— PIPC Submits Comments on ICER Assessment of Sickle Cell Treatments. Click here to read the comment letter.
— Biden Administration Pushed to Address Disability Discrimination in Health Care. Click here to read the article.
— Commentary: Hope is Its Own Form of Healing. See details below.
— PIPC Submits Comments on Maryland Prescription Drug Affordability Board Draft Regulations. Click here to read the 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. 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.
2. Biden Administration Pushed to Address Disability Discrimination in Health Care. An article in Disability Scoop covers recent efforts by advocates to update regulations that prevent medical providers from discriminating against people with disabilities. “Section 504 regulations have not been updated for decades and do not adequately address issues facing people with disabilities today,” wrote 30 advocacy groups in a recent letter to Susan E. Rice, director of the White House Domestic Policy Council, and Melanie Fontes Rainer, director of the Office for Civil Rights at HHS. “We are concerned that if the notice of proposed rulemaking is not issued imminently, that there will not be sufficient time for meaningful comment and response prior to the end of the administration’s first term.” Click here to read the article.
3. Commentary: Hope is Its Own Form of Healing. In a column for WBUR, Jennifer Handt offers her perspective on what the accelerated approval of a new gene therapy for Duchenne muscular dystrophy (DMD) would mean for her and her family, emphasizing the need for patients and providers to have a voice in the process. "Accelerated approval has enormous potential to write a new and promising chapter in the history of this relentless disease. If we enable a therapy to reach these boys’ muscles with a functional level of dystrophin, the protein their bodies lack, it could transform everyday life for them, their caregivers and their doctors — starting now. That’s why their stories are essential to the conversation on May 12 and beyond." Click here to read the column. Click here to view information about the advisory committee’s decision to recommend approval.
4. PIPC Submits Comments on Maryland Prescription Drug Affordability Board Draft Regulations. In a comment letter to the Maryland Prescription Drug Affordability Board, PIPC highlighted concerns of patients and people with disabilities related to the Board's potential use of discriminatory cost-effectiveness analyses. "We are concerned that the draft regulations ignore the letter referenced above, instead specifically calling for information on cost effectiveness 'derived from health economics and outcomes research' which is known to rely on biased and discriminatory measures such as QALYs," wrote PIPC Chairman Tony Coelho. "By devaluing people with disabilities, whether in terms of their life extension or quality of life, cost effectiveness analyses relying on QALYs and similar measures have no place in our health care system." Click here to read the letter.
5. 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. It does not include a bar on using QALYs or other metrics that discriminate as a consideration for selecting the treatments to be considered, nor does the bill bar the use of QALYs as a consideration in establishing an upper payment limit, instead explicitly referencing prices established by third parties that may consider QALYs. The bill specifically references Canadian prices as a consideration for establishing the upper payment limit for drugs, prices which are explicitly based on QALYs. Click here to view the testimony from the ALS Association.
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.
- 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
Connecting Real-World Data and Domain Expertise to Enhance Trial Design and Planning
May 18, 2023
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PCORnet Studies Applicant Workshop
May 18, 2023
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PCORI Board of Governors Meeting
June 5-6, 2023
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PCORI Board of Governors Meeting
September 11, 2023
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2023 PCORI Annual Meeting
October 4-6, 2023
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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 Update
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.
Systematic Review: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder. Click here to view.
Systematic Review: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. 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.