In this week's edition...
— Proposed Use of QALYs and Similar Measures from Foreign Countries in States - MD, OR and CO. 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.
— Join PCORI's Advisory Panels: Applications, Nominations are Due March 7! Click here for additional details and to submit an application.
— PIPC Calls for Robust Engagement and Transparency as CMS Reviews 15 Additional Drugs under Medicare Drug Negotiation Program. 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.
— Proposed Use of QALYs and Similar Measures from Foreign Countries in States - MD, OR and CO. 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.
— Join PCORI's Advisory Panels: Applications, Nominations are Due March 7! Click here for additional details and to submit an application.
— PIPC Calls for Robust Engagement and Transparency as CMS Reviews 15 Additional Drugs under Medicare Drug Negotiation Program. 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. Proposed Use of QALYs and Similar Measures from Foreign Countries in States - MD, OR and CO. PIPC is 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 in states including Maryland, Oregon and Colorado to share and explain how discriminatory value assessments are used internationally to establish coverage and reimbursement policies. As background, the National Council on Disability, an independent federal agency advising Congress and the administration on disability policy, highlighted how foreign countries use QALYs and similar measures to discriminate as part of its Ethics Series in 2019 and in policy recommendations to Congress and the administration. Click here to view what the experts have said about policies importing QALYs and similar measures to the United States.
2. 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 February 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 White House, Democrats supported the original QALY ban in the Affordable Car Act and the nondiscrimination provisions in the IRA. 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 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.
3. 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.
4. 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.
5. Join PCORI's Advisory Panels: Applications, Nominations are Due March 7! PCORI is accepting applications and nominations for members on its multi-stakeholder advisory panels for the 2024-2025 cycle. These research advisory panels are an important way we bring voices from across the health care community into our work. The panels focus on clinical effectiveness and decision science, clinical trials, healthcare delivery and disparities research, patient engagement, and rare diseases. Click here for additional details and to submit an application.
6. PIPC Calls for Robust Engagement and Transparency as CMS Reviews 15 Additional Drugs under Medicare Drug Negotiation Program. On January 17, 2025, CMS released the second list of drugs that will be subject to Medicare negotiation under the Inflation Reduction Act (IRA). PIPC reiterated in a statement that many patients rely on these treatments for their health and quality of life. PIPC also highlighted concern that ICER and NICE were cited in the explanations for the most recent drugs reviewed by the program and urged CMS to avoid relying on outside research entities whose methods inherently devalue the lives of people with disabilities as they review the second list of drugs. It is not clear how the new administration may change the process to center on patients and people with disabilities.
PIPC stated, "It is imperative that they are consistently able to access the medications they need and that payers and government bureaucrats do not come between patients and their doctors...Unfortunately, CMS’ process over the past year did not provide a level of robust engagement and transparency that would allow patients and people with disabilities to trust that their voices were heard in the process and that different patients’ needs were considered. Therefore, we encourage the incoming administration to take the time necessary to revisit the processes for implementation of the program and address any shortcomings going forward.” View PIPC’s statement here.
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.
Colorado
PDAB meets March 7! The full PDAB Board's next meeting will take place March 7, 2025 and patients may register to attend here.
The Colorado PDAB released its 2025 meeting and staff office hour schedule, which can be accessed here. The Prescription Drug Affordability Advisory Council (PDAAC) met on January 23, 2025.
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 voted to adopt a rule (PDAB Rule 3 CCR 702-9) that would allow the PDAB to consider QALYs, despite state and federal law barring its consideration of QALYs and similar measures. The PDAB also confirmed that they will be proceeding with a UPL rulemaking for Enbrel starting in January.
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 legislative committees met on Feb. 6, 2025 to review HB424/ SB357, legislation requiring the Prescription Drug Affordability Board, under certain circumstances, to establish a process for setting upper payment limits for all purchases and payor reimbursements of prescription drug products in the State that the Board determines have led or will lead to affordability challenges. The bill does not bar the use of QALYs and similar measures and does not bar consideration of information from third parties that utilize QALYs and similar measures. View PIPC’s comments here reiterating past comments and concerns.
PDASC meeting CANCELLED Feb 24!
Comments on the Maryland PDAB proposal at 14.01.05 in Section .06 Policy Review—Process for Establishing a UPL, were due on Feb. 10, 2025. The proposed rule explicitly calls or use of cost effectiveness analysis and does not discuss any restrictions against the use of QALYs and similar measures. View PIPC’s comments here, reiterating several comment letters sent in the past opposing the Board’s proposal to reference international prices and cost effectiveness without protections against use of discriminatory value assessments.
The Maryland PDAB released its 2025 meeting schedule, which can be found here. The next PDAB meeting is TBD.
At the 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.
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 Wednesday, February 19, 2025. Register to attend here. Provide testimony here. To speak, submit a form 24 hours beforehand. To submit written comments, submit 48 hours beforehand.
The Oregon PDAB responded to advocates opposing its proposed use of ICER’s measure known as the evLYG, expressing support for its use and stating "Therefore, we believe that evLYG data for analyses or reports would be permissible to use during affordability reviews and we welcome your further input on why they cannot be so used.” PIPC submitted additional comments to the Board, again highlighting how QALYs, evLYG and similar measures are contrary to nondiscrimination laws.
As background, at the Oregon PDAB meeting on November 20, 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
The Washington PDAB will meet again on March 19, 2025. At the Nov. 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.
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.
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
March 18-19, 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. Proposed Use of QALYs and Similar Measures from Foreign Countries in States - MD, OR and CO. PIPC is 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 in states including Maryland, Oregon and Colorado to share and explain how discriminatory value assessments are used internationally to establish coverage and reimbursement policies. As background, the National Council on Disability, an independent federal agency advising Congress and the administration on disability policy, highlighted how foreign countries use QALYs and similar measures to discriminate as part of its Ethics Series in 2019 and in policy recommendations to Congress and the administration. Click here to view what the experts have said about policies importing QALYs and similar measures to the United States.
2. 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 February 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 White House, Democrats supported the original QALY ban in the Affordable Car Act and the nondiscrimination provisions in the IRA. 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 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.
3. 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.
4. 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.
5. Join PCORI's Advisory Panels: Applications, Nominations are Due March 7! PCORI is accepting applications and nominations for members on its multi-stakeholder advisory panels for the 2024-2025 cycle. These research advisory panels are an important way we bring voices from across the health care community into our work. The panels focus on clinical effectiveness and decision science, clinical trials, healthcare delivery and disparities research, patient engagement, and rare diseases. Click here for additional details and to submit an application.
6. PIPC Calls for Robust Engagement and Transparency as CMS Reviews 15 Additional Drugs under Medicare Drug Negotiation Program. On January 17, 2025, CMS released the second list of drugs that will be subject to Medicare negotiation under the Inflation Reduction Act (IRA). PIPC reiterated in a statement that many patients rely on these treatments for their health and quality of life. PIPC also highlighted concern that ICER and NICE were cited in the explanations for the most recent drugs reviewed by the program and urged CMS to avoid relying on outside research entities whose methods inherently devalue the lives of people with disabilities as they review the second list of drugs. It is not clear how the new administration may change the process to center on patients and people with disabilities.
PIPC stated, "It is imperative that they are consistently able to access the medications they need and that payers and government bureaucrats do not come between patients and their doctors...Unfortunately, CMS’ process over the past year did not provide a level of robust engagement and transparency that would allow patients and people with disabilities to trust that their voices were heard in the process and that different patients’ needs were considered. Therefore, we encourage the incoming administration to take the time necessary to revisit the processes for implementation of the program and address any shortcomings going forward.” View PIPC’s statement here.
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
PDAB meets March 7! The full PDAB Board's next meeting will take place March 7, 2025 and patients may register to attend here.
The Colorado PDAB released its 2025 meeting and staff office hour schedule, which can be accessed here. The Prescription Drug Affordability Advisory Council (PDAAC) met on January 23, 2025.
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 voted to adopt a rule (PDAB Rule 3 CCR 702-9) that would allow the PDAB to consider QALYs, despite state and federal law barring its consideration of QALYs and similar measures. The PDAB also confirmed that they will be proceeding with a UPL rulemaking for Enbrel starting in January.
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 legislative committees met on Feb. 6, 2025 to review HB424/ SB357, legislation requiring the Prescription Drug Affordability Board, under certain circumstances, to establish a process for setting upper payment limits for all purchases and payor reimbursements of prescription drug products in the State that the Board determines have led or will lead to affordability challenges. The bill does not bar the use of QALYs and similar measures and does not bar consideration of information from third parties that utilize QALYs and similar measures. View PIPC’s comments here reiterating past comments and concerns.
PDASC meeting CANCELLED Feb 24!
Comments on the Maryland PDAB proposal at 14.01.05 in Section .06 Policy Review—Process for Establishing a UPL, were due on Feb. 10, 2025. The proposed rule explicitly calls or use of cost effectiveness analysis and does not discuss any restrictions against the use of QALYs and similar measures. View PIPC’s comments here, reiterating several comment letters sent in the past opposing the Board’s proposal to reference international prices and cost effectiveness without protections against use of discriminatory value assessments.
The Maryland PDAB released its 2025 meeting schedule, which can be found here. The next PDAB meeting is TBD.
At the 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.
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 Wednesday, February 19, 2025. Register to attend here. Provide testimony here. To speak, submit a form 24 hours beforehand. To submit written comments, submit 48 hours beforehand.
The Oregon PDAB responded to advocates opposing its proposed use of ICER’s measure known as the evLYG, expressing support for its use and stating "Therefore, we believe that evLYG data for analyses or reports would be permissible to use during affordability reviews and we welcome your further input on why they cannot be so used.” PIPC submitted additional comments to the Board, again highlighting how QALYs, evLYG and similar measures are contrary to nondiscrimination laws.
As background, at the Oregon PDAB meeting on November 20, 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
The Washington PDAB will meet again on March 19, 2025. At the Nov. 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.
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.
- Canada. Children with rare diseases get placed in the crosshairs over arbitrary coverage decisions that limit access and raise costs.
- United Kingdom. The NHS has discontinued coverage for a variety of over-the-counter remedies for the sole purpose of cutting expenditures, shifting the access burden to patients.
- Korea. Drug price negotiations and cost controls have incentivized some companies to roll back their presence, shrinking access for patients.
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.
- Spinal Muscular Atrophy: Revised Scoping Document, Research Protocol available.
- Multiple Sclerosis — SPMS: Revised Scoping Document, Research Protocol available.
- Special Assessment to Inform CMS Drug Price Negotiation: Research Protocol available. Final report due March 4, 2025.
- Retinitis Pigmentosa: Draft Evidence Report available. Comments due March 6, 2025.
- Acute Pain: Draft Evidence Report available. Public meeting: Feb. 28, 2025.
Upcoming Events and Webinars
PCORI Board of Governors Meeting
March 18-19, 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.
