For most of my professional life I have avoided writing about gender inequity in the field of surgery – not because I believe it does not exist, but because the reasons it does exist are manifold and complicated.

To be sure, implicit and explicit bias remain important reasons why only a minority of female medical students still choose surgery as a career in 2018, why female surgeons still earn only 82% of the salary of their male counterparts, and why women occupy only 12% of the chairs in academic surgical departments in the United States. It has long been tempting to lay the blame for those inequities on a stereotypical macho surgical culture that prevailed as the 20th century came to a close. But the factors that perpetuate male/female imbalance in our profession are complicated and run deep in the psyches of both men and women. We are all products of our generations, our culture, upbringing, and environment – men as well as women. Correcting the imbalance requires much more than simply passing laws that require equitable pay or treatment.

I realized how deeply ingrained biases are in all of us while still in my surgical residency in the 1970s. On a rare occasion when neither my husband (also a surgical resident) nor I was on call, we had a dinner party with friends at our house. The telephone rang (no iPhones, just a land line), and I ran to answer our only telephone in another room. It was the hospital operator, who asked, “Is Dr. Deveney there?” Without thinking, I answered, “Just a moment, I’ll get him!” I had taken only three steps away from the phone when it occurred to me what I had just said. I turned back, picked up the phone, and asked, “Which Dr. Deveney were you looking for?”

If even I had been conditioned to think automatically of “doctor” being a man after all of my effort to earn a place in the ranks, was there any hope that equality could be achieved? When I finished my residency and was offered a surgery position at our VA, I asked no questions about salary or other particulars; I was simply grateful that I had been given a job.

That was 1978 – a different time, a different generation. Since then, women have made dramatic progress toward equity in our profession, as in many others. The support, mentoring, and consciousness-raising efforts of the Association of Women Surgeons (AWS) are responsible for much of this progress. The American College of Surgeons was very supportive of the AWS early on and continues to encourage female medical students to choose surgery as a career and to help the advancement of women into leadership roles in surgery. Many surgical residency programs have 50% women in their ranks; half of our residents in Oregon have been women for over a decade. Our program director is a woman, as are 28% of the faculty; not 50%, but definitely progress, since I was the lone woman on the faculty when I arrived in 1987.

Although women occupy only 12% of the surgical chairs in U.S. surgical departments, this number has soared in the past 2 years from 7 in 2016 to 21 this year after languishing in the low single digits since 1987, when the late Olga Jonasson, MD, FACS, became the first female chair at Ohio State University. And yet, women have not reached full equality with men across the United States in surgical training, leadership, or pay. Why not?

Multiple factors have played roles in impeding the progress of women in achieving equality in surgery. Traditional cultural expectations of male and female roles in society affect both genders as they grow up, even when their parents make deliberate efforts to raise their children in as “gender-neutral” a way as possible and encourage their daughters to strive for success to the same degree as their sons. Explicit bias against women remains easier to recognize and combat than implicit or unconscious bias, to which we are all subject.

Dr. Deveney is professor of surgery and vice chair of education in the department of surgery, Oregon Health & Science University, Portland. She is the coeditor of ACS Surgery News.

For most of my professional life I have avoided writing about gender inequity in the field of surgery – not because I believe it does not exist, but because the reasons it does exist are manifold and complicated.

To be sure, implicit and explicit bias remain important reasons why only a minority of female medical students still choose surgery as a career in 2018, why female surgeons still earn only 82% of the salary of their male counterparts, and why women occupy only 12% of the chairs in academic surgical departments in the United States. It has long been tempting to lay the blame for those inequities on a stereotypical macho surgical culture that prevailed as the 20th century came to a close. But the factors that perpetuate male/female imbalance in our profession are complicated and run deep in the psyches of both men and women. We are all products of our generations, our culture, upbringing, and environment – men as well as women. Correcting the imbalance requires much more than simply passing laws that require equitable pay or treatment.

I realized how deeply ingrained biases are in all of us while still in my surgical residency in the 1970s. On a rare occasion when neither my husband (also a surgical resident) nor I was on call, we had a dinner party with friends at our house. The telephone rang (no iPhones, just a land line), and I ran to answer our only telephone in another room. It was the hospital operator, who asked, “Is Dr. Deveney there?” Without thinking, I answered, “Just a moment, I’ll get him!” I had taken only three steps away from the phone when it occurred to me what I had just said. I turned back, picked up the phone, and asked, “Which Dr. Deveney were you looking for?”

If even I had been conditioned to think automatically of “doctor” being a man after all of my effort to earn a place in the ranks, was there any hope that equality could be achieved? When I finished my residency and was offered a surgery position at our VA, I asked no questions about salary or other particulars; I was simply grateful that I had been given a job.

That was 1978 – a different time, a different generation. Since then, women have made dramatic progress toward equity in our profession, as in many others. The support, mentoring, and consciousness-raising efforts of the Association of Women Surgeons (AWS) are responsible for much of this progress. The American College of Surgeons was very supportive of the AWS early on and continues to encourage female medical students to choose surgery as a career and to help the advancement of women into leadership roles in surgery. Many surgical residency programs have 50% women in their ranks; half of our residents in Oregon have been women for over a decade. Our program director is a woman, as are 28% of the faculty; not 50%, but definitely progress, since I was the lone woman on the faculty when I arrived in 1987.

Although women occupy only 12% of the surgical chairs in U.S. surgical departments, this number has soared in the past 2 years from 7 in 2016 to 21 this year after languishing in the low single digits since 1987, when the late Olga Jonasson, MD, FACS, became the first female chair at Ohio State University. And yet, women have not reached full equality with men across the United States in surgical training, leadership, or pay. Why not?

Multiple factors have played roles in impeding the progress of women in achieving equality in surgery. Traditional cultural expectations of male and female roles in society affect both genders as they grow up, even when their parents make deliberate efforts to raise their children in as “gender-neutral” a way as possible and encourage their daughters to strive for success to the same degree as their sons. Explicit bias against women remains easier to recognize and combat than implicit or unconscious bias, to which we are all subject.

Dr. Deveney is professor of surgery and vice chair of education in the department of surgery, Oregon Health & Science University, Portland. She is the coeditor of ACS Surgery News.

For most of my professional life I have avoided writing about gender inequity in the field of surgery – not because I believe it does not exist, but because the reasons it does exist are manifold and complicated.

To be sure, implicit and explicit bias remain important reasons why only a minority of female medical students still choose surgery as a career in 2018, why female surgeons still earn only 82% of the salary of their male counterparts, and why women occupy only 12% of the chairs in academic surgical departments in the United States. It has long been tempting to lay the blame for those inequities on a stereotypical macho surgical culture that prevailed as the 20th century came to a close. But the factors that perpetuate male/female imbalance in our profession are complicated and run deep in the psyches of both men and women. We are all products of our generations, our culture, upbringing, and environment – men as well as women. Correcting the imbalance requires much more than simply passing laws that require equitable pay or treatment.

I realized how deeply ingrained biases are in all of us while still in my surgical residency in the 1970s. On a rare occasion when neither my husband (also a surgical resident) nor I was on call, we had a dinner party with friends at our house. The telephone rang (no iPhones, just a land line), and I ran to answer our only telephone in another room. It was the hospital operator, who asked, “Is Dr. Deveney there?” Without thinking, I answered, “Just a moment, I’ll get him!” I had taken only three steps away from the phone when it occurred to me what I had just said. I turned back, picked up the phone, and asked, “Which Dr. Deveney were you looking for?”

If even I had been conditioned to think automatically of “doctor” being a man after all of my effort to earn a place in the ranks, was there any hope that equality could be achieved? When I finished my residency and was offered a surgery position at our VA, I asked no questions about salary or other particulars; I was simply grateful that I had been given a job.

That was 1978 – a different time, a different generation. Since then, women have made dramatic progress toward equity in our profession, as in many others. The support, mentoring, and consciousness-raising efforts of the Association of Women Surgeons (AWS) are responsible for much of this progress. The American College of Surgeons was very supportive of the AWS early on and continues to encourage female medical students to choose surgery as a career and to help the advancement of women into leadership roles in surgery. Many surgical residency programs have 50% women in their ranks; half of our residents in Oregon have been women for over a decade. Our program director is a woman, as are 28% of the faculty; not 50%, but definitely progress, since I was the lone woman on the faculty when I arrived in 1987.

Although women occupy only 12% of the surgical chairs in U.S. surgical departments, this number has soared in the past 2 years from 7 in 2016 to 21 this year after languishing in the low single digits since 1987, when the late Olga Jonasson, MD, FACS, became the first female chair at Ohio State University. And yet, women have not reached full equality with men across the United States in surgical training, leadership, or pay. Why not?

Multiple factors have played roles in impeding the progress of women in achieving equality in surgery. Traditional cultural expectations of male and female roles in society affect both genders as they grow up, even when their parents make deliberate efforts to raise their children in as “gender-neutral” a way as possible and encourage their daughters to strive for success to the same degree as their sons. Explicit bias against women remains easier to recognize and combat than implicit or unconscious bias, to which we are all subject.

Dr. Deveney is professor of surgery and vice chair of education in the department of surgery, Oregon Health & Science University, Portland. She is the coeditor of ACS Surgery News.

Many are submitted, but few are chosen.

Concerned about the quality of submitted research papers based on large surgical databases that are not accepted for publication, the editorial board of JAMA Surgery has taken the initiative by giving some pointers to would-be authors. The journal editors have published a 10-point checklist of dos and don’ts to address commonly seen problems with submitted manuscripts. In addition, JAMA Surgery collaborated with the Surgical Outcomes Club to commission a series of practical guides on the most widely used data sets in an effort to improve the quality of surgical database research. The Surgical Outcomes Club is a consortium of surgeons and scientists who work to advance health services and outcomes research in surgery that was launched in 2005 at the American College of Surgeons Clinical Congress.

The series concludes with tips from the statistical editors of JAMA Surgery – Amy H. Kaji, MD, PhD, of Harbor–University of California Los Angeles Medical Center in Torrance; Alfred W. Rademaker, PhD, of Northwestern University, Chicago; and Terry Hyslop, PhD, of Duke University, Durham, N.C. – for performing statistical analysis of large data sets: “With bigger data, random signals may denote statistical significance, and precision may be incorrectly inferred because of narrow confidence intervals,” the statistical editors noted. “While many principles apply to all studies, the importance of these methodological issues is amplified in large, complex data sets.”

However, they noted that large data sets are prone to bias and measurement errors. “It is important to respect and acknowledge the limitations of the data,” the statistical team wrote. They also reprise the introductory editorial’s call for a clear hypothesis and take-home message. “The challenge with Big Data is that it requires a carefully thought-out research question and a transparent analytic strategy,” the statistical editors said.

He said, “A problem we frequently see is that these databases change the variable definitions over time – in fact, change the variables over time. So if researchers aren’t checking to see if that variable was reported the same every year of the study and in the same way, they will get spurious results. Similarly, the number of hospitals reporting is important as well since hospitals come in and out of these data sets.”

In their introductory editorial, the JAMA Surgery team noted that the checklist, practical guides, and statistical tips are a three-pronged approach that authors should consult before submitting their manuscripts. “We hope that by following these simple guides, authors can benefit from the collective wisdom of so many colleagues who have successfully completed similar analyses in the past,” they wrote.

Dr. Bilimoria sees great strengths in database research, such as giving researchers a population-level view of how care is being delivered, insights into the outcomes of care, indications of the effects of policy decisions, and data on rare diseases and operations.

Big Data of all kinds will be increasingly available for researchers. Dr. Kibbe commented that, “In the future, having a comprehensive (not sampling) country- or worldwide electronic medical record that will allow for robust inclusion of all medical data at the individual as well as cohort level will greatly contribute to the era of personalized medicine. In my opinion, this would be a real-time inclusive medical database that would allow for individual as well as population-based prospective studies.”

Dr. Haider receives support from the Henry M. Jackson Foundation of the Department of Defense, the Orthopaedic Research and Education Foundation, and the National Institutes of Health, and nonfinancial research support the Centers for Medicare and Medicaid Services Office of Minority Health. Dr. Bilimoria was the president of the Surgical Outcomes Club from 2016 to 2017.

Many are submitted, but few are chosen.

Concerned about the quality of submitted research papers based on large surgical databases that are not accepted for publication, the editorial board of JAMA Surgery has taken the initiative by giving some pointers to would-be authors. The journal editors have published a 10-point checklist of dos and don’ts to address commonly seen problems with submitted manuscripts. In addition, JAMA Surgery collaborated with the Surgical Outcomes Club to commission a series of practical guides on the most widely used data sets in an effort to improve the quality of surgical database research. The Surgical Outcomes Club is a consortium of surgeons and scientists who work to advance health services and outcomes research in surgery that was launched in 2005 at the American College of Surgeons Clinical Congress.

The series concludes with tips from the statistical editors of JAMA Surgery – Amy H. Kaji, MD, PhD, of Harbor–University of California Los Angeles Medical Center in Torrance; Alfred W. Rademaker, PhD, of Northwestern University, Chicago; and Terry Hyslop, PhD, of Duke University, Durham, N.C. – for performing statistical analysis of large data sets: “With bigger data, random signals may denote statistical significance, and precision may be incorrectly inferred because of narrow confidence intervals,” the statistical editors noted. “While many principles apply to all studies, the importance of these methodological issues is amplified in large, complex data sets.”

However, they noted that large data sets are prone to bias and measurement errors. “It is important to respect and acknowledge the limitations of the data,” the statistical team wrote. They also reprise the introductory editorial’s call for a clear hypothesis and take-home message. “The challenge with Big Data is that it requires a carefully thought-out research question and a transparent analytic strategy,” the statistical editors said.

He said, “A problem we frequently see is that these databases change the variable definitions over time – in fact, change the variables over time. So if researchers aren’t checking to see if that variable was reported the same every year of the study and in the same way, they will get spurious results. Similarly, the number of hospitals reporting is important as well since hospitals come in and out of these data sets.”

In their introductory editorial, the JAMA Surgery team noted that the checklist, practical guides, and statistical tips are a three-pronged approach that authors should consult before submitting their manuscripts. “We hope that by following these simple guides, authors can benefit from the collective wisdom of so many colleagues who have successfully completed similar analyses in the past,” they wrote.

Dr. Bilimoria sees great strengths in database research, such as giving researchers a population-level view of how care is being delivered, insights into the outcomes of care, indications of the effects of policy decisions, and data on rare diseases and operations.

Big Data of all kinds will be increasingly available for researchers. Dr. Kibbe commented that, “In the future, having a comprehensive (not sampling) country- or worldwide electronic medical record that will allow for robust inclusion of all medical data at the individual as well as cohort level will greatly contribute to the era of personalized medicine. In my opinion, this would be a real-time inclusive medical database that would allow for individual as well as population-based prospective studies.”

Dr. Haider receives support from the Henry M. Jackson Foundation of the Department of Defense, the Orthopaedic Research and Education Foundation, and the National Institutes of Health, and nonfinancial research support the Centers for Medicare and Medicaid Services Office of Minority Health. Dr. Bilimoria was the president of the Surgical Outcomes Club from 2016 to 2017.

Many are submitted, but few are chosen.

Concerned about the quality of submitted research papers based on large surgical databases that are not accepted for publication, the editorial board of JAMA Surgery has taken the initiative by giving some pointers to would-be authors. The journal editors have published a 10-point checklist of dos and don’ts to address commonly seen problems with submitted manuscripts. In addition, JAMA Surgery collaborated with the Surgical Outcomes Club to commission a series of practical guides on the most widely used data sets in an effort to improve the quality of surgical database research. The Surgical Outcomes Club is a consortium of surgeons and scientists who work to advance health services and outcomes research in surgery that was launched in 2005 at the American College of Surgeons Clinical Congress.

The series concludes with tips from the statistical editors of JAMA Surgery – Amy H. Kaji, MD, PhD, of Harbor–University of California Los Angeles Medical Center in Torrance; Alfred W. Rademaker, PhD, of Northwestern University, Chicago; and Terry Hyslop, PhD, of Duke University, Durham, N.C. – for performing statistical analysis of large data sets: “With bigger data, random signals may denote statistical significance, and precision may be incorrectly inferred because of narrow confidence intervals,” the statistical editors noted. “While many principles apply to all studies, the importance of these methodological issues is amplified in large, complex data sets.”

However, they noted that large data sets are prone to bias and measurement errors. “It is important to respect and acknowledge the limitations of the data,” the statistical team wrote. They also reprise the introductory editorial’s call for a clear hypothesis and take-home message. “The challenge with Big Data is that it requires a carefully thought-out research question and a transparent analytic strategy,” the statistical editors said.

He said, “A problem we frequently see is that these databases change the variable definitions over time – in fact, change the variables over time. So if researchers aren’t checking to see if that variable was reported the same every year of the study and in the same way, they will get spurious results. Similarly, the number of hospitals reporting is important as well since hospitals come in and out of these data sets.”

In their introductory editorial, the JAMA Surgery team noted that the checklist, practical guides, and statistical tips are a three-pronged approach that authors should consult before submitting their manuscripts. “We hope that by following these simple guides, authors can benefit from the collective wisdom of so many colleagues who have successfully completed similar analyses in the past,” they wrote.

Dr. Bilimoria sees great strengths in database research, such as giving researchers a population-level view of how care is being delivered, insights into the outcomes of care, indications of the effects of policy decisions, and data on rare diseases and operations.

Big Data of all kinds will be increasingly available for researchers. Dr. Kibbe commented that, “In the future, having a comprehensive (not sampling) country- or worldwide electronic medical record that will allow for robust inclusion of all medical data at the individual as well as cohort level will greatly contribute to the era of personalized medicine. In my opinion, this would be a real-time inclusive medical database that would allow for individual as well as population-based prospective studies.”

Dr. Haider receives support from the Henry M. Jackson Foundation of the Department of Defense, the Orthopaedic Research and Education Foundation, and the National Institutes of Health, and nonfinancial research support the Centers for Medicare and Medicaid Services Office of Minority Health. Dr. Bilimoria was the president of the Surgical Outcomes Club from 2016 to 2017.

SILVER SPRING, Md. – A novel drug to treat a rare metabolic disorder was recommended for approval by the Endocrinologic and Metabolic Drugs Advisory Committee in a May 10 meeting.
Advisors voted 12 to 8 to recommend volanesorsen, a novel antisense drug, as an adjunct to diet for the treatment of familial chylomicronemia syndrome (FCS).
“The sponsor provided compelling evidence that the drug lowers triglyceride levels, substantially,” said panelist Jean-Pierre Raufman, MD, of the University of Maryland. I think that there is clearly a group of patients with this disease that can benefit from this agent.”

FCS is an extremely rare genetic disorder characterized by severe hypertriglyceridemia and recurrent pancreatitis caused by a deficiency in lipoprotein lipase. Pancreatitis in the setting of hypertriglyceridemia is particularly severe, often leading to multi-organ failure, pancreatic necrosis, and death.

Volanesorsen targets apolipoprotein C-III (apoC-III), a key regulator of lipoprotein lipase, the essential enzyme involved in chylomicron and triglyceride clearance.
Because of the small number of patients affected, estimated at just 3,000-5,000 patients worldwide, the FDA considers FCS to be an orphan disease.

The application, submitted by Akcea Pharmaceuticals, was based on the results of several phase 3 clinical trials conducted both in FCS patients and, because of the rarity of the disorder, patients with high triglycerides. The APPROACH study looked at patients with FCS. The COMPASS study examined patients with hypertriglyceridemia. Additionally, an ongoing, open-label extension of the APPROACH study examined patients with FCS who had completed APPROACH,  COMPASS, and FCS patients who had not participated in a previous volanesorsen trial.

APPROACH APPROACH was a phase 3, double-blind clinical trial to assess the safety and efficacy of volanesorsen in patients with FCS. Because of the extremely small number of patients affected by FCS, non-FCS patients with hypertriglyceridemia were included in the trial.

All patients who had completed a 6 week diet, lifestyle, and medication stabilization period were enrolled in the study. In total, 67 patients were randomized to receive a weekly, subcutaneous 300 mg dose of volanesorsen or a placebo for 52 weeks. Due to the risk of platelet reduction, the study allowed for dosing schedule interruptions or pauses, if needed. To assess efficacy, the study looked at the percent change in fasting triglycerides at month 3 of the study in all randomized patients who received at least one dose of the study drug and who had baseline fasting plasma trigylcerides recorded.

Volanesorsen proved quite effective in reducing fasting triglyceride levels, with a 94% (P < 0.0001) reduction in plasma concentrations at 3 months, compared with placebo.

The effectiveness of volanesorsen extended beyond month 3, with statistically significant reductions in triglyceride concentrations at both month 6 and month 12 compared with placebo. Patients taking volanesorsen had 53% and 40% reductions at month 6 and 12 compared to baseline. Conversely, patients taking placebo experienced increases of 25% and 9%, respectively. The differences between volanesorsen and placebo patients at month 6 (–78%) and month 12 (–49%) were both statistically significant.

Another important finding from this study was that 77% of patients responded to treatment, evidenced by a reduction of triglyceride levels greater than 750 mg/dL, a much better response than the 10% of placebo patients (P =.0001).

Nine patients (27%) discontinued the study due to adverse events (AE). Five of the discontinuations were related to platelet reductions with the other four related to nonplatelet-related adverse events.

COMPASS

The COMPASS study primarily looked at patients without FCS, but severe hypertriglyceridemia, to evaluate the safety and efficacy of volanesorsen in a similarly ill, but less severe, population over a 6 month study period. Patients with FCS had baseline fasting plasma triglyceride levels of 2,644 mg/dL and 2,134 mg/dL in the placebo and volanesorsen groups, respectively. This is nearly double what was seen in non-FCS patients in the study, and is 14-17 higher than the upper limit of normal (ULN).
Researchers enrolled 114 patients and randomized them 2:1 to receive volanesorsen or placebo. This led to 75 patients taking volanesorsen and 34 placebo.
The efficacy results were similar to those in the FCS-focused APPROACH trial, with volanesorsen treatment leading to reductions in triglyceride levels after 3 months, with further reductions over the 6-month study treatment.
Weekly treatment with volanesorsen reduced triglycerides by nearly 71%  in the total patient population, compared to placebo, which only achieved a 0.9% decrease in triglycerides. These reductions were conserved at the end of week 26 in patients who had to reduce their dosing to biweekly (62%) and patients who maintained weekly dosing treatments (78%). Overall, a greater than 40% reduction of fasting triglyceride levels after 3 months of treatment was achieved in 87% of volanesorsen-treated patients, compared to 13% of placebo-treated patients (P < 0.0001).
Serious adverse events (SAEs) occurred in nine patients (8%). In the placebo group, two patients (5%) reported acute pancreatitis. Apart from the bouts of pancreatitis, some of the most common AEs were decreased platelet count, thrombocytopenia, and nasopharyngitis.

APPROACH OPEN LABEL

The open label portion of the APPROACH study is ongoing and was designed to assess the safety and efficacy of extended treatment with volanesorsen in patients with FCS who had previously completed APPROACH or COMPASS studies or had never received volanesorsen treatment. Patients received 300 mg of volanesorsen once weekly for 52 weeks. Patients who completed the trial were eligible to continue treatment for an additional 52 weeks or until a product is available. As of August 31, 2017, after the data cutoff, 60 patients were enrolled.
The results of this open label portion of the APPROACH trial were similar to that of the APPROACH trial itself. Patients who transitioned from APPROACH or COMPASS showed decreases in their open label extension (OLE) baseline triglyceride measurements to OLE month 3 of 48.1% and 52.1%, respectively. Treatment-naive patients displayed a nearly 60% decrease in their triglyceride levels 3 months.
Of the 60 patients who started the study, 12 have discontinued the study prematurely. The majority of patients, eight, who discontinued did so because of adverse events and four withdrew voluntarily.
Adverse events were not uncommon, with 56 patients (93%) experiencing an AE during the course of the study. Some of the most common AEs were decreased platelet count, thrombocytopenia, and nasopharyngitis.
Akcea has developed a Risk Evaluation and Mitigation program to mitigate the risk of serious bleeding related to severe thrombocytopenia with volanesorsen use in patients with FCS.
Panelist Susan Z. Yanovksi, MD, of the National Institutes of Health, said she voted no over safety concerns, but felt conflicted.
“There’s no question that volanesorsen is effective in dramatically reducing triglycerides, but I had a lot of concerns whether the data presented by the sponsor actually established favorable risk-benefit ratio.”

The FDA is expected to decide on the application by August 30. The FDA usually follows the recommendations of its advisory panels, which are not binding.

[email protected]

SILVER SPRING, Md. – A novel drug to treat a rare metabolic disorder was recommended for approval by the Endocrinologic and Metabolic Drugs Advisory Committee in a May 10 meeting.
Advisors voted 12 to 8 to recommend volanesorsen, a novel antisense drug, as an adjunct to diet for the treatment of familial chylomicronemia syndrome (FCS).
“The sponsor provided compelling evidence that the drug lowers triglyceride levels, substantially,” said panelist Jean-Pierre Raufman, MD, of the University of Maryland. I think that there is clearly a group of patients with this disease that can benefit from this agent.”

FCS is an extremely rare genetic disorder characterized by severe hypertriglyceridemia and recurrent pancreatitis caused by a deficiency in lipoprotein lipase. Pancreatitis in the setting of hypertriglyceridemia is particularly severe, often leading to multi-organ failure, pancreatic necrosis, and death.

Volanesorsen targets apolipoprotein C-III (apoC-III), a key regulator of lipoprotein lipase, the essential enzyme involved in chylomicron and triglyceride clearance.
Because of the small number of patients affected, estimated at just 3,000-5,000 patients worldwide, the FDA considers FCS to be an orphan disease.

The application, submitted by Akcea Pharmaceuticals, was based on the results of several phase 3 clinical trials conducted both in FCS patients and, because of the rarity of the disorder, patients with high triglycerides. The APPROACH study looked at patients with FCS. The COMPASS study examined patients with hypertriglyceridemia. Additionally, an ongoing, open-label extension of the APPROACH study examined patients with FCS who had completed APPROACH,  COMPASS, and FCS patients who had not participated in a previous volanesorsen trial.

APPROACH APPROACH was a phase 3, double-blind clinical trial to assess the safety and efficacy of volanesorsen in patients with FCS. Because of the extremely small number of patients affected by FCS, non-FCS patients with hypertriglyceridemia were included in the trial.

All patients who had completed a 6 week diet, lifestyle, and medication stabilization period were enrolled in the study. In total, 67 patients were randomized to receive a weekly, subcutaneous 300 mg dose of volanesorsen or a placebo for 52 weeks. Due to the risk of platelet reduction, the study allowed for dosing schedule interruptions or pauses, if needed. To assess efficacy, the study looked at the percent change in fasting triglycerides at month 3 of the study in all randomized patients who received at least one dose of the study drug and who had baseline fasting plasma trigylcerides recorded.

Volanesorsen proved quite effective in reducing fasting triglyceride levels, with a 94% (P < 0.0001) reduction in plasma concentrations at 3 months, compared with placebo.

The effectiveness of volanesorsen extended beyond month 3, with statistically significant reductions in triglyceride concentrations at both month 6 and month 12 compared with placebo. Patients taking volanesorsen had 53% and 40% reductions at month 6 and 12 compared to baseline. Conversely, patients taking placebo experienced increases of 25% and 9%, respectively. The differences between volanesorsen and placebo patients at month 6 (–78%) and month 12 (–49%) were both statistically significant.

Another important finding from this study was that 77% of patients responded to treatment, evidenced by a reduction of triglyceride levels greater than 750 mg/dL, a much better response than the 10% of placebo patients (P =.0001).

Nine patients (27%) discontinued the study due to adverse events (AE). Five of the discontinuations were related to platelet reductions with the other four related to nonplatelet-related adverse events.

COMPASS

The COMPASS study primarily looked at patients without FCS, but severe hypertriglyceridemia, to evaluate the safety and efficacy of volanesorsen in a similarly ill, but less severe, population over a 6 month study period. Patients with FCS had baseline fasting plasma triglyceride levels of 2,644 mg/dL and 2,134 mg/dL in the placebo and volanesorsen groups, respectively. This is nearly double what was seen in non-FCS patients in the study, and is 14-17 higher than the upper limit of normal (ULN).
Researchers enrolled 114 patients and randomized them 2:1 to receive volanesorsen or placebo. This led to 75 patients taking volanesorsen and 34 placebo.
The efficacy results were similar to those in the FCS-focused APPROACH trial, with volanesorsen treatment leading to reductions in triglyceride levels after 3 months, with further reductions over the 6-month study treatment.
Weekly treatment with volanesorsen reduced triglycerides by nearly 71%  in the total patient population, compared to placebo, which only achieved a 0.9% decrease in triglycerides. These reductions were conserved at the end of week 26 in patients who had to reduce their dosing to biweekly (62%) and patients who maintained weekly dosing treatments (78%). Overall, a greater than 40% reduction of fasting triglyceride levels after 3 months of treatment was achieved in 87% of volanesorsen-treated patients, compared to 13% of placebo-treated patients (P < 0.0001).
Serious adverse events (SAEs) occurred in nine patients (8%). In the placebo group, two patients (5%) reported acute pancreatitis. Apart from the bouts of pancreatitis, some of the most common AEs were decreased platelet count, thrombocytopenia, and nasopharyngitis.

APPROACH OPEN LABEL

The open label portion of the APPROACH study is ongoing and was designed to assess the safety and efficacy of extended treatment with volanesorsen in patients with FCS who had previously completed APPROACH or COMPASS studies or had never received volanesorsen treatment. Patients received 300 mg of volanesorsen once weekly for 52 weeks. Patients who completed the trial were eligible to continue treatment for an additional 52 weeks or until a product is available. As of August 31, 2017, after the data cutoff, 60 patients were enrolled.
The results of this open label portion of the APPROACH trial were similar to that of the APPROACH trial itself. Patients who transitioned from APPROACH or COMPASS showed decreases in their open label extension (OLE) baseline triglyceride measurements to OLE month 3 of 48.1% and 52.1%, respectively. Treatment-naive patients displayed a nearly 60% decrease in their triglyceride levels 3 months.
Of the 60 patients who started the study, 12 have discontinued the study prematurely. The majority of patients, eight, who discontinued did so because of adverse events and four withdrew voluntarily.
Adverse events were not uncommon, with 56 patients (93%) experiencing an AE during the course of the study. Some of the most common AEs were decreased platelet count, thrombocytopenia, and nasopharyngitis.
Akcea has developed a Risk Evaluation and Mitigation program to mitigate the risk of serious bleeding related to severe thrombocytopenia with volanesorsen use in patients with FCS.
Panelist Susan Z. Yanovksi, MD, of the National Institutes of Health, said she voted no over safety concerns, but felt conflicted.
“There’s no question that volanesorsen is effective in dramatically reducing triglycerides, but I had a lot of concerns whether the data presented by the sponsor actually established favorable risk-benefit ratio.”

The FDA is expected to decide on the application by August 30. The FDA usually follows the recommendations of its advisory panels, which are not binding.

[email protected]

SILVER SPRING, Md. – A novel drug to treat a rare metabolic disorder was recommended for approval by the Endocrinologic and Metabolic Drugs Advisory Committee in a May 10 meeting.
Advisors voted 12 to 8 to recommend volanesorsen, a novel antisense drug, as an adjunct to diet for the treatment of familial chylomicronemia syndrome (FCS).
“The sponsor provided compelling evidence that the drug lowers triglyceride levels, substantially,” said panelist Jean-Pierre Raufman, MD, of the University of Maryland. I think that there is clearly a group of patients with this disease that can benefit from this agent.”

FCS is an extremely rare genetic disorder characterized by severe hypertriglyceridemia and recurrent pancreatitis caused by a deficiency in lipoprotein lipase. Pancreatitis in the setting of hypertriglyceridemia is particularly severe, often leading to multi-organ failure, pancreatic necrosis, and death.

Volanesorsen targets apolipoprotein C-III (apoC-III), a key regulator of lipoprotein lipase, the essential enzyme involved in chylomicron and triglyceride clearance.
Because of the small number of patients affected, estimated at just 3,000-5,000 patients worldwide, the FDA considers FCS to be an orphan disease.

The application, submitted by Akcea Pharmaceuticals, was based on the results of several phase 3 clinical trials conducted both in FCS patients and, because of the rarity of the disorder, patients with high triglycerides. The APPROACH study looked at patients with FCS. The COMPASS study examined patients with hypertriglyceridemia. Additionally, an ongoing, open-label extension of the APPROACH study examined patients with FCS who had completed APPROACH,  COMPASS, and FCS patients who had not participated in a previous volanesorsen trial.

APPROACH APPROACH was a phase 3, double-blind clinical trial to assess the safety and efficacy of volanesorsen in patients with FCS. Because of the extremely small number of patients affected by FCS, non-FCS patients with hypertriglyceridemia were included in the trial.

All patients who had completed a 6 week diet, lifestyle, and medication stabilization period were enrolled in the study. In total, 67 patients were randomized to receive a weekly, subcutaneous 300 mg dose of volanesorsen or a placebo for 52 weeks. Due to the risk of platelet reduction, the study allowed for dosing schedule interruptions or pauses, if needed. To assess efficacy, the study looked at the percent change in fasting triglycerides at month 3 of the study in all randomized patients who received at least one dose of the study drug and who had baseline fasting plasma trigylcerides recorded.

Volanesorsen proved quite effective in reducing fasting triglyceride levels, with a 94% (P < 0.0001) reduction in plasma concentrations at 3 months, compared with placebo.

The effectiveness of volanesorsen extended beyond month 3, with statistically significant reductions in triglyceride concentrations at both month 6 and month 12 compared with placebo. Patients taking volanesorsen had 53% and 40% reductions at month 6 and 12 compared to baseline. Conversely, patients taking placebo experienced increases of 25% and 9%, respectively. The differences between volanesorsen and placebo patients at month 6 (–78%) and month 12 (–49%) were both statistically significant.

Another important finding from this study was that 77% of patients responded to treatment, evidenced by a reduction of triglyceride levels greater than 750 mg/dL, a much better response than the 10% of placebo patients (P =.0001).

Nine patients (27%) discontinued the study due to adverse events (AE). Five of the discontinuations were related to platelet reductions with the other four related to nonplatelet-related adverse events.

COMPASS

The COMPASS study primarily looked at patients without FCS, but severe hypertriglyceridemia, to evaluate the safety and efficacy of volanesorsen in a similarly ill, but less severe, population over a 6 month study period. Patients with FCS had baseline fasting plasma triglyceride levels of 2,644 mg/dL and 2,134 mg/dL in the placebo and volanesorsen groups, respectively. This is nearly double what was seen in non-FCS patients in the study, and is 14-17 higher than the upper limit of normal (ULN).
Researchers enrolled 114 patients and randomized them 2:1 to receive volanesorsen or placebo. This led to 75 patients taking volanesorsen and 34 placebo.
The efficacy results were similar to those in the FCS-focused APPROACH trial, with volanesorsen treatment leading to reductions in triglyceride levels after 3 months, with further reductions over the 6-month study treatment.
Weekly treatment with volanesorsen reduced triglycerides by nearly 71%  in the total patient population, compared to placebo, which only achieved a 0.9% decrease in triglycerides. These reductions were conserved at the end of week 26 in patients who had to reduce their dosing to biweekly (62%) and patients who maintained weekly dosing treatments (78%). Overall, a greater than 40% reduction of fasting triglyceride levels after 3 months of treatment was achieved in 87% of volanesorsen-treated patients, compared to 13% of placebo-treated patients (P < 0.0001).
Serious adverse events (SAEs) occurred in nine patients (8%). In the placebo group, two patients (5%) reported acute pancreatitis. Apart from the bouts of pancreatitis, some of the most common AEs were decreased platelet count, thrombocytopenia, and nasopharyngitis.

APPROACH OPEN LABEL

The open label portion of the APPROACH study is ongoing and was designed to assess the safety and efficacy of extended treatment with volanesorsen in patients with FCS who had previously completed APPROACH or COMPASS studies or had never received volanesorsen treatment. Patients received 300 mg of volanesorsen once weekly for 52 weeks. Patients who completed the trial were eligible to continue treatment for an additional 52 weeks or until a product is available. As of August 31, 2017, after the data cutoff, 60 patients were enrolled.
The results of this open label portion of the APPROACH trial were similar to that of the APPROACH trial itself. Patients who transitioned from APPROACH or COMPASS showed decreases in their open label extension (OLE) baseline triglyceride measurements to OLE month 3 of 48.1% and 52.1%, respectively. Treatment-naive patients displayed a nearly 60% decrease in their triglyceride levels 3 months.
Of the 60 patients who started the study, 12 have discontinued the study prematurely. The majority of patients, eight, who discontinued did so because of adverse events and four withdrew voluntarily.
Adverse events were not uncommon, with 56 patients (93%) experiencing an AE during the course of the study. Some of the most common AEs were decreased platelet count, thrombocytopenia, and nasopharyngitis.
Akcea has developed a Risk Evaluation and Mitigation program to mitigate the risk of serious bleeding related to severe thrombocytopenia with volanesorsen use in patients with FCS.
Panelist Susan Z. Yanovksi, MD, of the National Institutes of Health, said she voted no over safety concerns, but felt conflicted.
“There’s no question that volanesorsen is effective in dramatically reducing triglycerides, but I had a lot of concerns whether the data presented by the sponsor actually established favorable risk-benefit ratio.”

The FDA is expected to decide on the application by August 30. The FDA usually follows the recommendations of its advisory panels, which are not binding.

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NEW ORLEANS – A reasonable stepwise approach to pharmacotherapy for agitation in outpatient dementia begins with a judiciously chosen SSRI as first-line therapy, with the second-line consisting of augmentation with a benzodiazepine, M. Philip Luber, MD, said at the annual meeting of the American College of Physicians.

If this combination doesn’t provide satisfactory improvement after adequate dosing for sufficient duration, only then is it time to turn to an atypical antipsychotic agent as a higher-risk/higher-reward third-line therapy, added Dr. Luber, professor of psychiatry and associate dean for graduate medical education at the University of Texas, San Antonio.

Bruce Jancin/MDedge News

He emphasized, however, that medications are not the first-line therapy for agitation in outpatients with dementia. That distinction belongs to psychosocial interventions.

“Utilize a social worker to assist the family in modifying the environment: family settings and people, regular routines, consistently orienting the patient, keeping stimulation at a low to moderate level. When that’s done right, it can really dampen down a lot of agitation,” he said.

Dr. Luber noted that the formal randomized trial evidence base for SSRIs as first-line pharmacotherapy and benzodiazepines as second-line is quite limited. But in conversations with geriatric psychiatrists and other health care professionals around the country who are working in this area, he has found broad agreement that this is a good strategy.

“A lot of folks don’t realize that [SSRIs] are actually very useful in the treatment of agitation in dementia. But you want to choose one with fewer side effects and drug-drug interactions,” Dr. Luber said.

Two SSRIs that fit the bill are escitalopram (Lexapro) and sertraline (Zoloft). Start low and increase the dose slowly, provided circumstances allow. Expect a therapeutic response to emerge in weeks, not days. And, in this elderly population, be sure to monitor for hyponatremia.

[email protected]

NEW ORLEANS – A reasonable stepwise approach to pharmacotherapy for agitation in outpatient dementia begins with a judiciously chosen SSRI as first-line therapy, with the second-line consisting of augmentation with a benzodiazepine, M. Philip Luber, MD, said at the annual meeting of the American College of Physicians.

If this combination doesn’t provide satisfactory improvement after adequate dosing for sufficient duration, only then is it time to turn to an atypical antipsychotic agent as a higher-risk/higher-reward third-line therapy, added Dr. Luber, professor of psychiatry and associate dean for graduate medical education at the University of Texas, San Antonio.

Bruce Jancin/MDedge News

He emphasized, however, that medications are not the first-line therapy for agitation in outpatients with dementia. That distinction belongs to psychosocial interventions.

“Utilize a social worker to assist the family in modifying the environment: family settings and people, regular routines, consistently orienting the patient, keeping stimulation at a low to moderate level. When that’s done right, it can really dampen down a lot of agitation,” he said.

Dr. Luber noted that the formal randomized trial evidence base for SSRIs as first-line pharmacotherapy and benzodiazepines as second-line is quite limited. But in conversations with geriatric psychiatrists and other health care professionals around the country who are working in this area, he has found broad agreement that this is a good strategy.

“A lot of folks don’t realize that [SSRIs] are actually very useful in the treatment of agitation in dementia. But you want to choose one with fewer side effects and drug-drug interactions,” Dr. Luber said.

Two SSRIs that fit the bill are escitalopram (Lexapro) and sertraline (Zoloft). Start low and increase the dose slowly, provided circumstances allow. Expect a therapeutic response to emerge in weeks, not days. And, in this elderly population, be sure to monitor for hyponatremia.

[email protected]

NEW ORLEANS – A reasonable stepwise approach to pharmacotherapy for agitation in outpatient dementia begins with a judiciously chosen SSRI as first-line therapy, with the second-line consisting of augmentation with a benzodiazepine, M. Philip Luber, MD, said at the annual meeting of the American College of Physicians.

If this combination doesn’t provide satisfactory improvement after adequate dosing for sufficient duration, only then is it time to turn to an atypical antipsychotic agent as a higher-risk/higher-reward third-line therapy, added Dr. Luber, professor of psychiatry and associate dean for graduate medical education at the University of Texas, San Antonio.

Bruce Jancin/MDedge News

He emphasized, however, that medications are not the first-line therapy for agitation in outpatients with dementia. That distinction belongs to psychosocial interventions.

“Utilize a social worker to assist the family in modifying the environment: family settings and people, regular routines, consistently orienting the patient, keeping stimulation at a low to moderate level. When that’s done right, it can really dampen down a lot of agitation,” he said.

Dr. Luber noted that the formal randomized trial evidence base for SSRIs as first-line pharmacotherapy and benzodiazepines as second-line is quite limited. But in conversations with geriatric psychiatrists and other health care professionals around the country who are working in this area, he has found broad agreement that this is a good strategy.

“A lot of folks don’t realize that [SSRIs] are actually very useful in the treatment of agitation in dementia. But you want to choose one with fewer side effects and drug-drug interactions,” Dr. Luber said.

Two SSRIs that fit the bill are escitalopram (Lexapro) and sertraline (Zoloft). Start low and increase the dose slowly, provided circumstances allow. Expect a therapeutic response to emerge in weeks, not days. And, in this elderly population, be sure to monitor for hyponatremia.

[email protected]

BOSTON – Cardiac contractility modulation (CCM), an electrical device–based modality, may improve exercise tolerance and quality of life in heart failure patients with ejection fraction between 25% and 45%, results of a randomized, controlled trial suggested.

Notably, clinical effectiveness appeared to be even greater in the subset of patient with ejection fractions between 35% and 45%, according to results presented at the annual scientific sessions of the Heart Rhythm Society.

SOURCE: Abraham WT et al. JACC Heart Fail. 2018 May 10. doi: 10.1016/j.jchf.2018.04.010.

BOSTON – Cardiac contractility modulation (CCM), an electrical device–based modality, may improve exercise tolerance and quality of life in heart failure patients with ejection fraction between 25% and 45%, results of a randomized, controlled trial suggested.

Notably, clinical effectiveness appeared to be even greater in the subset of patient with ejection fractions between 35% and 45%, according to results presented at the annual scientific sessions of the Heart Rhythm Society.

SOURCE: Abraham WT et al. JACC Heart Fail. 2018 May 10. doi: 10.1016/j.jchf.2018.04.010.

BOSTON – Cardiac contractility modulation (CCM), an electrical device–based modality, may improve exercise tolerance and quality of life in heart failure patients with ejection fraction between 25% and 45%, results of a randomized, controlled trial suggested.

Notably, clinical effectiveness appeared to be even greater in the subset of patient with ejection fractions between 35% and 45%, according to results presented at the annual scientific sessions of the Heart Rhythm Society.

SOURCE: Abraham WT et al. JACC Heart Fail. 2018 May 10. doi: 10.1016/j.jchf.2018.04.010.

Although skin biopsy and the “presence of deep dermal mixed infiltrate with abundant neutrophils surrounding eccrine sweat glands” is considered the preferred method for diagnosing palmoplantar eccrine hidradenitis (PEH), Paola Piccini, MD, and her colleagues at the University of Florence (Italy) caution that biopsy frequently is not needed.

In the days prior to the appearance of erythematous and painful nodules on the soles of his feet, a healthy 8-year-old boy sustained “thermal and mechanical trauma playing football and cycling,” that made walking difficult. The week prior to the injury, he had complained of diarrhea in the absence of fever.

Ika84/iStock/Getty Images

No disclosures were noted.

SOURCE: Piccini, P et al. J. Pediatr. 2018. doi: 10.1016/j.peds.2018.03.017.

Although skin biopsy and the “presence of deep dermal mixed infiltrate with abundant neutrophils surrounding eccrine sweat glands” is considered the preferred method for diagnosing palmoplantar eccrine hidradenitis (PEH), Paola Piccini, MD, and her colleagues at the University of Florence (Italy) caution that biopsy frequently is not needed.

In the days prior to the appearance of erythematous and painful nodules on the soles of his feet, a healthy 8-year-old boy sustained “thermal and mechanical trauma playing football and cycling,” that made walking difficult. The week prior to the injury, he had complained of diarrhea in the absence of fever.

Ika84/iStock/Getty Images

No disclosures were noted.

SOURCE: Piccini, P et al. J. Pediatr. 2018. doi: 10.1016/j.peds.2018.03.017.

Although skin biopsy and the “presence of deep dermal mixed infiltrate with abundant neutrophils surrounding eccrine sweat glands” is considered the preferred method for diagnosing palmoplantar eccrine hidradenitis (PEH), Paola Piccini, MD, and her colleagues at the University of Florence (Italy) caution that biopsy frequently is not needed.

In the days prior to the appearance of erythematous and painful nodules on the soles of his feet, a healthy 8-year-old boy sustained “thermal and mechanical trauma playing football and cycling,” that made walking difficult. The week prior to the injury, he had complained of diarrhea in the absence of fever.

Ika84/iStock/Getty Images

No disclosures were noted.

SOURCE: Piccini, P et al. J. Pediatr. 2018. doi: 10.1016/j.peds.2018.03.017.

SAN DIEGO – Delaying surgery in certain cases of non–small cell lung cancer (NSCLC) can mean patients will be upstaged and consequently have worse prognoses, a study suggests.

“There is significant upstaging with time from completion of clinical staging to surgical resection, with a 4% increase of upstaging per week for the overall study population,” said study coauthor Harmik J. Soukiasian, MD, FACS, of Cedars-Sinai Medical Center, Los Angeles, in an interview. “Upstaging impacts lung cancer prognosis as more advanced stages portend to a poorer prognosis.”

SOURCE: Soukiasian HJ et al. AATS 2018, Abstract 67.

SAN DIEGO – Delaying surgery in certain cases of non–small cell lung cancer (NSCLC) can mean patients will be upstaged and consequently have worse prognoses, a study suggests.

“There is significant upstaging with time from completion of clinical staging to surgical resection, with a 4% increase of upstaging per week for the overall study population,” said study coauthor Harmik J. Soukiasian, MD, FACS, of Cedars-Sinai Medical Center, Los Angeles, in an interview. “Upstaging impacts lung cancer prognosis as more advanced stages portend to a poorer prognosis.”

SOURCE: Soukiasian HJ et al. AATS 2018, Abstract 67.

SAN DIEGO – Delaying surgery in certain cases of non–small cell lung cancer (NSCLC) can mean patients will be upstaged and consequently have worse prognoses, a study suggests.

“There is significant upstaging with time from completion of clinical staging to surgical resection, with a 4% increase of upstaging per week for the overall study population,” said study coauthor Harmik J. Soukiasian, MD, FACS, of Cedars-Sinai Medical Center, Los Angeles, in an interview. “Upstaging impacts lung cancer prognosis as more advanced stages portend to a poorer prognosis.”

SOURCE: Soukiasian HJ et al. AATS 2018, Abstract 67.

The White House is requesting cuts of about $7 billion to the Children’s Health Insurance Program as part of $15.4 billion budget cut using a rare maneuver known as rescission.

The package also includes an $800 million cut to the Centers for Medicare & Medicaid Innovation (CMMI), a policy incubator that the Centers for Medicare & Medicaid Services uses to test innovative payment approaches.

Kativ/iStockphoto

“While White House officials insist that the CHIP cuts would not harm access to care for children and families, that is simply not the case,” according to the letter signed by more than 500 organizations. “Our nation is facing an increasing number of natural disasters ... [and] each catastrophe leaves families more vulnerable and more likely to qualify for CHIP. In addition to natural disasters, the Child Enrollment Contingency Fund provides states needed protection and security should their CHIP enrollment suddenly spike due to an economic recession or a public health crisis.”

CMMI “is our best hope to innovate & achieve better health outcomes at lower costs,” Patrick Conway, MD, former deputy administrator and chief medical officer at CMS said in a May 8 tweet. “Cutting $800M or any amount would be a bad decision. CMS spends over $1 trillion per year; over $100M/hour. CMMI is key to better system.”

American Academy of Family Physicians President Michael Munger, MD, agreed.

“We’re also concerned about the $800 million rescission in funding for the CMS Innovation Center,” he said. “This initiative has been at the forefront of innovative demonstrations that can pave the way for a health care system that improves the quality of care and cuts health care spending.”

Congress has 45 days to act on the White House’s rescission proposal. If it takes no action, the proposal expires and the funds cannot be targeted by the same mechanism in the future. If Congress addresses the rescission request, it would need just a simple majority to pass each chamber.

Despite slashing more than $15 billion from the budget, the White House notes that the rescission request would reduce spending by only $3 billion.

[email protected]

The White House is requesting cuts of about $7 billion to the Children’s Health Insurance Program as part of $15.4 billion budget cut using a rare maneuver known as rescission.

The package also includes an $800 million cut to the Centers for Medicare & Medicaid Innovation (CMMI), a policy incubator that the Centers for Medicare & Medicaid Services uses to test innovative payment approaches.

Kativ/iStockphoto

“While White House officials insist that the CHIP cuts would not harm access to care for children and families, that is simply not the case,” according to the letter signed by more than 500 organizations. “Our nation is facing an increasing number of natural disasters ... [and] each catastrophe leaves families more vulnerable and more likely to qualify for CHIP. In addition to natural disasters, the Child Enrollment Contingency Fund provides states needed protection and security should their CHIP enrollment suddenly spike due to an economic recession or a public health crisis.”

CMMI “is our best hope to innovate & achieve better health outcomes at lower costs,” Patrick Conway, MD, former deputy administrator and chief medical officer at CMS said in a May 8 tweet. “Cutting $800M or any amount would be a bad decision. CMS spends over $1 trillion per year; over $100M/hour. CMMI is key to better system.”

American Academy of Family Physicians President Michael Munger, MD, agreed.

“We’re also concerned about the $800 million rescission in funding for the CMS Innovation Center,” he said. “This initiative has been at the forefront of innovative demonstrations that can pave the way for a health care system that improves the quality of care and cuts health care spending.”

Congress has 45 days to act on the White House’s rescission proposal. If it takes no action, the proposal expires and the funds cannot be targeted by the same mechanism in the future. If Congress addresses the rescission request, it would need just a simple majority to pass each chamber.

Despite slashing more than $15 billion from the budget, the White House notes that the rescission request would reduce spending by only $3 billion.

[email protected]

The White House is requesting cuts of about $7 billion to the Children’s Health Insurance Program as part of $15.4 billion budget cut using a rare maneuver known as rescission.

The package also includes an $800 million cut to the Centers for Medicare & Medicaid Innovation (CMMI), a policy incubator that the Centers for Medicare & Medicaid Services uses to test innovative payment approaches.

Kativ/iStockphoto

“While White House officials insist that the CHIP cuts would not harm access to care for children and families, that is simply not the case,” according to the letter signed by more than 500 organizations. “Our nation is facing an increasing number of natural disasters ... [and] each catastrophe leaves families more vulnerable and more likely to qualify for CHIP. In addition to natural disasters, the Child Enrollment Contingency Fund provides states needed protection and security should their CHIP enrollment suddenly spike due to an economic recession or a public health crisis.”

CMMI “is our best hope to innovate & achieve better health outcomes at lower costs,” Patrick Conway, MD, former deputy administrator and chief medical officer at CMS said in a May 8 tweet. “Cutting $800M or any amount would be a bad decision. CMS spends over $1 trillion per year; over $100M/hour. CMMI is key to better system.”

American Academy of Family Physicians President Michael Munger, MD, agreed.

“We’re also concerned about the $800 million rescission in funding for the CMS Innovation Center,” he said. “This initiative has been at the forefront of innovative demonstrations that can pave the way for a health care system that improves the quality of care and cuts health care spending.”

Congress has 45 days to act on the White House’s rescission proposal. If it takes no action, the proposal expires and the funds cannot be targeted by the same mechanism in the future. If Congress addresses the rescission request, it would need just a simple majority to pass each chamber.

Despite slashing more than $15 billion from the budget, the White House notes that the rescission request would reduce spending by only $3 billion.

[email protected]

The vast majority of oncologists discuss medical marijuana use with their patients, and around one-half recommend it to patients, yet many also say they do not feel equipped to make clinical recommendations on its use, new research has found.

A survey on medical marijuana was mailed to a nationally-representative, random sample of 400 medical oncologists and had a response rate of 63%; results from the 237 responders revealed that 79.8% had discussed medical marijuana use with patients or their families and 45.9% had recommended medical marijuana for cancer-related issues to at least one patient in the previous year.

Oncologists in the western United States were significantly more likely to recommend medical marijuana use, compared with those in the south of the country (84.2% vs. 34.7%, respectively; P less than .001), Ilana M. Braun, MD, and her associates reported in Journal of Clinical Oncology.

Doctors who practiced outside a hospital setting were also significantly more likely to recommend medical marijuana to their patients, as were medical oncologists with higher practice volumes.

Among the oncologists who reported discussing medical marijuana use with patients, 78% said these conversations were more likely to be initiated by the patient and their family than by the oncologist themselves.

However only 29.4% of oncologists surveyed said they felt “sufficiently knowledgeable” to make recommendations to patients about medical marijuana. Even among those who said they had recommended medical marijuana to a patient in the past year, 56.2% said they didn’t feel they had enough knowledge to make a recommendation.

Overall, oncologists had mixed views about medical marijuana. About one-third viewed it as equal to or more effective than standard pain treatments, one-third viewed it as less effective, and one-third said they did not know. However two-thirds viewed medical marijuana as a useful adjunct to standard pain therapies.

Two-thirds of oncologists surveyed believed medical marijuana was as good as or better than standard treatments for poor appetite or cachexia, but less than half felt it was equal to or better than standard antinausea therapies.

The data revealed a “clinically problematic discrepancy” between medical oncologists’ their perceived knowledge about medical marijuana use and their actual beliefs and practices, said Dr. Braun of the Dana-Farber Cancer Institute and her associates.

“Although our survey could not determine why oncologists recommend medical marijuana, it may be because they regard medical marijuana as an alternative therapy that is difficult to evaluate given sparse randomized, controlled trial data,” they wrote.

“[The results] highlight a crucial need for expedited clinical trials exploring marijuana’s potential medicinal effects in oncology (e.g. as an adjunctive pain management strategy or as a treatment of anorexia/cachexia) and the need for educational programs about medical marijuana to inform oncologists who frequently confront questions regarding medical marijuana in daily practice.”

No funding was declared. Two authors declared royalties and honoraria from medical publishing and a research institute, and one declared fees for expert testimony.

SOURCE: Braun I et al. J Clin Oncol. 2018 May 10. doi: 10.1200/JCO.2017.76.1221.
 

The vast majority of oncologists discuss medical marijuana use with their patients, and around one-half recommend it to patients, yet many also say they do not feel equipped to make clinical recommendations on its use, new research has found.

A survey on medical marijuana was mailed to a nationally-representative, random sample of 400 medical oncologists and had a response rate of 63%; results from the 237 responders revealed that 79.8% had discussed medical marijuana use with patients or their families and 45.9% had recommended medical marijuana for cancer-related issues to at least one patient in the previous year.

Oncologists in the western United States were significantly more likely to recommend medical marijuana use, compared with those in the south of the country (84.2% vs. 34.7%, respectively; P less than .001), Ilana M. Braun, MD, and her associates reported in Journal of Clinical Oncology.

Doctors who practiced outside a hospital setting were also significantly more likely to recommend medical marijuana to their patients, as were medical oncologists with higher practice volumes.

Among the oncologists who reported discussing medical marijuana use with patients, 78% said these conversations were more likely to be initiated by the patient and their family than by the oncologist themselves.

However only 29.4% of oncologists surveyed said they felt “sufficiently knowledgeable” to make recommendations to patients about medical marijuana. Even among those who said they had recommended medical marijuana to a patient in the past year, 56.2% said they didn’t feel they had enough knowledge to make a recommendation.

Overall, oncologists had mixed views about medical marijuana. About one-third viewed it as equal to or more effective than standard pain treatments, one-third viewed it as less effective, and one-third said they did not know. However two-thirds viewed medical marijuana as a useful adjunct to standard pain therapies.

Two-thirds of oncologists surveyed believed medical marijuana was as good as or better than standard treatments for poor appetite or cachexia, but less than half felt it was equal to or better than standard antinausea therapies.

The data revealed a “clinically problematic discrepancy” between medical oncologists’ their perceived knowledge about medical marijuana use and their actual beliefs and practices, said Dr. Braun of the Dana-Farber Cancer Institute and her associates.

“Although our survey could not determine why oncologists recommend medical marijuana, it may be because they regard medical marijuana as an alternative therapy that is difficult to evaluate given sparse randomized, controlled trial data,” they wrote.

“[The results] highlight a crucial need for expedited clinical trials exploring marijuana’s potential medicinal effects in oncology (e.g. as an adjunctive pain management strategy or as a treatment of anorexia/cachexia) and the need for educational programs about medical marijuana to inform oncologists who frequently confront questions regarding medical marijuana in daily practice.”

No funding was declared. Two authors declared royalties and honoraria from medical publishing and a research institute, and one declared fees for expert testimony.

SOURCE: Braun I et al. J Clin Oncol. 2018 May 10. doi: 10.1200/JCO.2017.76.1221.
 

The vast majority of oncologists discuss medical marijuana use with their patients, and around one-half recommend it to patients, yet many also say they do not feel equipped to make clinical recommendations on its use, new research has found.

A survey on medical marijuana was mailed to a nationally-representative, random sample of 400 medical oncologists and had a response rate of 63%; results from the 237 responders revealed that 79.8% had discussed medical marijuana use with patients or their families and 45.9% had recommended medical marijuana for cancer-related issues to at least one patient in the previous year.

Oncologists in the western United States were significantly more likely to recommend medical marijuana use, compared with those in the south of the country (84.2% vs. 34.7%, respectively; P less than .001), Ilana M. Braun, MD, and her associates reported in Journal of Clinical Oncology.

Doctors who practiced outside a hospital setting were also significantly more likely to recommend medical marijuana to their patients, as were medical oncologists with higher practice volumes.

Among the oncologists who reported discussing medical marijuana use with patients, 78% said these conversations were more likely to be initiated by the patient and their family than by the oncologist themselves.

However only 29.4% of oncologists surveyed said they felt “sufficiently knowledgeable” to make recommendations to patients about medical marijuana. Even among those who said they had recommended medical marijuana to a patient in the past year, 56.2% said they didn’t feel they had enough knowledge to make a recommendation.

Overall, oncologists had mixed views about medical marijuana. About one-third viewed it as equal to or more effective than standard pain treatments, one-third viewed it as less effective, and one-third said they did not know. However two-thirds viewed medical marijuana as a useful adjunct to standard pain therapies.

Two-thirds of oncologists surveyed believed medical marijuana was as good as or better than standard treatments for poor appetite or cachexia, but less than half felt it was equal to or better than standard antinausea therapies.

The data revealed a “clinically problematic discrepancy” between medical oncologists’ their perceived knowledge about medical marijuana use and their actual beliefs and practices, said Dr. Braun of the Dana-Farber Cancer Institute and her associates.

“Although our survey could not determine why oncologists recommend medical marijuana, it may be because they regard medical marijuana as an alternative therapy that is difficult to evaluate given sparse randomized, controlled trial data,” they wrote.

“[The results] highlight a crucial need for expedited clinical trials exploring marijuana’s potential medicinal effects in oncology (e.g. as an adjunctive pain management strategy or as a treatment of anorexia/cachexia) and the need for educational programs about medical marijuana to inform oncologists who frequently confront questions regarding medical marijuana in daily practice.”

No funding was declared. Two authors declared royalties and honoraria from medical publishing and a research institute, and one declared fees for expert testimony.

SOURCE: Braun I et al. J Clin Oncol. 2018 May 10. doi: 10.1200/JCO.2017.76.1221.
 

NEW YORK – The Mental Health Parity and Addiction Equity Act, passed in 2008 and strengthened within the Affordable Care Act, continues to be routinely violated, but there are avenues for complaint that can help keep pressure on third-party payers, according to an update on this topic presented at the annual meeting of the American Psychiatric Association.

“There are basically two approaches: One is the top-down approach, which is actual government enforcement; the other is the bottom-up approach, which is to work through the courts with class action suits,” reported Daniel Knoepflmacher, MD, assistant professor of clinical psychiatry at Cornell University, New York.

NEW YORK – The Mental Health Parity and Addiction Equity Act, passed in 2008 and strengthened within the Affordable Care Act, continues to be routinely violated, but there are avenues for complaint that can help keep pressure on third-party payers, according to an update on this topic presented at the annual meeting of the American Psychiatric Association.

“There are basically two approaches: One is the top-down approach, which is actual government enforcement; the other is the bottom-up approach, which is to work through the courts with class action suits,” reported Daniel Knoepflmacher, MD, assistant professor of clinical psychiatry at Cornell University, New York.

NEW YORK – The Mental Health Parity and Addiction Equity Act, passed in 2008 and strengthened within the Affordable Care Act, continues to be routinely violated, but there are avenues for complaint that can help keep pressure on third-party payers, according to an update on this topic presented at the annual meeting of the American Psychiatric Association.

“There are basically two approaches: One is the top-down approach, which is actual government enforcement; the other is the bottom-up approach, which is to work through the courts with class action suits,” reported Daniel Knoepflmacher, MD, assistant professor of clinical psychiatry at Cornell University, New York.