In this week's edition...
— CMS Publishes Final Guidance for 2026-2027 Implementation of Negotiation Program. See details below.
— PIPC Urges Oregon PDAB to Reject Discriminatory Metrics. See details below.
— AUCD Webinar: What do Adults with Autism and Other Neurodevelopmental Disorders Value in Adult Neurodevelopmental Care? Click here to register.
— Next Week! PCORI Annual Meeting October 22-23, 2024. See details below.
— Global Coalition on Aging Publishes Best Practices for Patient Engagement in PDABs. Click here to view the report.
— PCORI Accepting Nominations for Methodology Committee: Applications Due 10/31! Click here to learn more and submit a nomination.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— Advancing H.R. 485, the Protecting Health Care for All Patients Act. See details below.
— Medicare Drug Price Negotiation Program: CMS Announced Prices of First 10 Drugs. 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.
— CMS Publishes Final Guidance for 2026-2027 Implementation of Negotiation Program. See details below.
— PIPC Urges Oregon PDAB to Reject Discriminatory Metrics. See details below.
— AUCD Webinar: What do Adults with Autism and Other Neurodevelopmental Disorders Value in Adult Neurodevelopmental Care? Click here to register.
— Next Week! PCORI Annual Meeting October 22-23, 2024. See details below.
— Global Coalition on Aging Publishes Best Practices for Patient Engagement in PDABs. Click here to view the report.
— PCORI Accepting Nominations for Methodology Committee: Applications Due 10/31! Click here to learn more and submit a nomination.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— Advancing H.R. 485, the Protecting Health Care for All Patients Act. See details below.
— Medicare Drug Price Negotiation Program: CMS Announced Prices of First 10 Drugs. 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.
What We're Reading
1. CMS Publishes Final Guidance for 2026-2027 Implementation of Negotiation Program. The Centers for Medicare and Medicaid Services (CMS) recently published final guidance governing its implementation of the Medicare Drug Price Negotiation Program for 2026-2027. The Partnership to Improve Patient Care (PIPC) shares the goal of advancing affordability of health care for all patients and people with disabilities. While we appreciate that CMS recognizes the need to take a patient-centered approach to negotiation, we are disappointed that the agency has declined to develop a process for continuous patient engagement. Further, we are also concerned that the agency did not outline a process for disclosing to the public in a transparent manner how its decisions are made, the evidence on which it is making decisions, and particularly the value of patient input in its decisions. We understand that the agency is willing to accept data and input – patients are not clear on how that information is used and therefore whether there is a return on their investment of time and resources in this process. We look forward to learning more about the roundtables that CMS intends to convene with patients and urge the agency to demonstrate to patients that their engagement and investment in this process is meaningful and impactful in its decisions. Click here to view PIPC’s comments. Click here to view the final CMS guidance.
2. PIPC Urges Oregon PDAB to Reject Discriminatory Metrics. PIPC recently submitted a comments to the Oregon Prescription Drug Affordability Board (PDAB) to avoid policies that would potentially discriminate by relying on discriminatory metrics such as the Quality-Adjusted Life Year (QALY) that have detrimental implications for access to needed care and treatment. The letter also encourages the Oregon PDAB to include patients and people with disabilities throughout its decision-making process.
"It is important to note that cost effectiveness analyses always come with risky tradeoffs for patients and people with disabilities. It is now widely recognized that traditional methods and metrics of value assessment – even beyond the QALY – have significant shortcomings. Well-intentioned development of other measures and approaches that developers assert to be nondiscriminatory and more patient-centered come with tradeoffs, need for improvement, and inherent methodological flaws."
3. AUCD Webinar: What do Adults with Autism and Other Neurodevelopmental Disorders Value in Adult Neurodevelopmental Care? The Association of University Centers on Disabilities (AUCD) will host a webinar discussion today @ 4 PM EST on how to better understand the values and how to best serve adults with neurodevelopmental disabilities and their caregivers. Key topics will include: (1) Insights into Caregiver and Patient Priorities: Exploring what truly matters in neurodevelopmental care for adults; (2) Challenges and Opportunities in Adult NDD Care: Understanding the specific needs to tailor better health services; and (3) Future Directions in Neurodevelopmental Services: Shaping an emerging field to better serve this unique population. Click here to register.
4. Next Week! PCORI Annual Meeting October 22-23, 2024. PCORI’s two-day event brings together patients, caregivers, researchers and the broader healthcare community to learn about the latest findings from PCORI-funded research and research-related projects as well as to network and share in rich discussions to advance patient-centered comparative clinical effectiveness research. Session topics include:
Click here to view information about scholarships provided to those wanting to attend. Click here for additional details.
5. PCORI Accepting Nominations for Methodology Committee: Applications Due 10/31! PCORI is seeking new members of the Methodology Committee with expertise in various scientific fields. Final decisions are made by PCORI’s Board of Governors. The Committee is responsible for making recommendations to the Board regarding methodological standards for patient-centered comparative clinical effectiveness research (CER). PIPC encourages nominations for individuals that have experience with novel research methods prioritizing engagement of patients and people with disabilities and measuring outcomes that matter to patients. Click here to learn more and submit a nomination.
6. Global Coalition on Aging Publishes Best Practices for Patient Engagement in PDABs. The Global Coalition on Aging (GCOA) published a policy brief, “The Risks of Prescription Drug Affordability Boards and the Importance of Innovation for Healthy Aging and Health Equity.” The brief outlines patient concerns with Prescription Drug Affordability Boards (PDABs) and upper payment limits (UPLs) and highlights seven best practices for meaningful patient engagement processes within both established PDABs and state legislatures considering PDAB legislation. The report was informed by a convening in June 2024 of cross-sector stakeholders at a roundtable discussion on PDABs, UPLs. The new policy brief captures themes from the conversation between experts representing patients, caregivers, academia, biopharmaceutical innovators, and business communities. PIPC’s Executive Director stated at the convening, "The only real way to make a difference is to build in and require engagement.” Click here to view the report.
7. 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.
8. Advancing H.R. 485, the Protecting Health Care for All Patients Act. H.R. 485, the Protecting Health Care for All Patients Act, passed the House of Representatives on February 7, 2024. It was encouraging that the Statement of Administration Policy from the White House limited its opposition to the use of an offset to cover the estimated cost of the bill, but did not oppose the bill’s policy, highlighting Democratic support for the original QALY ban in the Affordable Care Act and the nondiscrimination provisions in the IRA. The need for an offset is expected to change going forward, giving this bipartisan policy change a strong chance of consideration this year. People with disabilities and serious chronic conditions continue to support legislation that would extend the law barring Medicare’s use of quality-adjusted life years (QALYs) and similar measures to other programs.
As CBO has stated, current law already prohibits the Secretary from using QALYs and similar measures in Medicare, thereby avoiding any impact on the Medicare Drug Price Negotiation Program with 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 H.R. 485) 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. A law that is consistent across federal programs will promote oversight and enforcement of laws barring the use of QALYs and similar measures.
We urge advocates to call on Congress to take action to pass H.R. 485 this year. Click here to view a one-pager about the bill. The National Down Syndrome Society has updated their FAQ on H.R. 485 here.
9. Medicare Drug Price Negotiation Program: CMS Announced Prices of First 10 Drugs On August 15, 2024, CMS announced the negotiated prices for Initial Price Applicability Year 2026 under the Medicare Drug Price Negotiation Program. As noted by Manatt Health attorneys, CMS is not required to publish an explanation of the rationale for their price decisions based on certain statutory factors until March 2025. Inside Health Policy reported that lawyers are eying whether CMS also referenced benchmarks such as international drug prices, which presumably would be made public in March 2025. As background, in 2019, the National Council on Disability recommended CMS avoid modeling US pricing after the pricing in other countries, which may heavily rely on QALYs and often deny people with disabilities access to needed care. Furthermore, Politico highlighted that the announcement is both a milestone for the administration - and complex. PIPC reiterated the comments and suggestions from nearly 60 organizations and individuals to CMS urging the agency to avoid use of evidence that devalues people with disabilities and serious chronic conditions, to continuously engage with patients and people with disabilities, and to adopt a concrete plan to monitor and respond to any unintended consequences for affordable and timely access to the specific treatment physicians determine to be most appropriate and effective for each individual patient. Similarly, the EACH Coalition urged CMS to closely monitor the ultimate impact on patients.
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
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).
Colorado's Prescription Drug Affordability Advisory Council (PDAAC) met on September 6, 2024 to discuss affordability reviews and upper payment limits. 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. The PDAAC met again on September 24. The next meeting of the full Board is on October 18. Click here to learn more and register. Comments may be sent to [email protected]. PIPC would urge the PDAB to pause its UPL work until it has a grasp on the impact of UPLs on patient access, utilization management etc. and encourage use of the patient-led survey HERE.
Maryland
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 UPL Plan is now to be considered for approval by the Maryland Legislative Policy Committee. Comments and concerns about the UPL Plan may be sent to the Committee chairs Senator Bill Ferguson at [email protected] and Del. Adrienne Jones at [email protected].
More information about the drugs and the evidence being reviewed by the PDAB is HERE. Note that it includes ICER reports, despite the Section 504 rules that bar use of discriminatory value assessments. We understand the Board will next meet on November 25, 2024 though the Board’s meeting dates have changed in the past.
Comments to the PDAB may be sent to [email protected].
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 Oregon PDAB met on October 2, during which the Board advanced its discussion of a UPL Plan. Register here for the next meeting on October 16. 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.
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.
The Oregon PDAB met on October 2, during which the Board advanced its discussion of a UPL Plan. We remain concerned that the Oregon PDAB has not made substantive changes to their patient and disability engagement strategies. It appears that they expect the patient and disability communities to pursue legislative changes to accomplish more robust engagement. Register here for the next meeting on October 16 and consider urging the PDAB to develop a robust plan for engagement, a transparent process for decisions and accessible information about the impact of UPLs on access to care, utilization management. Also, encourage use of the patient-led survey here.
Washington State
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. The Board will meet again on November 13. Click here to view the schedule and review prior meetings.
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 Annual Meeting
October 22-23, 2024
Click here to view.
Quality Medication Use in Rare Disease
November 10, 2024
Click here to view.
PCORI Board of Governors Meeting
December 10, 2024
Click here to view.
AHRQ Effective Program Updates
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. CMS Publishes Final Guidance for 2026-2027 Implementation of Negotiation Program. The Centers for Medicare and Medicaid Services (CMS) recently published final guidance governing its implementation of the Medicare Drug Price Negotiation Program for 2026-2027. The Partnership to Improve Patient Care (PIPC) shares the goal of advancing affordability of health care for all patients and people with disabilities. While we appreciate that CMS recognizes the need to take a patient-centered approach to negotiation, we are disappointed that the agency has declined to develop a process for continuous patient engagement. Further, we are also concerned that the agency did not outline a process for disclosing to the public in a transparent manner how its decisions are made, the evidence on which it is making decisions, and particularly the value of patient input in its decisions. We understand that the agency is willing to accept data and input – patients are not clear on how that information is used and therefore whether there is a return on their investment of time and resources in this process. We look forward to learning more about the roundtables that CMS intends to convene with patients and urge the agency to demonstrate to patients that their engagement and investment in this process is meaningful and impactful in its decisions. Click here to view PIPC’s comments. Click here to view the final CMS guidance.
2. PIPC Urges Oregon PDAB to Reject Discriminatory Metrics. PIPC recently submitted a comments to the Oregon Prescription Drug Affordability Board (PDAB) to avoid policies that would potentially discriminate by relying on discriminatory metrics such as the Quality-Adjusted Life Year (QALY) that have detrimental implications for access to needed care and treatment. The letter also encourages the Oregon PDAB to include patients and people with disabilities throughout its decision-making process.
"It is important to note that cost effectiveness analyses always come with risky tradeoffs for patients and people with disabilities. It is now widely recognized that traditional methods and metrics of value assessment – even beyond the QALY – have significant shortcomings. Well-intentioned development of other measures and approaches that developers assert to be nondiscriminatory and more patient-centered come with tradeoffs, need for improvement, and inherent methodological flaws."
3. AUCD Webinar: What do Adults with Autism and Other Neurodevelopmental Disorders Value in Adult Neurodevelopmental Care? The Association of University Centers on Disabilities (AUCD) will host a webinar discussion today @ 4 PM EST on how to better understand the values and how to best serve adults with neurodevelopmental disabilities and their caregivers. Key topics will include: (1) Insights into Caregiver and Patient Priorities: Exploring what truly matters in neurodevelopmental care for adults; (2) Challenges and Opportunities in Adult NDD Care: Understanding the specific needs to tailor better health services; and (3) Future Directions in Neurodevelopmental Services: Shaping an emerging field to better serve this unique population. Click here to register.
4. Next Week! PCORI Annual Meeting October 22-23, 2024. PCORI’s two-day event brings together patients, caregivers, researchers and the broader healthcare community to learn about the latest findings from PCORI-funded research and research-related projects as well as to network and share in rich discussions to advance patient-centered comparative clinical effectiveness research. Session topics include:
- How Are PCORI-Funded Palliative Care Trials Transforming the Delivery of Care for Individuals Living With Advanced Illnesses?
- Make Way for Mental Health: Pathways to Promoting Mental Wellness for Children and Youth
- Learn Why and How To Include People With Intellectual and Developmental Disabilities in Research
- Approaches to Hepatitis C Virus Care and Cure: Findings From the PCORI Research Portfolio
- Advancing Equity Through Patient-Centered Research
- Achieving Patient-Centeredness in Artificial Intelligence
Click here to view information about scholarships provided to those wanting to attend. Click here for additional details.
5. PCORI Accepting Nominations for Methodology Committee: Applications Due 10/31! PCORI is seeking new members of the Methodology Committee with expertise in various scientific fields. Final decisions are made by PCORI’s Board of Governors. The Committee is responsible for making recommendations to the Board regarding methodological standards for patient-centered comparative clinical effectiveness research (CER). PIPC encourages nominations for individuals that have experience with novel research methods prioritizing engagement of patients and people with disabilities and measuring outcomes that matter to patients. Click here to learn more and submit a nomination.
6. Global Coalition on Aging Publishes Best Practices for Patient Engagement in PDABs. The Global Coalition on Aging (GCOA) published a policy brief, “The Risks of Prescription Drug Affordability Boards and the Importance of Innovation for Healthy Aging and Health Equity.” The brief outlines patient concerns with Prescription Drug Affordability Boards (PDABs) and upper payment limits (UPLs) and highlights seven best practices for meaningful patient engagement processes within both established PDABs and state legislatures considering PDAB legislation. The report was informed by a convening in June 2024 of cross-sector stakeholders at a roundtable discussion on PDABs, UPLs. The new policy brief captures themes from the conversation between experts representing patients, caregivers, academia, biopharmaceutical innovators, and business communities. PIPC’s Executive Director stated at the convening, "The only real way to make a difference is to build in and require engagement.” Click here to view the report.
7. 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.
8. Advancing H.R. 485, the Protecting Health Care for All Patients Act. H.R. 485, the Protecting Health Care for All Patients Act, passed the House of Representatives on February 7, 2024. It was encouraging that the Statement of Administration Policy from the White House limited its opposition to the use of an offset to cover the estimated cost of the bill, but did not oppose the bill’s policy, highlighting Democratic support for the original QALY ban in the Affordable Care Act and the nondiscrimination provisions in the IRA. The need for an offset is expected to change going forward, giving this bipartisan policy change a strong chance of consideration this year. People with disabilities and serious chronic conditions continue to support legislation that would extend the law barring Medicare’s use of quality-adjusted life years (QALYs) and similar measures to other programs.
As CBO has stated, current law already prohibits the Secretary from using QALYs and similar measures in Medicare, thereby avoiding any impact on the Medicare Drug Price Negotiation Program with 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 H.R. 485) 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. A law that is consistent across federal programs will promote oversight and enforcement of laws barring the use of QALYs and similar measures.
We urge advocates to call on Congress to take action to pass H.R. 485 this year. Click here to view a one-pager about the bill. The National Down Syndrome Society has updated their FAQ on H.R. 485 here.
9. Medicare Drug Price Negotiation Program: CMS Announced Prices of First 10 Drugs On August 15, 2024, CMS announced the negotiated prices for Initial Price Applicability Year 2026 under the Medicare Drug Price Negotiation Program. As noted by Manatt Health attorneys, CMS is not required to publish an explanation of the rationale for their price decisions based on certain statutory factors until March 2025. Inside Health Policy reported that lawyers are eying whether CMS also referenced benchmarks such as international drug prices, which presumably would be made public in March 2025. As background, in 2019, the National Council on Disability recommended CMS avoid modeling US pricing after the pricing in other countries, which may heavily rely on QALYs and often deny people with disabilities access to needed care. Furthermore, Politico highlighted that the announcement is both a milestone for the administration - and complex. PIPC reiterated the comments and suggestions from nearly 60 organizations and individuals to CMS urging the agency to avoid use of evidence that devalues people with disabilities and serious chronic conditions, to continuously engage with patients and people with disabilities, and to adopt a concrete plan to monitor and respond to any unintended consequences for affordable and timely access to the specific treatment physicians determine to be most appropriate and effective for each individual patient. Similarly, the EACH Coalition urged CMS to closely monitor the ultimate impact on patients.
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
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).
Colorado's Prescription Drug Affordability Advisory Council (PDAAC) met on September 6, 2024 to discuss affordability reviews and upper payment limits. 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. The PDAAC met again on September 24. The next meeting of the full Board is on October 18. Click here to learn more and register. Comments may be sent to [email protected]. PIPC would urge the PDAB to pause its UPL work until it has a grasp on the impact of UPLs on patient access, utilization management etc. and encourage use of the patient-led survey HERE.
Maryland
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 UPL Plan is now to be considered for approval by the Maryland Legislative Policy Committee. Comments and concerns about the UPL Plan may be sent to the Committee chairs Senator Bill Ferguson at [email protected] and Del. Adrienne Jones at [email protected].
More information about the drugs and the evidence being reviewed by the PDAB is HERE. Note that it includes ICER reports, despite the Section 504 rules that bar use of discriminatory value assessments. We understand the Board will next meet on November 25, 2024 though the Board’s meeting dates have changed in the past.
Comments to the PDAB may be sent to [email protected].
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 Oregon PDAB met on October 2, during which the Board advanced its discussion of a UPL Plan. Register here for the next meeting on October 16. 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.
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.
The Oregon PDAB met on October 2, during which the Board advanced its discussion of a UPL Plan. We remain concerned that the Oregon PDAB has not made substantive changes to their patient and disability engagement strategies. It appears that they expect the patient and disability communities to pursue legislative changes to accomplish more robust engagement. Register here for the next meeting on October 16 and consider urging the PDAB to develop a robust plan for engagement, a transparent process for decisions and accessible information about the impact of UPLs on access to care, utilization management. Also, encourage use of the patient-led survey here.
Washington State
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. The Board will meet again on November 13. Click here to view the schedule and review prior meetings.
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. Unfair coverage decisions are forcing patients to get sicker in order to qualify for publicly-available treatments.
- New Zealand. A breast cancer patient had to spend $98k for early-stage treatment after Pharmac limited funding to late-stage triple-negative breast cancer cases.
- United Kingdom. Campaigners criticize the NHS for denying access to life-changing drugs due to high approval standards.
- United Kingdom. Breast Cancer Now has raised concerns over the NHS’s decision not to approve Enhertu for advanced breast cancer patients. An estimated 1,000 women a year could benefit from the treatment, which is available in many other countries.
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.
- Special Assessment to Inform CMS Drug Price Negotiation: Research protocol available. Final report due March 4, 2024.
- Retinitis Pigmentosa: Draft scoping document available. Comments open until October 16, 2024.
- Acute Pain: Revised Scoping Document, Research Protocol available. Public meeting: Feb. 28, 2025.
- Fair Access: Coverage Policies in 2024: Protocol available. Final report to be published Dec. 19, 2024.
- Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease: Draft Scoping Document, Research Protocol, Model Analysis Plan available. Public meeting: Nov. 2024
- Transthyretin Amyloidosis Cardiomyopathy: Draft Evidence Report available. Final Evidence Report to be published Oct. 21, 2024.
Upcoming Events and Webinars
PCORI Annual Meeting
October 22-23, 2024
Click here to view.
Quality Medication Use in Rare Disease
November 10, 2024
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PCORI Board of Governors Meeting
December 10, 2024
Click here to view.
AHRQ Effective Program Updates
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.
