1. PIPC to MedPAC: Avoid Use of QALY in Medicare Coverage Policy. Click here to read the letter.
2. PCORI Funds Patients' and Pharmacists' Work to Address Social Determinants of Health. Click here to read the article.
3. IVI Announces Health Equity Initiative. Click here to view the press release.
4. Lawmakers Introduce Legislation Barring the Use of QALYs in Federal Programs. Click here to view the press release.
5. PIPC Joins Over 60 Organizations Urging CMS to Reject QALY's in OR Waiver Renewal Application. Click here to read the letter.
6. Upcoming PCORI Funding Announcement to Fund Studies on the Science of Engagement - Postponed until July 12, 2022. Click here for more details.
7. Speaking Up on ADA with Tony Coelho. Click here to view the one pager.
8. NCD Health Equity Framework Includes Recommendation Against Use of QALYs, Following BBB Recommendation. Click here to view the framework.
9. Value Our Health: Advancing Value Assessment Principles Centered on Patients and People with Disabilities. See below for details.
10. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
11. Emerging Threats in States for Use of Discriminatory Metrics, see details below.
12. ICER's QALY-Based Study Topics. Click here to provide patient input.
13. Upcoming Events and Webinars, see details below.
14. Medical Journal Articles, see details below.
15. AHRQ Effective Program Updates, see details below.
In a letter to the Medicare Payment Advisory Commission (MedPAC), the Partnership to Improve Patient Care (PIPC) urged the commission to avoid reliance on discriminatory value assessment metrics as the basis for Medicare coverage policy. PIPC’s concerns stem from comments by MedPAC commissioners during the April 2022 public meeting, where metrics of clinical and cost effectiveness were discussed, particularly the quality-adjusted-life-year (QALY).
"Past policy recommendations relying on studies from entities such as the Institute for Clinical and Economic Review (ICER) have consistently been met with opposition from patients and people with disabilities due to the flawed data and metrics on which they rely to make conclusions,” wrote PIPC Chair Tony Coelho. “We urge the commission not to use discriminatory metrics as part of any proposed policy related to coverage and reimbursement.”
2. PCORI Funds Patients' and Pharmacists' Work to Address Social Determinants of Health
Supported by funding through a Patient-Centered Outcomes Research Institute (PCORI) Eugene Washington PCORI Engagement Award (EASCS-23304), the Pharmacy Quality Alliance (PQA and the Patient Advocate Foundation (PFA) are working to improve screening and interventions for social determinants of health in community pharmacies. PQA and PAF are convening diverse patients and pharmacy practitioners and researchers to work on this project. The project has a focus on patient-centered outcomes (social need, non-clinical outcomes). It will ensure patient perspectives and needs are incorporated into research on SDOH screenings and interventions as they become more widespread in pharmacies. Click here to read more about the project.
3. IVI Announces Health Equity Initiative
The Innovation and Value Initiative (IVI), a non-profit research organization committed to advancing the science, practice, and use of value assessment in health care, on April 27, 2022, announced the launch of its Health Equity Initiative, a 2-year initiative that aims to define gaps in value assessment methodology and develop best practices that support health equity. Click here to view the press release.
4. Lawmakers Introduce Legislation Barring the Use of QALYs in Federal Programs
House Energy and Commerce Republican Leader and Republican Study Committee Member Cathy McMorris Rodgers, RSC Chairman Jim Banks, and RSC Member and GOP Doctors Caucus Co-Chair Brad Wenstrup, D.P.M, introduced The Protecting Health Care for All Patients Act, important legislation that would bar the use of the quality-adjusted life year (QALY) metric in federal programs, consistent with the recommendations of the National Council on Disability. The QALY metric was barred from use in Medicare decisions as part of the Affordable Care Act, but the statute did not extend too other federal health programs. Democrats have also expressed opposition to use of the QALY as part of the DNC Platform. Click here to view the press release. Click here to view the DNC Platform. Click here to view a news article about the new legislation. Click here to view the bill.
5. PIPC Joins Over 60 Organizations Urging CMS to Reject QALY's in OR Waiver Renewal Application
The Partnership to Improve Patient Care (PIPC), along with 62 leading advocacy groups representing patients and people with disabilities, submitted a comment letter to the Centers for Medicare and Medicaid Services (CMS) on Oregon's 1115(a) Demonstration Waiver renewal application. The letter expresses concern that Oregon's application would further entrench health inequalities and deny access to care for those with disabilities and chronic conditions through the use of the quality-adjusted-life-year (QALY) metric. The state's defense of its use of the discriminatory QALY, as well as its efforts to exclude FDA-approved drugs from coverage, are inconsistent with Oregon's stated goals of health equity. "We urge CMS to reject Oregon’s waiver renewal application, in accordance with its decision in 1992," the letter states. "A consistent policy across Medicare and Medicaid barring the use of QALYs will serve to improve health equity, access to medically necessary care, and compliance with disability rights laws." Click here to view the letter.
6. Upcoming PCORI Funding Announcement to Fund Studies on the Science of Engagement - Postponed until July 12, 2022
The Patient-Centered Outcomes Research Institute (PCORI) intends to release a PCORI Funding Announcement (PFA) on July 12, 2022, seeking to fund studies that build an evidence base on engagement in research. Initial funding will primarily focus on measure development and/or validation, particularly rapid measure development, leveraging opportunities to support teams who may have already begun measure development work. Applicants can also validate or adapt existing measures initially developed for this or different contexts (e.g., community-based participatory research, engagement in health care delivery). A smaller number of early awards will test engagement techniques using a combination of limited existing validated measures of engagement and measures of other research outcomes (e.g., participant recruitment and retention; diversity, equity and inclusion outcomes).
PCORI anticipates multiple funding cycles for this initiative, with the focus of subsequent PFAs to be updated as the evidence base grows. PCORI invites interested participants to review the full preannouncement on the website for details and register for a town hall on July 19, 2022, at 12:00 pm ET where researchers and stakeholders will learn more about this upcoming PFA. Please feel free to share this information with anyone who may be interested.
7. Speaking Up on ADA with Tony Coelho.
The Disability Garrison podcast, hosted by Holly Carmichael and Michael Murray, seeks to inform listeners about issues that people with disabilities face every day. A series of podcasts were recorded with PIPC Chairman Tony Coelho. Tony spoke at length about his upbringing, disability diagnosis, darkest times, and finding his voice, and how it all ultimately led to his involvement in the Americans with Disabilities Act. Episodes include: Discovering Disability Identity with Tony Coelho (Part 1), Speaking up with Tony Coelho (Part 2), Shaping the ADA with Tony Coelho (Part 3), The Americans with Disabilities Act with Tony Coelho (Part 4). Click here to view entries from the "Because of ADA I Can" Campaign. Click here to view the one pager.
8. NCD Health Equity Framework Includes Recommendation Against Use of QALYs, Following BBB Recommendation
The National Council on Disability (NCD) released a Health Equity Framework in February, 2022. NCD’s framework included a recommendation to mandate a blanket prohibition on the use of Quality Adjusted Life Years by any federal agency, or recipients of federal financial assistance from the Department of Health and Human Services, both directly and through third-party assessments. This recommendations follows the NCD’s recommendation for an unambiguous ban on the quality adjusted life year (QALY) within the text of the Build Back Better Act (H.R. 5367), responding to their increasing reference in coverage, reimbursement and incentive programs. Click here to view the framework. Click here to view the NCD’s letter to Congress and here to view NCD’s presentation at the 2021 PIPC forum.
The NCD’s recommendations are supported by the law and public opinion. Morning Consult ran a poll on behalf of Partnership to Improve Patient Care focusing on the use of cost-effectiveness assessments to determine the value of coverage and treatment costs. The survey found that Americans want patients and their doctors in charge of health care decision making and are opposed to the use of cost assessments such as Quality-Adjusted Life Year (QALY). PIPC Chairman Tony Coelho published an opinion piece in Morning Consult about the poll concluding that it is time to finally end the ambiguity and prohibit QALYs altogether. DREDF published a paper explaining 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. Click here to view the DREDF report. Click here to view the one-pager. Click here to find summaries in both English and Spanish.
9. Value Our Health: Advancing Value Assessment Principles Centered on Patients and People with Disabilities
Advocates are facing challenges and opportunities at the state and federal level to convince policymakers to advance policies that value outcomes that matter to patients and people with disabilities. Organizations supporting Value Our Health have developed a set of principles supporting value assessment methodologies centered on patients and people with disabilities, opposing summary measures such as the cost-per-quality-adjusted life year or QALY in favor of value frameworks that meaningfully acknowledge diversity and differences among patients and people with disabilities, that are developed using transparent processes and methods and meaningfully engage patients and people with disabilities, therefore capturing costs and benefits that matter to the patient. As states consider legislation that ranges from seeking to bar the use of QALYs to authorizing increased use of QALYs, opportunities exist for advocacy consistent with these principles and the recommendations of entities such as the National Council on Disability, an independent federal agency recommending QALYs be barred across federal programs. Click here to view the Value Our Health principles. Click here to view template legislation for states to consider that would improve the evidence used in Medicaid decisions, require stakeholder engagement and bar state use of QALYs and similar discriminatory metrics. Click here to view the Open Letter from over 90 organizations to state and federal policymakers. Click here for more information about Value Our Health.
10. 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.
- New Zealand: CF patients are holding their breath for Trikafta, the lifesaving drug, while Pharmac continues to refuse to provide access. MS patients are outraged that they are unable to access lifesaving treatment because Pharmac does not fund the medication.
- United Kingdom: MS patient is contacting (link) medical professionals in Mexico to obtain access to the chemo and stem cell therapy he needs, because it is unavailable for him on the NHS as he doesn't meet the required criteria.
11. 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. We encourage you to keep track of all state-based threats using this new website tracking state activities. Key issues are highlighted below.
- 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.
Oregon's waiver application, submitted to CMS on February 18, 2022 defended the state’s use of QALYs and continues to request authority to conduct reviews of drugs approved through FDA’s accelerated pathway to allow for restricted coverage. Disability and patient stakeholders have submitted comments to CMS asking that it reject Oregon’s use of the discriminatory QALY. Click here to read comments submitted by PIPC along with 62 other groups. Click here to view letters from Disability Rights Oregon and Paul Terdal. Click here to view the letter from Patients Rights Action Fund.
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. Governor Charlie Baker filed his health care bill, SB 2774, An Act Investing in the Future of our Health, on March 17, 2022. The bill expands the Health Policy Commission’s authority to determine the value of a drug beyond Medicaid to private payers. As written, the bill allows for use of cost-effectiveness assessments and analysis by a third-party, and it does not include a QALY ban. The bill requires disclosure of methods used by third parties and disclosures of potential limitations of that research. In a hearing on SB 2774 on April 11, patient advocates testified that the bill as written has the potential to discriminate and must include a QALY ban.
12. ICER's QALY-Based Study Topics
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.
- Alzheimer's Disease: ICER has decided to extend its timeline for its assessment of the beta-amyloid antibodies for early Alzheimer’s disease, so the timeline is to be determined. Research Protocol available. Draft Scoping Document available.
- Amyotrophic Lateral Sclerosis (ALS): Model Analysis Plan available. Research Protocol available. Revised Scoping Document available. 6/14/2022: Draft Evidence Report.
- Beta Thalassemia: 6/2/2022: Evidence Report. 6/17/2022: Evidence Presentation. Draft Evidence Report available. Public comments closed. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available.
- COVID-19: Final Evidence Report and Meeting Summary available. Evidence Presentation and Report available. Draft Evidence Report Available. Public comments closed. Model Analysis Plan available. Draft Scoping Document and Research Protocol available.
- Obesity Management: Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. Public comments closed. 5/25/2022: Model Analysis Plan. 7/13/2022: Draft Evidence Report.
- Multiple Sclerosis: CIS, RRMS, and SPMS: 5/27/2022: Revised Scoping Document. 6/6/2022: Research Protocol. Draft Scoping Document available. Public comments closed.
- Gene Therapies for Hemophilia A and B: Draft Scoping Document available. Public comments open until 5/25/2022. 6/6/22: Revised Scoping Document.
- Unsupported Price Increases Occurring in 2020 in California: Protocol available. 10/13/2022: Final Report.
- Type 2 Diabetes: Final Evidence Report and Meeting Summary available. Evidence Presentation available. Evidence Report available. Draft Scoping Document and Research Protocol available.
- Hypertrophic Cardiomyopathy: Final Evidence Report and Meeting Summary available. Evidence Report Available. Evidence Presentation available.
- Unsupported Price Increase: Final Report available.
- Asthma: Final Evidence Report and Meeting Summary available. Evidence Presentation available. Evidence Report available.
- Fair Access: Coverage Policies in 2020: Protocol Available. Final Report available.
13. Upcoming Events and Webinars
Advisory Panel on Patient Engagement Spring 2022 Meeting
May 16, 2022
Click here for details.
Advisory Panel on Rare Disease Spring 2022 Meeting
May 18, 2022
Click here for details.
Advisory Panel on Clinical Effectiveness and Decision Science Spring 2022 Meeting
May 20, 2022
Click here for details.
Health Affairs Policy Spotlight: One-on-One with PCORI Executive Director Nakela L. Cook, MD, MPH
May 23, 2022
Click here for details.
14. 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.
15. AHRQ Effective Program Updates
Systematic Review: Treatments for Acute Pain. Click here to view.
Systematic Review: Models of Care That Include Primary Care for Adult Survivors of Childhood Cancer: A Realist Review. Click here to view.
Systematic Review: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Systematic Review: Prehabilitation and Rehabilitation for Major Joint Replacement. Click here to view.
Integrated and Comprehensive Pain Management Programs: Effectiveness and Harms. Click here to view.
Systematic Review: Management of High-Need, High-Cost Patients: A “Best Fit” Framework Synthesis, Realist Review, and Systematic Review. Click here to view.
Research Protocol: Partial Breast Irradiation for Breast Cancer. Click here to view.
Systematic Review: Physical Activity and the Health of Wheelchair Users: A Systematic Review in Multiple Sclerosis, Cerebral Palsy, and Spinal Cord Injury. Click here to view.
Research Report: Developing and Piloting a Tool to Create Dot Plots to Summarize Pooled Data for Multiple Outcomes in Systematic Reviews. Click here to view.
Systematic Review: Malnutrition in Hospitalized Adults. Click here to view.
Research Protocol: Nutrition as Prevention for Improved Cancer Outcomes. Click here to view.
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.