PIPC Forum 2022: Inclusion
On December 13, 2022, the Partnership to Improve Patient Care held its 13th Annual Forum highlighting the goals of Inclusion in Health Care Research and Value Assessment.
Sara van Geertruyden, Executive Director of PIPC, highlighted PIPC’s leadership in the Congressional authorization of the Patient-Centered Outcomes Research Institute, an organization conducting comparative clinical effectiveness research, with a statutory charge to include patients and people with disabilities at every step of the research process and protecting against the use of discriminatory metrics in research and Medicare decisions. Sara described the quality-adjusted life year or QALY as a discriminatory metric used to measure the cost effectiveness of treatments that is a relic of discrimination and bias in healthcare – a metric that was created to ration health care against people that society deemed not worthy. She highlighted that the National Council on Disability, an independent federal agency advising Congress and the administration on disability policy, recommended a consistent ban on the use of QALYs be extended to all federal health programs. She also discussed the administration’s implementation of the Inflation Reduction Act (IRA), which requires them to use comparative clinical effectiveness research and other sources of evidence to establish the required fair price for prescription drugs. She urged that the administration enforce the statute’s bar on referencing QALYs and create robust mechanisms for engaging affected patients and people with disabilities. In closing, Sara expressed optimism for bipartisanship on policies supporting patients and people with disabilities.
The first panel involved a bipartisan, bicameral discussion about priorities for the next Congress, including efforts to extend a ban on use of QALYs across federal programs and support robust engagement of patients and people with disabilities as the administration implements the IRA’s policies related to drug pricing.
The second panel included Donna Cryer, founder and CEO of the Global Liver Institute, Kenny Mendez, President and CEO of the Asthma and Allergy Foundation of America (AAFA), and Thayer Roberts, Deputy Director of PIPC.
Donna began by describing a published article she co-authored entitled, “Is the QALY Fit for Purpose?” As a liver transplant patient, Donna shared the concerns of others on the ability to reduce people’s experiences with a condition to a number, given their varying experiences and heterogeneity. She highlighted that value and affordability are different things, both worthwhile, but perfect health is not equal to a valuable life. Also, measuring what matters to individual patients is essential. For example, cognitive function is a metric that is important to liver transplant patients that was not being measured. She concluded the QALY is not fit for the purpose of defining coverage and reimbursement and urged that the Centers for Medicare and Medicaid Services (CMS) not rely on it as they implement the new drug pricing provisions of the IRA. She urged emphasis on filling research gaps, suggesting collaboration with Historically Black Colleges and Universities (HBCUs), more participatory research and more targeted research responding to patient needs. She also shared concerns with other panelists about the comparator used in studies, such as the need to include interventions other than medication. When treatments are valued using clinical trials early in their development, we know the least about them, and therefore must continue collecting real-world evidence. Donna strongly supported CMS having an ongoing dialogue with patients and people with disabilities, with clear pathways to engage and expectations for information to be shared, similar to other agencies with robust engagement processes.
Kenny described AAFA’s Asthma Disparities in America Report published by the foundation as part of AAFA’s strategic emphasis on asthma disparities and health equity. Black Americans are 3 times more likely to die from asthma, 5 times more likely to be treated in the emergency room and Black Women have the highest rate of mortality of any gender and ethnic group. FDA is focused on whether a treatment is safe and effective based on randomized clinical trials. Kenny discussed the lack of diversity within clinical trials and over-reliance on that clinical trial data as part of QALY-based value assessments that result in fundamental flaws. He discussed a new AAFA program, HEAL Innovation, investing in community-based asthma programs shown to reduce emergency room visits, hospitalizations, and improve quality of life by removing triggers in the home for asthma, providing valuable cost savings that should be considered outside of the impact of the medication itself. Kenny advised that his organization is active in collecting data and sharing it with payors to urge their investment in what works. He also supported PIPC’s work against discrimination and use of QALYs.
Thayer discussed a new report published by PIPC describing the German system for valuing treatments. Germany has a body, the joint federal commission (G-BA) that does not use QALYs but exists to assess the treatment’s value, doing so by comparing with other treatments they choose - yet the comparator is often not the best comparator based on clinical outcome or patient need, just the lowest cost drug for that indication. Thayer found that the research that the G-BA will accept to value treatments is stringent, not accepting surrogate end points and only accepting randomized clinical trials instead of real-world evidence. There is also a very minimal role for the impacted population to engage other than submitting formal feedback different from the FDA’s Patient-Focused Drug Development process. For example, Thayer noted that hepatitis C drugs cure about 90% of hepatitis C patients yet were found by GBA to have no additional clinical benefit because the sustained virological response was not considered an end point in the GBA's assessment. Not being able to detect hepatitis C for 12 weeks was not a benefit by their standards. To underscore the challenge, Thayer noted that the National Institute for Health and Care Excellence (NICE) in the U.K. found more treatments effective than the German system, highlighting then that swapping out the QALY without better evidence does not solve the problem. Thayer hoped this is instructive for CMS to establish standards for the research used to assess the value of treatments under the IRA – to make sure that patients and people with disabilities have a seat at the table, relevant providers have a seat at the table, and they have a full data set and are not relying on flawed data and academic studies that may not present the full picture. She emphasized that patient groups have the most knowledge of the patient experience and the evidence base for consideration by policymakers. Thayer suggested an Ombudsman at CMS to be the point of contacts for patients and people with disabilities, as well as for notice and comment opportunities related to the implementation of the IRA’s drug pricing provisions.
Sara van Geertruyden, Executive Director of PIPC, highlighted PIPC’s leadership in the Congressional authorization of the Patient-Centered Outcomes Research Institute, an organization conducting comparative clinical effectiveness research, with a statutory charge to include patients and people with disabilities at every step of the research process and protecting against the use of discriminatory metrics in research and Medicare decisions. Sara described the quality-adjusted life year or QALY as a discriminatory metric used to measure the cost effectiveness of treatments that is a relic of discrimination and bias in healthcare – a metric that was created to ration health care against people that society deemed not worthy. She highlighted that the National Council on Disability, an independent federal agency advising Congress and the administration on disability policy, recommended a consistent ban on the use of QALYs be extended to all federal health programs. She also discussed the administration’s implementation of the Inflation Reduction Act (IRA), which requires them to use comparative clinical effectiveness research and other sources of evidence to establish the required fair price for prescription drugs. She urged that the administration enforce the statute’s bar on referencing QALYs and create robust mechanisms for engaging affected patients and people with disabilities. In closing, Sara expressed optimism for bipartisanship on policies supporting patients and people with disabilities.
The first panel involved a bipartisan, bicameral discussion about priorities for the next Congress, including efforts to extend a ban on use of QALYs across federal programs and support robust engagement of patients and people with disabilities as the administration implements the IRA’s policies related to drug pricing.
The second panel included Donna Cryer, founder and CEO of the Global Liver Institute, Kenny Mendez, President and CEO of the Asthma and Allergy Foundation of America (AAFA), and Thayer Roberts, Deputy Director of PIPC.
Donna began by describing a published article she co-authored entitled, “Is the QALY Fit for Purpose?” As a liver transplant patient, Donna shared the concerns of others on the ability to reduce people’s experiences with a condition to a number, given their varying experiences and heterogeneity. She highlighted that value and affordability are different things, both worthwhile, but perfect health is not equal to a valuable life. Also, measuring what matters to individual patients is essential. For example, cognitive function is a metric that is important to liver transplant patients that was not being measured. She concluded the QALY is not fit for the purpose of defining coverage and reimbursement and urged that the Centers for Medicare and Medicaid Services (CMS) not rely on it as they implement the new drug pricing provisions of the IRA. She urged emphasis on filling research gaps, suggesting collaboration with Historically Black Colleges and Universities (HBCUs), more participatory research and more targeted research responding to patient needs. She also shared concerns with other panelists about the comparator used in studies, such as the need to include interventions other than medication. When treatments are valued using clinical trials early in their development, we know the least about them, and therefore must continue collecting real-world evidence. Donna strongly supported CMS having an ongoing dialogue with patients and people with disabilities, with clear pathways to engage and expectations for information to be shared, similar to other agencies with robust engagement processes.
Kenny described AAFA’s Asthma Disparities in America Report published by the foundation as part of AAFA’s strategic emphasis on asthma disparities and health equity. Black Americans are 3 times more likely to die from asthma, 5 times more likely to be treated in the emergency room and Black Women have the highest rate of mortality of any gender and ethnic group. FDA is focused on whether a treatment is safe and effective based on randomized clinical trials. Kenny discussed the lack of diversity within clinical trials and over-reliance on that clinical trial data as part of QALY-based value assessments that result in fundamental flaws. He discussed a new AAFA program, HEAL Innovation, investing in community-based asthma programs shown to reduce emergency room visits, hospitalizations, and improve quality of life by removing triggers in the home for asthma, providing valuable cost savings that should be considered outside of the impact of the medication itself. Kenny advised that his organization is active in collecting data and sharing it with payors to urge their investment in what works. He also supported PIPC’s work against discrimination and use of QALYs.
Thayer discussed a new report published by PIPC describing the German system for valuing treatments. Germany has a body, the joint federal commission (G-BA) that does not use QALYs but exists to assess the treatment’s value, doing so by comparing with other treatments they choose - yet the comparator is often not the best comparator based on clinical outcome or patient need, just the lowest cost drug for that indication. Thayer found that the research that the G-BA will accept to value treatments is stringent, not accepting surrogate end points and only accepting randomized clinical trials instead of real-world evidence. There is also a very minimal role for the impacted population to engage other than submitting formal feedback different from the FDA’s Patient-Focused Drug Development process. For example, Thayer noted that hepatitis C drugs cure about 90% of hepatitis C patients yet were found by GBA to have no additional clinical benefit because the sustained virological response was not considered an end point in the GBA's assessment. Not being able to detect hepatitis C for 12 weeks was not a benefit by their standards. To underscore the challenge, Thayer noted that the National Institute for Health and Care Excellence (NICE) in the U.K. found more treatments effective than the German system, highlighting then that swapping out the QALY without better evidence does not solve the problem. Thayer hoped this is instructive for CMS to establish standards for the research used to assess the value of treatments under the IRA – to make sure that patients and people with disabilities have a seat at the table, relevant providers have a seat at the table, and they have a full data set and are not relying on flawed data and academic studies that may not present the full picture. She emphasized that patient groups have the most knowledge of the patient experience and the evidence base for consideration by policymakers. Thayer suggested an Ombudsman at CMS to be the point of contacts for patients and people with disabilities, as well as for notice and comment opportunities related to the implementation of the IRA’s drug pricing provisions.