Male HPV Vaccination Rates Soar With Intensive QI Project

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BALTIMORE – A 3-year quality improvement (QI) measure succeeded in delivering the full human papillomavirus series to more than four out of five eligible boys.

The high rate of completed vaccinations – nearly triple the national average – was accomplished at a clinic with a high-need population that includes many newly-arrived immigrants, said Dr. Pilar Gonzalez, a pediatrician at Mount Sinai Health System, New York.

©dina2001/thinkstockphotos.com

The improvements occurred against the backdrop of a clinic where physicians and staff already had a very strong commitment to achieving high immunization rates, Dr. Gonzalez said during a poster session at the annual meeting of the Pediatric Academic Societies. “We feel this is one of the most important things we can do for our population.”

To assess the efficacy of the pediatric primary care QI program, Dr. Gonzalez and her coinvestigators conducted a retrospective chart review to assess how many of the clinic’s male patients aged 13-17 years had initiated or completed the HPV series. The chart review looked back to Jan. 1, 2012, a full year before the QI initiative was implemented, and ended in December 2015.

Implementing the comprehensive QI program first involved engaging clinic staff, patients, and families through an educational curriculum that gave them facts about the HPV vaccine, and also provided information about oral, genital, and cervical cancer risks from HPV infection. The second arm of the QI project was outreach to the community. This included such interventions as Saturday vaccination clinics focused on delivering the full series to the target patients.

At the end of 2012, 50% of the 731 eligible male patients had received the full HPV vaccination series. One year later, 67% had completed the series and 24% more had started the series, for a total of 91% of eligible male patients who had received at least one HPV immunization. After 2 years of the QI project, a total of 93% of the eligible male patients had at least begun the HPV series.

The final data collection in December 2015, after a full 3 years of the QI project, showed that nearly all eligible male patients – 97% – had received at least one dose of the HPV vaccination.

“At the time we designed the QI project, we did not have the data” showing good efficacy with fewer doses than the full series of three immunizations, said Dr. Gonzalez, so the initiative was focused on getting all eligible adolescent males to completion of the series.

“Educating clinical staff and families, routinely offering the vaccine during clinic visits, sending patient reminders and recalls, and creating a catch-up immunization clinic helped increase the rate of HPV vaccination,” wrote Dr. Gonzalez and her coauthors, emphasizing that success of the effort in a high-need community was really a team effort.

Dr. Gonzalez and her coauthors reported no conflicts of interest.

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BALTIMORE – A 3-year quality improvement (QI) measure succeeded in delivering the full human papillomavirus series to more than four out of five eligible boys.

The high rate of completed vaccinations – nearly triple the national average – was accomplished at a clinic with a high-need population that includes many newly-arrived immigrants, said Dr. Pilar Gonzalez, a pediatrician at Mount Sinai Health System, New York.

©dina2001/thinkstockphotos.com

The improvements occurred against the backdrop of a clinic where physicians and staff already had a very strong commitment to achieving high immunization rates, Dr. Gonzalez said during a poster session at the annual meeting of the Pediatric Academic Societies. “We feel this is one of the most important things we can do for our population.”

To assess the efficacy of the pediatric primary care QI program, Dr. Gonzalez and her coinvestigators conducted a retrospective chart review to assess how many of the clinic’s male patients aged 13-17 years had initiated or completed the HPV series. The chart review looked back to Jan. 1, 2012, a full year before the QI initiative was implemented, and ended in December 2015.

Implementing the comprehensive QI program first involved engaging clinic staff, patients, and families through an educational curriculum that gave them facts about the HPV vaccine, and also provided information about oral, genital, and cervical cancer risks from HPV infection. The second arm of the QI project was outreach to the community. This included such interventions as Saturday vaccination clinics focused on delivering the full series to the target patients.

At the end of 2012, 50% of the 731 eligible male patients had received the full HPV vaccination series. One year later, 67% had completed the series and 24% more had started the series, for a total of 91% of eligible male patients who had received at least one HPV immunization. After 2 years of the QI project, a total of 93% of the eligible male patients had at least begun the HPV series.

The final data collection in December 2015, after a full 3 years of the QI project, showed that nearly all eligible male patients – 97% – had received at least one dose of the HPV vaccination.

“At the time we designed the QI project, we did not have the data” showing good efficacy with fewer doses than the full series of three immunizations, said Dr. Gonzalez, so the initiative was focused on getting all eligible adolescent males to completion of the series.

“Educating clinical staff and families, routinely offering the vaccine during clinic visits, sending patient reminders and recalls, and creating a catch-up immunization clinic helped increase the rate of HPV vaccination,” wrote Dr. Gonzalez and her coauthors, emphasizing that success of the effort in a high-need community was really a team effort.

Dr. Gonzalez and her coauthors reported no conflicts of interest.

BALTIMORE – A 3-year quality improvement (QI) measure succeeded in delivering the full human papillomavirus series to more than four out of five eligible boys.

The high rate of completed vaccinations – nearly triple the national average – was accomplished at a clinic with a high-need population that includes many newly-arrived immigrants, said Dr. Pilar Gonzalez, a pediatrician at Mount Sinai Health System, New York.

©dina2001/thinkstockphotos.com

The improvements occurred against the backdrop of a clinic where physicians and staff already had a very strong commitment to achieving high immunization rates, Dr. Gonzalez said during a poster session at the annual meeting of the Pediatric Academic Societies. “We feel this is one of the most important things we can do for our population.”

To assess the efficacy of the pediatric primary care QI program, Dr. Gonzalez and her coinvestigators conducted a retrospective chart review to assess how many of the clinic’s male patients aged 13-17 years had initiated or completed the HPV series. The chart review looked back to Jan. 1, 2012, a full year before the QI initiative was implemented, and ended in December 2015.

Implementing the comprehensive QI program first involved engaging clinic staff, patients, and families through an educational curriculum that gave them facts about the HPV vaccine, and also provided information about oral, genital, and cervical cancer risks from HPV infection. The second arm of the QI project was outreach to the community. This included such interventions as Saturday vaccination clinics focused on delivering the full series to the target patients.

At the end of 2012, 50% of the 731 eligible male patients had received the full HPV vaccination series. One year later, 67% had completed the series and 24% more had started the series, for a total of 91% of eligible male patients who had received at least one HPV immunization. After 2 years of the QI project, a total of 93% of the eligible male patients had at least begun the HPV series.

The final data collection in December 2015, after a full 3 years of the QI project, showed that nearly all eligible male patients – 97% – had received at least one dose of the HPV vaccination.

“At the time we designed the QI project, we did not have the data” showing good efficacy with fewer doses than the full series of three immunizations, said Dr. Gonzalez, so the initiative was focused on getting all eligible adolescent males to completion of the series.

“Educating clinical staff and families, routinely offering the vaccine during clinic visits, sending patient reminders and recalls, and creating a catch-up immunization clinic helped increase the rate of HPV vaccination,” wrote Dr. Gonzalez and her coauthors, emphasizing that success of the effort in a high-need community was really a team effort.

Dr. Gonzalez and her coauthors reported no conflicts of interest.

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Male HPV Vaccination Rates Soar With Intensive QI Project
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Male HPV vaccination rates soar with intensive QI project

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BALTIMORE – A 3-year quality improvement (QI) measure succeeded in delivering the full human papillomavirus series to more than four out of five eligible boys.

The high rate of completed vaccinations – nearly triple the national average – was accomplished at a clinic with a high-need population that includes many newly-arrived immigrants, said Dr. Pilar Gonzalez, a pediatrician at Mount Sinai Health System, New York.

©dina2001/thinkstockphotos.com

The improvements occurred against the backdrop of a clinic where physicians and staff already had a very strong commitment to achieving high immunization rates, Dr. Gonzalez said during a poster session at the annual meeting of the Pediatric Academic Societies. “We feel this is one of the most important things we can do for our population.”

To assess the efficacy of the pediatric primary care QI program, Dr. Gonzalez and her coinvestigators conducted a retrospective chart review to assess how many of the clinic’s male patients aged 13-17 years had initiated or completed the HPV series. The chart review looked back to Jan. 1, 2012, a full year before the QI initiative was implemented, and ended in December 2015.

Implementing the comprehensive QI program first involved engaging clinic staff, patients, and families through an educational curriculum that gave them facts about the HPV vaccine, and also provided information about oral, genital, and cervical cancer risks from HPV infection. The second arm of the QI project was outreach to the community. This included such interventions as Saturday vaccination clinics focused on delivering the full series to the target patients.

At the end of 2012, 50% of the 731 eligible male patients had received the full HPV vaccination series. One year later, 67% had completed the series and 24% more had started the series, for a total of 91% of eligible male patients who had received at least one HPV immunization. After 2 years of the QI project, a total of 93% of the eligible male patients had at least begun the HPV series.

The final data collection in December 2015, after a full 3 years of the QI project, showed that nearly all eligible male patients – 97% – had received at least one dose of the HPV vaccination.

“At the time we designed the QI project, we did not have the data” showing good efficacy with fewer doses than the full series of three immunizations, said Dr. Gonzalez, so the initiative was focused on getting all eligible adolescent males to completion of the series.

“Educating clinical staff and families, routinely offering the vaccine during clinic visits, sending patient reminders and recalls, and creating a catch-up immunization clinic helped increase the rate of HPV vaccination,” wrote Dr. Gonzalez and her coauthors, emphasizing that success of the effort in a high-need community was really a team effort.

Dr. Gonzalez and her coauthors reported no conflicts of interest.

[email protected]

On Twitter @karioakes

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BALTIMORE – A 3-year quality improvement (QI) measure succeeded in delivering the full human papillomavirus series to more than four out of five eligible boys.

The high rate of completed vaccinations – nearly triple the national average – was accomplished at a clinic with a high-need population that includes many newly-arrived immigrants, said Dr. Pilar Gonzalez, a pediatrician at Mount Sinai Health System, New York.

©dina2001/thinkstockphotos.com

The improvements occurred against the backdrop of a clinic where physicians and staff already had a very strong commitment to achieving high immunization rates, Dr. Gonzalez said during a poster session at the annual meeting of the Pediatric Academic Societies. “We feel this is one of the most important things we can do for our population.”

To assess the efficacy of the pediatric primary care QI program, Dr. Gonzalez and her coinvestigators conducted a retrospective chart review to assess how many of the clinic’s male patients aged 13-17 years had initiated or completed the HPV series. The chart review looked back to Jan. 1, 2012, a full year before the QI initiative was implemented, and ended in December 2015.

Implementing the comprehensive QI program first involved engaging clinic staff, patients, and families through an educational curriculum that gave them facts about the HPV vaccine, and also provided information about oral, genital, and cervical cancer risks from HPV infection. The second arm of the QI project was outreach to the community. This included such interventions as Saturday vaccination clinics focused on delivering the full series to the target patients.

At the end of 2012, 50% of the 731 eligible male patients had received the full HPV vaccination series. One year later, 67% had completed the series and 24% more had started the series, for a total of 91% of eligible male patients who had received at least one HPV immunization. After 2 years of the QI project, a total of 93% of the eligible male patients had at least begun the HPV series.

The final data collection in December 2015, after a full 3 years of the QI project, showed that nearly all eligible male patients – 97% – had received at least one dose of the HPV vaccination.

“At the time we designed the QI project, we did not have the data” showing good efficacy with fewer doses than the full series of three immunizations, said Dr. Gonzalez, so the initiative was focused on getting all eligible adolescent males to completion of the series.

“Educating clinical staff and families, routinely offering the vaccine during clinic visits, sending patient reminders and recalls, and creating a catch-up immunization clinic helped increase the rate of HPV vaccination,” wrote Dr. Gonzalez and her coauthors, emphasizing that success of the effort in a high-need community was really a team effort.

Dr. Gonzalez and her coauthors reported no conflicts of interest.

[email protected]

On Twitter @karioakes

BALTIMORE – A 3-year quality improvement (QI) measure succeeded in delivering the full human papillomavirus series to more than four out of five eligible boys.

The high rate of completed vaccinations – nearly triple the national average – was accomplished at a clinic with a high-need population that includes many newly-arrived immigrants, said Dr. Pilar Gonzalez, a pediatrician at Mount Sinai Health System, New York.

©dina2001/thinkstockphotos.com

The improvements occurred against the backdrop of a clinic where physicians and staff already had a very strong commitment to achieving high immunization rates, Dr. Gonzalez said during a poster session at the annual meeting of the Pediatric Academic Societies. “We feel this is one of the most important things we can do for our population.”

To assess the efficacy of the pediatric primary care QI program, Dr. Gonzalez and her coinvestigators conducted a retrospective chart review to assess how many of the clinic’s male patients aged 13-17 years had initiated or completed the HPV series. The chart review looked back to Jan. 1, 2012, a full year before the QI initiative was implemented, and ended in December 2015.

Implementing the comprehensive QI program first involved engaging clinic staff, patients, and families through an educational curriculum that gave them facts about the HPV vaccine, and also provided information about oral, genital, and cervical cancer risks from HPV infection. The second arm of the QI project was outreach to the community. This included such interventions as Saturday vaccination clinics focused on delivering the full series to the target patients.

At the end of 2012, 50% of the 731 eligible male patients had received the full HPV vaccination series. One year later, 67% had completed the series and 24% more had started the series, for a total of 91% of eligible male patients who had received at least one HPV immunization. After 2 years of the QI project, a total of 93% of the eligible male patients had at least begun the HPV series.

The final data collection in December 2015, after a full 3 years of the QI project, showed that nearly all eligible male patients – 97% – had received at least one dose of the HPV vaccination.

“At the time we designed the QI project, we did not have the data” showing good efficacy with fewer doses than the full series of three immunizations, said Dr. Gonzalez, so the initiative was focused on getting all eligible adolescent males to completion of the series.

“Educating clinical staff and families, routinely offering the vaccine during clinic visits, sending patient reminders and recalls, and creating a catch-up immunization clinic helped increase the rate of HPV vaccination,” wrote Dr. Gonzalez and her coauthors, emphasizing that success of the effort in a high-need community was really a team effort.

Dr. Gonzalez and her coauthors reported no conflicts of interest.

[email protected]

On Twitter @karioakes

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Key clinical point: A multipronged QI project boosted male human papillomavirus (HPV) vaccination rates in a high-need population.

Major finding: The percentage of eligible male patients who completed the HPV series climbed from 50% to 86% after a 3-year QI initiative.

Data source: Retrospective chart review of eligible male patients at a university-associated pediatric primary care clinic before, during, and after a QI project to boost HPV vaccination rates in eligible male patients.

Disclosures: The study investigators reported no disclosures.

Paperwork snarls stand between kids and at-school asthma medications

Statewide forms for children with asthma would be helpful
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Paperwork snarls stand between kids and at-school asthma medications

BALTIMORE – Four out of five children with asthma didn’t have access to their medication at school because the proper paperwork was missing, according to a survey of 10 inner-city Milwaukee elementary schools.

The number of students who had the required physician-signed authorization forms remained low throughout the school year, said Dr. Santiago Encalada, a pulmonary fellow at the Medical College of Wisconsin, Milwaukee.

Dr. Encalada cited administrative hurdles, lack of standardization, and challenges in school-physician-family communication as barriers to children’s access to asthma medication at school. Although school nurses in Milwaukee have standing orders for emergency albuterol administration, they otherwise need physician signatures on school-generated forms to administer both rescue and prophylactic asthma administration.

Kari Oakes/Frontline Medical News
Dr. Santiago Encalada

In a study whose purpose was to assess the percentage of children with asthma who had appropriate orders on file in a sample of 10 Milwaukee inner-city schools, the schools had orders on file for just 11% of students, on average, at the beginning of the 2014-2015 school year. At the second assessment in January 2015, the average number of students with orders on file at each school had risen to 22%, with schools that had performed better earlier also showing greater gains at mid-year. However, the June 2015 assessment showed that the gains did not continue, with the schools’ aggregate average of 21% of students with appropriate orders showing no improvement from mid-year.

The number of students with asthma in schools varied from about 40 to nearly 200. Numbers varied through the school year as enrollments shifted in these high-need schools, said Dr. Encalada, who presented his findings during a poster session at the annual meeting of the Pediatric Academic Societies. In general, the schools with lower enrollments tended to do better with having orders on file, although statistical analysis was not performed for this variable.

“On average, 80% of asthmatic students in the inner city schools we studied did not have school forms or orders available for life-saving asthma rescue medications, with significant variation between schools. Our findings show that access to even basic asthma care necessities are lagging for this vulnerable population, and a significant disparity exists even within this population,” said senior author Nicholas Antos*, associate director of the Cystic Fibrosis Center at Milwaukee’s Children’s Hospital of Wisconsin.

In interviews and discussion with school nurses and physicians’ offices, Dr. Antos* and Dr. Encalada found that there were often simple but fundamental misunderstandings that impeded the proper flow of paperwork. For example, schools in Milwaukee do not have standardized forms that authorize administration of prescription medications at school, so forms may be confusing to providers and their staff. Privacy concerns sometimes impeded the ability of clinic staff to authorize treatment for students. Also, the inevitable shuffle of paperwork in school-aged families meant that the forms sometimes were simply lost on the way to school.

Understanding the barriers in the process both on the school side and in physician offices has helped Dr. Antos*, Dr. Encalada, and their colleagues to start to build a better pathway. For example, a module has been built into the EHR asthma visit template that allows easy generation of a school form and asks for patient consent for release of information to the schools.

Dr. Antos* said in an interview that the work is ongoing: “To help address these problems, we have devised interventions to improve the way school nurses can contact clinicians, and helped design innovative standardized Asthma Action Plans that can double as school orders.”

In addition to working with local providers and schools, Dr. Encalada and Dr. Antos* have reached out to pediatric societies and the American Academy of Asthma, Allergy, and Immunology (AAAAI). Emphasizing the need for “education of stakeholders of all types,” Dr. Antos* said that change “may be difficult, but we hope with the support of pediatric organizations, the AAAAI, and school administrators, we can begin to break down the barriers preventing quality and timely communication with school nurses.”

The authors had no financial disclosures. The study was funded by the Centers for Disease Control and Prevention through the Wisconsin Asthma Coalition (WAC).

[email protected]

On Twitter @karioakes

*In a previous version, Dr. Antos' name was misspelled.

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Dr. Susan Millard, FCCP: comments: The issues identified in this article are huge and not just an occurrence in the inner cities.  The critical problem is that the children are even more at risk when living in the inner cities and for sudden death due to asthma.  Having one form for the whole state would help tremendously because we could print out an asthma action plan and the form for the school and then fax it directly!

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Dr. Susan Millard, FCCP: comments: The issues identified in this article are huge and not just an occurrence in the inner cities.  The critical problem is that the children are even more at risk when living in the inner cities and for sudden death due to asthma.  Having one form for the whole state would help tremendously because we could print out an asthma action plan and the form for the school and then fax it directly!

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Dr. Susan Millard, FCCP: comments: The issues identified in this article are huge and not just an occurrence in the inner cities.  The critical problem is that the children are even more at risk when living in the inner cities and for sudden death due to asthma.  Having one form for the whole state would help tremendously because we could print out an asthma action plan and the form for the school and then fax it directly!

Title
Statewide forms for children with asthma would be helpful
Statewide forms for children with asthma would be helpful

BALTIMORE – Four out of five children with asthma didn’t have access to their medication at school because the proper paperwork was missing, according to a survey of 10 inner-city Milwaukee elementary schools.

The number of students who had the required physician-signed authorization forms remained low throughout the school year, said Dr. Santiago Encalada, a pulmonary fellow at the Medical College of Wisconsin, Milwaukee.

Dr. Encalada cited administrative hurdles, lack of standardization, and challenges in school-physician-family communication as barriers to children’s access to asthma medication at school. Although school nurses in Milwaukee have standing orders for emergency albuterol administration, they otherwise need physician signatures on school-generated forms to administer both rescue and prophylactic asthma administration.

Kari Oakes/Frontline Medical News
Dr. Santiago Encalada

In a study whose purpose was to assess the percentage of children with asthma who had appropriate orders on file in a sample of 10 Milwaukee inner-city schools, the schools had orders on file for just 11% of students, on average, at the beginning of the 2014-2015 school year. At the second assessment in January 2015, the average number of students with orders on file at each school had risen to 22%, with schools that had performed better earlier also showing greater gains at mid-year. However, the June 2015 assessment showed that the gains did not continue, with the schools’ aggregate average of 21% of students with appropriate orders showing no improvement from mid-year.

The number of students with asthma in schools varied from about 40 to nearly 200. Numbers varied through the school year as enrollments shifted in these high-need schools, said Dr. Encalada, who presented his findings during a poster session at the annual meeting of the Pediatric Academic Societies. In general, the schools with lower enrollments tended to do better with having orders on file, although statistical analysis was not performed for this variable.

“On average, 80% of asthmatic students in the inner city schools we studied did not have school forms or orders available for life-saving asthma rescue medications, with significant variation between schools. Our findings show that access to even basic asthma care necessities are lagging for this vulnerable population, and a significant disparity exists even within this population,” said senior author Nicholas Antos*, associate director of the Cystic Fibrosis Center at Milwaukee’s Children’s Hospital of Wisconsin.

In interviews and discussion with school nurses and physicians’ offices, Dr. Antos* and Dr. Encalada found that there were often simple but fundamental misunderstandings that impeded the proper flow of paperwork. For example, schools in Milwaukee do not have standardized forms that authorize administration of prescription medications at school, so forms may be confusing to providers and their staff. Privacy concerns sometimes impeded the ability of clinic staff to authorize treatment for students. Also, the inevitable shuffle of paperwork in school-aged families meant that the forms sometimes were simply lost on the way to school.

Understanding the barriers in the process both on the school side and in physician offices has helped Dr. Antos*, Dr. Encalada, and their colleagues to start to build a better pathway. For example, a module has been built into the EHR asthma visit template that allows easy generation of a school form and asks for patient consent for release of information to the schools.

Dr. Antos* said in an interview that the work is ongoing: “To help address these problems, we have devised interventions to improve the way school nurses can contact clinicians, and helped design innovative standardized Asthma Action Plans that can double as school orders.”

In addition to working with local providers and schools, Dr. Encalada and Dr. Antos* have reached out to pediatric societies and the American Academy of Asthma, Allergy, and Immunology (AAAAI). Emphasizing the need for “education of stakeholders of all types,” Dr. Antos* said that change “may be difficult, but we hope with the support of pediatric organizations, the AAAAI, and school administrators, we can begin to break down the barriers preventing quality and timely communication with school nurses.”

The authors had no financial disclosures. The study was funded by the Centers for Disease Control and Prevention through the Wisconsin Asthma Coalition (WAC).

[email protected]

On Twitter @karioakes

*In a previous version, Dr. Antos' name was misspelled.

BALTIMORE – Four out of five children with asthma didn’t have access to their medication at school because the proper paperwork was missing, according to a survey of 10 inner-city Milwaukee elementary schools.

The number of students who had the required physician-signed authorization forms remained low throughout the school year, said Dr. Santiago Encalada, a pulmonary fellow at the Medical College of Wisconsin, Milwaukee.

Dr. Encalada cited administrative hurdles, lack of standardization, and challenges in school-physician-family communication as barriers to children’s access to asthma medication at school. Although school nurses in Milwaukee have standing orders for emergency albuterol administration, they otherwise need physician signatures on school-generated forms to administer both rescue and prophylactic asthma administration.

Kari Oakes/Frontline Medical News
Dr. Santiago Encalada

In a study whose purpose was to assess the percentage of children with asthma who had appropriate orders on file in a sample of 10 Milwaukee inner-city schools, the schools had orders on file for just 11% of students, on average, at the beginning of the 2014-2015 school year. At the second assessment in January 2015, the average number of students with orders on file at each school had risen to 22%, with schools that had performed better earlier also showing greater gains at mid-year. However, the June 2015 assessment showed that the gains did not continue, with the schools’ aggregate average of 21% of students with appropriate orders showing no improvement from mid-year.

The number of students with asthma in schools varied from about 40 to nearly 200. Numbers varied through the school year as enrollments shifted in these high-need schools, said Dr. Encalada, who presented his findings during a poster session at the annual meeting of the Pediatric Academic Societies. In general, the schools with lower enrollments tended to do better with having orders on file, although statistical analysis was not performed for this variable.

“On average, 80% of asthmatic students in the inner city schools we studied did not have school forms or orders available for life-saving asthma rescue medications, with significant variation between schools. Our findings show that access to even basic asthma care necessities are lagging for this vulnerable population, and a significant disparity exists even within this population,” said senior author Nicholas Antos*, associate director of the Cystic Fibrosis Center at Milwaukee’s Children’s Hospital of Wisconsin.

In interviews and discussion with school nurses and physicians’ offices, Dr. Antos* and Dr. Encalada found that there were often simple but fundamental misunderstandings that impeded the proper flow of paperwork. For example, schools in Milwaukee do not have standardized forms that authorize administration of prescription medications at school, so forms may be confusing to providers and their staff. Privacy concerns sometimes impeded the ability of clinic staff to authorize treatment for students. Also, the inevitable shuffle of paperwork in school-aged families meant that the forms sometimes were simply lost on the way to school.

Understanding the barriers in the process both on the school side and in physician offices has helped Dr. Antos*, Dr. Encalada, and their colleagues to start to build a better pathway. For example, a module has been built into the EHR asthma visit template that allows easy generation of a school form and asks for patient consent for release of information to the schools.

Dr. Antos* said in an interview that the work is ongoing: “To help address these problems, we have devised interventions to improve the way school nurses can contact clinicians, and helped design innovative standardized Asthma Action Plans that can double as school orders.”

In addition to working with local providers and schools, Dr. Encalada and Dr. Antos* have reached out to pediatric societies and the American Academy of Asthma, Allergy, and Immunology (AAAAI). Emphasizing the need for “education of stakeholders of all types,” Dr. Antos* said that change “may be difficult, but we hope with the support of pediatric organizations, the AAAAI, and school administrators, we can begin to break down the barriers preventing quality and timely communication with school nurses.”

The authors had no financial disclosures. The study was funded by the Centers for Disease Control and Prevention through the Wisconsin Asthma Coalition (WAC).

[email protected]

On Twitter @karioakes

*In a previous version, Dr. Antos' name was misspelled.

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Key clinical point: Four out of five high-risk elementary school children lacked proper orders for at-school asthma medication administration.

Major finding: The average number of elementary school children with asthma medication orders on file was 21% at year’s end.

Data source: Yearlong study of 10 inner-city Milwaukee elementary schools; enrollees with asthma ranged from about 40 to nearly 200.

Disclosures: The study was funded by the Centers for Disease Control and Prevention through the Wisconsin Asthma Coalition (WAC).

mHealth shows promise in getting teens moving

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mHealth shows promise in getting teens moving

BALTIMORE – More than two-thirds of teens consistently wore and synced a digital activity tracker in a small study of an integrating mobile health (mHealth) approach for teens who had or were at risk to acquire obesity.

The devices were well accepted by the adolescent participants, whose activity increased for several months after beginning the study. Of the teens who participated for the full 9 months of the study, 85% of them had maintained or decreased their body mass index.

Dr. Raquel Hernandez

Set in a high school in St. Petersburg, Fla., an intervention called “Teens Tracking for Health,” or TT4H, examined 9th graders with a BMI greater than the 85th percentile, Dr. Raquel Hernandez said at the annual meeting of the Pediatric Academic Societies. The study population was identified through a schoolwide health assessment.

“You wouldn’t be surprised to learn that physical activity exposure was quite minimal” at the participants’ high school, said Dr. Hernandez, professor of pediatrics at Johns Hopkins University, based at All Children’s Hospital in St. Petersburg. “We were also not surprised, but concerned, to learn that the prevalence of morbid obesity in this student body was over 9%, which is twice what is observed nationally.”

Nutrition and fitness sessions were conducted within the school day during lunch hours. Motivational counseling was offered twice a month after school.

Each student received individual tailored texts, as well as group texts reinforcing the content delivered in group sessions. Investigators created Instagram and Twitter accounts to help the teens connect as well.

All participants received a digital tracking device – a Fitbit Flex – that was used for the duration of the study period. The step goal for participants was set at 8,000 steps per day.

Participants were also asked to track food intake via MyFitnessPal.

The 18 participants were mostly African American (11, 61%) and female (11, 61%), and most (11, 61%) participated in the free and reduced lunch program.

“There were significant fluctuations in activity of the entire group based on seasonality,” noted Dr. Hernandez. Dips in activity were seen during schoolwide standardized testing, during the winter holiday, and at the beginning of summer.

The greatest activity and consistency in syncing devices was seen in the first 20 weeks of the 9-month program.

“We noted an increase in activity in our male participants of about 1,000 steps early in the study; that, unfortunately, waned as we followed the teens,” said Dr. Hernandez. Female participants also had a progressive decline in activity over the course of the study. Overall, females were substantially less active than males in the study; data analysis is ongoing to quantify the difference and to identify associations with the lower activity level.

Data from individual activity monitors were exported into the research database whenever a device was synced, allowing for real time monitoring and feedback for participants in the form of individualized texts.

Messaging in the texts sent to students was congratulatory and encouraging, rather than nagging, Dr. Hernandez said in an interview. “So if a participant was 2,000 steps away from their goal for the day, we could send them a text cheering them on and encouraging them to be a little more active for the rest of the day,” she said.

Dietary tracking was “problematic” among the adolescent participants. “Teens had many reasons for not logging their food, primarily confusion and uncertainty in terms of logging their food in the app,” said Dr. Hernandez. Efforts at further education were unsuccessful in improving the rate of diet tracking.

The study was funded by a Building Healthy Communities Grant from the Florida Blue Foundation.

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BALTIMORE – More than two-thirds of teens consistently wore and synced a digital activity tracker in a small study of an integrating mobile health (mHealth) approach for teens who had or were at risk to acquire obesity.

The devices were well accepted by the adolescent participants, whose activity increased for several months after beginning the study. Of the teens who participated for the full 9 months of the study, 85% of them had maintained or decreased their body mass index.

Dr. Raquel Hernandez

Set in a high school in St. Petersburg, Fla., an intervention called “Teens Tracking for Health,” or TT4H, examined 9th graders with a BMI greater than the 85th percentile, Dr. Raquel Hernandez said at the annual meeting of the Pediatric Academic Societies. The study population was identified through a schoolwide health assessment.

“You wouldn’t be surprised to learn that physical activity exposure was quite minimal” at the participants’ high school, said Dr. Hernandez, professor of pediatrics at Johns Hopkins University, based at All Children’s Hospital in St. Petersburg. “We were also not surprised, but concerned, to learn that the prevalence of morbid obesity in this student body was over 9%, which is twice what is observed nationally.”

Nutrition and fitness sessions were conducted within the school day during lunch hours. Motivational counseling was offered twice a month after school.

Each student received individual tailored texts, as well as group texts reinforcing the content delivered in group sessions. Investigators created Instagram and Twitter accounts to help the teens connect as well.

All participants received a digital tracking device – a Fitbit Flex – that was used for the duration of the study period. The step goal for participants was set at 8,000 steps per day.

Participants were also asked to track food intake via MyFitnessPal.

The 18 participants were mostly African American (11, 61%) and female (11, 61%), and most (11, 61%) participated in the free and reduced lunch program.

“There were significant fluctuations in activity of the entire group based on seasonality,” noted Dr. Hernandez. Dips in activity were seen during schoolwide standardized testing, during the winter holiday, and at the beginning of summer.

The greatest activity and consistency in syncing devices was seen in the first 20 weeks of the 9-month program.

“We noted an increase in activity in our male participants of about 1,000 steps early in the study; that, unfortunately, waned as we followed the teens,” said Dr. Hernandez. Female participants also had a progressive decline in activity over the course of the study. Overall, females were substantially less active than males in the study; data analysis is ongoing to quantify the difference and to identify associations with the lower activity level.

Data from individual activity monitors were exported into the research database whenever a device was synced, allowing for real time monitoring and feedback for participants in the form of individualized texts.

Messaging in the texts sent to students was congratulatory and encouraging, rather than nagging, Dr. Hernandez said in an interview. “So if a participant was 2,000 steps away from their goal for the day, we could send them a text cheering them on and encouraging them to be a little more active for the rest of the day,” she said.

Dietary tracking was “problematic” among the adolescent participants. “Teens had many reasons for not logging their food, primarily confusion and uncertainty in terms of logging their food in the app,” said Dr. Hernandez. Efforts at further education were unsuccessful in improving the rate of diet tracking.

The study was funded by a Building Healthy Communities Grant from the Florida Blue Foundation.

BALTIMORE – More than two-thirds of teens consistently wore and synced a digital activity tracker in a small study of an integrating mobile health (mHealth) approach for teens who had or were at risk to acquire obesity.

The devices were well accepted by the adolescent participants, whose activity increased for several months after beginning the study. Of the teens who participated for the full 9 months of the study, 85% of them had maintained or decreased their body mass index.

Dr. Raquel Hernandez

Set in a high school in St. Petersburg, Fla., an intervention called “Teens Tracking for Health,” or TT4H, examined 9th graders with a BMI greater than the 85th percentile, Dr. Raquel Hernandez said at the annual meeting of the Pediatric Academic Societies. The study population was identified through a schoolwide health assessment.

“You wouldn’t be surprised to learn that physical activity exposure was quite minimal” at the participants’ high school, said Dr. Hernandez, professor of pediatrics at Johns Hopkins University, based at All Children’s Hospital in St. Petersburg. “We were also not surprised, but concerned, to learn that the prevalence of morbid obesity in this student body was over 9%, which is twice what is observed nationally.”

Nutrition and fitness sessions were conducted within the school day during lunch hours. Motivational counseling was offered twice a month after school.

Each student received individual tailored texts, as well as group texts reinforcing the content delivered in group sessions. Investigators created Instagram and Twitter accounts to help the teens connect as well.

All participants received a digital tracking device – a Fitbit Flex – that was used for the duration of the study period. The step goal for participants was set at 8,000 steps per day.

Participants were also asked to track food intake via MyFitnessPal.

The 18 participants were mostly African American (11, 61%) and female (11, 61%), and most (11, 61%) participated in the free and reduced lunch program.

“There were significant fluctuations in activity of the entire group based on seasonality,” noted Dr. Hernandez. Dips in activity were seen during schoolwide standardized testing, during the winter holiday, and at the beginning of summer.

The greatest activity and consistency in syncing devices was seen in the first 20 weeks of the 9-month program.

“We noted an increase in activity in our male participants of about 1,000 steps early in the study; that, unfortunately, waned as we followed the teens,” said Dr. Hernandez. Female participants also had a progressive decline in activity over the course of the study. Overall, females were substantially less active than males in the study; data analysis is ongoing to quantify the difference and to identify associations with the lower activity level.

Data from individual activity monitors were exported into the research database whenever a device was synced, allowing for real time monitoring and feedback for participants in the form of individualized texts.

Messaging in the texts sent to students was congratulatory and encouraging, rather than nagging, Dr. Hernandez said in an interview. “So if a participant was 2,000 steps away from their goal for the day, we could send them a text cheering them on and encouraging them to be a little more active for the rest of the day,” she said.

Dietary tracking was “problematic” among the adolescent participants. “Teens had many reasons for not logging their food, primarily confusion and uncertainty in terms of logging their food in the app,” said Dr. Hernandez. Efforts at further education were unsuccessful in improving the rate of diet tracking.

The study was funded by a Building Healthy Communities Grant from the Florida Blue Foundation.

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Key clinical point: More than two-thirds of teens consistently wore and synced a digital activity tracker in a pilot study.

Major finding: Body mass index was maintained or decreased in 85% of participating teens over a 9-month period.

Data source: A pilot study of 18 at-risk 9th graders who participated in a school-based program that integrated face-to-face counseling with mobile health (mHealth) approaches.

Disclosures: The study was funded by a grant from the Florida Blue Foundation.

Reframing the problem seen as way to ease inpatient bed shortage

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Reframing the problem seen as way to ease inpatient bed shortage

If an individual with schizophrenia presents to the emergency department, there’s about a 1 in 2 chance that person will wind up in an inpatient psychiatric bed, or transferred to a residential psychiatric facility. As reimbursement to hospitals for psychiatric beds decreases, there’s decreasing incentive for hospitals to maintain inpatient psychiatry services.

Decreasing numbers of hospital beds means strategic thinking about outpatient services is more important than ever, to help avert the crises that bring patients to EDs and to run-ins with the justice system. In some parts of the country, though, the downstream effects of cutbacks and increased demand are overwhelming the system.

For Dr. Carl C. Bell, the combination of shrinking resources and growing need feels like a prescription for disaster in Chicago. Dr. Bell, a psychiatrist who has spent decades providing community mental health services there, saw a relatively robust mental health infrastructure crumble when municipal belt tightening resulted in the consolidation of 13 mental health centers down to just 6.

As individuals with serious mental illness lost access to such outpatient resources as therapy, medication management, supported housing, and employment assistance, jail populations swelled. The Cook County jail became known as “the largest mental health center in the state of Illinois,” said Dr. Bell. He’s not sure he sees a good solution for the near term, but he holds out hope that innovative solutions are on the horizon.

Telepsychiatry offers an eminently workable solution to scarcity and geographic separation in some areas. Dr. David Baldes, a psychiatrist at St. Luke’s Health Care System in Duluth, Minn., “sees” patients via his computer several hours a week. He’s able to care for the sickest of the patients with mental illness served by primary care clinics along the Iron Range in northern Minnesota, helping keep this population out of the emergency department and fending off brushes with the law that are all too common among those with serious mental illnesses such as schizophrenia and severe bipolar disorder.

Erin Metzger
A rural primary care doctor consults with Dr. David Baldes, a psychiatrist in Minnesota.

“The people I see tend to be really sick,” said Dr. Baldes, “and the number of psychiatrists per capita is basically zero” on the Iron Range. Although the area is served by a federally funded community mental health center, it’s extremely difficult to attract and retain psychiatrists to the remote area.

His ability to provide care for patients with serious mental illness helps their primary care providers “not feel so much like they’re on an island,” he said. He enjoys the collaboration and support he’s able to provide for the primary physicians as well.

Getting things started wasn’t hard: “The technology was actually quite simple to set up,” he said, noting that psychiatry is an ideal discipline for virtual care. “We don’t touch the patient. Our exam is our conversation with the patient,” he said.

Another advantage of telepsychiatry, Dr. Baldes said, is that there’s no stigma associated with visiting one’s primary care provider. “My patients go to their regular doctor’s office, they check in with the receptionist, and nobody really knows why they are there.” This can be a particular advantage in some of the more conservative rural communities served by the St. Luke’s program.

This mode of care soon feels completely natural for physician and patient, he said. “Especially for our generation; we’re very comfortable with FaceTime, with Skype, and generally with communicating electronically,” Dr. Baldes said.

“What patients really want is to be able to do these visits from their home,” he said. Because of privacy and security concerns, patients still go to the primary care office to have their virtual visits with Dr. Baldes.

Telepsychiatry’s promise is not limited to rural areas. “Any time people are resource limited, transportation is always an issue,” Dr. Baldes said. The suburbs and exurbs of many American cities are increasingly populated by low-income individuals forced out of gentrifying city centers into areas with fewer mental health resources and fewer transportation options. Telepsychiatry could be useful in many settings, he said.

A more fully integrated suite of services, the Collaborative Care Model (CCM), has been piloted in five locations nationwide and was the subject of an April 14, 2016, congressional briefing. This care model goes beyond co-location and collaboration to encompass a specific set of team members providing specific services, with ongoing tracking of validated outcome measures.

Dr. Erik Vanderlip, professor of psychiatry and medical informatics at the University of Oklahoma, Tulsa, coauthored a recent report sharing evidence of the successful implementation of collaborative care. He said the CCM really represents a shift in thinking. “The lack of psychiatric beds isn’t the problem. The problem is the lack of affordable, accessible, high-quality mental health services,” and collaborative care seeks to meet that need.

 

 

Dr. Vanderlip is a double-boarded psychiatrist and family medicine physician; he said that during training, “I discovered quickly that we have to redesign the way we deliver health care services to meet the needs of the most vulnerable.” He began working with Dr. Wayne Katon, now deceased, who pioneered the collaborative care model in Washington state.

In practice, this means that a psychiatrist works with a primary care provider and other team members to provide intensive care and monitoring. Clinical trials have shown impressive results in the treatment of depression, with response rates approaching 70%, Dr. Vanderlip said. “This stuff is the solution,” he said.

“So you have these little ‘teamlets’ of the psychiatrist, the primary care provider, the care manager, and the nurse working together to take care of a cohort of patients,” Dr. Vanderlip said. Typically, a care manager will have from 40 to as many as 100 patients under his or her care.

Key to measuring the success of the care model is an objective, validated measure that changes in relation to improvement or worsening of the target chronic condition. For example, in depression, that measure is the Patient Health Questionnaire (PHQ-9).

In the CCM, a psychiatrist will log in to the secure patient management system and pull up the entire registry of the care manager’s patients. One by one, patients are briefly reviewed, and the care plan and medications are adjusted as needed. The psychiatrist completes a brief note for each patient during the session; notes have a disclaimer that makes clear that the physician did not have a face-to-face encounter with the patient.

The psychiatrists also are available for “curbside” consults to the primary care provider, so they may collaborate on patients’ care plans. For one care manager’s panel of 40-100 patients, a psychiatrist will typically devote about a half day per week of consultative time.

Dr. Vanderlip has found that for some psychiatrists, the new role of “care quarterback” can be a tough sell. “Providers have a hard time comprehending that they are not going to see people directly.” Most psychiatrists involved in collaborative care also see patients in the traditional model as well, he said.

A critical piece of the puzzle for the success of integrated care is reimbursement – and the CCM now has its own CPT code. “There’s reimbursement for the psychiatrist’s time, for the care manager’s time, and for the primary care provider’s time,” Dr. Vanderlip said. The American Psychiatric Association is in discussion with the Centers for Medicare & Medicaid Services and the American Medical Association to fine-tune valuation.

“This is a great candidate for value-based reimbursement,” Dr. Vanderlip said. Depression scores can be tracked over time; successful care teams could be rewarded – and less successful ones docked – depending on patient outcome measures.

As reimbursers seek to find more ways to recognize the burden that chronic care places on the health care system, collaborative care should find more takers. “Collaborative care is chronic care incarnate,” Dr. Vanderlip said. He said he thinks it’s the solution for the care crunch in America. “This is not a bed shortage problem,” he reiterated.

Availability of inpatient services wide ranging

The number of psychiatric hospital beds per capita varies widely by state, as does the availability of psychiatrists and outpatient mental health facilities. In 2011, the American Hospital Association reported that psychiatric bed allocations ranged from a low of about 5 beds per 100,000 persons in Colorado to a high of more than 50 beds per 100,000 persons in both Missouri and Mississippi.

Reported rates of hospital admission among adults with a diagnosis of any mental illness also varies, from 1.1% in Louisiana, to 4.9% in New York (2010-2011 Substance Abuse and Mental Health Services Administration report).

State-by-state estimates of the prevalence of serious mental illness in adults ranges from just under 3% to about 7% (2012 revised SAMHSA report).

[email protected]

On Twitter @karioakes

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If an individual with schizophrenia presents to the emergency department, there’s about a 1 in 2 chance that person will wind up in an inpatient psychiatric bed, or transferred to a residential psychiatric facility. As reimbursement to hospitals for psychiatric beds decreases, there’s decreasing incentive for hospitals to maintain inpatient psychiatry services.

Decreasing numbers of hospital beds means strategic thinking about outpatient services is more important than ever, to help avert the crises that bring patients to EDs and to run-ins with the justice system. In some parts of the country, though, the downstream effects of cutbacks and increased demand are overwhelming the system.

For Dr. Carl C. Bell, the combination of shrinking resources and growing need feels like a prescription for disaster in Chicago. Dr. Bell, a psychiatrist who has spent decades providing community mental health services there, saw a relatively robust mental health infrastructure crumble when municipal belt tightening resulted in the consolidation of 13 mental health centers down to just 6.

As individuals with serious mental illness lost access to such outpatient resources as therapy, medication management, supported housing, and employment assistance, jail populations swelled. The Cook County jail became known as “the largest mental health center in the state of Illinois,” said Dr. Bell. He’s not sure he sees a good solution for the near term, but he holds out hope that innovative solutions are on the horizon.

Telepsychiatry offers an eminently workable solution to scarcity and geographic separation in some areas. Dr. David Baldes, a psychiatrist at St. Luke’s Health Care System in Duluth, Minn., “sees” patients via his computer several hours a week. He’s able to care for the sickest of the patients with mental illness served by primary care clinics along the Iron Range in northern Minnesota, helping keep this population out of the emergency department and fending off brushes with the law that are all too common among those with serious mental illnesses such as schizophrenia and severe bipolar disorder.

Erin Metzger
A rural primary care doctor consults with Dr. David Baldes, a psychiatrist in Minnesota.

“The people I see tend to be really sick,” said Dr. Baldes, “and the number of psychiatrists per capita is basically zero” on the Iron Range. Although the area is served by a federally funded community mental health center, it’s extremely difficult to attract and retain psychiatrists to the remote area.

His ability to provide care for patients with serious mental illness helps their primary care providers “not feel so much like they’re on an island,” he said. He enjoys the collaboration and support he’s able to provide for the primary physicians as well.

Getting things started wasn’t hard: “The technology was actually quite simple to set up,” he said, noting that psychiatry is an ideal discipline for virtual care. “We don’t touch the patient. Our exam is our conversation with the patient,” he said.

Another advantage of telepsychiatry, Dr. Baldes said, is that there’s no stigma associated with visiting one’s primary care provider. “My patients go to their regular doctor’s office, they check in with the receptionist, and nobody really knows why they are there.” This can be a particular advantage in some of the more conservative rural communities served by the St. Luke’s program.

This mode of care soon feels completely natural for physician and patient, he said. “Especially for our generation; we’re very comfortable with FaceTime, with Skype, and generally with communicating electronically,” Dr. Baldes said.

“What patients really want is to be able to do these visits from their home,” he said. Because of privacy and security concerns, patients still go to the primary care office to have their virtual visits with Dr. Baldes.

Telepsychiatry’s promise is not limited to rural areas. “Any time people are resource limited, transportation is always an issue,” Dr. Baldes said. The suburbs and exurbs of many American cities are increasingly populated by low-income individuals forced out of gentrifying city centers into areas with fewer mental health resources and fewer transportation options. Telepsychiatry could be useful in many settings, he said.

A more fully integrated suite of services, the Collaborative Care Model (CCM), has been piloted in five locations nationwide and was the subject of an April 14, 2016, congressional briefing. This care model goes beyond co-location and collaboration to encompass a specific set of team members providing specific services, with ongoing tracking of validated outcome measures.

Dr. Erik Vanderlip, professor of psychiatry and medical informatics at the University of Oklahoma, Tulsa, coauthored a recent report sharing evidence of the successful implementation of collaborative care. He said the CCM really represents a shift in thinking. “The lack of psychiatric beds isn’t the problem. The problem is the lack of affordable, accessible, high-quality mental health services,” and collaborative care seeks to meet that need.

 

 

Dr. Vanderlip is a double-boarded psychiatrist and family medicine physician; he said that during training, “I discovered quickly that we have to redesign the way we deliver health care services to meet the needs of the most vulnerable.” He began working with Dr. Wayne Katon, now deceased, who pioneered the collaborative care model in Washington state.

In practice, this means that a psychiatrist works with a primary care provider and other team members to provide intensive care and monitoring. Clinical trials have shown impressive results in the treatment of depression, with response rates approaching 70%, Dr. Vanderlip said. “This stuff is the solution,” he said.

“So you have these little ‘teamlets’ of the psychiatrist, the primary care provider, the care manager, and the nurse working together to take care of a cohort of patients,” Dr. Vanderlip said. Typically, a care manager will have from 40 to as many as 100 patients under his or her care.

Key to measuring the success of the care model is an objective, validated measure that changes in relation to improvement or worsening of the target chronic condition. For example, in depression, that measure is the Patient Health Questionnaire (PHQ-9).

In the CCM, a psychiatrist will log in to the secure patient management system and pull up the entire registry of the care manager’s patients. One by one, patients are briefly reviewed, and the care plan and medications are adjusted as needed. The psychiatrist completes a brief note for each patient during the session; notes have a disclaimer that makes clear that the physician did not have a face-to-face encounter with the patient.

The psychiatrists also are available for “curbside” consults to the primary care provider, so they may collaborate on patients’ care plans. For one care manager’s panel of 40-100 patients, a psychiatrist will typically devote about a half day per week of consultative time.

Dr. Vanderlip has found that for some psychiatrists, the new role of “care quarterback” can be a tough sell. “Providers have a hard time comprehending that they are not going to see people directly.” Most psychiatrists involved in collaborative care also see patients in the traditional model as well, he said.

A critical piece of the puzzle for the success of integrated care is reimbursement – and the CCM now has its own CPT code. “There’s reimbursement for the psychiatrist’s time, for the care manager’s time, and for the primary care provider’s time,” Dr. Vanderlip said. The American Psychiatric Association is in discussion with the Centers for Medicare & Medicaid Services and the American Medical Association to fine-tune valuation.

“This is a great candidate for value-based reimbursement,” Dr. Vanderlip said. Depression scores can be tracked over time; successful care teams could be rewarded – and less successful ones docked – depending on patient outcome measures.

As reimbursers seek to find more ways to recognize the burden that chronic care places on the health care system, collaborative care should find more takers. “Collaborative care is chronic care incarnate,” Dr. Vanderlip said. He said he thinks it’s the solution for the care crunch in America. “This is not a bed shortage problem,” he reiterated.

Availability of inpatient services wide ranging

The number of psychiatric hospital beds per capita varies widely by state, as does the availability of psychiatrists and outpatient mental health facilities. In 2011, the American Hospital Association reported that psychiatric bed allocations ranged from a low of about 5 beds per 100,000 persons in Colorado to a high of more than 50 beds per 100,000 persons in both Missouri and Mississippi.

Reported rates of hospital admission among adults with a diagnosis of any mental illness also varies, from 1.1% in Louisiana, to 4.9% in New York (2010-2011 Substance Abuse and Mental Health Services Administration report).

State-by-state estimates of the prevalence of serious mental illness in adults ranges from just under 3% to about 7% (2012 revised SAMHSA report).

[email protected]

On Twitter @karioakes

If an individual with schizophrenia presents to the emergency department, there’s about a 1 in 2 chance that person will wind up in an inpatient psychiatric bed, or transferred to a residential psychiatric facility. As reimbursement to hospitals for psychiatric beds decreases, there’s decreasing incentive for hospitals to maintain inpatient psychiatry services.

Decreasing numbers of hospital beds means strategic thinking about outpatient services is more important than ever, to help avert the crises that bring patients to EDs and to run-ins with the justice system. In some parts of the country, though, the downstream effects of cutbacks and increased demand are overwhelming the system.

For Dr. Carl C. Bell, the combination of shrinking resources and growing need feels like a prescription for disaster in Chicago. Dr. Bell, a psychiatrist who has spent decades providing community mental health services there, saw a relatively robust mental health infrastructure crumble when municipal belt tightening resulted in the consolidation of 13 mental health centers down to just 6.

As individuals with serious mental illness lost access to such outpatient resources as therapy, medication management, supported housing, and employment assistance, jail populations swelled. The Cook County jail became known as “the largest mental health center in the state of Illinois,” said Dr. Bell. He’s not sure he sees a good solution for the near term, but he holds out hope that innovative solutions are on the horizon.

Telepsychiatry offers an eminently workable solution to scarcity and geographic separation in some areas. Dr. David Baldes, a psychiatrist at St. Luke’s Health Care System in Duluth, Minn., “sees” patients via his computer several hours a week. He’s able to care for the sickest of the patients with mental illness served by primary care clinics along the Iron Range in northern Minnesota, helping keep this population out of the emergency department and fending off brushes with the law that are all too common among those with serious mental illnesses such as schizophrenia and severe bipolar disorder.

Erin Metzger
A rural primary care doctor consults with Dr. David Baldes, a psychiatrist in Minnesota.

“The people I see tend to be really sick,” said Dr. Baldes, “and the number of psychiatrists per capita is basically zero” on the Iron Range. Although the area is served by a federally funded community mental health center, it’s extremely difficult to attract and retain psychiatrists to the remote area.

His ability to provide care for patients with serious mental illness helps their primary care providers “not feel so much like they’re on an island,” he said. He enjoys the collaboration and support he’s able to provide for the primary physicians as well.

Getting things started wasn’t hard: “The technology was actually quite simple to set up,” he said, noting that psychiatry is an ideal discipline for virtual care. “We don’t touch the patient. Our exam is our conversation with the patient,” he said.

Another advantage of telepsychiatry, Dr. Baldes said, is that there’s no stigma associated with visiting one’s primary care provider. “My patients go to their regular doctor’s office, they check in with the receptionist, and nobody really knows why they are there.” This can be a particular advantage in some of the more conservative rural communities served by the St. Luke’s program.

This mode of care soon feels completely natural for physician and patient, he said. “Especially for our generation; we’re very comfortable with FaceTime, with Skype, and generally with communicating electronically,” Dr. Baldes said.

“What patients really want is to be able to do these visits from their home,” he said. Because of privacy and security concerns, patients still go to the primary care office to have their virtual visits with Dr. Baldes.

Telepsychiatry’s promise is not limited to rural areas. “Any time people are resource limited, transportation is always an issue,” Dr. Baldes said. The suburbs and exurbs of many American cities are increasingly populated by low-income individuals forced out of gentrifying city centers into areas with fewer mental health resources and fewer transportation options. Telepsychiatry could be useful in many settings, he said.

A more fully integrated suite of services, the Collaborative Care Model (CCM), has been piloted in five locations nationwide and was the subject of an April 14, 2016, congressional briefing. This care model goes beyond co-location and collaboration to encompass a specific set of team members providing specific services, with ongoing tracking of validated outcome measures.

Dr. Erik Vanderlip, professor of psychiatry and medical informatics at the University of Oklahoma, Tulsa, coauthored a recent report sharing evidence of the successful implementation of collaborative care. He said the CCM really represents a shift in thinking. “The lack of psychiatric beds isn’t the problem. The problem is the lack of affordable, accessible, high-quality mental health services,” and collaborative care seeks to meet that need.

 

 

Dr. Vanderlip is a double-boarded psychiatrist and family medicine physician; he said that during training, “I discovered quickly that we have to redesign the way we deliver health care services to meet the needs of the most vulnerable.” He began working with Dr. Wayne Katon, now deceased, who pioneered the collaborative care model in Washington state.

In practice, this means that a psychiatrist works with a primary care provider and other team members to provide intensive care and monitoring. Clinical trials have shown impressive results in the treatment of depression, with response rates approaching 70%, Dr. Vanderlip said. “This stuff is the solution,” he said.

“So you have these little ‘teamlets’ of the psychiatrist, the primary care provider, the care manager, and the nurse working together to take care of a cohort of patients,” Dr. Vanderlip said. Typically, a care manager will have from 40 to as many as 100 patients under his or her care.

Key to measuring the success of the care model is an objective, validated measure that changes in relation to improvement or worsening of the target chronic condition. For example, in depression, that measure is the Patient Health Questionnaire (PHQ-9).

In the CCM, a psychiatrist will log in to the secure patient management system and pull up the entire registry of the care manager’s patients. One by one, patients are briefly reviewed, and the care plan and medications are adjusted as needed. The psychiatrist completes a brief note for each patient during the session; notes have a disclaimer that makes clear that the physician did not have a face-to-face encounter with the patient.

The psychiatrists also are available for “curbside” consults to the primary care provider, so they may collaborate on patients’ care plans. For one care manager’s panel of 40-100 patients, a psychiatrist will typically devote about a half day per week of consultative time.

Dr. Vanderlip has found that for some psychiatrists, the new role of “care quarterback” can be a tough sell. “Providers have a hard time comprehending that they are not going to see people directly.” Most psychiatrists involved in collaborative care also see patients in the traditional model as well, he said.

A critical piece of the puzzle for the success of integrated care is reimbursement – and the CCM now has its own CPT code. “There’s reimbursement for the psychiatrist’s time, for the care manager’s time, and for the primary care provider’s time,” Dr. Vanderlip said. The American Psychiatric Association is in discussion with the Centers for Medicare & Medicaid Services and the American Medical Association to fine-tune valuation.

“This is a great candidate for value-based reimbursement,” Dr. Vanderlip said. Depression scores can be tracked over time; successful care teams could be rewarded – and less successful ones docked – depending on patient outcome measures.

As reimbursers seek to find more ways to recognize the burden that chronic care places on the health care system, collaborative care should find more takers. “Collaborative care is chronic care incarnate,” Dr. Vanderlip said. He said he thinks it’s the solution for the care crunch in America. “This is not a bed shortage problem,” he reiterated.

Availability of inpatient services wide ranging

The number of psychiatric hospital beds per capita varies widely by state, as does the availability of psychiatrists and outpatient mental health facilities. In 2011, the American Hospital Association reported that psychiatric bed allocations ranged from a low of about 5 beds per 100,000 persons in Colorado to a high of more than 50 beds per 100,000 persons in both Missouri and Mississippi.

Reported rates of hospital admission among adults with a diagnosis of any mental illness also varies, from 1.1% in Louisiana, to 4.9% in New York (2010-2011 Substance Abuse and Mental Health Services Administration report).

State-by-state estimates of the prevalence of serious mental illness in adults ranges from just under 3% to about 7% (2012 revised SAMHSA report).

[email protected]

On Twitter @karioakes

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References

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Neoadjuvant chemotherapy improves survival for some with pancreatic cancer

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Neoadjuvant chemotherapy improves survival for some with pancreatic cancer

MONTREAL – For individuals with stage III pancreatic cancer, neoadjuvant therapy may improve survival, compared with surgery-first treatment. “This study supports, however does not prove, the hypothesis that early and continued control of occult metastatic disease prolongs survival in surgical patients,” said Dr. Christopher Shubert.

Dr. Shubert and his colleagues used an intention-to-treat (ITT) analysis to compare overall survival (OS) for 377 patients who were to receive neoadjuvant chemotherapy with 216 patients who received surgery first. Median OS for the neoadjuvant group was 20.7 months, compared with 13.7 months for the surgery-first group (log-rank P less than .0001).

Courtesy Dr. Lance Liotta Laboratory

This study was the first to utilize national data to look at how patients who received neoadjuvant chemotherapy for stage III pancreatic cancer fared, when compared with those treated with a surgery-first approach, Dr. Shubert, a general surgery resident at the Mayo Clinic in Rochester, Minn., said at the annual meeting of the Central Surgical Association.

Stage III pancreatic cancer (T4, any N, M0) means that the cancer has invaded the celiac trunk, or there is superior mesenteric artery involvement, he noted.

Using data from the National Cancer Database from 2002 to 2011, the investigators identified patients with clinical stage III pancreatic adenocarcinoma of the head or body of the pancreas. The ITT neoadjuvant therapy cohort included all patients whose treatment recommendations included curative-intent surgery and neoadjuvant chemotherapy, regardless of what therapies the patients received. The surgery-first cohort included those who were recommended to receive adjuvant therapy.

A total of 593 patients were identified, of whom 377 (63.5%) were in the neoadjuvant group. Of these, 104 (27.6%) were lost to presurgical attrition. The surgery-first group included 216 patients (36.3%), 30 (13.9%) of whom did not receive the intended adjuvant chemotherapy. Comparing the two ITT groups yielded an adjusted hazard ratio of 0.68 (P = .001).

A secondary aim of the study was to see which aspects of therapy, and which pathologic features, were associated with longer OS. The addition of postsurgical therapy was associated with additional survival benefit (31.6 vs. 22.6 months for no postsurgical therapy; HR, 0.60; P = .002). Node-negative and R0 status were both also significantly increased among those receiving neoadjuvant chemotherapy, and both disease characteristics were associated with increased OS, he reported.

Dr. Shubert said that study limitations included its review of a prospective database. Also, investigators could not determine the type or duration of chemotherapy; they also were unable to tell when systemic chemotherapy plus chemoradiation or just chemoradiation alone had been used. No recurrence data were available, and all cases were grouped under one procedure code, limiting information about vascular resections.

“Neoadjuvant therapy may offer survival advantages compared to a surgery-first approach,” said Dr. Shubert, and control of small occult metastases may be the mechanism behind this advantage. Still to be determined, however, are the optimal type, duration, and sequencing of neoadjuvant chemotherapy and chemoradiation, he said.

Dr. Shubert reported no relevant disclosures.

[email protected]

On Twitter @karioakes

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MONTREAL – For individuals with stage III pancreatic cancer, neoadjuvant therapy may improve survival, compared with surgery-first treatment. “This study supports, however does not prove, the hypothesis that early and continued control of occult metastatic disease prolongs survival in surgical patients,” said Dr. Christopher Shubert.

Dr. Shubert and his colleagues used an intention-to-treat (ITT) analysis to compare overall survival (OS) for 377 patients who were to receive neoadjuvant chemotherapy with 216 patients who received surgery first. Median OS for the neoadjuvant group was 20.7 months, compared with 13.7 months for the surgery-first group (log-rank P less than .0001).

Courtesy Dr. Lance Liotta Laboratory

This study was the first to utilize national data to look at how patients who received neoadjuvant chemotherapy for stage III pancreatic cancer fared, when compared with those treated with a surgery-first approach, Dr. Shubert, a general surgery resident at the Mayo Clinic in Rochester, Minn., said at the annual meeting of the Central Surgical Association.

Stage III pancreatic cancer (T4, any N, M0) means that the cancer has invaded the celiac trunk, or there is superior mesenteric artery involvement, he noted.

Using data from the National Cancer Database from 2002 to 2011, the investigators identified patients with clinical stage III pancreatic adenocarcinoma of the head or body of the pancreas. The ITT neoadjuvant therapy cohort included all patients whose treatment recommendations included curative-intent surgery and neoadjuvant chemotherapy, regardless of what therapies the patients received. The surgery-first cohort included those who were recommended to receive adjuvant therapy.

A total of 593 patients were identified, of whom 377 (63.5%) were in the neoadjuvant group. Of these, 104 (27.6%) were lost to presurgical attrition. The surgery-first group included 216 patients (36.3%), 30 (13.9%) of whom did not receive the intended adjuvant chemotherapy. Comparing the two ITT groups yielded an adjusted hazard ratio of 0.68 (P = .001).

A secondary aim of the study was to see which aspects of therapy, and which pathologic features, were associated with longer OS. The addition of postsurgical therapy was associated with additional survival benefit (31.6 vs. 22.6 months for no postsurgical therapy; HR, 0.60; P = .002). Node-negative and R0 status were both also significantly increased among those receiving neoadjuvant chemotherapy, and both disease characteristics were associated with increased OS, he reported.

Dr. Shubert said that study limitations included its review of a prospective database. Also, investigators could not determine the type or duration of chemotherapy; they also were unable to tell when systemic chemotherapy plus chemoradiation or just chemoradiation alone had been used. No recurrence data were available, and all cases were grouped under one procedure code, limiting information about vascular resections.

“Neoadjuvant therapy may offer survival advantages compared to a surgery-first approach,” said Dr. Shubert, and control of small occult metastases may be the mechanism behind this advantage. Still to be determined, however, are the optimal type, duration, and sequencing of neoadjuvant chemotherapy and chemoradiation, he said.

Dr. Shubert reported no relevant disclosures.

[email protected]

On Twitter @karioakes

MONTREAL – For individuals with stage III pancreatic cancer, neoadjuvant therapy may improve survival, compared with surgery-first treatment. “This study supports, however does not prove, the hypothesis that early and continued control of occult metastatic disease prolongs survival in surgical patients,” said Dr. Christopher Shubert.

Dr. Shubert and his colleagues used an intention-to-treat (ITT) analysis to compare overall survival (OS) for 377 patients who were to receive neoadjuvant chemotherapy with 216 patients who received surgery first. Median OS for the neoadjuvant group was 20.7 months, compared with 13.7 months for the surgery-first group (log-rank P less than .0001).

Courtesy Dr. Lance Liotta Laboratory

This study was the first to utilize national data to look at how patients who received neoadjuvant chemotherapy for stage III pancreatic cancer fared, when compared with those treated with a surgery-first approach, Dr. Shubert, a general surgery resident at the Mayo Clinic in Rochester, Minn., said at the annual meeting of the Central Surgical Association.

Stage III pancreatic cancer (T4, any N, M0) means that the cancer has invaded the celiac trunk, or there is superior mesenteric artery involvement, he noted.

Using data from the National Cancer Database from 2002 to 2011, the investigators identified patients with clinical stage III pancreatic adenocarcinoma of the head or body of the pancreas. The ITT neoadjuvant therapy cohort included all patients whose treatment recommendations included curative-intent surgery and neoadjuvant chemotherapy, regardless of what therapies the patients received. The surgery-first cohort included those who were recommended to receive adjuvant therapy.

A total of 593 patients were identified, of whom 377 (63.5%) were in the neoadjuvant group. Of these, 104 (27.6%) were lost to presurgical attrition. The surgery-first group included 216 patients (36.3%), 30 (13.9%) of whom did not receive the intended adjuvant chemotherapy. Comparing the two ITT groups yielded an adjusted hazard ratio of 0.68 (P = .001).

A secondary aim of the study was to see which aspects of therapy, and which pathologic features, were associated with longer OS. The addition of postsurgical therapy was associated with additional survival benefit (31.6 vs. 22.6 months for no postsurgical therapy; HR, 0.60; P = .002). Node-negative and R0 status were both also significantly increased among those receiving neoadjuvant chemotherapy, and both disease characteristics were associated with increased OS, he reported.

Dr. Shubert said that study limitations included its review of a prospective database. Also, investigators could not determine the type or duration of chemotherapy; they also were unable to tell when systemic chemotherapy plus chemoradiation or just chemoradiation alone had been used. No recurrence data were available, and all cases were grouped under one procedure code, limiting information about vascular resections.

“Neoadjuvant therapy may offer survival advantages compared to a surgery-first approach,” said Dr. Shubert, and control of small occult metastases may be the mechanism behind this advantage. Still to be determined, however, are the optimal type, duration, and sequencing of neoadjuvant chemotherapy and chemoradiation, he said.

Dr. Shubert reported no relevant disclosures.

[email protected]

On Twitter @karioakes

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AT THE ANNUAL MEETING OF THE CENTRAL SURGICAL ASSOCIATION

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Key clinical point: Overall survival in stage III pancreatic cancer was better with neoadjuvant chemotherapy.

Major finding: Median survival with neoadjuvant chemotherapy was 20.7 months, compared with 13.7 months for surgery-first patients.

Data source: Review of stage III pancreatic cancer patients in the prospective National Cancer Database, comparing 377 patients recommended to receive neoadjuvant chemotherapy with 216 patients who were to receive surgery first.

Disclosures: Dr. Shubert reported no relevant disclosures.

Critical new elements in GI product innovation

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Critical new elements in GI product innovation

BOSTON – Dr. Tom Shehab is a gastroenterologist who now works in venture capital. As a principal at the Ann Arbor, Mich., firm Arboretum Ventures, he was able to give his colleagues at the AGA Tech Summit a realistic perspective on what’s needed to show value in GI innovation.

“I look at oncology products, I look at cardiology products, at urology products, at women’s health products. And I would say that we, as a profession in GI, we’re a bit behind” in the ability to present and articulate a convincing value proposition for innovations, said Dr. Shehab, formerly a physician executive.

Courtesy Arboretum Ventures
Dr. Tom Shehab

Though many clinician innovators have new and better ways to solve technology or work-flow problems in the clinic or operating room, Dr. Shehab said that he looks for more. He wants to see thought given to a plan for each stage of the design cycle. “If you have a value proposition that’s just about discovery and ideation, and you can’t talk to me or my partners about the rest of this pathway, you’re dead in the water,” he said.

“Vision without execution is hallucination,” he added, quoting Thomas Edison. “That’s why our funnel gets very narrow very fast.”

When he and his partners evaluate new technology to assess whether they will invest, Dr. Shehab said he makes sure the innovator understands and can fully explain the “Who, What, Why, and How” of the innovation.

Who: Dr. Shehab said that developers need to know not just who the end user is, but who the actual buyer will be. Is it the patient, the payer, the hospital executive, or the physician? Understanding this key point will help frame the kind and amount of information needed to prove value.

Importantly, a business plan has to include the possibility that shifts in the reimbursement language will change the answer to the question. “The buyer in health care is changing all the time,” said Dr. Shehab.

What: Dr. Shehab said that he wants innovators to be able to tell them how their product is different, and how is it clinically superior to available options. What about this product can demonstrate cost savings?

“There was a time that if I showed noninferiority – that I wasn’t worse – and if I showed quality of life was adequate, that was enough to move forward. I think now that the bar has changed,” he said. Innovators now need to show “not just efficacy but effectiveness.”

Why: An entrepreneur with a new medical technology must be able to understand – and explain – why the product is compelling to the end user. Dr. Shehab said he asks, “Why will it displace what is already being used in clinical practice?”

Recommending that entrepreneurs talk to a wide variety of people about their ideas, Dr. Shehab said, “The person you need to listen to the most is the person who told you this is the worst idea ever.” They may see something that the innovator is missing, usually one of three things: “Buyers don’t want it, the payers aren’t going to pay for it, or it doesn’t fit into the clinical flow. “

How: More and more, said Dr. Shehab, he makes sure that he knows how technologies he supports will work in an evolving reimbursement system. “How would this product fit into a dramatically different payment model?” he asks entrepreneurs.

He advised keeping a close eye on the trend toward bundled reimbursement, since even a small incremental expense has to show value to justify purchase by the hospital or health care organization when reimbursement is fixed. “Your reimbursement pathway is not a checkbox any more. It’s the most important item,” he said. “An effective value proposition tells the story of your device, but a great value proposition shows that you have a terrific device that also fits into the ‘real world’ of health care.”

The summit was sponsored by the AGA Center for GI Innovation and Technology. Dr. Shehab disclosed that he is a partner and investor in Arboretum Ventures.

[email protected]

On Twitter @karioakes

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BOSTON – Dr. Tom Shehab is a gastroenterologist who now works in venture capital. As a principal at the Ann Arbor, Mich., firm Arboretum Ventures, he was able to give his colleagues at the AGA Tech Summit a realistic perspective on what’s needed to show value in GI innovation.

“I look at oncology products, I look at cardiology products, at urology products, at women’s health products. And I would say that we, as a profession in GI, we’re a bit behind” in the ability to present and articulate a convincing value proposition for innovations, said Dr. Shehab, formerly a physician executive.

Courtesy Arboretum Ventures
Dr. Tom Shehab

Though many clinician innovators have new and better ways to solve technology or work-flow problems in the clinic or operating room, Dr. Shehab said that he looks for more. He wants to see thought given to a plan for each stage of the design cycle. “If you have a value proposition that’s just about discovery and ideation, and you can’t talk to me or my partners about the rest of this pathway, you’re dead in the water,” he said.

“Vision without execution is hallucination,” he added, quoting Thomas Edison. “That’s why our funnel gets very narrow very fast.”

When he and his partners evaluate new technology to assess whether they will invest, Dr. Shehab said he makes sure the innovator understands and can fully explain the “Who, What, Why, and How” of the innovation.

Who: Dr. Shehab said that developers need to know not just who the end user is, but who the actual buyer will be. Is it the patient, the payer, the hospital executive, or the physician? Understanding this key point will help frame the kind and amount of information needed to prove value.

Importantly, a business plan has to include the possibility that shifts in the reimbursement language will change the answer to the question. “The buyer in health care is changing all the time,” said Dr. Shehab.

What: Dr. Shehab said that he wants innovators to be able to tell them how their product is different, and how is it clinically superior to available options. What about this product can demonstrate cost savings?

“There was a time that if I showed noninferiority – that I wasn’t worse – and if I showed quality of life was adequate, that was enough to move forward. I think now that the bar has changed,” he said. Innovators now need to show “not just efficacy but effectiveness.”

Why: An entrepreneur with a new medical technology must be able to understand – and explain – why the product is compelling to the end user. Dr. Shehab said he asks, “Why will it displace what is already being used in clinical practice?”

Recommending that entrepreneurs talk to a wide variety of people about their ideas, Dr. Shehab said, “The person you need to listen to the most is the person who told you this is the worst idea ever.” They may see something that the innovator is missing, usually one of three things: “Buyers don’t want it, the payers aren’t going to pay for it, or it doesn’t fit into the clinical flow. “

How: More and more, said Dr. Shehab, he makes sure that he knows how technologies he supports will work in an evolving reimbursement system. “How would this product fit into a dramatically different payment model?” he asks entrepreneurs.

He advised keeping a close eye on the trend toward bundled reimbursement, since even a small incremental expense has to show value to justify purchase by the hospital or health care organization when reimbursement is fixed. “Your reimbursement pathway is not a checkbox any more. It’s the most important item,” he said. “An effective value proposition tells the story of your device, but a great value proposition shows that you have a terrific device that also fits into the ‘real world’ of health care.”

The summit was sponsored by the AGA Center for GI Innovation and Technology. Dr. Shehab disclosed that he is a partner and investor in Arboretum Ventures.

[email protected]

On Twitter @karioakes

BOSTON – Dr. Tom Shehab is a gastroenterologist who now works in venture capital. As a principal at the Ann Arbor, Mich., firm Arboretum Ventures, he was able to give his colleagues at the AGA Tech Summit a realistic perspective on what’s needed to show value in GI innovation.

“I look at oncology products, I look at cardiology products, at urology products, at women’s health products. And I would say that we, as a profession in GI, we’re a bit behind” in the ability to present and articulate a convincing value proposition for innovations, said Dr. Shehab, formerly a physician executive.

Courtesy Arboretum Ventures
Dr. Tom Shehab

Though many clinician innovators have new and better ways to solve technology or work-flow problems in the clinic or operating room, Dr. Shehab said that he looks for more. He wants to see thought given to a plan for each stage of the design cycle. “If you have a value proposition that’s just about discovery and ideation, and you can’t talk to me or my partners about the rest of this pathway, you’re dead in the water,” he said.

“Vision without execution is hallucination,” he added, quoting Thomas Edison. “That’s why our funnel gets very narrow very fast.”

When he and his partners evaluate new technology to assess whether they will invest, Dr. Shehab said he makes sure the innovator understands and can fully explain the “Who, What, Why, and How” of the innovation.

Who: Dr. Shehab said that developers need to know not just who the end user is, but who the actual buyer will be. Is it the patient, the payer, the hospital executive, or the physician? Understanding this key point will help frame the kind and amount of information needed to prove value.

Importantly, a business plan has to include the possibility that shifts in the reimbursement language will change the answer to the question. “The buyer in health care is changing all the time,” said Dr. Shehab.

What: Dr. Shehab said that he wants innovators to be able to tell them how their product is different, and how is it clinically superior to available options. What about this product can demonstrate cost savings?

“There was a time that if I showed noninferiority – that I wasn’t worse – and if I showed quality of life was adequate, that was enough to move forward. I think now that the bar has changed,” he said. Innovators now need to show “not just efficacy but effectiveness.”

Why: An entrepreneur with a new medical technology must be able to understand – and explain – why the product is compelling to the end user. Dr. Shehab said he asks, “Why will it displace what is already being used in clinical practice?”

Recommending that entrepreneurs talk to a wide variety of people about their ideas, Dr. Shehab said, “The person you need to listen to the most is the person who told you this is the worst idea ever.” They may see something that the innovator is missing, usually one of three things: “Buyers don’t want it, the payers aren’t going to pay for it, or it doesn’t fit into the clinical flow. “

How: More and more, said Dr. Shehab, he makes sure that he knows how technologies he supports will work in an evolving reimbursement system. “How would this product fit into a dramatically different payment model?” he asks entrepreneurs.

He advised keeping a close eye on the trend toward bundled reimbursement, since even a small incremental expense has to show value to justify purchase by the hospital or health care organization when reimbursement is fixed. “Your reimbursement pathway is not a checkbox any more. It’s the most important item,” he said. “An effective value proposition tells the story of your device, but a great value proposition shows that you have a terrific device that also fits into the ‘real world’ of health care.”

The summit was sponsored by the AGA Center for GI Innovation and Technology. Dr. Shehab disclosed that he is a partner and investor in Arboretum Ventures.

[email protected]

On Twitter @karioakes

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Surgeons commonly off the mark in estimating blood loss

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MONTREAL – Surgeons, nurses, and anesthesia providers were all pretty bad at estimating surgical blood loss in a small study. And more experience doesn’t improve accuracy, though experienced providers were more confident in their estimates.

These were the findings from a study that simulated operating room scenarios and asked providers to estimate blood loss. “Estimation of blood loss is inaccurate and unreliable,” Dr. Luke Rothermel said at the Central Surgical Association’s annual meeting.

Dr. Rothermel, a resident at Case Western Reserve University, Cleveland, noted that although the Joint Commission requires operative notes to contain estimated blood loss, “no study in the United States has compared the characteristics of operating room personnel or conditions associated with improved accuracy or reliability of blood loss estimation.”

©Svisio/Thinkstock

Beyond the required reporting, estimating blood loss (EBL) also provides important guidance in perioperative care. Still, said Dr. Rothermel, previous studies have shown that EBL is typically inaccurate.

To assess providers’ ability to be accurate and reliable in estimating blood loss, Dr. Rothermel and his collaborator, Dr. Jeremy Lipman, assistant residency director at MetroHealth, Cleveland, designed a study to simulate three different operating room scenarios, involving high, medium, and low blood loss volumes. The materials used, such as blood-soaked sponges and suction canisters, were identical to what’s actually used in the operating room (porcine blood was used in the simulations).

Before the study, Dr. Rothermel said that he and Dr. Lipman hypothesized that those providers who had more experience and those who were working at the operating field would be more accurate in estimating blood loss. They also hypothesized that estimations in procedures with lower volumes of blood loss would be more accurate.

The study recruited providers from the surgery, anesthesia, and nursing services at an urban level 1 trauma center. Each scenario included a written description of the procedure performed and the course of surgery, and participants could handle study materials for each scenario under the supervision of study staff.

A total of 60 participants (22 from surgery, 17 from anesthesia, and 21 from nursing) participated; they had an average of 12.8 years of experience. The surgical participants included surgical scrub techs, trainees, and attending physicians. Anesthesia participants included anesthesia assistants, CRNAs, trainees, and attending physicians. Nursing participants were all RNs.

The findings? All over the board: “There was no association between specialty, years of experience, or confidence in ability with the consistency or accuracy of estimated blood loss,” said Dr. Rothermel.

Most participants were far shy of the mark, with just 5% of study participants overall able to come within 25% accuracy in judging EBL in all scenarios. Just over a quarter were consistent in over- or underestimating blood loss.

These findings held true across scenarios, across disciplines, and regardless of the number of years of experience. “Increased years of experience trended toward increased error,” said Dr. Rothermel, though the difference was not statistically significant. However, those with more years of experience tended to be more confident of their judgments.

Dr. Rothermel noted the small study size and single institution studied as limitations. Also, “this model was not a high fidelity representation of the OR experience, “ he said, explaining that during surgery, caregivers continually assess intraoperative blood loss and may form an estimate in a different – and potentially more accurate – manner than occurs when presented with the contrived presentation of a scenario.

The study calls into question the validity of using EBL as a quality indicator in assessing physician performance and patient outcomes, said Dr. Rothermel, who had no financial disclosures.

[email protected]

On Twitter @karioakes

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MONTREAL – Surgeons, nurses, and anesthesia providers were all pretty bad at estimating surgical blood loss in a small study. And more experience doesn’t improve accuracy, though experienced providers were more confident in their estimates.

These were the findings from a study that simulated operating room scenarios and asked providers to estimate blood loss. “Estimation of blood loss is inaccurate and unreliable,” Dr. Luke Rothermel said at the Central Surgical Association’s annual meeting.

Dr. Rothermel, a resident at Case Western Reserve University, Cleveland, noted that although the Joint Commission requires operative notes to contain estimated blood loss, “no study in the United States has compared the characteristics of operating room personnel or conditions associated with improved accuracy or reliability of blood loss estimation.”

©Svisio/Thinkstock

Beyond the required reporting, estimating blood loss (EBL) also provides important guidance in perioperative care. Still, said Dr. Rothermel, previous studies have shown that EBL is typically inaccurate.

To assess providers’ ability to be accurate and reliable in estimating blood loss, Dr. Rothermel and his collaborator, Dr. Jeremy Lipman, assistant residency director at MetroHealth, Cleveland, designed a study to simulate three different operating room scenarios, involving high, medium, and low blood loss volumes. The materials used, such as blood-soaked sponges and suction canisters, were identical to what’s actually used in the operating room (porcine blood was used in the simulations).

Before the study, Dr. Rothermel said that he and Dr. Lipman hypothesized that those providers who had more experience and those who were working at the operating field would be more accurate in estimating blood loss. They also hypothesized that estimations in procedures with lower volumes of blood loss would be more accurate.

The study recruited providers from the surgery, anesthesia, and nursing services at an urban level 1 trauma center. Each scenario included a written description of the procedure performed and the course of surgery, and participants could handle study materials for each scenario under the supervision of study staff.

A total of 60 participants (22 from surgery, 17 from anesthesia, and 21 from nursing) participated; they had an average of 12.8 years of experience. The surgical participants included surgical scrub techs, trainees, and attending physicians. Anesthesia participants included anesthesia assistants, CRNAs, trainees, and attending physicians. Nursing participants were all RNs.

The findings? All over the board: “There was no association between specialty, years of experience, or confidence in ability with the consistency or accuracy of estimated blood loss,” said Dr. Rothermel.

Most participants were far shy of the mark, with just 5% of study participants overall able to come within 25% accuracy in judging EBL in all scenarios. Just over a quarter were consistent in over- or underestimating blood loss.

These findings held true across scenarios, across disciplines, and regardless of the number of years of experience. “Increased years of experience trended toward increased error,” said Dr. Rothermel, though the difference was not statistically significant. However, those with more years of experience tended to be more confident of their judgments.

Dr. Rothermel noted the small study size and single institution studied as limitations. Also, “this model was not a high fidelity representation of the OR experience, “ he said, explaining that during surgery, caregivers continually assess intraoperative blood loss and may form an estimate in a different – and potentially more accurate – manner than occurs when presented with the contrived presentation of a scenario.

The study calls into question the validity of using EBL as a quality indicator in assessing physician performance and patient outcomes, said Dr. Rothermel, who had no financial disclosures.

[email protected]

On Twitter @karioakes

MONTREAL – Surgeons, nurses, and anesthesia providers were all pretty bad at estimating surgical blood loss in a small study. And more experience doesn’t improve accuracy, though experienced providers were more confident in their estimates.

These were the findings from a study that simulated operating room scenarios and asked providers to estimate blood loss. “Estimation of blood loss is inaccurate and unreliable,” Dr. Luke Rothermel said at the Central Surgical Association’s annual meeting.

Dr. Rothermel, a resident at Case Western Reserve University, Cleveland, noted that although the Joint Commission requires operative notes to contain estimated blood loss, “no study in the United States has compared the characteristics of operating room personnel or conditions associated with improved accuracy or reliability of blood loss estimation.”

©Svisio/Thinkstock

Beyond the required reporting, estimating blood loss (EBL) also provides important guidance in perioperative care. Still, said Dr. Rothermel, previous studies have shown that EBL is typically inaccurate.

To assess providers’ ability to be accurate and reliable in estimating blood loss, Dr. Rothermel and his collaborator, Dr. Jeremy Lipman, assistant residency director at MetroHealth, Cleveland, designed a study to simulate three different operating room scenarios, involving high, medium, and low blood loss volumes. The materials used, such as blood-soaked sponges and suction canisters, were identical to what’s actually used in the operating room (porcine blood was used in the simulations).

Before the study, Dr. Rothermel said that he and Dr. Lipman hypothesized that those providers who had more experience and those who were working at the operating field would be more accurate in estimating blood loss. They also hypothesized that estimations in procedures with lower volumes of blood loss would be more accurate.

The study recruited providers from the surgery, anesthesia, and nursing services at an urban level 1 trauma center. Each scenario included a written description of the procedure performed and the course of surgery, and participants could handle study materials for each scenario under the supervision of study staff.

A total of 60 participants (22 from surgery, 17 from anesthesia, and 21 from nursing) participated; they had an average of 12.8 years of experience. The surgical participants included surgical scrub techs, trainees, and attending physicians. Anesthesia participants included anesthesia assistants, CRNAs, trainees, and attending physicians. Nursing participants were all RNs.

The findings? All over the board: “There was no association between specialty, years of experience, or confidence in ability with the consistency or accuracy of estimated blood loss,” said Dr. Rothermel.

Most participants were far shy of the mark, with just 5% of study participants overall able to come within 25% accuracy in judging EBL in all scenarios. Just over a quarter were consistent in over- or underestimating blood loss.

These findings held true across scenarios, across disciplines, and regardless of the number of years of experience. “Increased years of experience trended toward increased error,” said Dr. Rothermel, though the difference was not statistically significant. However, those with more years of experience tended to be more confident of their judgments.

Dr. Rothermel noted the small study size and single institution studied as limitations. Also, “this model was not a high fidelity representation of the OR experience, “ he said, explaining that during surgery, caregivers continually assess intraoperative blood loss and may form an estimate in a different – and potentially more accurate – manner than occurs when presented with the contrived presentation of a scenario.

The study calls into question the validity of using EBL as a quality indicator in assessing physician performance and patient outcomes, said Dr. Rothermel, who had no financial disclosures.

[email protected]

On Twitter @karioakes

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AT THE ANNUAL MEETING OF THE CENTRAL SURGICAL ASSOCIATION

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Inside the Article

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Key clinical point: Surgery, anesthesia, and nursing providers were inaccurate and unreliable in estimating surgical blood loss.

Major finding: Only 5% of providers could come within 25% accuracy of simulated surgical blood loss.

Data source: Simulations of surgical scenarios depicting varying amounts of blood loss using porcine blood, presented to 60 providers.

Disclosures: The study authors reported no relevant disclosures.

Obeticholic acid gains recommendation for accelerated approval in primary biliary cholangitis

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Obeticholic acid gains recommendation for accelerated approval in primary biliary cholangitis

SILVER SPRING, MD. – A Food and Drug Administration advisory panel unanimously voted in favor of accelerated approval for a novel drug to treat primary biliary cholangitis on April 7, 2016. The accelerated drug approval process allows for the use of alkaline phosphatase levels as a surrogate endpoint of efficacy.*

At the meeting of the FDA Gastrointestinal Drugs Advisory Committee, panelists wrestled with additional questions from the FDA regarding the use of obeticholic acid (OCA) for primary biliary cholangitis. Presented as discussion items, these included how to select appropriate populations for the novel drug, the proper dosing schema, and how to go forward in postmarketing confirmatory trials of OCA.

Courtesy Wikimedia Commons/FitzColinGerald/Creative Commons License

The exact indication for which the applicant, Intercept Pharmaceuticals, is seeking obeticholic acid approval is “treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.”

In discussion after the unanimous vote for accelerated approval, Dr. Maria Sjogren, a hepatologist at Walter Reed National Military Medical Center, Bethesda, Md. said, “I welcome this drug in the clinic. It will be a great addition for many patients.” Dr. Marina Silveira, of the division of gastroenterology at the Louis Stokes Cleveland VA Medical Center, agreed: “There is an unmet need, and there are no significant safety or tolerability concerns. But I do think there is more study that’s going to be needed.”

The pivotal phase III clinical trial, POISE [PBC OCA International Study of Efficacy], showed that at the final 1-year assessment, 46% of patients treated with an initial 5 mg daily dose of OCA, and 47% of those treated with 10 mg daily, achieved the primary efficacy endpoint of an alkaline phosphatase (ALP) level less than 1.67 times the upper limit of normal, and/or had bilirubin levels less than the upper limit of normal and a 15% or greater reduction in ALP.

The drug was generally well tolerated at the doses used in the POISE trial. Pruritus was common but well-managed in both OCA treatment arms. No concerning hepatic safety signals were seen in the 5-mg titration arm or the 10-mg arm.

PBC (formerly known as primary biliary cirrhosis) is a progressive autoimmune disease of unknown etiology that causes cholestasis, resulting in destruction of the biliary system over time.

Currently, the only approved treatment for PBC is UDCA. Obeticholic acid (to be marketed as Ocaliva by Intercept Pharmaceuticals) is an agonist of the farnesoid X receptor (FXR), a nuclear receptor that controls bile acid homeostasis. The direct FXR agonist activity differs from the mechanism of action of UDCA, which has no nuclear receptor action. Dosing for OCA can thus be much lower, making it attractive for PBC patients who face a considerable pill burden with weight-based dosing of UDCA.

Though committee members acknowledged unmet needs for more and better therapies for PBC patients who don’t respond to or can’t tolerate UDCA, they repeatedly asked for high-quality longitudinal data collection and analysis. Clinical trial data show a signal for early low-density lipoprotein elevation with OCA, as well as long-term lowering of high-density lipoprotein levels.

Regarding the proposed dosing of OCA, FDA analysts agreed with the applicant that a starting dose of 5 mg daily is appropriate, since a dose-dependent increase in pruritus-related discontinuations was seen in phase III clinical trials. Also, a better tolerability profile is seen over time with a lower starting dose, with fewer discontinuations, fewer days of severe pruritus, and delayed time to the first episode of pruritus with the 5-mg starting dose.

Additionally, efficacy was preserved with dose titration, with similar efficacy seen at 1 year for the titration arm, compared with the 10-mg fixed-dose arm in the POISE trial that compared titration from 5 to 10 mg and fixed 10-mg dosing with placebo.

However, the FDA took issue with Intercept’s proposal not to adjust dosing in moderate or severe hepatic impairment, instead proposing a starting dose of 5 mg once weekly, and titrating up to 5 mg twice weekly and finally to a maximum of 10 mg twice weekly, depending on response and tolerability. The FDA’s rationale for this was the lack of clear benefit of high exposures to OCA in moderate or severe hepatic impairment. Another factor was the increased incidence of hepatic adverse events and the higher rate of discontinuation at higher exposures that were seen in earlier clinical trials that used higher doses of OCA.

Panelists, in their critique of the FDA’s proposed hepatic impairment dose adjustments, felt there was insufficient data to support this adjusted regimen and called for more study.

 

 

An especially thorny issue for panelists and the FDA was the design of the phase IV trial, which is a double-blind, placebo-controlled, multi-center international trial comparing OCA with placebo for PBC. This trial has already begun to enroll patients and will continue to recruit for a total of 2 years; follow-up will continue for 6 years with quarterly visits. Historical control data from a global PBC database are also available for analysis.

Panelist Dr. Timothy Lipman, emeritus chief of the GI-hepatology-nutrition center at the Department of Veterans Affairs Medical Center in Washington, said, “As a clinician who is very interested in clinical study methodology, I am concerned about possible bias. And the use of historical control data is a nonstarter,” since the study quality would suffer. “Changes in protocol as a study goes on are always problematic,” so the FDA’s request for feedback on how to tinker with study design was a concern.

But the biggest concern voiced by Dr. Lipman and others on the committee, including FDA representatives, was the huge barrier to enrollment that’s presented by a placebo-controlled study for a drug that has already been approved. “This is always a major issue for the FDA in approving drugs under accelerated approval,” acknowledged Dr. Amy Egan, deputy director of the FDA’s office of drug evaluation III, office of new drugs. The anticipation of difficulty in enrolling is one reason the historical control arm is held in reserve, she said. Intercept’s vice president for clinical development Dr. Leigh MacConell concurred, saying of the discussions with the FDA about study design, “It was a very difficult conversation, because we agree with your assessment regarding the feasibility” of the study.

The FDA noted remaining issues requiring ongoing study of OCA for PBC. Among these is the need to confirm the clinical benefit of OCA across the full spectrum of severity of PBC, from early stage to advanced disease. “FDA would also like to evaluate additional data on use of OCA as monotherapy,” said Dr. Lara Dimick-Santos, cross-discipline team leader at the FDA.

The course of PBC is variable; it affects women approximately 10 times more frequently than men. Occurring in approximately 1 in 1,000 women over the age of 40, its prevalence is thought to be increasing. PBC is usually asymptomatic for some time; when symptoms occur, fatigue and pruritis are the most common. Concomitant autoimmune diseases are common and an earlier age at diagnosis is often associated with a worse disease course. A significant number of those affected by PBC will progress to death or liver transplantation.

Intercept Pharmaceuticals is also studying OCA for use in other liver conditions, including nonalcoholic steatohepatitis (NASH).

All committee members submitted information to the FDA regarding conflicts of interest. The FDA usually follows the recommendations of its advisory panels.

*Changes were made to this story on April 8, 2016.

[email protected]

On Twitter @karioakes

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SILVER SPRING, MD. – A Food and Drug Administration advisory panel unanimously voted in favor of accelerated approval for a novel drug to treat primary biliary cholangitis on April 7, 2016. The accelerated drug approval process allows for the use of alkaline phosphatase levels as a surrogate endpoint of efficacy.*

At the meeting of the FDA Gastrointestinal Drugs Advisory Committee, panelists wrestled with additional questions from the FDA regarding the use of obeticholic acid (OCA) for primary biliary cholangitis. Presented as discussion items, these included how to select appropriate populations for the novel drug, the proper dosing schema, and how to go forward in postmarketing confirmatory trials of OCA.

Courtesy Wikimedia Commons/FitzColinGerald/Creative Commons License

The exact indication for which the applicant, Intercept Pharmaceuticals, is seeking obeticholic acid approval is “treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.”

In discussion after the unanimous vote for accelerated approval, Dr. Maria Sjogren, a hepatologist at Walter Reed National Military Medical Center, Bethesda, Md. said, “I welcome this drug in the clinic. It will be a great addition for many patients.” Dr. Marina Silveira, of the division of gastroenterology at the Louis Stokes Cleveland VA Medical Center, agreed: “There is an unmet need, and there are no significant safety or tolerability concerns. But I do think there is more study that’s going to be needed.”

The pivotal phase III clinical trial, POISE [PBC OCA International Study of Efficacy], showed that at the final 1-year assessment, 46% of patients treated with an initial 5 mg daily dose of OCA, and 47% of those treated with 10 mg daily, achieved the primary efficacy endpoint of an alkaline phosphatase (ALP) level less than 1.67 times the upper limit of normal, and/or had bilirubin levels less than the upper limit of normal and a 15% or greater reduction in ALP.

The drug was generally well tolerated at the doses used in the POISE trial. Pruritus was common but well-managed in both OCA treatment arms. No concerning hepatic safety signals were seen in the 5-mg titration arm or the 10-mg arm.

PBC (formerly known as primary biliary cirrhosis) is a progressive autoimmune disease of unknown etiology that causes cholestasis, resulting in destruction of the biliary system over time.

Currently, the only approved treatment for PBC is UDCA. Obeticholic acid (to be marketed as Ocaliva by Intercept Pharmaceuticals) is an agonist of the farnesoid X receptor (FXR), a nuclear receptor that controls bile acid homeostasis. The direct FXR agonist activity differs from the mechanism of action of UDCA, which has no nuclear receptor action. Dosing for OCA can thus be much lower, making it attractive for PBC patients who face a considerable pill burden with weight-based dosing of UDCA.

Though committee members acknowledged unmet needs for more and better therapies for PBC patients who don’t respond to or can’t tolerate UDCA, they repeatedly asked for high-quality longitudinal data collection and analysis. Clinical trial data show a signal for early low-density lipoprotein elevation with OCA, as well as long-term lowering of high-density lipoprotein levels.

Regarding the proposed dosing of OCA, FDA analysts agreed with the applicant that a starting dose of 5 mg daily is appropriate, since a dose-dependent increase in pruritus-related discontinuations was seen in phase III clinical trials. Also, a better tolerability profile is seen over time with a lower starting dose, with fewer discontinuations, fewer days of severe pruritus, and delayed time to the first episode of pruritus with the 5-mg starting dose.

Additionally, efficacy was preserved with dose titration, with similar efficacy seen at 1 year for the titration arm, compared with the 10-mg fixed-dose arm in the POISE trial that compared titration from 5 to 10 mg and fixed 10-mg dosing with placebo.

However, the FDA took issue with Intercept’s proposal not to adjust dosing in moderate or severe hepatic impairment, instead proposing a starting dose of 5 mg once weekly, and titrating up to 5 mg twice weekly and finally to a maximum of 10 mg twice weekly, depending on response and tolerability. The FDA’s rationale for this was the lack of clear benefit of high exposures to OCA in moderate or severe hepatic impairment. Another factor was the increased incidence of hepatic adverse events and the higher rate of discontinuation at higher exposures that were seen in earlier clinical trials that used higher doses of OCA.

Panelists, in their critique of the FDA’s proposed hepatic impairment dose adjustments, felt there was insufficient data to support this adjusted regimen and called for more study.

 

 

An especially thorny issue for panelists and the FDA was the design of the phase IV trial, which is a double-blind, placebo-controlled, multi-center international trial comparing OCA with placebo for PBC. This trial has already begun to enroll patients and will continue to recruit for a total of 2 years; follow-up will continue for 6 years with quarterly visits. Historical control data from a global PBC database are also available for analysis.

Panelist Dr. Timothy Lipman, emeritus chief of the GI-hepatology-nutrition center at the Department of Veterans Affairs Medical Center in Washington, said, “As a clinician who is very interested in clinical study methodology, I am concerned about possible bias. And the use of historical control data is a nonstarter,” since the study quality would suffer. “Changes in protocol as a study goes on are always problematic,” so the FDA’s request for feedback on how to tinker with study design was a concern.

But the biggest concern voiced by Dr. Lipman and others on the committee, including FDA representatives, was the huge barrier to enrollment that’s presented by a placebo-controlled study for a drug that has already been approved. “This is always a major issue for the FDA in approving drugs under accelerated approval,” acknowledged Dr. Amy Egan, deputy director of the FDA’s office of drug evaluation III, office of new drugs. The anticipation of difficulty in enrolling is one reason the historical control arm is held in reserve, she said. Intercept’s vice president for clinical development Dr. Leigh MacConell concurred, saying of the discussions with the FDA about study design, “It was a very difficult conversation, because we agree with your assessment regarding the feasibility” of the study.

The FDA noted remaining issues requiring ongoing study of OCA for PBC. Among these is the need to confirm the clinical benefit of OCA across the full spectrum of severity of PBC, from early stage to advanced disease. “FDA would also like to evaluate additional data on use of OCA as monotherapy,” said Dr. Lara Dimick-Santos, cross-discipline team leader at the FDA.

The course of PBC is variable; it affects women approximately 10 times more frequently than men. Occurring in approximately 1 in 1,000 women over the age of 40, its prevalence is thought to be increasing. PBC is usually asymptomatic for some time; when symptoms occur, fatigue and pruritis are the most common. Concomitant autoimmune diseases are common and an earlier age at diagnosis is often associated with a worse disease course. A significant number of those affected by PBC will progress to death or liver transplantation.

Intercept Pharmaceuticals is also studying OCA for use in other liver conditions, including nonalcoholic steatohepatitis (NASH).

All committee members submitted information to the FDA regarding conflicts of interest. The FDA usually follows the recommendations of its advisory panels.

*Changes were made to this story on April 8, 2016.

[email protected]

On Twitter @karioakes

SILVER SPRING, MD. – A Food and Drug Administration advisory panel unanimously voted in favor of accelerated approval for a novel drug to treat primary biliary cholangitis on April 7, 2016. The accelerated drug approval process allows for the use of alkaline phosphatase levels as a surrogate endpoint of efficacy.*

At the meeting of the FDA Gastrointestinal Drugs Advisory Committee, panelists wrestled with additional questions from the FDA regarding the use of obeticholic acid (OCA) for primary biliary cholangitis. Presented as discussion items, these included how to select appropriate populations for the novel drug, the proper dosing schema, and how to go forward in postmarketing confirmatory trials of OCA.

Courtesy Wikimedia Commons/FitzColinGerald/Creative Commons License

The exact indication for which the applicant, Intercept Pharmaceuticals, is seeking obeticholic acid approval is “treatment of primary biliary cholangitis (PBC) in combination with ursodeoxycholic acid (UDCA) in adults with an inadequate response to UDCA, or as monotherapy in patients unable to tolerate UDCA.”

In discussion after the unanimous vote for accelerated approval, Dr. Maria Sjogren, a hepatologist at Walter Reed National Military Medical Center, Bethesda, Md. said, “I welcome this drug in the clinic. It will be a great addition for many patients.” Dr. Marina Silveira, of the division of gastroenterology at the Louis Stokes Cleveland VA Medical Center, agreed: “There is an unmet need, and there are no significant safety or tolerability concerns. But I do think there is more study that’s going to be needed.”

The pivotal phase III clinical trial, POISE [PBC OCA International Study of Efficacy], showed that at the final 1-year assessment, 46% of patients treated with an initial 5 mg daily dose of OCA, and 47% of those treated with 10 mg daily, achieved the primary efficacy endpoint of an alkaline phosphatase (ALP) level less than 1.67 times the upper limit of normal, and/or had bilirubin levels less than the upper limit of normal and a 15% or greater reduction in ALP.

The drug was generally well tolerated at the doses used in the POISE trial. Pruritus was common but well-managed in both OCA treatment arms. No concerning hepatic safety signals were seen in the 5-mg titration arm or the 10-mg arm.

PBC (formerly known as primary biliary cirrhosis) is a progressive autoimmune disease of unknown etiology that causes cholestasis, resulting in destruction of the biliary system over time.

Currently, the only approved treatment for PBC is UDCA. Obeticholic acid (to be marketed as Ocaliva by Intercept Pharmaceuticals) is an agonist of the farnesoid X receptor (FXR), a nuclear receptor that controls bile acid homeostasis. The direct FXR agonist activity differs from the mechanism of action of UDCA, which has no nuclear receptor action. Dosing for OCA can thus be much lower, making it attractive for PBC patients who face a considerable pill burden with weight-based dosing of UDCA.

Though committee members acknowledged unmet needs for more and better therapies for PBC patients who don’t respond to or can’t tolerate UDCA, they repeatedly asked for high-quality longitudinal data collection and analysis. Clinical trial data show a signal for early low-density lipoprotein elevation with OCA, as well as long-term lowering of high-density lipoprotein levels.

Regarding the proposed dosing of OCA, FDA analysts agreed with the applicant that a starting dose of 5 mg daily is appropriate, since a dose-dependent increase in pruritus-related discontinuations was seen in phase III clinical trials. Also, a better tolerability profile is seen over time with a lower starting dose, with fewer discontinuations, fewer days of severe pruritus, and delayed time to the first episode of pruritus with the 5-mg starting dose.

Additionally, efficacy was preserved with dose titration, with similar efficacy seen at 1 year for the titration arm, compared with the 10-mg fixed-dose arm in the POISE trial that compared titration from 5 to 10 mg and fixed 10-mg dosing with placebo.

However, the FDA took issue with Intercept’s proposal not to adjust dosing in moderate or severe hepatic impairment, instead proposing a starting dose of 5 mg once weekly, and titrating up to 5 mg twice weekly and finally to a maximum of 10 mg twice weekly, depending on response and tolerability. The FDA’s rationale for this was the lack of clear benefit of high exposures to OCA in moderate or severe hepatic impairment. Another factor was the increased incidence of hepatic adverse events and the higher rate of discontinuation at higher exposures that were seen in earlier clinical trials that used higher doses of OCA.

Panelists, in their critique of the FDA’s proposed hepatic impairment dose adjustments, felt there was insufficient data to support this adjusted regimen and called for more study.

 

 

An especially thorny issue for panelists and the FDA was the design of the phase IV trial, which is a double-blind, placebo-controlled, multi-center international trial comparing OCA with placebo for PBC. This trial has already begun to enroll patients and will continue to recruit for a total of 2 years; follow-up will continue for 6 years with quarterly visits. Historical control data from a global PBC database are also available for analysis.

Panelist Dr. Timothy Lipman, emeritus chief of the GI-hepatology-nutrition center at the Department of Veterans Affairs Medical Center in Washington, said, “As a clinician who is very interested in clinical study methodology, I am concerned about possible bias. And the use of historical control data is a nonstarter,” since the study quality would suffer. “Changes in protocol as a study goes on are always problematic,” so the FDA’s request for feedback on how to tinker with study design was a concern.

But the biggest concern voiced by Dr. Lipman and others on the committee, including FDA representatives, was the huge barrier to enrollment that’s presented by a placebo-controlled study for a drug that has already been approved. “This is always a major issue for the FDA in approving drugs under accelerated approval,” acknowledged Dr. Amy Egan, deputy director of the FDA’s office of drug evaluation III, office of new drugs. The anticipation of difficulty in enrolling is one reason the historical control arm is held in reserve, she said. Intercept’s vice president for clinical development Dr. Leigh MacConell concurred, saying of the discussions with the FDA about study design, “It was a very difficult conversation, because we agree with your assessment regarding the feasibility” of the study.

The FDA noted remaining issues requiring ongoing study of OCA for PBC. Among these is the need to confirm the clinical benefit of OCA across the full spectrum of severity of PBC, from early stage to advanced disease. “FDA would also like to evaluate additional data on use of OCA as monotherapy,” said Dr. Lara Dimick-Santos, cross-discipline team leader at the FDA.

The course of PBC is variable; it affects women approximately 10 times more frequently than men. Occurring in approximately 1 in 1,000 women over the age of 40, its prevalence is thought to be increasing. PBC is usually asymptomatic for some time; when symptoms occur, fatigue and pruritis are the most common. Concomitant autoimmune diseases are common and an earlier age at diagnosis is often associated with a worse disease course. A significant number of those affected by PBC will progress to death or liver transplantation.

Intercept Pharmaceuticals is also studying OCA for use in other liver conditions, including nonalcoholic steatohepatitis (NASH).

All committee members submitted information to the FDA regarding conflicts of interest. The FDA usually follows the recommendations of its advisory panels.

*Changes were made to this story on April 8, 2016.

[email protected]

On Twitter @karioakes

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Adding chemo to radiation boosts survival from low-grade gliomas

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Adding chemo to radiation boosts survival from low-grade gliomas

Adding a chemotherapy combination to radiation therapy for initial treatment of low-grade gliomas significantly improved overall survival and progression-free survival, regardless of the tumor type, investigators report in the New England Journal of Medicine.

Grade 2 glioma patients who received radiation plus the combination of procarbazine, lomustine (also called CCNU), and vincristine (PCV) had a longer median overall survival than those who received radiation alone (13.3 vs. 7.8 years; hazard ratio for death, 0.59; P = .003). Of those who received radiation plus chemotherapy, the progression-free survival rate at 10 years was 51%, compared with 21% for the group who received radiation alone, Dr. Jan Buckner and his colleagues report (N Engl J Med. 2016;374:1344-55. doi: 10.1056/NEJMoa1500925).

“The magnitude of treatment benefit from combined chemotherapy plus radiation therapy is substantial, but the toxic effects are greater than those observed with radiation therapy alone,” wrote Dr. Buckner, professor of oncology at the Mayo Clinic, Rochester, Minn., and associates. Patients who received radiation plus PCV had more toxic side effects from their therapy, though most effects were grade 1 and 2.

For this study, 254 patients were randomized, with 128 assigned to radiotherapy alone, and 126 assigned to radiotherapy plus PCV. A total of 126 patients in the radiotherapy arm were included in the analysis, with one patient not receiving the intervention and 14 patients lost to follow-up at some point during the 10 years of the study. In the radiotherapy plus PCV arm, 125 patients were eligible for evaluation, and all of those patients were included in the analysis. Twenty-six patients in this arm were lost to follow-up, and 72 patients discontinued the intervention in this arm (this figure included four patients who died).

Tumor types included in the study were grade 2 astrocytoma, oligoastrocytoma, or oligodendroglioma.

Patients were included if they were between 18 and 39 years of age and had received a subtotal resection or biopsy of their tumor, or if they were 40 years of age or older and had any resection or biopsy of their tumor. Exclusion criteria included previous radiation to the head or neck, any previous chemotherapy, significant pulmonary disease, and a 5-year history of other cancers except cervical cancer in situ and non-melanoma skin cancer. Tumors could not have spread to noncontiguous leptomeninges, and patients could not have gliomatosis cerebri. Patients had to have a Karnofsky performance score of 60 or higher, and a neurological function score of 3 or less.

Dr. Buckner and his collaborators also assessed tumors for IDH1 mutational status by performing immunostaining with the mutation-specific monoclonal antibody IDH1 R132H; appropriate tissue was available for testing in slightly less than half of the patients in each study arm. The mutation was present in 35/57 patients (61%) in the radiotherapy-only arm, and 36/56 patients (64%) in the radiotherapy plus PCV arm. Patients with oligodendroglioma were most likely to have IDH1 R132H mutations. Sample sizes were too small to determine the effect of other IDH mutations or co-deletion of chromosome arms 1p and 19q.

In multivariable analysis, the presence of the IDH1 R132H mutation was identified as an independent prognostic factor for better overall survival (OS) and progression-free survival (PFS), regardless of the treatment administered. Those with the mutation still benefited significantly from receiving radiotherapy plus PCV rather than radiotherapy alone (P = .02 for OS, P less than .001 for PFS).

Exploratory analysis that broke down OS and PFS by cancer type showed that “the superiority of radiation therapy plus chemotherapy over radiation therapy alone was seen with all histologic diagnoses, although the difference did not reach significance among patients with astrocytoma,” wrote Dr. Buckner and his collaborators.

When all patients lost to follow-up in both groups were assessed as having died, the sensitivity analysis still showed benefit for radiotherapy plus PCV (HR for death, compared with radiotherapy alone, 0.72; P = .03).

The value of the long-term follow-up, wrote Dr. Buckner and his colleagues, was that “The separation of the progression-free survival curves of the two treatment groups did not begin until 2 to 3 years after randomization, although approximately 25% of the patients in each group had disease progression by then.”

Dr. Buckner and his collaborators emphasized that the patient-physician team should consider all factors in making treatment decisions, saying, “Patients and their physicians will have to weigh whether the longer survival justified the more toxic therapeutic approach.“

[email protected]

On Twitter @karioakes

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Adding a chemotherapy combination to radiation therapy for initial treatment of low-grade gliomas significantly improved overall survival and progression-free survival, regardless of the tumor type, investigators report in the New England Journal of Medicine.

Grade 2 glioma patients who received radiation plus the combination of procarbazine, lomustine (also called CCNU), and vincristine (PCV) had a longer median overall survival than those who received radiation alone (13.3 vs. 7.8 years; hazard ratio for death, 0.59; P = .003). Of those who received radiation plus chemotherapy, the progression-free survival rate at 10 years was 51%, compared with 21% for the group who received radiation alone, Dr. Jan Buckner and his colleagues report (N Engl J Med. 2016;374:1344-55. doi: 10.1056/NEJMoa1500925).

“The magnitude of treatment benefit from combined chemotherapy plus radiation therapy is substantial, but the toxic effects are greater than those observed with radiation therapy alone,” wrote Dr. Buckner, professor of oncology at the Mayo Clinic, Rochester, Minn., and associates. Patients who received radiation plus PCV had more toxic side effects from their therapy, though most effects were grade 1 and 2.

For this study, 254 patients were randomized, with 128 assigned to radiotherapy alone, and 126 assigned to radiotherapy plus PCV. A total of 126 patients in the radiotherapy arm were included in the analysis, with one patient not receiving the intervention and 14 patients lost to follow-up at some point during the 10 years of the study. In the radiotherapy plus PCV arm, 125 patients were eligible for evaluation, and all of those patients were included in the analysis. Twenty-six patients in this arm were lost to follow-up, and 72 patients discontinued the intervention in this arm (this figure included four patients who died).

Tumor types included in the study were grade 2 astrocytoma, oligoastrocytoma, or oligodendroglioma.

Patients were included if they were between 18 and 39 years of age and had received a subtotal resection or biopsy of their tumor, or if they were 40 years of age or older and had any resection or biopsy of their tumor. Exclusion criteria included previous radiation to the head or neck, any previous chemotherapy, significant pulmonary disease, and a 5-year history of other cancers except cervical cancer in situ and non-melanoma skin cancer. Tumors could not have spread to noncontiguous leptomeninges, and patients could not have gliomatosis cerebri. Patients had to have a Karnofsky performance score of 60 or higher, and a neurological function score of 3 or less.

Dr. Buckner and his collaborators also assessed tumors for IDH1 mutational status by performing immunostaining with the mutation-specific monoclonal antibody IDH1 R132H; appropriate tissue was available for testing in slightly less than half of the patients in each study arm. The mutation was present in 35/57 patients (61%) in the radiotherapy-only arm, and 36/56 patients (64%) in the radiotherapy plus PCV arm. Patients with oligodendroglioma were most likely to have IDH1 R132H mutations. Sample sizes were too small to determine the effect of other IDH mutations or co-deletion of chromosome arms 1p and 19q.

In multivariable analysis, the presence of the IDH1 R132H mutation was identified as an independent prognostic factor for better overall survival (OS) and progression-free survival (PFS), regardless of the treatment administered. Those with the mutation still benefited significantly from receiving radiotherapy plus PCV rather than radiotherapy alone (P = .02 for OS, P less than .001 for PFS).

Exploratory analysis that broke down OS and PFS by cancer type showed that “the superiority of radiation therapy plus chemotherapy over radiation therapy alone was seen with all histologic diagnoses, although the difference did not reach significance among patients with astrocytoma,” wrote Dr. Buckner and his collaborators.

When all patients lost to follow-up in both groups were assessed as having died, the sensitivity analysis still showed benefit for radiotherapy plus PCV (HR for death, compared with radiotherapy alone, 0.72; P = .03).

The value of the long-term follow-up, wrote Dr. Buckner and his colleagues, was that “The separation of the progression-free survival curves of the two treatment groups did not begin until 2 to 3 years after randomization, although approximately 25% of the patients in each group had disease progression by then.”

Dr. Buckner and his collaborators emphasized that the patient-physician team should consider all factors in making treatment decisions, saying, “Patients and their physicians will have to weigh whether the longer survival justified the more toxic therapeutic approach.“

[email protected]

On Twitter @karioakes

Adding a chemotherapy combination to radiation therapy for initial treatment of low-grade gliomas significantly improved overall survival and progression-free survival, regardless of the tumor type, investigators report in the New England Journal of Medicine.

Grade 2 glioma patients who received radiation plus the combination of procarbazine, lomustine (also called CCNU), and vincristine (PCV) had a longer median overall survival than those who received radiation alone (13.3 vs. 7.8 years; hazard ratio for death, 0.59; P = .003). Of those who received radiation plus chemotherapy, the progression-free survival rate at 10 years was 51%, compared with 21% for the group who received radiation alone, Dr. Jan Buckner and his colleagues report (N Engl J Med. 2016;374:1344-55. doi: 10.1056/NEJMoa1500925).

“The magnitude of treatment benefit from combined chemotherapy plus radiation therapy is substantial, but the toxic effects are greater than those observed with radiation therapy alone,” wrote Dr. Buckner, professor of oncology at the Mayo Clinic, Rochester, Minn., and associates. Patients who received radiation plus PCV had more toxic side effects from their therapy, though most effects were grade 1 and 2.

For this study, 254 patients were randomized, with 128 assigned to radiotherapy alone, and 126 assigned to radiotherapy plus PCV. A total of 126 patients in the radiotherapy arm were included in the analysis, with one patient not receiving the intervention and 14 patients lost to follow-up at some point during the 10 years of the study. In the radiotherapy plus PCV arm, 125 patients were eligible for evaluation, and all of those patients were included in the analysis. Twenty-six patients in this arm were lost to follow-up, and 72 patients discontinued the intervention in this arm (this figure included four patients who died).

Tumor types included in the study were grade 2 astrocytoma, oligoastrocytoma, or oligodendroglioma.

Patients were included if they were between 18 and 39 years of age and had received a subtotal resection or biopsy of their tumor, or if they were 40 years of age or older and had any resection or biopsy of their tumor. Exclusion criteria included previous radiation to the head or neck, any previous chemotherapy, significant pulmonary disease, and a 5-year history of other cancers except cervical cancer in situ and non-melanoma skin cancer. Tumors could not have spread to noncontiguous leptomeninges, and patients could not have gliomatosis cerebri. Patients had to have a Karnofsky performance score of 60 or higher, and a neurological function score of 3 or less.

Dr. Buckner and his collaborators also assessed tumors for IDH1 mutational status by performing immunostaining with the mutation-specific monoclonal antibody IDH1 R132H; appropriate tissue was available for testing in slightly less than half of the patients in each study arm. The mutation was present in 35/57 patients (61%) in the radiotherapy-only arm, and 36/56 patients (64%) in the radiotherapy plus PCV arm. Patients with oligodendroglioma were most likely to have IDH1 R132H mutations. Sample sizes were too small to determine the effect of other IDH mutations or co-deletion of chromosome arms 1p and 19q.

In multivariable analysis, the presence of the IDH1 R132H mutation was identified as an independent prognostic factor for better overall survival (OS) and progression-free survival (PFS), regardless of the treatment administered. Those with the mutation still benefited significantly from receiving radiotherapy plus PCV rather than radiotherapy alone (P = .02 for OS, P less than .001 for PFS).

Exploratory analysis that broke down OS and PFS by cancer type showed that “the superiority of radiation therapy plus chemotherapy over radiation therapy alone was seen with all histologic diagnoses, although the difference did not reach significance among patients with astrocytoma,” wrote Dr. Buckner and his collaborators.

When all patients lost to follow-up in both groups were assessed as having died, the sensitivity analysis still showed benefit for radiotherapy plus PCV (HR for death, compared with radiotherapy alone, 0.72; P = .03).

The value of the long-term follow-up, wrote Dr. Buckner and his colleagues, was that “The separation of the progression-free survival curves of the two treatment groups did not begin until 2 to 3 years after randomization, although approximately 25% of the patients in each group had disease progression by then.”

Dr. Buckner and his collaborators emphasized that the patient-physician team should consider all factors in making treatment decisions, saying, “Patients and their physicians will have to weigh whether the longer survival justified the more toxic therapeutic approach.“

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Adding chemo to radiation boosts survival from low-grade gliomas
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Adding chemo to radiation boosts survival from low-grade gliomas
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FROM THE NEW ENGLAND JOURNAL OF MEDICINE

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Key clinical point: Adding chemotherapy to radiotherapy improved progression-free survival and overall survival in patients with low-grade glioma.

Major finding: Median overall survival was 13.3 years for those receiving radiotherapy plus chemotherapy, compared with 7.8 years for radiotherapy alone (hazard ratio for death, 0.59; P = .003).

Data source: Longitudinal study of 254 patients with grade 2 gliomas receiving radiation therapy alone or radiation therapy plus procarbazine, lomustine, and vincristine.

Disclosures: The study was supported by the National Cancer Institute and did not receive funding from commercial sources. Dr. Bell and Dr. Chakravarti reported a planned patent application related to this work. Dr. Buckner, Dr. Gilbert, Dr. Mehta, and Dr. Suh reported support from pharmaceutical companies outside the scope of this study.