— PIPC Submits Letter With 100 Signatures to HHS Supporting Health Care Nondiscrimination. See details below.
— Make Sure People with Disabilities Are Included in Clinical Trials: Your Participation Is Needed! See details below.
— QALY Alternatives Abound, but None Fully Addresses Discrimination Concerns. Click here to read the blog.
— AHRQ Announces Interest in Health Services Research to Improve Healthcare for Persons Living with Disabilities. Click here to read the announcement.
— PCORI Seeks Nominations for Advisory Panels. Click here to learn more, to apply to serve on an advisory panel, or submit a nomination.
— Emerging Threats in States for Use of Discriminatory Metrics. See details below.
— What Happens in Countries Using QALYs and Cost-Based Thresholds? See details below
— ICER's QALY-Based Study Topics. See details below.
— Upcoming Events and Webinars. See details below.
— AHRQ Effective Program Updates. See details below.
1. PIPC Submits Letter With 100 Signatures to HHS Supporting Health Care Nondiscrimination. On November 13, 2023, PIPC submitted a letter to CMS with 100 signatures supporting the agency’s efforts to advancing a rulemaking that would update regulations implementing Section 504 of the Rehabilitation Act. The signatories recommended that HHS should advance a final rule that uses language consistent with Section 1182(e) of the Affordable Care Act to restrict the use of discriminatory value assessments. Doing so would be consistent with current developments and laws and discourage confusion; allow for consideration of how value assessments may discriminate by classifying people with disabilities as inferior whether in measures of life extension or in quality-of-life improvement; be consistent with NIH efforts to address ableist assumptions about quality of life that may also drive value assessments; and spur meaningful innovation in the development and use of measures of quality of life and improvement that do not discriminate based on the assumed “worth” of patients with disabilities to treat.
The signing organizations also recommended that HHS explain that the final rule related to Medical Treatment applies to payer policies advanced by recipients of federal financial assistance, recognizing how recipient payers cannot categorially exclude or limit access to care that is not futile for individuals with disabilities and clarifying that it is not a legitimate, nondiscriminatory reason to selectively deny or limit care to a person with a disability based on the determination the person’s quality of life is not worth the cost of treatment. The letter also recommended HHS collect, analyze, and publicly report disability data.
Also posted on the PIPC website are similar comments from the Alliance for Aging Research signed by 44 groups, ALS Association, the Consortium for Constituents with Disabilities signed by 68 groups, and nHelp sharing concerns expressed by PIPC related to quality of life measures and health utilities used in cost effectiveness analyses.
2. Make Sure People with Disabilities Are Included in Clinical Trials: Your Participation Is Needed! U.S. Food and Drug Administration (FDA) is holding a two-day virtual public workshop to promote diversity in clinical trials on November 29 and 30. Notable sessions include: (1) November 29: Approaches to Support the Inclusion and Clinical Study Participation of Individuals with Disabilities Including Intellectual or Developmental Disabilities and (2) Approaches to Support the Inclusion and Clinical Study Participation of Individuals with Mental Illness. Click to view agenda for the two-day workshop to register.
3. QALY Alternatives Abound, but None Fully Addresses Discrimination Concerns. A blog by Kimberly Westrich overviews a recent journal article on alternatives to the QALY and the risk of health care discrimination. The authors critique 4 of the approaches (evLY, HYT, GRACE, and severity weighting) are aligned with a cost-effectiveness framework for informing Medicare price negotiation under the Inflation Reduction Act, stating that 'none fully addresses all aspects of the stated concerns that QALYs may be used to unintentionally implement discrimination.' The authors recommend a hybrid approach that uses a quantitative assessment of value elements to inform a structured deliberation process, positing that such an approach could reduce cognitive burden, mitigate deliberative biases, improve accountability and transparency, and ultimately lead to higher-quality decisions.
4. AHRQ Announces Interest in Health Services Research to Improve Healthcare for Persons Living with Disabilities. The Agency for Healthcare Research and Quality (AHRQ) announced a new grant opportunity that seeks to advance the Nation’s goal of improving health outcomes for persons living with disabilities through enhanced or innovative care. Proposed study designs and methods should demonstrate innovation, a high potential impact, and meaningful collaboration and inclusion of diverse partners and communities. This includes participation of persons with disabilities in all stages of proposed research and use of methods that ensure equal partnership with the scientific and program staff.
5. PCORI Seeks Nominations for Advisory Panels. PCORI is currently seeking nominations for appointments to its advisory panels. These multi-stakeholder advisory panels must include patients, caregivers, or patient advocates. PCORI advisory panels do not serve in an official decision-making capacity, but their recommendations and advice will be taken into consideration by PCORI. PIPC encourages patients, caregivers, and patient advocates to apply or to submit nominations to serve on PCORI's five advisory panels on: Clinical Effectiveness and Decision Science, Clinical Trials, Healthcare Delivery & Disparities Research, Patient Engagement, and Rare Disease. The application is open through March 29, 2024. Click here to learn more, to apply to serve on an advisory panel, or submit a nomination.
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.
The Oregon legislature passed SB 844 in 2021 creating a Prescription Drug Affordability Board, which was updated based on the Board’s recommendations in 2023 by SB 192. While the legislation included language barring consideration of QALYs and similar formulas, the Board has engaged the Program on Regulation, Therapeutics, and Law (PORTAL), per their meeting agenda for November 15. PORTAL has a subcontract with the Institute for Clinical and Economic Review (ICER) for its work with the Massachusetts Health Policy Commission and has presented on the merits of the QALY and evLYG measures to the Colorado Prescription Drug Affordability Board. This raises concerns about the evidentiary basis for the Board’s decisions and the potential for reference to discriminatory measures of cost effectiveness.
The Oregon Board voted to advance an affordability review of 26 drugs at the October 18, 2023 meeting, with an ambitious plan to conduct the reviews by February. The Board’s rules for conducting an affordability review does not include a robust process for engaging patients and people with disabilities in their decisions. The Oregon PDAB will hold a public hearing on December 13, 2023 from 1:30-4pm Pacific time. Guidance for providing public testimony is available here. Separately, the Oregon Drug Price Transparency Program will hold their annual meeting on December 7, 2023, with registration available here.
Click here to join Disability Rights Oregon in advocating for legislation banning use of QALYs. Click here to join the Pacific Northwest Advocates Confab in their engagement efforts.
Colorado passed legislation in 2021 creating a Prescription Drug Affordability Review Board. The legislation included language stating that the Upper Payment Limit for selected drugs "shall not consider research or methods that employ a dollars-per-quality adjusted life year, or similar measure, that discounts the value of a life because of an individual's disability or age.” However, this limitation did not clearly extend to the process for determining the selected drugs or the affordability review. Presentations to the Colorado Board from entities such as the Program on Regulation, Therapeutics, and Law (PORTAL) related to cost effectiveness analyses have referenced the merits of using of a cost-per-QALY or the equal value of life-year gained (evLYG) in estimating cost effectiveness of treatments, indicating that these discriminatory measures may have influenced how Colorado selected the drugs and may influence the affordability review. Concerns about the potential for discrimination were described in a letter to the Board from 16 patient and disability organizations. The PDAB offers several opportunities for patients to engage and ensure their voice is heard on these topics. The next PDAB Meeting will be held Friday, December 8 at 10 am MT. Click here to register. Click here to learn more from a Colorado patient with Cystic Fibrosis.
The Massachusetts Senate’s Ways and Means Committees was referred the “PACT Act”, reported favorably on October 30, 2023 by the Joint Committee on Health Care Financing. The legislation includes a provision calling for the health policy commission not to base its determinations on measures such as QALYs. The Senate rejected an amendment (#19) pushed by Massachusetts advocates calling for transparency related to the use of cost effectiveness analyses by the commission, as well as increased requirements for stakeholder engagement. The Senate passed the bill on November 15, 2023. Massachusetts advocates will work with the House toward language that more clearly provides for public transparency of the evidence under consideration and opportunities for engagement from patients and people with disabilities.
International News: What Happens in Countries Using QALYs and Cost-Based Thresholds?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- Australia: Families are calling for greater access to a medication that helps manage a severe form of epilepsy because they have been left desperate and disappointed after being told it is not being covered by the PBS.
- Canada: Cystic fibrosis patients are struggling with arbitrary barriers to access for life-saving treatment.
- New Zealand: A treatment for myeloma is the default treatment for patients in at least 49 countries, but not in New Zealand as patients search for alternatives.
- United Kingdom: Kids with cystic fibrosis are struggling to gain access to a treatment that regulators say is too expensive to be covered by the NHS.
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.
- Chronic Obstructive Pulmonary Disease: Revised Scoping Document available. Public meeting: June 2024
- Post-Traumatic Stress Disorder: Revised Scoping Document available. Main review: May 30, 2024.
- Paroxysmal Nocturnal Hemoglobinuria: Evidence Report available.
- Schizophrenia: Draft Evidence Report available.
- Pulmonary Arterial Hypertension: Main review: December 1, 2023.
Upcoming Events and Webinars
PCORI Board of Governors Meeting
December 12, 2023
Click here to view.
Medical Journal Articles
Alternative approaches to measuring value: an update on innovative methods in the context of the United States Medicare drug price negotiation program. Click here to read the article.
Economic Outcomes in Patient-Centered Outcomes Research: A Paradigm Shift. Click here to read the article.
Visualization of Evidence for Shared Decision Making. Click here to read the article.
Avenues for Strengthening PCORnet’s Capacity to Advance Patient-Centered Economic Outcomes in Patient-Centered Outcomes Research. Click here to read the article.
Linking Medicare-Medicaid Claims for Patient-Centered Outcomes Research Among Dual-Eligible Beneficiaries. Click here to read the article.
Gene Therapies for Sickle Cell Disease: Effectiveness and Value. Click here to read the article.
White Paper: Improving Patient Subgroup Representation with Real-World Data. Click here to read the article.
A Clinical Pathway to Well-Being: Putting Patient Priorities at the Center of Care. Click here to read the article.
Comparative Effectiveness of Treatments for Rheumatoid Arthritis in Clinical Practice: A Systematic Review. Click here to read the article.
QALYs: The Math Doesn’t Work. Click here to read the article.
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Perspectives on Patient-Reported Outcome Data After Treatment Discontinuation in Cancer Clinical Trials. Click here to view.
AHRQ Effective Program Updates
Technical Brief: Measuring Healthcare Organization Characteristics in Cancer Care Delivery Research. Click here to view.
Systematic Review: Postpartum Care up to 1 Year After Pregnancy: A Systematic Review and Meta-analysis. Click here to view.
Draft Report: Impact of Healthcare Algorithms on Racial and Ethnic Disparities in Health and Healthcare. Click here to view.
Systematic Review: Partian Breast Irradiation for Breast Cancer. Click here to view.
Systematic Review: Use of Telehealth During the COVID-19 Era. Click here to view.
Research Report: Analysis of Requirements for Coverage with Evidence Development (CED) - Topic Refinement. Click here to view.
Technical Brief: Infection Prevention and Control for the Emergency Medical Services and 911 Workforce. Click here to view.
Systematic Review: Management of Infantile Epilepsies. Click here to view.