1. DREDF Releases Report Responding to a Flawed ICER Analyses, Instead Finds that QALY Violates Disability Nondiscrimination Law. Click here to view the report.
2. PCORI Invites Your Input: Science of Engagement Potential Funding Initiative. See below for details.
3. Issue Brief: Implications of Reliance on Health Technology Assessment in the VA and TRICARE Formularies. Click here to read the full report.
4. Washington Must Help Patients Choose, Not Dictate Their Care. Click here to read the article.
5. DREDF Comments on Tenncare III and Impact on People with Disabilities. Click here to view the comments.
6. PIPC Chairman Tony Coelho Applauds HHS Report Acknowledgment of Equity and Access Implications of Discriminatory Metrics and Urges Policymakers to Avoid One-Size Fits-All Value Judgements. See details below.
7. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Hypertrophic Cardiomyopathy. Click here to read the letter.
8. PIPC Comments on PCORI Proposed National Priorities for Health, click here to read the letter.
9. PCORI Announces Engagement Award on Consideration of the Full Range of Outcomes Data - DUE October 1, 2021, see details below.
10. Upcoming opportunity within the Center for Evidence and Practice Improvement at AHRQ, see details below.
11. New Disability Equity Collaborative Paper Identifies Research Priorities for Advancing Equitable Care for People with Disabilities. Click here to read the press release.
12. It is Time to Let Go of the QALY’s Legacy of Discrimination. Click here to read the article.
13. 2021 PCORI Annual Meeting Scheduled for Nov. 17-19, click here for details.
14. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
15. Emerging Threats in States for Use of Discriminatory Metrics, see details below.
16. IVI to Hold Second Methods Summit, click here to learn more.
17. ICER's QALY-Based Study Topics: Hypertrophic Cardiomyopathy, Myasthenia Gravis, Atopic Dermatitis, Alzheimer's Disease, Asthma, Type 2 Diabetes, click here to provide patient input.
18. Upcoming Events and Webinars, see details below.
19. Medical Journal Articles, see details below.
20. AHRQ Effective Program Updates, see details below.
This week, the Disability Rights Education and Defense Fund ("DREDF") responded directly to a report commissioned for the Institute for Clinical and Economic Review (“ICER”), an organization specializing in clinical cost-effectiveness analyses relying on the QALY, in which its hired legal consultants posit that the use of QALY as a measure of the cost-effectiveness of specific drugs and therapies “poses absolutely no risk of discrimination against any patient group.” DREDF strongly disagrees with this conclusion. In its response, DREDF challenges the factual and legal assumptions of ICER's report and explains how the use of the QALY, even in tandem with alternative measures such as the Equal Value of Life Years Gained (“evLYG”), violates disability nondiscrimination law.
Later this month, DREDF will join Familia Unida to celebrate National Disabilities Awareness Month and share information about its new report and the ongoing work of the disability community against disability discrimination in healthcare. At that time, information about the report will be made available in Spanish as well. We will provide more details about the webinar soon! Click here to view the DREDF report. Click here to view the one-pager.
2. PCORI Invites Your Input: Science of Engagement Potential Funding Initiative
PCORI has opened a Request for Information (RFI) to inform the development of a potential funding initiative around the Science of Engagement. Research funding opportunities for the Science of Engagement could expand the evidence base on the approaches for effectively engaging diverse patients and stakeholders throughout the research process. PCORI is seeking input from potential applicants, patients and other stakeholders, and organizations who participate in research or engage communities. Feedback is requested on the research topics that could be most useful to research teams for implementation of more effective engagement. Additionally, they are inviting input on the resources and facilitators necessary to successfully support studies that address these research topics.
The RFI is open from September 27 – November 19, 2021. PCORI will also host two webinars to overview the Science of Engagement and present an opportunity to ask questions about this RFI. You can find more information and download the RFI here. If you have any questions, please email SoE-RFI@pcori.org.
3. Issue Brief: Implications of Reliance on Health Technology Assessment in the VA and TRICARE Formularies
This issue brief from the Partnership to Improve Patient Care (PIPC) provides an overview of the U.S. Department of Veterans Affairs (VA) and the Department of Defense (DoD) TRICARE program policies that rely on health technology assessment to make reimbursement and coverage decisions.
Veterans, members of the military and their families often have unique health care needs and preferences. It is important they are given options for high quality health care that meets their needs. No patient is average, which is why there has been a movement towards personalized medicine in health care with the goal of empowering the patient and clinician so that each person receives the right treatment at the right time. Yet, the U.S. Department of Veterans Affairs (VA) and the Department of Defense (DoD) TRICARE program rely on methods to lower costs by relying on assessments of clinical and cost effectiveness that use flawed and discriminatory methods to determine which treatments it will and will not cover, without considering the holistic value of treatments to the patient and his or her family.
4. Washington Must Help Patients Choose, Not Dictate Their Care
PIPC Chair Tony Coelho wrote an opinion piece published in The Hill. In 2010, he notes that the Affordable Care Act (ACA) “recognizes not only the importance of strong clinical evidence to support decision-making but also the need to use the research to support doctors’ and patients’ decisions, not to dictate what those decisions should be.” Coelho explains that this principle is again under debate "as Congress considers new strategies for informing Medicare reimbursement and coverage" and "the bipartisan history PCORI's creation should provide some lessons." Click here to read the full article.
5. DREDF Comments on Tenncare III and Impact on People with Disabilities
The State of Tennessee submitted a waiver application to the Centers for Medicare and Medicaid Services, which recently received comments on it. The Disability Rights Education and Defense Fund is concerned that the waiver "permitted TennCare to change its financial structure to an inflexible block grant.” DREDF is concerned its approval creates new barriers to health care for hundreds of thousands of low-income people, including people with disabilities, and will result in large health coverage losses, thus undermining the express purposes of the Medicaid program. Consistent with comments provided by advocates related to the earlier version of the waiver application (TennCare II), DREDF urged HHS to rescind the TennCare III approval in order to avoid the harms that the program changes will inflict on low-income Tennesseans. Click here to view DREDF comments. Click here to view the letter from advocates on the TennCare II application.
6. PIPC Chairman Tony Coelho Applauds HHS Report Acknowledgment of Equity and Access Implications of Discriminatory Metrics and Urges Policymakers to Avoid One-Size Fits-All Value Judgements
In a report from the U.S. Department of Health and Human Services (HHS) to the White House laying out a “Comprehensive Plan for Addressing High Drug Prices,” HHS acknowledged the equity implications of certain methodologies, such as quality-adjusted-life-years (QALYs), for people of all ages with disabilities and chronic conditions and stated that drug pricing reforms should avoid utilization of methodologies that adversely impact access to needed medications for vulnerable populations.
In response to the report, PIPC Chairman Tony Coelho issued a statement expressing appreciation to Secretary Becerra for acknowledging the dangers of discriminatory metrics, like the QALY, while cautioning the Administration to avoid one-size-fits all value judgements that could interfere with individualized care and personalized medicine. Chairman Coelho then urged the Administration and Congress to strengthen the essential protections against the misuse of comparative effectiveness research that advocates fought so hard for in the Affordable Care Act.” Click here to read the entire press release. Click here to view the HHS report to the White House.
7. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Hypertrophic Cardiomyopathy
In a letter to the Institute for Clinical and Economic Review (ICER), the Partnership to Improve Patient Care (PIPC) offered comments on ICER's draft evidence report for Hypertrophic Cardiomyopathy (HCM) treatments.
The letter criticizes ICER's assessment of mayacamten, the HCM treatment at the focal point of this report, for being conducted prior the completion of ongoing studies into the drug's long-tern effectiveness. PIPC also took issue with ICER for its reliance on the discriminatory quality-adjusted-life-years (QALY) metric, as well as refusing to incorporate key outcomes requested by patients and clinicians in constructing this model.
8. PIPC Comments on PCORI Proposed National Priorities for Health
In a comment letter to the Patient-Centered Outcomes Research Institute (PCORI), the Partnership to Improve Patient Care (PIPC) offered feedback on PCORI's Proposed National Priorities for Health.
The letter encourages PCORI work closely with stakeholders in identifying the topics to be prioritized within each broad area, the research agenda for each topic, and the outcomes data to be collected and analyzed as part of the research. PIPC also reiterated its strong support for PCORI’s mandate of conducting research on the comparative clinical effectiveness of medical treatments and services, as well as the statutory prohibition against cost- effectiveness analysis.
9. PCORI Announces Engagement Award on Consideration of the Full Range of Outcomes Data
As a component of PCORI’s reauthorization, a provision was included requiring PCORI-funded research to consider the full range of outcomes data, including potential burdens and economic impacts. PCORI has adopted principles for collecting this data. As PCORI moves forward in this process, it is accepting applications for Engagement Awards to fund convenings focused on defining appropriate and relevant burdens and economic impact data, identifying sources of and gaps in data, and opportunities to advance the rigor and standards when conducting research that considers a full range of outcomes data. PCORI will play an important role in defining what factors on cost and economic burden are important to the individuals our healthcare system serves and how to collect this data in a rigorous and patient-centric manner. Organizations wishing to apply for these Engagement Awards may do so here. Applications are due October 1, 2021.
10. Upcoming Opportunity Within the Center for Evidence and Practice Improvement at AHRQ
AHRQ will soon be hiring a Senior Staff Service Fellow in CEPI’s Office of the Director to support activities funded by the Patient-Centered Outcomes Research Trust Fund (PCOR TF) under the National Center for Excellence in Primary Care Research (NCEPCR). The Senior Staff Service Fellow will support AHRQ’s mandated roles under the PCOR TF in evidence dissemination and implementation in primary care as well as of training and capacity building for comparative effectiveness research in primary care settings.
The Senior Staff Service Fellow designs, executes and evaluates projects funded by the PCOR TF to increase the uptake of evidence in primary care including by using new models of primary/ambulatory care delivery, advancing health equity and engaging learning health systems. AHRQ is looking for a team player to lead a multidisciplinary team to design, that leads national initiatives to disseminate and implement PCOR findings related to transforming primary/ambulatory care delivery to make care more equitable and patient-centered including a focus on people living with multiple chronic conditions and other at risk populations.
This Senior Staff Service Fellow will also advise other members of CEPI and AHRQ on dissemination and implementation activities related to the PCOR TF; supports and aligns ongoing primary care projects across all divisions within the Center as well as across AHRQ. Serves as a recognized expert within the areas of primary care, primary care research and implementation science.
11. New DEC Paper Identifies Research Priorities for Advancing Equitable Care for People with Disabilities.
A research workgroup of the Disability Equity Collaborative (DEC) released its first paper, "Patient-Centered Outcomes Research Priorities to Advance Disability Equity" funded by the Patient-Centered Outcomes Research Institute (PCORI). The publication provides a clear, much needed blueprint for the field, identifying critical areas for research, providing guidance for funding agencies, and outlining gaps that policy makers need to address to assure equitable health care for people with disabilities. Click here to read the white paper.
12. It is Time to Let Go of the QALY's Legacy of Discrimination
In a piece published by The Center for Dignity in Healthcare for People with Disabilities, authors Kelly Israel of Autistic Self Advocacy Network and Sara van Geertruyden of PIPC explain the Quality-Adjusted Life Year (QALY), how it is used, and its discriminatory implications. The authors explain the politics of the QALY as well as some of the challenges we face as we try to move beyond this discriminatory metric. Ultimately they conclude that investment is needed to develop methods to assess values of treatments that do not discriminate and sustained advocacy is needed to remind states and other entities of their obligations of nondiscrimination under the ADA and other disability rights laws. Click here to read the full article.
13. 2021 PCORI Annual Meeting Scheduled for Nov. 17-19
PCORI announced that its 2021 annual meeting will be held virtually on Wednesday, November 17 through Friday, November 19. The event serves as a report to the nation on PCORI’s progress in funding initiatives to determine which care approaches work best, for whom, and under what circumstances. Click here for details.
14. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- Canada: CF patients are suffering due to the restricted access of Trikafta, a life saving drug, which is only available to the very sick. Advocates in Ontario will not stop fighting to ensure the government approves funding for Trikafta for all CF patients. Mother of CF patients is criticizing the the government's decision to restrict access to the life saving drug.
15. Emerging Threats in States for Use of Discriminatory Metrics
Several states are considering policies that would reference entities such as the Institute for Clinical and Economic Review (ICER), which calls the discriminatory quality-adjusted life year (QALY) the “gold standard” for value assessment, and others are considering policies to import QALY-based coverage and reimbursement decisions from other countries that restrict access to care. Yet, federal policymakers have emphasized that the use of discriminatory metrics is subject to civil rights laws such as the Americans with Disabilities Act. We encourage sharing resources for advocates in key states mentioned below to ensure protections against discrimination!
- Click here to learn more about the potential for Prescription Drug Affordability Boards to discriminate by incorporating the use of QALYS.
- Click here to view the Value Our Health state template legislation that would protect people with disabilities and chronic conditions from the use of QALYs and similar metrics developed by third parties such as ICER in decisions related to reimbursement and coverage, as well as ensure their engagement in decision-making.
- Click here to view a one pager about the flaws in ICER’s methodology.
- Click here to view information from experts on the downside of referencing foreign countries.
- Click here to learn about statutory protections against use of QALYs.
Other states to watch: Rhode Island, Minnesota and Washington State are also potential targets for future legislation that could trigger the use of discriminatory QALYs.
Efforts are underway to advance legislation that would create a Prescription Drug Affordability Board in the State of Virginia, triggering concerns about potential discrimination, according to recent news. It is not clear that these efforts will include provisions to bar discrimination and protect patient access to medications, such as achieved by disability advocates in other states such as Oregon where the legislature barred the use of QALYs in similar Board deliberations.
Renewed efforts are anticipated in New Mexico to advance legislation creating a Prescription Drug Affordability Board, triggering concerns about potential discrimination. It is not clear that these efforts will include provisions to bar discrimination and protect patient access to medications, such as achieved by disability advocates in other states. The news about the effort has not mentioned including a ban on the use of discriminatory QALYs nor including patient and disability representation or other patient protections from restricted access to care.
The Maryland legislature passed legislation earlier this year creating a Prescription Drug Affordability Board. The legislation did not include protections for patients and people with disabilities such as barring the use of discriminatory QALYs. The Board has begun meeting to hear from stakeholders, including hearing a presentation from the Institute for Clinical and Economic Review which relies on QALYs to assess treatment value. Legislation was introduced this year to bar the use of QALYs and implement patient protections that has not advanced in the legislature. On August 3, organizations representing patients and people with disabilities sent a letter urging the Board to avoid policies that would potentially discriminate by relying on discriminatory metrics such as QALYs.
The Governor of Colorado has signed into law SB 21-175, which creates a prescription drug affordability review board. SB 21-75 did include an amendment that prohibits the use of QALYs in the section of the bill that determines an upper payment limit. In some states these types of boards and commissions have referenced value assessments based on QALYs from the Institute for Clinical and Economic Review (ICER). In Massachusetts, the Health Policy Commission went so far as to contract with ICER. In order to mitigate this, it is important that the Board has representation from patients and people with disabilities. The Governors office is accepting applications for both the Colorado Prescription Drug Affordability Review Board and the Colorado Prescription Drug Affordability Advisory Council. It is essential that the Board and Advisory Committee include representation from patients and people with disabilities to ensure that that the Board’s deliberations do not include consideration of QALYs and other metrics that may discriminate or lead to restricted access for people with disabilities and chronic conditions or older adults.
The Massachusetts House and Senate have each taken the positive step of introducing An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with Disabilities, H.201 and S.753. This bill would enshrine essential patient protections including a ban on the use of the QALY, a requirement for research to meet patient-centeredness criteria, and robust engagement of the patient and disability communities in health care decision making.
16. IVI to Hold Second Methods Summit
The Innovation and Value Initiative (IVI) announced that it will hold a second methods summit to drive patient-centered value assessment. The multi-stakeholder event is intended to drive consensus on priority patient inputs, methods, and research that more fully represent the patient perspective in comparative effectiveness research and economic evaluations of health care interventions. The three-part series will take place in Fall 2021. Overall, the goal of IVI’s summit is to define an action agenda for patient-centered outcomes research and cost-effectiveness research by exploring patient outcomes, beyond traditional clinical factors, that represent important impacts to patients and must be accounted for in economic analyses. Click here to learn more.
17. ICER's QALY-Based Study Topics: Hypertrophic Myocardiopathy, Myasthenia Gravis, Atopic Dermatitis, Alzheimer's Disease, Asthma, Type 2 Diabetes
The Institute for Clinical Economic Review (ICER) conducts cost effectiveness studies for insurers using the cost-per-QALY methodology. ICER provides guidance on its website for patients and patient advocates to provide direct input related to their experiences with the disease. Click here to provide patient input. Click here to view the topics and deadlines.
- Type 2 Diabetes: Draft Scoping Document and Research Protocol available. 11/09/2021: Draft Evidence Report. Meeting 1/20/2022: Deliberation and vote on evidence presented in ICER's report on therapies for Type 2 Diabetes.
- Hypertrophic Cardiomyopathy: Evidence Report Available. Meeting 10/22/2021: CTAF will deliberate and vote on evidence presented in ICER’s report on therapies for obstructive hypertrophic cardiomyopathy.
- Myasthenia Gravis: Evidence Report available. Evidence Presentation available. 10/20/2021: Final Evidence Report and Meeting Summary.
- Unsupported Price Increase: 11/16/2021: Final Report.
- Asthma: Draft Evidence Report available. Public Comments open through 10/14/2021. Model Analysis Plan available. Meeting 11/19/2021: New England CEPAC will deliberate and vote on evidence presented in ICER’s report on therapies for severe asthma.
- Fair Access: Coverage Policies in 2020: Protocol Available. 12/01/2021: Final Report.
18. Upcoming Events and Webinars
Maryland Virtual Community Convening on Research and Health
October 13, 2021
Click here for details.
Colorado Virtual Community Convening on Research and Health
October 21, 2021
Click here for details.
Board of Governors Meeting
October 26, 2021
Click here for details.
19. Medical Journal Articles
“It’s Time to Represent”: Shifting the Paradigm to Improve the Quality of Inputs into Value Assessment Frameworks, click here to view.
Evaluation of Financial Outcomes Under a Value-Based Payment Program for Community Pharmacies, click here to view.
Limited Role of Patient Input in Specialty Drug Coverage Policies, click here to view.
The Importance of Collaboration in Pursuit of Patient-Centered Value Assessment, click here to view.
Toward Better Data Dashboards for US Drug Value Assessments, click here to view.
Prevalence of Avoidable and Bias-Inflicting Methodological Pitfalls in Real-World Studies of Medication Safety and Effectiveness, click here to view.
Association of Co-Pay Elimination With Medication Adherence and Total Cost, click here to view.
The Utility of Patient Engagement in Drug Research and Development, click here to view.
Patient-Powered Research Networks of the Autoimmune Research Collaborative: Rationale, Capacity, and Future Directions, click here to view.
Enhancing Patient Research Partner Engagement: Research in Psoriatic Arthritis, click here to view.
Patient Voices in Value-Based Cancer Care: Priorities for the Biden Administration, click here to view.
The Dollar or Disease Burden: Caps on Healthcare Spending May Save Money, but at What “Cost” to Patients?, click here to view.
20. AHRQ Effective Program Updates
Research Protocol: Telehealth During COVID-19. Click here to view.
Research Protocol: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder: An Update of the PTSD Repository Evidence Base. Click here to view.
Systematic Review: Transitions of Care From Pediatric to Adult Services For Children With Special Healthcare Needs. Click here to view.
Systematic Review: Interventional Treatments for Acute and Chronic Pain. Click here to view.
Research Protocol: Creating Efficiencies in the Extraction of Data From Randomized Trials: A Prospective Evaluation of a Machine Learning and Text Mining Tool. Click here to view.
Research Protocol: Schedule of Visits and Use of Telemedicine for Routine Antenatal Care. Click here to view.
Systematic Review: Breast Reconstruction After Mastectomy, click here to view.
Surveillance Report: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Systematic Review: Prehospital Airway Management. Click here to view.
Systematic Review: Radiation Therapy for Brain Metastases. Click here to view.
Systematic Review: Safety of Vaccines Used for Routine Immunization in the United States: An Update. Click here to view.
Research Protocol: Evaluation of Mental Health Applications. Click here to view.
Maternal, Fetal, and Child Outcomes of Mental Health Treatments in Women: A Systematic Review of Perinatal Pharmacologic Interventions. Click here to view.