— Patient Engagement & Experience Data: Missing Ingredients For CMS’ Successful IRA Implementation. Click here to read the article.
— Column: Washington Must Heed Patient Recommendations as Policymakers Implement the Inflation Reduction Act. Click here to read the column.
— PIPC Submits Comments on ICER Assessment of Sickle Cell Treatments. Click here to read the comment letter.
— 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. Patient Engagement & Experience Data: Missing Ingredients For CMS’ Successful IRA Implementation. In an article for Health Affairs, Jennifer Bright, Elisabeth Oehrlein, Joe Vandigo, and Eleanor Perfetto explain how patient engagement and experience data can help inform successful implementation efforts of the Inflation Reduction Act (IRA). "As it prepares to implement the new Medicare DPNP and other mechanisms called for by the IRA, CMS must be equally committed to early, pre-assessment work to incorporate patient insight and experience—directly from patients and caregivers rather than filtered through clinicians and academic experts. CMS must also continuously engage the patient community to ensure they have a voice in how the evidence is used and impacts decision making. By learning from other organizations and individuals’ experience with patient-centered approaches, CMS can efficiently co-develop a proactive, formalized approach to engaging patient communities and leveraging patient experience data." Click here to read the article.
2. Column: Washington Must Heed Patient Recommendations as Policymakers Implement the Inflation Reduction Act. In a column for RealClearHealth, Liz Helms writes that policymakers must factor in the patient perspective as they work through implementation of the Inflation Reduction Act (IRA). "CMS and the Biden Administration can prove their commitment to patients and health equity by ensuring that diverse patient perspectives are considered at every point throughout the IRA’s implementation process. To overcome barriers to engagement with federal processes and ensure that those most impacted are given a voice, regulators can host regional roundtables with broad patient representation and continue to request written comments on draft guidance to capture patient perspectives. CMS can also meaningfully engage patient communities in determining the Maximum Fair Price (MFP) of each drug as part of the price setting process." Click here to read the column.
3. PIPC Submits Comments on ICER Assessment of Sickle Cell Treatments. In a letter to the Institute for Clinical and Economic Review (ICER), the Partnership to Improve Patient Care (PIPC) submitted comments on ICER's assessment of gene therapies for Sickle Cell Disease (SCD). PIPC Chair Tony Coelho expressed disappointment that ICER is still incorporating some of the same flaws that were pointed out in PIPC's 2020 comment letter, including ICER's continued use of the discriminatory quality-adjusted-life-years (QALY) metric. The assessment also fails to to acknowledge that standard of care is often not standardized, and does not adequately incorporate pain and other concerns such as fatigue into its model. Click here to read the comment letter.
4. 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.
The Pennsylvania legislature held an informational meeting on Prescription Drug Affordability Boards in the House Health Committee on April 12 at 10am. While new text has not been released, a version of legislation creating a Board was introduced in 2021, HB 1722. In the prior Congress, the legislation did not bar the use of quality-adjusted life years (QALYs) in making determinations of the treatments subject to a cost review and upper payment limit. The bill explicitly allowed for consideration of "the estimated value or cost-effectiveness of the prescription drug product” without any restriction on use of discriminatory measures of cost effectiveness such as those using QALYs. 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.
Nevada has introduced AB 250 which would set prices for prescription drugs in line with the new federally determined maximum fair price (MFP). The MFP price will be newly established through implementation of the IRA, and CMS has not yet released their final methodology to determine the MFP. It will be important that CMS avoid the use of QALYs and similar measures with implications for bias and discrimination and appropriately engage impacted populations in determining the outcomes for measuring a drug's therapeutic benefit. 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. Click here to view testimony from Nevada Chronic Care Collaborative. Click here to view testimony from Epilepsy Foundation Nevada.
The Minnesota state legislature has introduced a bill to create a Prescription Drug Affordability Board, SF168 and HF17. The bill is expected to be signed into law by the governor. Due to robust advocacy by patient and disability groups, it now includes a QALY ban. It does still however reference Canadian prices as a consideration for establishing the upper payment limit for drugs, prices which are explicitly based on QALYs. 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, including proposals to redact the word QALY from the HERC’s deliberations without barring the use of the metric in making decisions. 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: Evidence Report available. Evidence Presentation to be held 4/28/2023
- Metachromatic Leukodystrophy: Revised Scoping Document and Research protocol available. Draft Evidence Report to be released 7/26/2023
Upcoming Events and Webinars
PCORI Board of Governors Meeting
June 5-6, 2023
Click here to view.
PCORI Board of Governors Meeting
September 11, 2023
Click here to view.
2023 PCORI Annual Meeting
October 4-6, 2023
Click here to view.
Medical Journal Articles
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.
A Perspective on Life-Cycle Health Technology Assessment and Real-World Evidence for Precision Oncology in Canada. 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.