— PIPC Statement on Drug Negotiation Guidance. See details below.
— Over 75 Leading Patient Groups Join PIPC Comment Letter on ICER 2024 Value Assessment Framework. Click here to read the letter.
— PIPC Submits Comments on FDA Guidance on Patient-Focused Drug Development. Click here to read the letter.
— Doing Research Differently: The Unique Value of Engagement in PCORI-Funded Research. Click here to read the blog.
— 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 Statement on Drug Negotiation Guidance. Last week, the Centers for Medicare and Medicaid Services (CMS) issued final guidance related to the Medicare Drug Price Negotiation Program, highlighting that current law already bars the agency from using QALYs and similar measures, thereby clearing up the mischaracterization that the pending H.R. 485, the Protecting Health Care for All Patients Act, would impact Medicare decisions. PIPC urged CMS to further clarify how the agency will judge whether the evidence being considered represents high quality, patient-centered evidence that is representative of the diverse patient population being treated, promotes health equity and excludes biased judgements of health improvement. PIPC also expressed concerns about metrics such as the evLYG that similarly incorporates unreliable calculations related to health improvement and quality of life. Finally, PIPC urged CMS to ensure that their listening sessions are part of a larger dialogue between CMS and stakeholders and recommended CMS appoint an ombudsman dedicated to patient engagement, providing a clear and easy path to ensure these important viewpoints are heard and incorporated throughout the process. Click here to view the PIPC statement. Click here to view the new CMS guidance.
2. Over 75 Leading Patient Groups Join PIPC Comment Letter on ICER 2024 Value Assessment Framework. More than 75 leading organizations representing patients, people with disabilities, and older Americans joined PIPC in submitting comments on the Institute for Clinical and Economic Review’s (ICER) proposed value assessment framework for 2024.
The letter criticizes ICER for continuing to rely on metrics that devalue patients, as well as failing to advance simple actions that would meaningfully advance health equity. Moving forward, ICER should pursue incorporation of caregiver benefits and costs in future modeling but also go further and rely on the societal perspective for its base case models. Click here to read the letter.
3. PIPC Submits Comments on FDA Guidance on Patient-Focused Drug Development. In a letter to the Food and Drug Administration (FDA), PIPC offered comments on the FDA’s guidance on “Patient-Focused Drug Development: Incorporating Clinical Outcome Assessments (COA) Into Endpoints for Regulatory Decision-Making.” The letter compliments the FDA’s efforts to provide stakeholders with a standardized process for collecting and submitting patient experience data and other relevant information from patients and caregivers and to use that information in the regulatory decision-making process.
“We applaud the FDA’s attention to the concepts of patient-centeredness and patient engagement in the conduct of research and the approval process,” wrote PIPC Chair Tony Coelho. “PIPC looks forward to continuing its efforts to bring the voices of patients and people with disabilities to the discussion of how to advance patient-centered principles throughout an evolving health care system.” Click here to read the letter.
4. Doing Research Differently: The Unique Value of Engagement in PCORI-Funded Research. In a recent blog post, PCORI Executive Director Nakela Cook explains the importance of patient engagement in PCORI-funded research. “...While we are excited by the advances within the field of patient engagement, we recognize there is much more work to be done...Methods for engaging and facilitating the inclusion of historically underrepresented populations as study partners is a further critical gap. To help address those needs, we launched the Science of Engagement funding opportunity to help accelerate building an actionable evidence base that clearly identifies the methods and approaches that lead to effective engagement in CER. We are also working on a revised engagement rubric that better reflects the growing body of research in this space.” Click here to read the blog.
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.
In the last legislative session, the legislature introduced, HB 1722, a bill creating a Prescription Drug Affordability Board that would not bar reference to QALYs and would explicitly authorize the Board to contract with a third party that could include ICER. The bill has not been reintroduced in 2023. 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.
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.
- Schizophrenia: Draft Scoping Document available. Comments due on July 26, 2023.
- 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
The Implementation of Value-Based Frameworks, Clinical Care Pathways, and Alternative Payment Models for Cancer Care in the United States. Click here to view.
Federal Data for Conducting Patient-Centered Outcomes Research on Economic Outcomes. Click here to view.
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.
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.