In this week's edition...
— Advancing H.R. 485, the Protecting Health Care for All Patients Act. See details below.
— 38 Organizations Comment on Maryland PDAB UPL Plan. Click here to view the letter.
— Register for Cancer Support Community's Second Annual Inflation Reduction Act (IRA) Stakeholder Summit. See details below.
— Register for AFPA National Policy & Advocacy Summit. See details below.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— NCD Releases Report on People with Disabilities in Clinical Trials. See details below.
— Medicare Drug Price Negotiation Program: CMS Announced Prices of First 10 Drugs. See details below.
— Aimed Alliance Publishes New Resource on PDAB & IRA Implications for Rare Disorders. Click here to view.
— PCORI Annual Meeting October 22-23, 2024. See details below.
— FDA’s CBER Hosting Patient Listening Session on Gene Therapy, September 20. See details below.
— PCORI Engagement Awards Now Open! 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.
— Advancing H.R. 485, the Protecting Health Care for All Patients Act. See details below.
— 38 Organizations Comment on Maryland PDAB UPL Plan. Click here to view the letter.
— Register for Cancer Support Community's Second Annual Inflation Reduction Act (IRA) Stakeholder Summit. See details below.
— Register for AFPA National Policy & Advocacy Summit. See details below.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— NCD Releases Report on People with Disabilities in Clinical Trials. See details below.
— Medicare Drug Price Negotiation Program: CMS Announced Prices of First 10 Drugs. See details below.
— Aimed Alliance Publishes New Resource on PDAB & IRA Implications for Rare Disorders. Click here to view.
— PCORI Annual Meeting October 22-23, 2024. See details below.
— FDA’s CBER Hosting Patient Listening Session on Gene Therapy, September 20. See details below.
— PCORI Engagement Awards Now Open! 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. 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.
2. 38 Organizations Comment on Maryland PDAB UPL Plan. 38 organizations representing patients and people with disabilities strongly urged the Maryland Prescription Drug Affordability Board (PDAB) to prioritize the perspectives of people whose care may be impacted by your decisions as it works to finalize a Plan of Action for Implementing the Process for Setting Upper Payment Limits. The groups expressed concerns with recommendations from academia to states implementing PDABs that are not centered on helping patients gain affordable access to the drugs that patients and doctors determine to be the most effective treatment. They recommended the Board develop a concrete plan to monitor and respond to potential increased use of utilization management strategies and adverse formulary placements for both selected drugs and their alternative treatments; improve the Board’s patient engagement practices and use of survey data; avoid the use of discriminatory value assessments, and; avoid reference to drug prices in other countries. Click here to view the letter.
3. Register for Cancer Support Community's Second Annual Inflation Reduction Act (IRA) Stakeholder Summit. Cancer Support Community (CSC) will host its IRA Stakeholder Engagement Summit in Washington, D.C., on Tuesday, September 24, 2024. PIPC Executive Director Sara van Geertruyden will join CSC, patient and caregiver advocates, policy experts, academics, and thought leaders for a discussion on the need to place patients and caregivers at the center ongoing conversations around implementation of the Medicare Drug Price Negotiation Program. Registration closes on Friday, September 13 at 11:59 EST. Click here to register.
4. Register for AFPA National Policy & Advocacy Summit. The Alliance for Patient Access is hosting the 9th annual National Policy & Advocacy Summit on September 24 in Washington, DC. PIPC Deputy Director Thayer Roberts will join policy experts, patient advocates, health care providers and decision-makers to discuss federal and state policies, patient cost-sharing, the effects of the IRA, and more. Click here to register.
5. 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.
6. NCD Releases Report on People with Disabilities in Clinical Trials. A new report from the National Council on Disability reveals that people with disabilities are often excluded from clinical trials due to outdated or unenforced federal rules. The report criticizes agencies like the FDA and NIH for allowing these exclusions, which contribute to poorer health outcomes for disabled individuals. The Council recommends increased oversight, updated eligibility criteria, and enhanced funding to ensure more inclusive trials and better health equity. Click here to learn more.
7. 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.
8. 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. Click here to view information about scholarships provided to those wanting to attend. Click here to learn more.
9. Aimed Alliance Publishes New Resource on PDAB & IRA Implications for Rare Disorders. On August 20, 2024, Aimed Alliance released a new fact sheet on, “IRA, PDABs, & Orphan Drugs: How Price Setting Mechanisms May Harm Patients with Rare Disorders.” This new resource reviews how the Inflation Reduction Act and Prescription Drug Affordability Boards may impact patients with rare disorders in terms of access to treatments as well as the investment in and research into treatments for these conditions. The resource highlights considerations for state and federal legislators that can help mitigate the impact of these programs on patients with rare disorders. The Aimed Alliance also has developed resources for all advocates to better understand the role of PDABs in states. Click here to view.
10. FDA’s CBER Hosting Patient Listening Session on Gene Therapy, September 20. The FDA Center for Biologics Evaluation and Research (CBER) is hosting a public patient listening meeting and opening a docket to better understand patient and care partner perspectives on safety considerations and long-term follow-up for approved gene therapy treatments for rare diseases. FDA states that the objectives of this listening meeting are to hear from patients and care partners on their perspectives on short-term and long-term risks of approved gene therapy products, to learn what types of information patients would find helpful in their decision-making when considering gene therapy, to learn about their considerations and experience with participating in long-term studies after receiving a gene therapy, and to help inform patient-centered protocols for long-term studies that include gene therapy outcomes. The virtual meeting will be held on September 20, 2024 from 11:00 AM - 4:30 PM ET. Click here for more information and to register!
11. PCORI Engagement Awards Are Now Open! PCORI's Online System opened on July 17 for the submission of Letters of Intent (LOI) for four PCORI Engagement Award Program funding announcements under the Fall 2024 Cycle. These awards are for Capacity Building, Dissemination Initiative, Stakeholder Convening Support, and Building Capacity for Small Organizations to Engage in Patient-Centered Comparative Clinical Effectiveness Research (CER) The PCORI Engagement Award Program is intended to bring more patients, caregivers, clinicians and other healthcare stakeholders into the research process. The goal is to support projects that will build a community better able to participate in patient-centered comparative clinical effectiveness research (CER) and serve as channels to disseminate study results. This is central to PCORI’s mission to fund useful CER that will help patients and those who care for them make better-informed healthcare decisions. 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
The next meeting of the Colorado Prescription Drug Affordability Board is September 6. You may register to participate here.
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).
Maryland
The Maryland Prescription Drug Affordability Board’s stakeholder council last met on July 22. Click here to register for today's Stakeholder Council meeting. Six Six drugs have been selected for cost reviews for drugs treating conditions such as type 2 diabetes, obesity, psoriasis, arthritis and Crohn's disease, eczema and asthma. On May 13, PIPC submitted a comment letter to the Maryland Prescription Drug Affordability Board (PDAB) on the Board's ongoing Cost Review Study process. 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 also 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. Click here to read the letter. Click here to learn more about the the Draft Upper Payment Limit Action Plan and here to read the letter from 38 organizations commenting on it
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
On July 24, 2024, the Oregon Prescription Drug Affordability Board (PDAB) held seven panels related to upper payment limits (UPLs), with each panel addressing 1-2 key questions from the Board. Click here to view the summary from Aimed Alliance.
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.
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. Click here for information about the next meeting on September 18.
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
HHS Section 504: Implications for Health Care Organizations and Teams
September 4, 2024
Click here to view.
PCORI Board of Governors Meeting
September 10-11, 2024
Click here to view.
PCORI Annual Meeting
October 22-23, 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. 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.
2. 38 Organizations Comment on Maryland PDAB UPL Plan. 38 organizations representing patients and people with disabilities strongly urged the Maryland Prescription Drug Affordability Board (PDAB) to prioritize the perspectives of people whose care may be impacted by your decisions as it works to finalize a Plan of Action for Implementing the Process for Setting Upper Payment Limits. The groups expressed concerns with recommendations from academia to states implementing PDABs that are not centered on helping patients gain affordable access to the drugs that patients and doctors determine to be the most effective treatment. They recommended the Board develop a concrete plan to monitor and respond to potential increased use of utilization management strategies and adverse formulary placements for both selected drugs and their alternative treatments; improve the Board’s patient engagement practices and use of survey data; avoid the use of discriminatory value assessments, and; avoid reference to drug prices in other countries. Click here to view the letter.
3. Register for Cancer Support Community's Second Annual Inflation Reduction Act (IRA) Stakeholder Summit. Cancer Support Community (CSC) will host its IRA Stakeholder Engagement Summit in Washington, D.C., on Tuesday, September 24, 2024. PIPC Executive Director Sara van Geertruyden will join CSC, patient and caregiver advocates, policy experts, academics, and thought leaders for a discussion on the need to place patients and caregivers at the center ongoing conversations around implementation of the Medicare Drug Price Negotiation Program. Registration closes on Friday, September 13 at 11:59 EST. Click here to register.
4. Register for AFPA National Policy & Advocacy Summit. The Alliance for Patient Access is hosting the 9th annual National Policy & Advocacy Summit on September 24 in Washington, DC. PIPC Deputy Director Thayer Roberts will join policy experts, patient advocates, health care providers and decision-makers to discuss federal and state policies, patient cost-sharing, the effects of the IRA, and more. Click here to register.
5. 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.
6. NCD Releases Report on People with Disabilities in Clinical Trials. A new report from the National Council on Disability reveals that people with disabilities are often excluded from clinical trials due to outdated or unenforced federal rules. The report criticizes agencies like the FDA and NIH for allowing these exclusions, which contribute to poorer health outcomes for disabled individuals. The Council recommends increased oversight, updated eligibility criteria, and enhanced funding to ensure more inclusive trials and better health equity. Click here to learn more.
7. 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.
8. 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. Click here to view information about scholarships provided to those wanting to attend. Click here to learn more.
9. Aimed Alliance Publishes New Resource on PDAB & IRA Implications for Rare Disorders. On August 20, 2024, Aimed Alliance released a new fact sheet on, “IRA, PDABs, & Orphan Drugs: How Price Setting Mechanisms May Harm Patients with Rare Disorders.” This new resource reviews how the Inflation Reduction Act and Prescription Drug Affordability Boards may impact patients with rare disorders in terms of access to treatments as well as the investment in and research into treatments for these conditions. The resource highlights considerations for state and federal legislators that can help mitigate the impact of these programs on patients with rare disorders. The Aimed Alliance also has developed resources for all advocates to better understand the role of PDABs in states. Click here to view.
10. FDA’s CBER Hosting Patient Listening Session on Gene Therapy, September 20. The FDA Center for Biologics Evaluation and Research (CBER) is hosting a public patient listening meeting and opening a docket to better understand patient and care partner perspectives on safety considerations and long-term follow-up for approved gene therapy treatments for rare diseases. FDA states that the objectives of this listening meeting are to hear from patients and care partners on their perspectives on short-term and long-term risks of approved gene therapy products, to learn what types of information patients would find helpful in their decision-making when considering gene therapy, to learn about their considerations and experience with participating in long-term studies after receiving a gene therapy, and to help inform patient-centered protocols for long-term studies that include gene therapy outcomes. The virtual meeting will be held on September 20, 2024 from 11:00 AM - 4:30 PM ET. Click here for more information and to register!
11. PCORI Engagement Awards Are Now Open! PCORI's Online System opened on July 17 for the submission of Letters of Intent (LOI) for four PCORI Engagement Award Program funding announcements under the Fall 2024 Cycle. These awards are for Capacity Building, Dissemination Initiative, Stakeholder Convening Support, and Building Capacity for Small Organizations to Engage in Patient-Centered Comparative Clinical Effectiveness Research (CER) The PCORI Engagement Award Program is intended to bring more patients, caregivers, clinicians and other healthcare stakeholders into the research process. The goal is to support projects that will build a community better able to participate in patient-centered comparative clinical effectiveness research (CER) and serve as channels to disseminate study results. This is central to PCORI’s mission to fund useful CER that will help patients and those who care for them make better-informed healthcare decisions. 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
The next meeting of the Colorado Prescription Drug Affordability Board is September 6. You may register to participate here.
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).
Maryland
The Maryland Prescription Drug Affordability Board’s stakeholder council last met on July 22. Click here to register for today's Stakeholder Council meeting. Six Six drugs have been selected for cost reviews for drugs treating conditions such as type 2 diabetes, obesity, psoriasis, arthritis and Crohn's disease, eczema and asthma. On May 13, PIPC submitted a comment letter to the Maryland Prescription Drug Affordability Board (PDAB) on the Board's ongoing Cost Review Study process. 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 also 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. Click here to read the letter. Click here to learn more about the the Draft Upper Payment Limit Action Plan and here to read the letter from 38 organizations commenting on it
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
On July 24, 2024, the Oregon Prescription Drug Affordability Board (PDAB) held seven panels related to upper payment limits (UPLs), with each panel addressing 1-2 key questions from the Board. Click here to view the summary from Aimed Alliance.
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.
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. Click here for information about the next meeting on September 18.
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: Absent significant reforms, rare disease treatments remain out of reach for most patients due to government price setting.
- Canada: Patients with Cystic Fibrosis struggle to get consistent and extended supplies of treatment without exorbitant out-of-pocket expenses.
- France: A breast cancer patient is litigating the Caisse Primaire d’Assurance Maladie (CPAM) for not reimbursing costs for her medication.
- New Zealand: Health New Zealand has blocked free early access to a cancer treatment, further delaying care for many 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.
- Acute Pain: Draft Scoping Document available. Public comments due: Aug. 16, 2024.
- Fair Access: Coverage Policies in 2024: Protocol available. Final report to be published December 19, 2024.
- Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease: Draft Scoping Document, Research Protocol, Model Analysis Plan available. Public meeting: November 2024
- Transthyretin Amyloidosis Cardiomyopathy: Draft Evidence Report available. Public meeting: Sept. 2024
- Myelodysplastic Syndrome: Final Evidence Report to be published in August 2024
Upcoming Events and Webinars
HHS Section 504: Implications for Health Care Organizations and Teams
September 4, 2024
Click here to view.
PCORI Board of Governors Meeting
September 10-11, 2024
Click here to view.
PCORI Annual Meeting
October 22-23, 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.
