In this week's edition...
— H.R. 485, the Protecting Health Care for All Patients Act, Has No Budget Impact. See details below.
— Center for Innovation & Value Research Seeking Stakeholder Interviews on Digital Health Therapies. See details below.
— Journal: Improving Access to Gene Therapies. Click here to read the article.
— National Alliance for Caregiving Report Highlights Critical Need to Support Transplant Caregivers. See details below.
— Help Shape the NIH Strategic Plan for Disability Research. See details below.
— CMS Publishes Final Guidance for 2026-2027 Implementation of 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.
— H.R. 485, the Protecting Health Care for All Patients Act, Has No Budget Impact. See details below.
— Center for Innovation & Value Research Seeking Stakeholder Interviews on Digital Health Therapies. See details below.
— Journal: Improving Access to Gene Therapies. Click here to read the article.
— National Alliance for Caregiving Report Highlights Critical Need to Support Transplant Caregivers. See details below.
— Help Shape the NIH Strategic Plan for Disability Research. See details below.
— CMS Publishes Final Guidance for 2026-2027 Implementation of 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. H.R. 485, the Protecting Health Care for All Patients Act, Has No Budget Impact. H.R. 485, the Protecting Health Care for All Patients Act, passed the House of Representatives on February 7, 2024. Since finalization of the new 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 White House 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 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. H.R. 485 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 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.
2. Center for Innovation & Value Research Seeking Stakeholder Interviews on Digital Health Therapies. The Center for Innovation & Value Research is conducting key informant interviews to learn from people who have experience with digital health solutions to manage anxiety or depression. All conversations are confidential, and your insights will directly impact the research study. If selected, you will participate in a 60-minute virtual interview and receive a $150 honorarium. If you have used a digital health app for the treatment of anxiety or depression, please complete this form to apply to participate.
3. Journal: Improving Access to Gene Therapies. An article in the Journal of Comparative Effectiveness Research. examines the flaws in ICER's value assessment methods and how they limit access to gene therapies and other innovative treatments. "Gene therapies hold the promise to transform healthcare and deliver groundbreaking improvements in patient health outcomes for previously intractable diseases. However, numerous challenges currently limit optimal patient access to gene therapy treatments. Moving forward, it is imperative that we collectively strive for policy and market solutions that are comprehensive and sensitive to the uniqueness of gene therapies and are geared toward improving patient health and society." Click here to read the article.
4. National Alliance for Caregiving Report Highlights Critical Need to Support Transplant Caregivers. The National Alliance for Caregiving (NAC) released new research revealing significant gaps in family caregiver support across U.S. transplant centers. The report, “Gaps and Opportunities: Family Caregiver Programs in U.S. Transplant Centers,” documents critical shortfalls in support services and programs for family caregivers – whose contributions are vital to successful transplant outcomes. NAC will host a webinar on December 11 @ 1 pm ET to learn more about the report, its findings, and recommendations. Click here to register. Click here to read the report.
5. Help Shape the NIH Strategic Plan for Disability Research. The National Institutes of Health is in the initial stage of developing an agency-wide NIH Strategic Plan for Disability Research. The agency has shared that the strategic plan will identify scientific themes and develop operational goals and objectives to advance research activities that promote the health and well-being of people with disabilities. Central to the strategic planning process is robust and meaningful community engagement to obtain a diverse range of input that will help inform the plan. Disability communities, including people with lived experience of disability, researchers, clinicians, advocacy organizations, and professional associations, are encouraged to participate in the strategic planning process. NIH has shared that the agency is holding a series of community engagement events over the next two months to better identify and understand disability communities’ health and research concerns and unique needs and preferences. Interested individuals are welcome to observe any of the Community Roundtables, join in on the Town Hall, and/or submit their input via a forthcoming Request for Information (RFI). The next roundtable will be held on Tuesday, Dec. 20. Click here to learn more about events and opportunities for input.
6. 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.
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.
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).
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.
The PDAB meets again on December 6, 2024. Click here to 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 Maryland Legislative Policy Committee approved the PDAB’s UPL Plan, despite comments from PIPC and others opposing its approval. As background, the PDAB reviewed a revised UPL Plan that did not respond to the comments from 38 organizations. The comment letter was not listed as a comment received for the meeting on their website, although receipt was confirmed by email. The revised UPL Plan continues to reference international prices from countries that use QALYs and similar measures and cost effectiveness analyses, with no safeguards against use of discriminatory value assessments, potentially violating federal law under Section 504 of the Rehabilitation Act.
The PDAB has asked for “emergency action” for updates to regulations covering the cost review process. The proposed changes, among other things, would allow for the PDAB to make a preliminary determination of affordability ahead of their final determination. Notably, the concept of the preliminary determination was already outlined and approved in the UPL Action Plan and cost reviews are currently underway.
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 November 20, during which the Board approved a UPL Plan despite concerns about the influence of QALYs and similar measures, as well as the impact on formulary placement and patient access and affordability. Click here to view the letter from PIPC to the Board responding to its discussion about the use of cost effectiveness analyses at the October 2 meeting. Click here to view the most recent letter with 30 signatures from Oregon advocates and organizations.
During the summer, in response to patient advocates’ concerns, the chair of the Oregon Prescription Drug Affordability Board proposed and the board voted in favor of pausing its assessments until 2025. The Board recognized that it does not have an accurate definition of “affordability” and is taking time to review the data collected and how it may be used in its work. The Board will also reconsider its selection of drugs to be assessed. The decision follows a webinar hosted by Caring Ambassadors and Disability Rights Oregon and others on the lack of a robust engagement process and understanding of the Board’s goals and decision-making process, particularly related to whether cost savings would translate to lower patient out-of-pocket costs. Patient advocates applauded the decision. Click here to view the June 26 meeting. Click here to view the webinar educating advocates and advocacy letters to the Board and legislature from patients. Click here to read the letter. Click here to view the opinion from Oregon advocate Lorren Sandt. Click here to view the letter from advocates to the legislature requesting pause of the board’s activities and legislative oversight as the board works on its engagement process. Click here to view the drugs under consideration at each meeting, including treatments for obesity, multiple sclerosis, ulcerative colitis, psoriasis, ADHD, HIV, and diabetes.
We remain concerned that the Oregon PDAB has not made substantive changes to their patient and disability engagement strategies. It appears that the patient and disability communities will need to pursue legislative changes to accomplish more robust engagement.
Washington State
At the most recent November 18, 2024 meeting, the PDAB staff discussed its use of ICER Analytics and cost effectiveness as part of its deliberations, indicating an intent to reference QALYs and similar measures in its assessments. Click here to view more information about the meeting, its agenda and transcripts.
At the May 22, 2024 meeting, the Program on Regulation, Therapeutics, and Law (PORTAL) provided information to the Prescription Drug Affordability Board on “Considerations for Conducting Affordability Reviews,” including cost effectiveness. PORTAL is known to be aligned with ICER, which considers QALY-based cost effectiveness to be the gold standard. PORTAL has also in the past referenced the evLYG as a measure to consider in affordability reviews. PIPC is following the PDAB’s implementation in Washington State closely for potential use of discriminatory measures of effectiveness, especially now that its engagement with PORTAL is official and public. On September 18, the Board met and discussed its affordability review process, including the potential reference to premiums as a component of affordability reviews. Click here to view the schedule and review prior meetings.
PIPC submitted a comment letter to the Washington Prescription Drug Affordability Board (PDAB) highlighting the concerns of patients and people with disabilities related to the PDAB’s potential use of discriminatory cost effectiveness analyses. Click here to read the letter.
International News: What Happens in Countries Using QALYs and Cost-Based Thresholds?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
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
December 10, 2024
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. H.R. 485, the Protecting Health Care for All Patients Act, Has No Budget Impact. H.R. 485, the Protecting Health Care for All Patients Act, passed the House of Representatives on February 7, 2024. Since finalization of the new 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 White House 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 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. H.R. 485 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 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.
2. Center for Innovation & Value Research Seeking Stakeholder Interviews on Digital Health Therapies. The Center for Innovation & Value Research is conducting key informant interviews to learn from people who have experience with digital health solutions to manage anxiety or depression. All conversations are confidential, and your insights will directly impact the research study. If selected, you will participate in a 60-minute virtual interview and receive a $150 honorarium. If you have used a digital health app for the treatment of anxiety or depression, please complete this form to apply to participate.
3. Journal: Improving Access to Gene Therapies. An article in the Journal of Comparative Effectiveness Research. examines the flaws in ICER's value assessment methods and how they limit access to gene therapies and other innovative treatments. "Gene therapies hold the promise to transform healthcare and deliver groundbreaking improvements in patient health outcomes for previously intractable diseases. However, numerous challenges currently limit optimal patient access to gene therapy treatments. Moving forward, it is imperative that we collectively strive for policy and market solutions that are comprehensive and sensitive to the uniqueness of gene therapies and are geared toward improving patient health and society." Click here to read the article.
4. National Alliance for Caregiving Report Highlights Critical Need to Support Transplant Caregivers. The National Alliance for Caregiving (NAC) released new research revealing significant gaps in family caregiver support across U.S. transplant centers. The report, “Gaps and Opportunities: Family Caregiver Programs in U.S. Transplant Centers,” documents critical shortfalls in support services and programs for family caregivers – whose contributions are vital to successful transplant outcomes. NAC will host a webinar on December 11 @ 1 pm ET to learn more about the report, its findings, and recommendations. Click here to register. Click here to read the report.
5. Help Shape the NIH Strategic Plan for Disability Research. The National Institutes of Health is in the initial stage of developing an agency-wide NIH Strategic Plan for Disability Research. The agency has shared that the strategic plan will identify scientific themes and develop operational goals and objectives to advance research activities that promote the health and well-being of people with disabilities. Central to the strategic planning process is robust and meaningful community engagement to obtain a diverse range of input that will help inform the plan. Disability communities, including people with lived experience of disability, researchers, clinicians, advocacy organizations, and professional associations, are encouraged to participate in the strategic planning process. NIH has shared that the agency is holding a series of community engagement events over the next two months to better identify and understand disability communities’ health and research concerns and unique needs and preferences. Interested individuals are welcome to observe any of the Community Roundtables, join in on the Town Hall, and/or submit their input via a forthcoming Request for Information (RFI). The next roundtable will be held on Tuesday, Dec. 20. Click here to learn more about events and opportunities for input.
6. 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.
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.
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).
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.
The PDAB meets again on December 6, 2024. Click here to 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 Maryland Legislative Policy Committee approved the PDAB’s UPL Plan, despite comments from PIPC and others opposing its approval. As background, the PDAB reviewed a revised UPL Plan that did not respond to the comments from 38 organizations. The comment letter was not listed as a comment received for the meeting on their website, although receipt was confirmed by email. The revised UPL Plan continues to reference international prices from countries that use QALYs and similar measures and cost effectiveness analyses, with no safeguards against use of discriminatory value assessments, potentially violating federal law under Section 504 of the Rehabilitation Act.
The PDAB has asked for “emergency action” for updates to regulations covering the cost review process. The proposed changes, among other things, would allow for the PDAB to make a preliminary determination of affordability ahead of their final determination. Notably, the concept of the preliminary determination was already outlined and approved in the UPL Action Plan and cost reviews are currently underway.
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 November 20, during which the Board approved a UPL Plan despite concerns about the influence of QALYs and similar measures, as well as the impact on formulary placement and patient access and affordability. Click here to view the letter from PIPC to the Board responding to its discussion about the use of cost effectiveness analyses at the October 2 meeting. Click here to view the most recent letter with 30 signatures from Oregon advocates and organizations.
During the summer, in response to patient advocates’ concerns, the chair of the Oregon Prescription Drug Affordability Board proposed and the board voted in favor of pausing its assessments until 2025. The Board recognized that it does not have an accurate definition of “affordability” and is taking time to review the data collected and how it may be used in its work. The Board will also reconsider its selection of drugs to be assessed. The decision follows a webinar hosted by Caring Ambassadors and Disability Rights Oregon and others on the lack of a robust engagement process and understanding of the Board’s goals and decision-making process, particularly related to whether cost savings would translate to lower patient out-of-pocket costs. Patient advocates applauded the decision. Click here to view the June 26 meeting. Click here to view the webinar educating advocates and advocacy letters to the Board and legislature from patients. Click here to read the letter. Click here to view the opinion from Oregon advocate Lorren Sandt. Click here to view the letter from advocates to the legislature requesting pause of the board’s activities and legislative oversight as the board works on its engagement process. Click here to view the drugs under consideration at each meeting, including treatments for obesity, multiple sclerosis, ulcerative colitis, psoriasis, ADHD, HIV, and diabetes.
We remain concerned that the Oregon PDAB has not made substantive changes to their patient and disability engagement strategies. It appears that the patient and disability communities will need to pursue legislative changes to accomplish more robust engagement.
Washington State
At the most recent November 18, 2024 meeting, the PDAB staff discussed its use of ICER Analytics and cost effectiveness as part of its deliberations, indicating an intent to reference QALYs and similar measures in its assessments. Click here to view more information about the meeting, its agenda and transcripts.
At the May 22, 2024 meeting, the Program on Regulation, Therapeutics, and Law (PORTAL) provided information to the Prescription Drug Affordability Board on “Considerations for Conducting Affordability Reviews,” including cost effectiveness. PORTAL is known to be aligned with ICER, which considers QALY-based cost effectiveness to be the gold standard. PORTAL has also in the past referenced the evLYG as a measure to consider in affordability reviews. PIPC is following the PDAB’s implementation in Washington State closely for potential use of discriminatory measures of effectiveness, especially now that its engagement with PORTAL is official and public. On September 18, the Board met and discussed its affordability review process, including the potential reference to premiums as a component of affordability reviews. Click here to view the schedule and review prior meetings.
PIPC submitted a comment letter to the Washington Prescription Drug Affordability Board (PDAB) highlighting the concerns of patients and people with disabilities related to the PDAB’s potential use of discriminatory cost effectiveness analyses. Click here to read the letter.
International News: What Happens in Countries Using QALYs and Cost-Based Thresholds?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- Australia. Restrictive coverage policies often times force patients with rare diseases to turn to crowdfunding and other means of raising funds while the government drags its feet on price negotiations.
- France. Despite the French High Authority for Health's (HAS) decision to expand access to a promising Alzheimer's treatment, patients still must jump through hoopsto gain access and the drug is still immediately available for most patients.
- South Korea. Cost-effectiveness and risk-sharing agreement policies are limiting spending on drugs for critical illnesses.
- United Kingdom. NICE says it was unable to reach a price agreement for a new breast cancer drug treatment, breaking a line of 21 positive recommendations for breast cancer therapies.
ICER's QALY-Based Study Topics
The Institute for Clinical Economic Review (ICER) conducts cost effectiveness studies for insurers using the cost-per-QALY methodology. ICER provides guidance on its website for patients and patient advocates to provide direct input related to their experiences with the disease. Click here to provide patient input. Click here to view the topics and deadlines.
- Multiple Sclerosis — SPMS: Draft Scoping Document available. Comments due Dec. 12, 2024.
- Special Assessment to Inform CMS Drug Price Negotiation: Research protocol available. Final report due March 4, 2024.
- Retinitis Pigmentosa: Revised Scoping Document, Research Protocol available. Public meeting: Apr. 2025.
- Acute Pain: Revised Scoping Document, Research Protocol, Model Analysis Plan 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: Evidence Report available. Final report to be published Dec. 16, 2024.
Upcoming Events and Webinars
PCORI Board of Governors Meeting
December 10, 2024
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.
