In this week's edition...
— Oregon Advocate Meeting: Drug Affordability Review Process Updates. See details below.
— Payer Perspectives Confirm UPLs Will Likely Raise Costs and Hinder Patient Access to Medicines. Click here to read the survey.
— New PCORnet Data Report Provides Insights on Patient Populations Receiving Gene Therapy. Click here to read the report.
— CMS Public Engagement Events for Medicare Drug Negotiation Program. Click here to read the report.
— Use of QALYs and Similar Measures Deemed Acceptable in MD, Considered in Congress. See details below.
— The Protecting Health Care for All Patients Act Has No Budget Impact. See details below.
— CMS References ICER and NICE in Explanations for Medicare Drug Price Negotiation Program. See details below.
— ICHOM Conference and Activities Geared Up for 2025. See details below.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— 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
— Oregon Advocate Meeting: Drug Affordability Review Process Updates. See details below.
— Payer Perspectives Confirm UPLs Will Likely Raise Costs and Hinder Patient Access to Medicines. Click here to read the survey.
— New PCORnet Data Report Provides Insights on Patient Populations Receiving Gene Therapy. Click here to read the report.
— CMS Public Engagement Events for Medicare Drug Negotiation Program. Click here to read the report.
— Use of QALYs and Similar Measures Deemed Acceptable in MD, Considered in Congress. See details below.
— The Protecting Health Care for All Patients Act Has No Budget Impact. See details below.
— CMS References ICER and NICE in Explanations for Medicare Drug Price Negotiation Program. See details below.
— ICHOM Conference and Activities Geared Up for 2025. See details below.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— 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
What We're Reading
1. Oregon Advocate Meeting: Drug Affordability Review Process Updates. PIPC will Join Caring Ambassadors on April 14 at 12pm PST (3pm EST) to discuss a new engagement opportunity. The Oregon Prescription Drug Affordability Board (PDAB) recently released their list of 27 drugs for affordability review as well as a request for information (RFI) from patients, people with disabilities, caregivers, advocacy groups and other stakeholders as part of its affordability review process. As stated in the invitation, "The PDAB is making decisions that will impact all Oregonians. The patient and disability voice needs to be heard in the debate around drug affordability in Oregon." The Board’s request for information deadline is April 30, making this a timely discussion. Don’t miss this important opportunity to learn how to share your feedback. Click here to register.
2. Payer Perspectives Confirm UPLs Will Likely Raise Costs and Hinder Patient Access to Medicines. The Partnership to Fight Chronic Disease commissioned a survey where payers admit that PDABs setting upper payment limits (UPLs) would likely raise patient out-of-pocket medicine and premium costs while disrupting medicine access. "While PDABs have a goal of improving patient affordability and overall financial sustainability for the state and larger healthcare system, these interviews and surveys demonstrate that UPLs would not achieve that goal, but rather could result in higher premiums, increased UM, and decreased patient access. Payers agreed that PDABs often simplify or fail to understand the complexities of the prescription drug supply chain, and that has led to proposed UPL effectuation plans that threaten to push a new administrative burden and cost onto various players in the system, including patients."
3. New PCORnet Data Report Provides Insights on Patient Populations Receiving Gene Therapy. A new PCORnet Gene Therapy Data Report is now available, offering researchers and the public timely insights into patients receiving gene therapies within healthcare settings. "The Gene Therapy Data Report demonstrates that the PCORnet® infrastructure has the capacity to support national-scale studies on gene therapy. The report describes the characteristics of patient populations who received gene therapy for different health conditions between January 1, 2016 and June 30, 2024, including distinct cohorts for cancer patients and noncancer patients. Data from 72 sites participating in PCORnet are included in this report, reflecting the experience of more than 53,000 patients. These data are standardized from healthcare settings using the PCORnet® Common Data Model, meaning they are ready for researcher analysis.” Click here to read the report.
4. CMS Public Engagement Events for Medicare Drug Negotiation Program. CMS will host public engagement events for patients, beneficiaries, caregivers, consumer and patient organizations, and other interested parties, such as clinicians and researchers to share input on drugs in second cycle of the Medicare Drug Negotiation Program. View details at the PIPC Action Center here. Registration information is here.
5. Use of QALYs and Similar Measures Deemed Acceptable in MD, Considered in Congress. As the 119th Congress works toward a budget resolution, importing QALYs from foreign countries is once again on the agenda. A Senate amendment allowing for legislation to import international policies and prices was considered and failed Feb. 21, 2025, raising concerns for the disability community. PIPC is also monitoring closely the ongoing consideration of importing the use of QALYs and similar measures from foreign countries as part of state-based affordability reviews and setting reimbursement. PIPC has provided a series of comments to entities known as Prescription Drug Affordability Boards (PDAB) in states including Maryland, Oregon and Colorado to share and explain how discriminatory value assessments are used internationally to establish coverage and reimbursement policies.
On Feb. 26, 2025, the Maryland PDAB openly considered PIPC’s comments opposing the use of QALY-based studies from other countries. In concluding not to take action to limit the Board’s use of QALYs and similar measures, one board member stated, "I don't think we should take data off the table. I think we should use as much data as we possibly can use. Some of us are good fans of QALY, some of us are not good fans of the QALYs. So I think having as much data as possible to make a decision is always a good thing.” PIPC is increasingly concerned that the Maryland legislature is advancing SB357, a bill to expand the reach of the state’s PDAB to the commercial insurance market despite patient and disability concerns.
6. The Protecting Health Care for All Patients Act Has No Budget Impact. Last Congress, the Protecting Health Care for All Patients Act (H.R. 485) passed the House of Representatives on Feb. 7, 2024. Since finalization of the Section 504 rules barring disability discrimination in May 2024, Congressional staff have been informed that the House-passed bill has no budget impact. When considered in the House, the Biden administration's concerns about the bill were limited to the use of a budget offset from the CDC, which is no longer needed. As highlighted by the previous administration, Democrats supported the original QALY ban in the Affordable Care Act and the nondiscrimination provisions in the IRA. The bill has not yet been reintroduced. Without the need for an offset, this bipartisan policy change has a stronger chance of consideration this year. People with disabilities and serious chronic conditions continue to support this legislation extending the law barring Medicare’s use of quality-adjusted life years (QALYs) and similar measures to other programs.
As CBO stated when the bill was introduced, current law already prohibits the Secretary from using QALYs and similar measures in Medicare, giving it no impact on the Medicare Drug Price Negotiation Program, which is subject to both the nondiscrimination provisions in the IRA and in the ACA. Entities receiving federal financial assistance (i.e. all the entities impacted by the bill) are also subject to the rules governing Section 504 of the Rehabilitation Act that similarly bar the use of use any measure, assessment, or tool that discounts the value of life extension on the basis of disability (i.e. QALYs) to deny or afford an unequal opportunity to qualified individuals with disabilities, with the agency also clarifying that methods of utility weight generation are also subject to Section 504 when they are used in a way that discriminates. The Protecting Health Care for All Patients Act would explicitly make the statute governing use of QALYs and similar measures also clear and consistent across federal and state programs.
We urge advocates to call on Congress to take action to pass The Protecting Health Care for All Patients Act this year. Click here to view a one-pager about the bill. The National Down Syndrome Society has updated their FAQ on the legislation here.
7. 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 to CMS, 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.
8. 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.
9. ICHOM Conference and Activities Geared Up for 2025. ICHOM is gearing up for its 2025 Conference, scheduled for September 29-October 1 in Dublin, Ireland. This annual event convenes global healthcare leaders, including providers, insurers, policymakers, and patient advocates, to exchange insights and establish best practices in value-based healthcare. Attendees can anticipate engaging workshops, panel discussions, and deep dive sessions aimed at equipping them with actionable strategies to implement real change. The event engages with the patients through specific patient-led sessions, a dedicated patient area and provides support through the Patient
Scholarship Program (opening June 2025). PPA Members and their communities can receive a 10% discount to attend with the code: PPA10. View more information here.
ICHOM has also convened a Learning Collaborative for Obesity, developed a heart valve disease outcome set to be published soon, and is developing sets for liver disease, major injury, and kidney stones.
Learn More About Emerging Threats in States for Use of Discriminatory Metrics
Several states are advancing policies that would reference quality-adjusted life years (QALYs) and similar measures known to discriminate and devalue people with disabilities and serious chronic conditions, despite laws barring their use. Contrary to the law, these measures are considered by some to be the “gold standard” for measuring cost effectiveness of treatments, including the Institute for Clinical and Economic Review (ICER) and by foreign countries. Their use as a reference for U.S. coverage and reimbursement decisions is concerning due to implications for access to care, as seen internationally where a focus on cost effectiveness disadvantages coverage of health care for people with conditions that are chronic or disabling. To keep track of state-based threats, several resources are available:
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.
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.
Upcoming Events and Webinars
PCORI Board of Governors Meeting
June 17-18, 2025
Click here to view.
PCORI Annual Meeting
Oct. 21-22, 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.
1. Oregon Advocate Meeting: Drug Affordability Review Process Updates. PIPC will Join Caring Ambassadors on April 14 at 12pm PST (3pm EST) to discuss a new engagement opportunity. The Oregon Prescription Drug Affordability Board (PDAB) recently released their list of 27 drugs for affordability review as well as a request for information (RFI) from patients, people with disabilities, caregivers, advocacy groups and other stakeholders as part of its affordability review process. As stated in the invitation, "The PDAB is making decisions that will impact all Oregonians. The patient and disability voice needs to be heard in the debate around drug affordability in Oregon." The Board’s request for information deadline is April 30, making this a timely discussion. Don’t miss this important opportunity to learn how to share your feedback. Click here to register.
2. Payer Perspectives Confirm UPLs Will Likely Raise Costs and Hinder Patient Access to Medicines. The Partnership to Fight Chronic Disease commissioned a survey where payers admit that PDABs setting upper payment limits (UPLs) would likely raise patient out-of-pocket medicine and premium costs while disrupting medicine access. "While PDABs have a goal of improving patient affordability and overall financial sustainability for the state and larger healthcare system, these interviews and surveys demonstrate that UPLs would not achieve that goal, but rather could result in higher premiums, increased UM, and decreased patient access. Payers agreed that PDABs often simplify or fail to understand the complexities of the prescription drug supply chain, and that has led to proposed UPL effectuation plans that threaten to push a new administrative burden and cost onto various players in the system, including patients."
3. New PCORnet Data Report Provides Insights on Patient Populations Receiving Gene Therapy. A new PCORnet Gene Therapy Data Report is now available, offering researchers and the public timely insights into patients receiving gene therapies within healthcare settings. "The Gene Therapy Data Report demonstrates that the PCORnet® infrastructure has the capacity to support national-scale studies on gene therapy. The report describes the characteristics of patient populations who received gene therapy for different health conditions between January 1, 2016 and June 30, 2024, including distinct cohorts for cancer patients and noncancer patients. Data from 72 sites participating in PCORnet are included in this report, reflecting the experience of more than 53,000 patients. These data are standardized from healthcare settings using the PCORnet® Common Data Model, meaning they are ready for researcher analysis.” Click here to read the report.
4. CMS Public Engagement Events for Medicare Drug Negotiation Program. CMS will host public engagement events for patients, beneficiaries, caregivers, consumer and patient organizations, and other interested parties, such as clinicians and researchers to share input on drugs in second cycle of the Medicare Drug Negotiation Program. View details at the PIPC Action Center here. Registration information is here.
- Events will be held April 16-April 30;
- One event will be live-streamed for the public on April 30 on all selected drugs.
- 15 private patient-focused events for each drug will be held for patients, caregivers and patient organizations.
5. Use of QALYs and Similar Measures Deemed Acceptable in MD, Considered in Congress. As the 119th Congress works toward a budget resolution, importing QALYs from foreign countries is once again on the agenda. A Senate amendment allowing for legislation to import international policies and prices was considered and failed Feb. 21, 2025, raising concerns for the disability community. PIPC is also monitoring closely the ongoing consideration of importing the use of QALYs and similar measures from foreign countries as part of state-based affordability reviews and setting reimbursement. PIPC has provided a series of comments to entities known as Prescription Drug Affordability Boards (PDAB) in states including Maryland, Oregon and Colorado to share and explain how discriminatory value assessments are used internationally to establish coverage and reimbursement policies.
On Feb. 26, 2025, the Maryland PDAB openly considered PIPC’s comments opposing the use of QALY-based studies from other countries. In concluding not to take action to limit the Board’s use of QALYs and similar measures, one board member stated, "I don't think we should take data off the table. I think we should use as much data as we possibly can use. Some of us are good fans of QALY, some of us are not good fans of the QALYs. So I think having as much data as possible to make a decision is always a good thing.” PIPC is increasingly concerned that the Maryland legislature is advancing SB357, a bill to expand the reach of the state’s PDAB to the commercial insurance market despite patient and disability concerns.
6. The Protecting Health Care for All Patients Act Has No Budget Impact. Last Congress, the Protecting Health Care for All Patients Act (H.R. 485) passed the House of Representatives on Feb. 7, 2024. Since finalization of the Section 504 rules barring disability discrimination in May 2024, Congressional staff have been informed that the House-passed bill has no budget impact. When considered in the House, the Biden administration's concerns about the bill were limited to the use of a budget offset from the CDC, which is no longer needed. As highlighted by the previous administration, Democrats supported the original QALY ban in the Affordable Care Act and the nondiscrimination provisions in the IRA. The bill has not yet been reintroduced. Without the need for an offset, this bipartisan policy change has a stronger chance of consideration this year. People with disabilities and serious chronic conditions continue to support this legislation extending the law barring Medicare’s use of quality-adjusted life years (QALYs) and similar measures to other programs.
As CBO stated when the bill was introduced, current law already prohibits the Secretary from using QALYs and similar measures in Medicare, giving it no impact on the Medicare Drug Price Negotiation Program, which is subject to both the nondiscrimination provisions in the IRA and in the ACA. Entities receiving federal financial assistance (i.e. all the entities impacted by the bill) are also subject to the rules governing Section 504 of the Rehabilitation Act that similarly bar the use of use any measure, assessment, or tool that discounts the value of life extension on the basis of disability (i.e. QALYs) to deny or afford an unequal opportunity to qualified individuals with disabilities, with the agency also clarifying that methods of utility weight generation are also subject to Section 504 when they are used in a way that discriminates. The Protecting Health Care for All Patients Act would explicitly make the statute governing use of QALYs and similar measures also clear and consistent across federal and state programs.
We urge advocates to call on Congress to take action to pass The Protecting Health Care for All Patients Act this year. Click here to view a one-pager about the bill. The National Down Syndrome Society has updated their FAQ on the legislation here.
7. 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 to CMS, 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.
8. 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.
9. ICHOM Conference and Activities Geared Up for 2025. ICHOM is gearing up for its 2025 Conference, scheduled for September 29-October 1 in Dublin, Ireland. This annual event convenes global healthcare leaders, including providers, insurers, policymakers, and patient advocates, to exchange insights and establish best practices in value-based healthcare. Attendees can anticipate engaging workshops, panel discussions, and deep dive sessions aimed at equipping them with actionable strategies to implement real change. The event engages with the patients through specific patient-led sessions, a dedicated patient area and provides support through the Patient
Scholarship Program (opening June 2025). PPA Members and their communities can receive a 10% discount to attend with the code: PPA10. View more information here.
ICHOM has also convened a Learning Collaborative for Obesity, developed a heart valve disease outcome set to be published soon, and is developing sets for liver disease, major injury, and kidney stones.
Learn More About Emerging Threats in States for Use of Discriminatory Metrics
Several states are advancing policies that would reference quality-adjusted life years (QALYs) and similar measures known to discriminate and devalue people with disabilities and serious chronic conditions, despite laws barring their use. Contrary to the law, these measures are considered by some to be the “gold standard” for measuring cost effectiveness of treatments, including the Institute for Clinical and Economic Review (ICER) and by foreign countries. Their use as a reference for U.S. coverage and reimbursement decisions is concerning due to implications for access to care, as seen internationally where a focus on cost effectiveness disadvantages coverage of health care for people with conditions that are chronic or disabling. To keep track of state-based threats, several resources are available:
- Patient Access Project State-by-state tracker of QALY-based policies and legislation, including Prescription Drug Affordability Boards, P&T Committees, and reference to discriminatory policies in foreign countries.
- Value Of Care Coalition advocacy tracker of state PDAB comment opportunities.
- AIMED Alliance resources and tracker of state PDAB activities.
- PIPC resources on use of QALYs and similar measures internationally.
- EACH/PIC Coalition resources.
- Value Our Health toolkit and resources on implications for disability discrimination.
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. "Facing high medical costs, the family has turned to fundraising to support her ongoing treatment.”
- Europe: Patients suffering with Alzheimer’s in the European Union will have to keep waiting for new treatments after regulators blocked a drug that’s already been approved in the United States and the United Kingdom.
- New Zealand. Physicians and patients are calling for more funding to broaden the eligibility criteria for the free shingles vaccine to stop people suffering from extraordinarily painful and debilitating symptoms.
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.
- Non-Cystic Fibrosis Bronchiectasis: Draft Scoping Document available. Public comments open until March 31, 2025. Public meeting: September 2025.
- Spinal Muscular Atrophy: Revised Scoping Document, Research Protocol available. Public meeting: August 2025.
- Multiple Sclerosis — SPMS: Revised Scoping Document, Research Protocol, Model Analysis Plan available. Public meeting: June 2025.
- Retinitis Pigmentosa: Evidence Report available. Comments were due March 6, 2025. Public meeting: April 11, 2025.
Upcoming Events and Webinars
PCORI Board of Governors Meeting
June 17-18, 2025
Click here to view.
PCORI Annual Meeting
Oct. 21-22, 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.
