant women and their doctors should be given the choice of whether or not to use the drug, given that pregnant women are at high risk for severe COVID-19 outcomes and infused therapies may not be available in all settings.
Other members of the committee said they were uncomfortable authorizing the drug given its potential to mutate the virus.
The drug, which forces the virus to mutate as it copies its RNA, eventually causes the virus to make so many errors in its genetic material that it can no longer make more of itself and the immune system clears it out of the body.
But it takes a few days to work — the drug is designed to be taken for 5 consecutive days -- and studies of the viral loads of patients taking the drug show that through the first 2 days, viral loads remain detectable as these mutations occur.
Studies by the FDA show some of those mutations in the spike protein are the same ones that have helped the virus become more transmissible and escape the protection of vaccines.
So the question is whether someone taking the medication could develop a dangerous mutation and then infect someone else, sparking the spread of a new variant.
Nicholas Kartsonis, MD, a vice president at Merck, said that the company was still analyzing data.
“Even if the probability is very low — 1 in 10,000 or 1 in 100,000 -- that this drug would induce an escape mutant for which the vaccines we have would not cover, that would be catastrophic for the whole world, actually,” said committee member James Hildreth, MD, an immunologist and president of Meharry Medical College, Nashville. “Do you have sufficient data on the likelihood of that happening?” he asked Dr. Kartsonis of Merck.
“So we don’t,” Dr. Kartsonis said.
He said, in theory, the risk of mutation with molnupiravir is the same as seen with the use of vaccines or monoclonal antibody therapies. Dr. Hildreth wasn’t satisfied with that answer.
“With all respect, the mechanism of your drug is to drive [genetic mutations], so it’s not the same as the vaccine. It’s not the same as monoclonal antibodies,” he said.
Dr. Hildreth later said he didn’t feel comfortable voting for authorization given the uncertainties around escape mutants. He voted no.
“It was an easy vote for me,” he said.
A version of this article first appeared on Medscape.com.
, according to a panel of experts that advises the Food and Drug Administration on its regulatory decisions for these types of drugs.
The FDA’s Antimicrobial Drugs Advisory Committee narrowly voted to authorize the drug molnupiravir, voting 13 to 10 to support emergency use, which requires a medication to meet a lower standard of evidence than does full approval.
The FDA is not bound by the committee’s vote but typically follows its advice.
If authorized by the agency, molnupiravir would be the first antiviral agent available as a pill to treat COVID-19. Other therapies to treat the infection are available — monoclonal antibodies and the drug remdesivir — but they are given by infusion.
The United Kingdom has already authorized the use of Merck’s drug.
“This was clearly a difficult decision,” said committee member Michael Green, MD, a pediatric infectious disease expert at the University of Pittsburg School of Medicine.
Green said he voted yes, and that the drug’s ability to prevent deaths in the study weighed heavily on his decision. He said given uncertainties around the drug both the company and FDA should keep a close eye on patients taking the drug going forward.
“Should an alternative oral agent become available that had a better safety profile and equal or better efficacy profile, the agency might reconsider its authorization,” he said.
Others didn’t agree that the drug should be allowed onto the market.
“I voted no,” said Jennifer Le, PharmD, a professor of clinical pharmacy at the University of California. Dr. Le said the modest benefit of the medication didn’t outweigh all the potential safety issues. “I think I just need more efficacy and safety data,” she said.
Initial results from the first half of people enrolled in the clinical trial found the pill cut the risk of hospitalization or death by 50% in patients at higher risk of severe outcomes from COVID-19.
But later results, released just days before the meeting, showed that the drug’s effectiveness had dropped to about 30%.
In the updated analysis, 48 patients out of the 709 who were taking the drug were hospitalized or died within 29 days compared to 68 out of 699 who randomly got the placebo. There was one death in the group that got molnupiravir compared to nine in the placebo group. Nearly all those deaths occurred during the first phase of the study.
On Nov. 30 Merck explained that the drug’s efficacy appeared to fall, in part, because the placebo group had experienced fewer hospitalizations and deaths than expected during the second half of the study, making the drug look less beneficial by comparison.
The company said it wasn’t sure why patients in the placebo group had fared so much better in later trial enrollments.
“The efficacy of this product is not overwhelmingly good,” said committee member David Hardy, MD, an infectious disease expert at Charles Drew University School of Medicine in Los Angeles. “And I think that makes all of us a little uncomfortable about whether this is an advanced therapeutic because it’s an oral medication rather than an intravenous medication,” he said during the panel’s deliberations.
“I think we have to be very careful about how we’re going to allow people to use this,” Dr. Hardy said.
Many who voted for authorization thought use of the drug should be restricted to unvaccinated people who were at high risk of severe COVID-19 outcomes, the same population enrolled in the clinical trial. People in the trial were considered at higher risk if they were over age 60, had cancer, chronic kidney disease, chronic obstructive pulmonary disease, were obese, or had heart disease or diabetes.
There are some significant limitations of the study that may affect how the drug is used. Vaccinated people couldn’t enroll in the study, so it’s not known if the medication would have any benefit for them. Nearly two-thirds of the U.S. population is fully vaccinated. The study found no additional benefit of the medication compared to the placebo in people who had detectable antibodies, presumably from a prior infection.
Animal studies found that the drug — which kills the virus by forcing it to make errors as it copies its genetic material inside cells — could disrupt bone formation. For that reason, the manufacturer and the FDA agreed that it should not be used in anyone younger than age 18.
Animal studies also indicated that the drug could cause birth defects. For that reason, the company said the drug shouldn’t be given to women who are pregnant or breastfeeding and said doctors should make sure women of childbearing age aren’t pregnant before taking the medication.
Some members of the panel felt that pregnant women and their doctors should be given the choice of whether or not to use the drug, given that pregnant women are at high risk for severe COVID-19 outcomes and infused therapies may not be available in all settings.
Other members of the committee said they were uncomfortable authorizing the drug given its potential to mutate the virus.
The drug, which forces the virus to mutate as it copies its RNA, eventually causes the virus to make so many errors in its genetic material that it can no longer make more of itself and the immune system clears it out of the body.
But it takes a few days to work — the drug is designed to be taken for 5 consecutive days -- and studies of the viral loads of patients taking the drug show that through the first 2 days, viral loads remain detectable as these mutations occur.
Studies by the FDA show some of those mutations in the spike protein are the same ones that have helped the virus become more transmissible and escape the protection of vaccines.
So the question is whether someone taking the medication could develop a dangerous mutation and then infect someone else, sparking the spread of a new variant.
Nicholas Kartsonis, MD, a vice president at Merck, said that the company was still analyzing data.
“Even if the probability is very low — 1 in 10,000 or 1 in 100,000 -- that this drug would induce an escape mutant for which the vaccines we have would not cover, that would be catastrophic for the whole world, actually,” said committee member James Hildreth, MD, an immunologist and president of Meharry Medical College, Nashville. “Do you have sufficient data on the likelihood of that happening?” he asked Dr. Kartsonis of Merck.
“So we don’t,” Dr. Kartsonis said.
He said, in theory, the risk of mutation with molnupiravir is the same as seen with the use of vaccines or monoclonal antibody therapies. Dr. Hildreth wasn’t satisfied with that answer.
“With all respect, the mechanism of your drug is to drive [genetic mutations], so it’s not the same as the vaccine. It’s not the same as monoclonal antibodies,” he said.
Dr. Hildreth later said he didn’t feel comfortable voting for authorization given the uncertainties around escape mutants. He voted no.
“It was an easy vote for me,” he said.
A version of this article first appeared on Medscape.com.
FDA approves imaging drug for detecting ovarian cancer lesions
The new drug “is designed to improve the ability to locate additional ovarian cancerous tissue that is normally difficult to detect during surgery,” according to the agency.
Pafolacianine, administered via intravenous injection prior to surgery, is the first FDA-approved tumor-targeted fluorescent agent for ovarian cancer.
In a press statement, drug inventor Philip Low, PhD, of Purdue University in West Lafayette, Ind., said the agent causes ovarian cancer lesions to “light up like stars against a night sky.”
Improving detection of ovarian cancer lesions is critical given that ovarian cancer is one of the “deadliest of all female reproductive system cancers,” according to the American Cancer Society. The organization estimates that there will be more than 21,000 new cases and more than 13,000 deaths in 2021.
Currently, surgeons use preoperative imaging as well as visual inspection of tumors under normal light and examination by touch to identify ovarian cancer lesions.
Pafolacianine offers a new tool to enhance surgeons’ ability “to identify deadly ovarian tumors that may otherwise go undetected,” Alex Gorovets, MD, deputy director of the office of specialty medicine in the FDA’s Center for Drug Evaluation and Research, said in a press statement.
Ovarian cancer often causes the body to overproduce the folate receptor protein in cell membranes. Pafolacianine, employed with a near-infrared fluorescence imaging system cleared by the FDA for use alongside the drug, binds to and illuminates these proteins under fluorescent light, “boosting surgeons’ ability to identify the cancerous tissue,” the agency in a statement.
The safety and effectiveness of pafolacianine was evaluated in a randomized, multi-center, open-label study of women diagnosed with ovarian cancer or with high clinical suspicion of ovarian cancer. Of the 134 women undergoing surgery who received a dose of pafolacianine and were evaluated under both normal and fluorescent light, 26.9% had at least one cancerous lesion detected that was not observed by standard visual or tactile inspection.
The most common side effects of pafolacianine were infusion-related reactions, including nausea, vomiting, abdominal pain, flushing, dyspepsia, chest discomfort, itching, and hypersensitivity.
Pafolacianine may cause fetal harm when administered to a pregnant woman. The use of folate, folic acid, or folate-containing supplements should be avoided within 48 hours before administration of pafolacianine.
The FDA also cautioned about the possible risk of image interpretation errors, including false negatives and false positives, with the use of the new drug and near-infrared fluorescence imaging system.
The FDA previously granted pafolacianine orphan-drug, priority, and fast track designations.
A version of this article first appeared on Medscape.com.
The new drug “is designed to improve the ability to locate additional ovarian cancerous tissue that is normally difficult to detect during surgery,” according to the agency.
Pafolacianine, administered via intravenous injection prior to surgery, is the first FDA-approved tumor-targeted fluorescent agent for ovarian cancer.
In a press statement, drug inventor Philip Low, PhD, of Purdue University in West Lafayette, Ind., said the agent causes ovarian cancer lesions to “light up like stars against a night sky.”
Improving detection of ovarian cancer lesions is critical given that ovarian cancer is one of the “deadliest of all female reproductive system cancers,” according to the American Cancer Society. The organization estimates that there will be more than 21,000 new cases and more than 13,000 deaths in 2021.
Currently, surgeons use preoperative imaging as well as visual inspection of tumors under normal light and examination by touch to identify ovarian cancer lesions.
Pafolacianine offers a new tool to enhance surgeons’ ability “to identify deadly ovarian tumors that may otherwise go undetected,” Alex Gorovets, MD, deputy director of the office of specialty medicine in the FDA’s Center for Drug Evaluation and Research, said in a press statement.
Ovarian cancer often causes the body to overproduce the folate receptor protein in cell membranes. Pafolacianine, employed with a near-infrared fluorescence imaging system cleared by the FDA for use alongside the drug, binds to and illuminates these proteins under fluorescent light, “boosting surgeons’ ability to identify the cancerous tissue,” the agency in a statement.
The safety and effectiveness of pafolacianine was evaluated in a randomized, multi-center, open-label study of women diagnosed with ovarian cancer or with high clinical suspicion of ovarian cancer. Of the 134 women undergoing surgery who received a dose of pafolacianine and were evaluated under both normal and fluorescent light, 26.9% had at least one cancerous lesion detected that was not observed by standard visual or tactile inspection.
The most common side effects of pafolacianine were infusion-related reactions, including nausea, vomiting, abdominal pain, flushing, dyspepsia, chest discomfort, itching, and hypersensitivity.
Pafolacianine may cause fetal harm when administered to a pregnant woman. The use of folate, folic acid, or folate-containing supplements should be avoided within 48 hours before administration of pafolacianine.
The FDA also cautioned about the possible risk of image interpretation errors, including false negatives and false positives, with the use of the new drug and near-infrared fluorescence imaging system.
The FDA previously granted pafolacianine orphan-drug, priority, and fast track designations.
A version of this article first appeared on Medscape.com.
The new drug “is designed to improve the ability to locate additional ovarian cancerous tissue that is normally difficult to detect during surgery,” according to the agency.
Pafolacianine, administered via intravenous injection prior to surgery, is the first FDA-approved tumor-targeted fluorescent agent for ovarian cancer.
In a press statement, drug inventor Philip Low, PhD, of Purdue University in West Lafayette, Ind., said the agent causes ovarian cancer lesions to “light up like stars against a night sky.”
Improving detection of ovarian cancer lesions is critical given that ovarian cancer is one of the “deadliest of all female reproductive system cancers,” according to the American Cancer Society. The organization estimates that there will be more than 21,000 new cases and more than 13,000 deaths in 2021.
Currently, surgeons use preoperative imaging as well as visual inspection of tumors under normal light and examination by touch to identify ovarian cancer lesions.
Pafolacianine offers a new tool to enhance surgeons’ ability “to identify deadly ovarian tumors that may otherwise go undetected,” Alex Gorovets, MD, deputy director of the office of specialty medicine in the FDA’s Center for Drug Evaluation and Research, said in a press statement.
Ovarian cancer often causes the body to overproduce the folate receptor protein in cell membranes. Pafolacianine, employed with a near-infrared fluorescence imaging system cleared by the FDA for use alongside the drug, binds to and illuminates these proteins under fluorescent light, “boosting surgeons’ ability to identify the cancerous tissue,” the agency in a statement.
The safety and effectiveness of pafolacianine was evaluated in a randomized, multi-center, open-label study of women diagnosed with ovarian cancer or with high clinical suspicion of ovarian cancer. Of the 134 women undergoing surgery who received a dose of pafolacianine and were evaluated under both normal and fluorescent light, 26.9% had at least one cancerous lesion detected that was not observed by standard visual or tactile inspection.
The most common side effects of pafolacianine were infusion-related reactions, including nausea, vomiting, abdominal pain, flushing, dyspepsia, chest discomfort, itching, and hypersensitivity.
Pafolacianine may cause fetal harm when administered to a pregnant woman. The use of folate, folic acid, or folate-containing supplements should be avoided within 48 hours before administration of pafolacianine.
The FDA also cautioned about the possible risk of image interpretation errors, including false negatives and false positives, with the use of the new drug and near-infrared fluorescence imaging system.
The FDA previously granted pafolacianine orphan-drug, priority, and fast track designations.
A version of this article first appeared on Medscape.com.
FDA approves first drug for treatment of resistant cytomegalovirus infection
The Food and Drug Administration has approved the first treatment for posttransplant cytomegalovirus (CMV) that is resistant to other drugs.
There are an estimated 200,000 adult transplants every year globally. CMV, a type of herpes virus, is one of the most common infections in transplant patients, occurring in 16%-56% of solid organ transplant recipients and 30%-70% of hematopoietic stem cell transplant recipients, according to Takeda Pharmaceutical Company Limited, the company that manufactures Livtencity. For immunosuppressed transplant patients, CMV infection can lead to complications that include loss of the transplanted or organ or even death.
“Cytomegalovirus infections that are resistant or do not respond to available drugs are of even greater concern,” John Farley, MD, MPH, the director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research, said in a statement. “Today’s approval helps meet a significant unmet medical need by providing a treatment option for this patient population.”
Livtencity, which is taken orally, works by preventing the activity of the enzyme responsible for virus replication. The approval, announced Nov. 23, was based on a phase 3 clinical trial that compared Livtencity with conventional antiviral treatments in the achievement of CMV DNA concentration levels below what is measurable in transplant patients with CMV infection that is refractory or treatment-resistant. After 8 weeks, of the 235 patients who received Livtencity, 56% achieved this primary endpoint, compared with 24% of the 117 patients who received conventional antiviral treatments, the press release says.
The most reported adverse reactions of Livtencity were taste disturbance, nausea, diarrhea, vomiting, and fatigue.
“We are grateful for the contributions of the patients and clinicians who participated in our clinical trials, as well as the dedication of our scientists and researchers,” Ramona Sequeira, president of the Takeda’s U.S. Business Unit and Global Portfolio Commercialization, said in a statement. “People undergoing transplants have a lengthy and complex health care journey; with the approval of this treatment, we’re proud to offer these individuals a new oral antiviral to fight CMV infection and disease.”
A version of this article first appeared on Medscape.com.
The Food and Drug Administration has approved the first treatment for posttransplant cytomegalovirus (CMV) that is resistant to other drugs.
There are an estimated 200,000 adult transplants every year globally. CMV, a type of herpes virus, is one of the most common infections in transplant patients, occurring in 16%-56% of solid organ transplant recipients and 30%-70% of hematopoietic stem cell transplant recipients, according to Takeda Pharmaceutical Company Limited, the company that manufactures Livtencity. For immunosuppressed transplant patients, CMV infection can lead to complications that include loss of the transplanted or organ or even death.
“Cytomegalovirus infections that are resistant or do not respond to available drugs are of even greater concern,” John Farley, MD, MPH, the director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research, said in a statement. “Today’s approval helps meet a significant unmet medical need by providing a treatment option for this patient population.”
Livtencity, which is taken orally, works by preventing the activity of the enzyme responsible for virus replication. The approval, announced Nov. 23, was based on a phase 3 clinical trial that compared Livtencity with conventional antiviral treatments in the achievement of CMV DNA concentration levels below what is measurable in transplant patients with CMV infection that is refractory or treatment-resistant. After 8 weeks, of the 235 patients who received Livtencity, 56% achieved this primary endpoint, compared with 24% of the 117 patients who received conventional antiviral treatments, the press release says.
The most reported adverse reactions of Livtencity were taste disturbance, nausea, diarrhea, vomiting, and fatigue.
“We are grateful for the contributions of the patients and clinicians who participated in our clinical trials, as well as the dedication of our scientists and researchers,” Ramona Sequeira, president of the Takeda’s U.S. Business Unit and Global Portfolio Commercialization, said in a statement. “People undergoing transplants have a lengthy and complex health care journey; with the approval of this treatment, we’re proud to offer these individuals a new oral antiviral to fight CMV infection and disease.”
A version of this article first appeared on Medscape.com.
The Food and Drug Administration has approved the first treatment for posttransplant cytomegalovirus (CMV) that is resistant to other drugs.
There are an estimated 200,000 adult transplants every year globally. CMV, a type of herpes virus, is one of the most common infections in transplant patients, occurring in 16%-56% of solid organ transplant recipients and 30%-70% of hematopoietic stem cell transplant recipients, according to Takeda Pharmaceutical Company Limited, the company that manufactures Livtencity. For immunosuppressed transplant patients, CMV infection can lead to complications that include loss of the transplanted or organ or even death.
“Cytomegalovirus infections that are resistant or do not respond to available drugs are of even greater concern,” John Farley, MD, MPH, the director of the Office of Infectious Diseases in the FDA’s Center for Drug Evaluation and Research, said in a statement. “Today’s approval helps meet a significant unmet medical need by providing a treatment option for this patient population.”
Livtencity, which is taken orally, works by preventing the activity of the enzyme responsible for virus replication. The approval, announced Nov. 23, was based on a phase 3 clinical trial that compared Livtencity with conventional antiviral treatments in the achievement of CMV DNA concentration levels below what is measurable in transplant patients with CMV infection that is refractory or treatment-resistant. After 8 weeks, of the 235 patients who received Livtencity, 56% achieved this primary endpoint, compared with 24% of the 117 patients who received conventional antiviral treatments, the press release says.
The most reported adverse reactions of Livtencity were taste disturbance, nausea, diarrhea, vomiting, and fatigue.
“We are grateful for the contributions of the patients and clinicians who participated in our clinical trials, as well as the dedication of our scientists and researchers,” Ramona Sequeira, president of the Takeda’s U.S. Business Unit and Global Portfolio Commercialization, said in a statement. “People undergoing transplants have a lengthy and complex health care journey; with the approval of this treatment, we’re proud to offer these individuals a new oral antiviral to fight CMV infection and disease.”
A version of this article first appeared on Medscape.com.