— CMS References ICER and NICE in Explanations for Medicare Drug Price Negotiation Program. See details below.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— New PCORI/AHRQ Research Shows Better Ways To Help Young People With OCD. Click here to read additional details on the review.
— New PCORI Funding Announcements Are Now Open! Click here for additional details.
— Center for Innovation & Value Research Seeking Stakeholder Interviews on Digital Health Therapies. See details below.
— 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. CMS References ICER and NICE in Explanations for Medicare Drug Price Negotiation Program. PIPC has long advocated against the reference of ICER’s value assessments that rely on the quality-adjusted life year (QALY) and the equal value of life year gained (evLYG), measures that are contrary to the laws governing Medicare and barring disability discrimination. In the explanations released on December 20, 2024, CMS explicitly listed studies from the Institute for Clinical and Economic Review (ICER) in its explanations for the calculation of Maximum Fair Prices (MFPs) for the drugs Eliquis, Enbrel, Farxiga, and Xarelto. The explanation for Xarelto report also cites data from the National Institute for Health and Care Excellence (NICE) in the United Kingdom which is know for its explicit reliance on QALYs. In its public comments, ICER defends the use of the evLYG, calling it a nondiscriminatory alternative and highlighting its use by the Veterans Administration, Medicaid programs and private insurers. CMS did not acknowledge that the evLYG is a similar measure to the QALY that is barred under Section 1182 of the Affordable Care Act and under §84.56 of the rules governing Section 504 of the Rehabilitation Act barring disability discrimination. Click here to view the explanations from CMS.
2. Learn More About Alternative Measures for Value Assessment. PIPC has developed new resources about alternative methods and metrics for value assessment. It is now widely recognized that traditional methods and metrics of value assessment – including the quality-adjusted life year (QALY) – have significant shortcomings. This has led to well-intentioned development of other measures and approaches that developers assert to be nondiscriminatory and more patient-centered. However, each approach comes with tradeoffs, need for improvement, and inherent methodological flaws. No patient is average, and no measure of value should assume so. Click here to learn more.
3. New PCORI Funding Announcements Are Now Open! PCORI's Online System is now open for the submission of Letters of Intent (LOI) for several new research PCORI Funding Announcements (PFAs) under Cycle 1 2025. Of note, PCORI is soliciting proposals for: (1) managing pain: (2) improving mental and behavioral health; (3) improving methods for conducting patient-centered comparative effectiveness research; and (4) advancing the science of engagement in research, among others. LOIs will be due Jan. 14, 2025. Click here for additional details.
4. New PCORI/AHRQ Research Shows Better Ways To Help Young People With OCD. Findings from a Patient-Centered Outcomes Research Institute (PCORI)-funded systematic review conducted by the Agency for Health Care Research and Quality (AHRQ) have provided important insights into diagnosing and treating obsessive compulsive disorder (OCD) in children and teens. "With OCD affecting one to two percent of young people in the United States, these findings are key to diagnosing and treatment options. Timely diagnosis and effective treatment can help to alleviate symptoms in childhood and promote healthy development through adulthood." Click here to read additional details on the review.
5. Center for Innovation & Value Research Seeking Stakeholder Interviews on Digital Health Therapies. The Center for Innovation & Value Research is conducting key informant interviews to learn from people who have experience with digital health solutions to manage anxiety or depression. All conversations are confidential, and your insights will directly impact the research study. If selected, you will participate in a 60-minute virtual interview and receive a $150 honorarium. If you have used a digital health app for the treatment of anxiety or depression, please complete this form to apply to participate.
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.
Colorado
The Colorado PDAB released its 2025 meeting and staff office hour schedule, which can be accessed here. The PDAB’s next meeting will take place on January 17, 2025 and patients may register to attend here.
PIPC and more than 40 leading patient organizations signed onto a letter led by The Ensuring Access through Collaborative Health (EACH) Coalition outlining recommendations for the Colorado PDAB to better utilize and engage patients in PDAB's Policy and Rule opportunities.
During their board meeting on Dec. 6, the Colorado PDAB confirmed that they will be proceeding with a UPL rulemaking for Enbrel starting in January. The rulemaking process will consist of 3 hearings, likely held in January, February and March.
On May 23, PIPC submitted a comment letter to the Colorado Prescription Drug Affordability Board (PDAB) on the Board's ongoing process for affordability reviews and establishment of upper payment limits. PIPC's comments come on the heels of additional correspondence to the PDAB urging the Board to reject the use of discriminatory metrics such as quality-adjusted-life-years (QALY) and others that create barriers to access for patients and people with disabilities. The letter notes that, pursuant to the recently-finalized rules governing Section 504 of the Rehabilitation Act, entities that receive federal financial assistance, including state Medicaid programs, are subject to requirements that bar the use of these discriminatory value assessments. The letter also responds to the Board’s discussion about foreign government policies, highlighting the concerns of patients and people with disabilities to such policies as well as the National Council on Disability recommendations against referencing other countries. Click here to read the letter. Click here to view the Board’s conversation about foreign methods and potential to import QALYs (begins at 2:41).
Stelara, Cosentyx and Enbrel were deemed unaffordable and the Board is considering UPLs. At the advisory council meeting, there was a robust discussion of developing a tool to understand the impact of UPLs, a process welcomed by the patient community although with many unanswered questions about what entity will conduct such a tool and its impact on the timeline for regulatory meetings and consideration of UPLs. Several advocates weighed in on October 15, 2024 to clarify again the legal implications under disability rights laws related to the use of QALYs and similar measures.
Maryland
The Maryland PDAB released its 2025 meeting schedule, which can be found here. The PDAB’s next meeting will take place January 27, 2025.
At the most recent November 25, 2024 PDAB meeting, cost effectiveness analysis and international reference pricing continued to be listed among the methodologies that could be used by the Board to establish a UPL, despite concerns raised by patients and people with disabilities. Comments to the PDAB may be sent to [email protected].
The Maryland Legislative Policy Committee approved the PDAB’s UPL Plan, despite comments from PIPC and others opposing its approval. As background, the PDAB reviewed a revised UPL Plan that did not respond to the comments from 38 organizations. The comment letter was not listed as a comment received for the meeting on their website, although receipt was confirmed by email. The revised UPL Plan continues to reference international prices from countries that use QALYs and similar measures and cost effectiveness analyses, with no safeguards against use of discriminatory value assessments, potentially violating federal law under Section 504 of the Rehabilitation Act.
The PDAB has asked for “emergency action” for updates to regulations covering the cost review process. The proposed changes, among other things, would allow for the PDAB to make a preliminary determination of affordability ahead of their final determination. Notably, the concept of the preliminary determination was already outlined and approved in the UPL Action Plan and cost reviews are currently underway.
Michigan
The Michigan legislature is similarly considering legislation SB 483 that would create a Prescription Drug Affordability Board. It includes language related to the use of quality-adjusted life years (QALYs) that was originally developed by the Institute for Clinical and Economic Review (ICER) for other states where it has not precluded boards from referencing QALYs and the equal value of life year gained (evLYG). As drafted, the language limits the use of QALYs only to identifying subpopulations and focuses on discrimination related to life extension, raising concerns that the legislation would allow the PDAB to partner with entities such as PORTAL and ICER that support the use of QALYs and evLYGs to value health care, as seen in other states. The bill has also been subject to critique related to its lack of patient engagement requirements and opportunities. Criteria for Board members would likely exclude patients or people with disabilities and even the stakeholder council does not require a patient perspective. View comments submitted by the Bonnell Foundation: Living with CF here. View NORD comments here. View comments from CANN here.
Oregon
The next meeting of the Oregon PDAB is January 15, 2025. Register to attend here.
The Oregon PDAB met on November 20, during which the Board approved a UPL Plan despite concerns about the influence of QALYs and similar measures, as well as the impact on formulary placement and patient access and affordability. Click here to view the letter from PIPC to the Board responding to its discussion about the use of cost effectiveness analyses at the October 2 meeting. Click here to view the most recent letter with 30 signatures from Oregon advocates and organizations.
During the summer, in response to patient advocates’ concerns, the chair of the Oregon Prescription Drug Affordability Board proposed and the board voted in favor of pausing its assessments until 2025. The Board recognized that it does not have an accurate definition of “affordability” and is taking time to review the data collected and how it may be used in its work. The Board will also reconsider its selection of drugs to be assessed. The decision follows a webinar hosted by Caring Ambassadors and Disability Rights Oregon and others on the lack of a robust engagement process and understanding of the Board’s goals and decision-making process, particularly related to whether cost savings would translate to lower patient out-of-pocket costs. Patient advocates applauded the decision. Click here to view the June 26 meeting. Click here to view the webinar educating advocates and advocacy letters to the Board and legislature from patients. Click here to read the letter. Click here to view the opinion from Oregon advocate Lorren Sandt. Click here to view the letter from advocates to the legislature requesting pause of the board’s activities and legislative oversight as the board works on its engagement process. Click here to view the drugs under consideration at each meeting, including treatments for obesity, multiple sclerosis, ulcerative colitis, psoriasis, ADHD, HIV, and diabetes.
We remain concerned that the Oregon PDAB has not made substantive changes to their patient and disability engagement strategies. It appears that the patient and disability communities will need to pursue legislative changes to accomplish more robust engagement.
Washington State
At the most recent November 18, 2024 meeting, the PDAB staff discussed its use of ICER Analytics and cost effectiveness as part of its deliberations, indicating an intent to reference QALYs and similar measures in its assessments. Click here to view more information about the meeting, its agenda and transcripts.
At the May 22, 2024 meeting, the Program on Regulation, Therapeutics, and Law (PORTAL) provided information to the Prescription Drug Affordability Board on “Considerations for Conducting Affordability Reviews,” including cost effectiveness. PORTAL is known to be aligned with ICER, which considers QALY-based cost effectiveness to be the gold standard. PORTAL has also in the past referenced the evLYG as a measure to consider in affordability reviews. PIPC is following the PDAB’s implementation in Washington State closely for potential use of discriminatory measures of effectiveness, especially now that its engagement with PORTAL is official and public. On September 18, the Board met and discussed its affordability review process, including the potential reference to premiums as a component of affordability reviews. Click here to view the schedule and review prior meetings.
PIPC submitted a comment letter to the Washington Prescription Drug Affordability Board (PDAB) highlighting the concerns of patients and people with disabilities related to the PDAB’s potential use of discriminatory cost effectiveness analyses. Click here to read the letter.
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. Arbitrary, condition-specific coverage decisions are restricting access to treatment for patients with a rare form of neurofibromatosis type 2.
- Canada. Some cancer patients are choosing to forgo treatment due to the lack of public and private coverage options for treatment.
- United Kingdom. NICE's latest guidance will slap a 12-year wait for patients to access certain obesity medications.
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.
- Multiple Sclerosis — SPMS: Draft Scoping Document available. Comments due Dec. 12, 2024.
- Special Assessment to Inform CMS Drug Price Negotiation: Research protocol available. Final report due March 4, 2024.
- Retinitis Pigmentosa: Revised Scoping Document, Research Protocol available. Public meeting: Apr. 2025.
- Acute Pain: Draft Evidence Report available. Public meeting: Feb. 28, 2025.
- Fair Access: Coverage Policies in 2024: Final report published.
- Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease: Final report published.
Upcoming Events and Webinars
PCORI 2025 & Beyond
January 21, 2025
Click here to view.
PCORI Advisory Panel Applicant Town Hall
February 10, 2025
Click here to view.
PCORI Board of Governors Meeting
February 11, 2025
Click here to view.
AHRQ Effective Program Updates
Systematic Review: Behavioral Interventions for Migraine Prevention. Click here to view.
Systematic Review: Healthcare Delivery of Clinical Preventive Services for People With Disabilities. Click here to view.
Systematic Review: Psychosocial and Pharmacologic Interventions for Disruptive Behavior in Children and Adolescents. Click here to view.
Research Protocol: Making Healthcare Safer IV: Programs for Responding to Harms Experienced by Patients during Clinical Care. Click here to view.
Research Protocol: Making Healthcare Safer IV: High Reliability Organization (HRO) as a Patient Safety Practice Click here to view.
Research Protocol: Making Healthcare Safer IV: Supply Chain Disruption Monitoring Programs Click here to view.