— PIPC Submits Comments to FDA on Patient Preference Information in Regulatory Submissions. Click here to read the letter.
— ICER Proposes Changes to Value Assessment Methods & Processes. Click here to read additional details.
— IVI Releases Economic Impacts Research Framework. Click here to view the report.
— PCORI to Award New Clinical Effectiveness Research Funding. Click here for additional details.
— H.R. 485: Protecting Health Care for All Patients Act. See details below.
— Follow PIPC on LinkedIn! Click here to view the page.
— 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.
— Job Openings. See details below.
1. PIPC Submits Comments to FDA on Patient Preference Information in Regulatory Submissions. PIPC recently submitted a comment letter to the Food and Drug Administration (FDA) offering comments on FDA's proposed revision of guidance on the voluntary use of patient preference information (PPI) in regulatory submissions. "There is no substitute for engaging with patients and people with disabilities that have personal experiences with the disease or illness to be treated in the development of a patient preference information study," wrote PIPC Chair Tony Coelho. "It is essential to not only understand the common preferences among patients, but also to understand how preferences may be weighted differently among patient subpopulations." Click here to read the letter.
2. ICER Proposes Changes to Value Assessment Methods & Processes. The Institute for Clinical and Economic Review (ICER) proposed a set of changes to its methods and processes for conducting value assessments, beginning in 2024. Areas with proposed changes include:
- Clinical trial diversity ratings and other methods adaptations related to health equity.
- Cost-effectiveness scenarios related to potential effects of Medicare drug price negotiation.
- New methods to ensure that cost-effectiveness analyses done according to a modified societal perspective have “non-zero” inputs for impacts on productivity for the patient and caregivers, even when direct data are lacking.
Click here to read additional details.
3. IVI Releases Economic Impacts Research Framework. The Innovation and Value Initiative (IVI), in partnership with AcademyHealth, a joint report to help researchers, value assessors, and other decision makers to address the full range of economic impacts on patients. This PCORI-funded resource offers structure, reference points, and guidance how to incorporate such data in comparative effectiveness, outcomes research, and health technology assessment. Click here to view the report.
4. PCORI to Award New Clinical Effectiveness Research Funding. The Patient-Centered Outcomes Research Institute (PCORI) has announced that it will award up to $600 million over three years in support of patient-centered comparative clinical effectiveness research (CER). Beginning in fiscal year 2024, PCORI will develop and issue funding announcements related to improving mental and behavioral health, improving cardiovascular health, preventing maternal morbidity and mortality, promoting health for older adults, addressing substance use, improving outcomes for people with intellectual and developmental disabilities, addressing rare diseases, and promoting sleep health. PCORI also announced it will consider funding four additional themes later this year: promoting child and adolescent health, addressing violence and trauma, managing pain, and addressing COVID-19. Click here for additional details.
5. H.R. 485: Protecting Health Care for All Patients Act. The Protecting Health Care for All Patients Act was passed out of the Energy and Commerce Committee on March 23, 2023. The bill was amended so that it simply extends current law barring use of QALYs and similar measures to other federal programs, incorporating language from the Inflation Reduction Act to consistently define the types of measures allowable for use in coverage, reimbursement and incentive programs. While the bill passed out of committee on a partisan vote, Health Subcommittee Ranking Member Anna Eshoo expressed support for the existing ban on use of QALYs and similar measures in current Medicare law and agreed to work with the Chair on language extending it to other programs, with the goal of bipartisan support on the House floor. The bill advances the recommendation of the National Council on Disability calling for the ban on use of QALYs and similar measures to be consistent across federal programs. Click here to view the bill marked up by the committee. Click here to view a summary. Click here to view a press release from advocates supporting the bill.
6. Follow PIPC on LinkedIn! Visit and follow PIPC's new page on LinkedIn to stay informed on all of our latest comment opportunities, letters, and other key resources. Click here to view the page.
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.
Pennsylvania on May 15 introduced SB 696 which, among other items relating to pharmaceutical price transparency, would establish a Pharmaceutical Transparency Review Board in the state. This Board would be charged with reviewing high price drugs and issuing a report as to how to lower their prices. The legislation does not include a QALY ban and does permit the entity to contract with third-parties to conduct its duties. The Pennsylvania legislature held an informational meeting on Prescription Drug Boards in the House Health Committee on April 12 at 10am. Click here to learn more about Prescription Drug Affordability Boards and the implications of discriminatory measures of cost effectiveness. Click here to learn more about the committee chair, Rep. Dan Frankel.
On June 5, 2023, Governor Lombardo vetoed AB 250. In his veto message, Governor Lombardo stated that “Since AB 250 would ultimately lead to higher costs and less accessibility to certain forms of care, I cannot support it.” The bill would have set prices for prescription drugs in line with the new federally determined maximum fair price (MFP). The MFP price will be newly established through the implementation of the IRA, and CMS has not yet released its final methodology to determine the MFP. Advocates are urging CMS to use a transparent methodology that is centered on outcomes that matter to patients and people with disabilities and that explicitly does not rely on discriminatory QALYs and similar measures. Therefore, the pending legislation raises serious concerns about states directly referencing prices that may rely on biased or discriminatory measures.
The Minnesota state legislature passed SF 2544, legislation establishing a process for setting upper payment limits for prescription drugs that included a provision barring the use of a cost effectiveness analysis using the quality-adjusted life year or similar measure in determinations related to affordability or upper payment limits. It also sets the upper payment limit for some drugs at the Medicare maximum fair price. The bill was signed into law by the governor on May 24, 2023. Patient and disability groups will be monitoring implementation closely.
An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with disabilities, SD. 2117 and HD. 3356 has been introduced in both the House and Senate. This bill includes important patient protections including a ban on the discriminatory Quality-Adjusted Life Year (QALY).
California Attorney General Rob Bonta has solicited information from hospital CEOs across the state about how healthcare facilities and other providers are identifying and addressing racial and ethnic disparities in commercial decision-making tools. In response to this letter, advocates urged Attorney General Bonta to include QALYs and similar metrics in his investigation. Click here to view the Attorney General’s letter. Click here to view a response from advocates.
The Oregon legislature held a hearing on March 27, 2023 on SB 492, legislation that would address the use of biased measures of quality of life, such as quality-adjusted life years or QALYs. PIPC Chairman Tony Coelho submitted testimony stating, "This legislation reflects decades of work to advance disability rights and to end the use of discriminatory measures of the effectiveness of health care that too often drive barriers to care for people with disabilities. In the past this may have been considered just a disability issue, but today we recognize the implications of these biased algorithms for health equity more broadly.” Click here to view testimony from PIPC Chairman Tony Coelho. Click here to view testimony submitted by the Caring Ambassadors Program. Click here to view the list of organizations in Oregon supporting the bill.
On October 6, 2022, the Oregon Health Evidence Review Commission (HERC) discussed a proposed plan for using QALYs. The meeting followed CMS approval of Oregon’s waiver application, which does not bar the use of QALYs in the interim. Click here to read comments submitted by PIPC along with 62 other groups asking CMS to reject the waiver. Click here to view the CMS-approved waiver. At the HERC meeting, the Oregon Health Authority expressed its intent to continue using a prioritized list, shifting it from use under a waiver to use as part of the State Plan Amendment after the waiver expires in 2027. The HERC decided to accept comments on their use of QALYs but has not made a decision as to whether they will adjust their methodology. Click here to view the letter from almost 50 advocacy groups asking the HERC to stop using QALYs. Click here to view testimony from PIPC Chairman Coelho to the Oregon HERC expressing his support for the Americans with Disabilities Act. Click here to view the options that continue to be under consideration by the HERC to continue using QALYs. Click here to view all of the comments shared with PIPC.
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: MS patients in Australia face limited choices with respect to effective treatments covered, and some are forced to raise money for treatment overseas.
- U.K: The family of a patient with MS has had to turn to crowdfunding for treatment that is not funded through 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.
- Pulmonary Arterial Hypertension: Draft Scoping Document available. Open for comments until June 5, 2023.
- Sickle Cell Disease: Draft Evidence Report Available. Public Comments open until 5/9/2023.
- Non-Alcoholic Steatohepatitis: Final Evidence Report now available.
- Metachromatic Leukodystrophy: Revised Scoping Document and Research protocol available. Draft Evidence Report to be released 7/26/2023
Upcoming Events and Webinars
A Deep Dive into the PCORnet Network, Common Data Model, and Engagement Resources
July 13, 2023
Click here to view.
PCORI Board of Governors Meeting
September 11, 2023
Click here to view.
2023 PCORI Annual Meeting
October 4-5, 2023
Click here to view.
Medical Journal Articles
Patient-Reported Outcomes for People with Diabetes: What and How to Measure? A Narrative Review. Click here to view.
Patient Engagement in Research; Benefits, Challenges, Importance, and Implications. Click here to view.
The Opportunity for Greater Patient and Public Involvement and Engagement in Drug Development and Regulation. Click here to view.
Data Governance for Real-World Data Management: A Proposal for a Checklist to Support Decision Making. Click here to view.
A Patient-Centered Comparative Effectiveness Research Study of Culturally Appropriate Options for Diabetes Self-Management. Click here to view.
Payer–patient Engagement Framework to Strengthen Ethical Formulary Decision-making in Rare Disease Arena in the USA. Click here to view.
The Impact on Cost-Effectiveness of Accounting for Generic Drug Pricing: Four Case Studies. Click here to view.
AHRQ Effective Program Updates
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.
- Associate Director, Public Engagement, Patient-Centered Outcomes Research Institute. Details.
- Engagement Officer, Public and Patient Engagement, Patient-Centered Outcomes Research Institute. Details.
- Program Officer, Science of Engagement, Patient-Centered Outcomes Research Institute. Details.