In this week's edition...
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— PCORI Annual Meeting October 22-23, 2024. See details below.
— PIPC Submits Comments on Maryland PDAB Cost Review Study. Click here to read the letter.
— Comment on PCORI Systematic Reviews by July 11, 2024. See details below.
— PCORI Engagement Awards opening July 17! Click here to learn more.
— Join Ensuring Access Through Collaborative Health. Click here to be included in the meeting.
— Building the Data Capacity for Patient-Centered Outcomes Research. Click here to read the report.
— CMS Releases Draft Guidance for Medicare Drug Negotiation Program. See details below.
— Chairman Tony Coelho's Statement on Final Passage of H.R. 485. See details below.
— PIPC Submits Comments to Colorado PDAB. Click here to read the letter.
— 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.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— PCORI Annual Meeting October 22-23, 2024. See details below.
— PIPC Submits Comments on Maryland PDAB Cost Review Study. Click here to read the letter.
— Comment on PCORI Systematic Reviews by July 11, 2024. See details below.
— PCORI Engagement Awards opening July 17! Click here to learn more.
— Join Ensuring Access Through Collaborative Health. Click here to be included in the meeting.
— Building the Data Capacity for Patient-Centered Outcomes Research. Click here to read the report.
— CMS Releases Draft Guidance for Medicare Drug Negotiation Program. See details below.
— Chairman Tony Coelho's Statement on Final Passage of H.R. 485. See details below.
— PIPC Submits Comments to Colorado PDAB. Click here to read the letter.
— 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. 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.
2. 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, 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.
3. Comment on PCORI Systematic Reviews by July 11, 2024. From May 28 through July 11, 2024, PCORI is seeking comments on three draft reports of audio-based care for managing mental health, diabetes, and other chronic conditions. PCORI is funding the systematic reviews to improve understanding of the evidence base on this topic and to inform the broader healthcare community and our future research investments. Click here to learn more.
4. PCORI Engagement Awards opening July 17! PCORI's Online System will open 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.
5. Join Ensuring Access Through Collaborative Health. The EACH Coalition will hold its next meeting on June 11 at 1pm EST. The coalition responds to states creating Prescription Drug Affordability Boards that are empowered to take actions that could limit patient access to prescription drugs, providing organizational support to engage. Click here to be included in the meeting.
6. Building the Data Capacity for Patient-Centered Outcomes Research. The Office of the Assistant Secretary for Planning and Evaluation at HHS published its annual report on building data capacity for patient-centered outcomes research, The highlights the accomplishments of 36 multi-agency projects that supports the four goals of the strategic plan: Data Capacity for National Health Priorities; Data Standards and Linkages for Longitudinal Research; Technology Solutions to Advance Research; and Person-Centeredness, Inclusion, and Equity. Click here to read the report.
7. CMS Releases Draft Guidance for Medicare Drug Negotiation Program. CMS released guidance for the Medicare Drug Negotiation Program that also discusses the agency’s use of value assessments, as well as its engagement process. Comments are due on July 2, 2024. In the guidance, CMS makes a strong, positive statement of commitment to “learning from, collaborating with, and engaging the public, including patients, consumer advocates, health and data experts, and pharmaceutical supply chain entities in the policy-making process.” The agency also expressed support for collecting real-world data and engaging patients related to its work to identify therapeutic alternatives.
Additionally, the new guidance states that CMS will “review cost-effectiveness measures used in studies relevant to a selected drug to determine whether the measure used is permitted in accordance with section 1194(e)(2), as well as with section 1182(e) of Title XI of the Act.” Yet, the guidance narrowly references the IRA’s statutory language, stating that the Negotiation Program will not use “information that treats extending the life of individuals in these populations as of lower value,” leaving out the ACA language barring similar measures that "discounts the value of a life because of an individual’s disability.”
PIPC looks forward to providing CMS with comments to ensure that the final guidance assures patients and people with disabilities that its implementation of the program will not only be aligned with current law governing the use of value assessment, but also provides concrete steps the agency will take to facilitate meaningful engagement of patients and people with disabilities and rely on high quality sources of evidence. View the guidance here.
8. Chairman Tony Coelho's Statement on Final Passage of H.R. 485. In response to the final passage of H.R. 485, PIPC Chairman Tony Coelho is pleased to share this statement.
It is disappointing that a bill to simply extend current law protections to all people with disabilities did not pass the House unanimously and instead passed on a partisan vote. Yet, it is important to note that Republicans and Democrats both stated support for the current law protections against use of QALYs & similar measures that devalue people with disabilities, and they support banning their use more broadly. This is the critical issue for those of us with disabilities. We expect both parties to keep that view and work with the disability community to act on the recommendations of the National Council on Disability, the independent federal agency that identified for policymakers how QALYs & similar measures discriminate and called for a consistent policy across federal programs to address it.
For additional information, you may click here to view the CCD letter supporting H.R. 485, click here to view the statement from supporting organizations and click here to view the letter from supportive organizations. You may view the White House Statement of Administration Policy here, which opposed how the bill would be paid for, while underscoring continued Democratic support for the current law barring QALYs and similar measures.
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). Click here to register for the next PDAB meeting on June 7.
Connecticut
The Governor’s bill No. 5054 would establish a Prescription Drug Affordability Board with authority to "(1) explore strategies to reduce out-of-pocket drug costs to consumers while supporting innovations in biotechnology and scientific discovery; (2) study the prescription drug supply chain and pharmaceutical pricing strategies to identify opportunities for consumer savings; (3) monitor prescription drug prices in the state; (4) promote innovative strategies for the use of more affordable drugs; and (5) recommend a range of options of prescription drug cost affordability tools to the executive director of the Office of Health Strategy.” The Board is to report on “cost effectiveness evaluations” among the topics to be covered, but is not precluded from referencing evaluations using quality-adjusted life years (QALYs) and similar measures. The board members "shall have an advanced degree and experience or expertise in health care economics, health services research, pharmoeconomics, pharmacology or clinical medicine. At least one such member shall have direct experience with consumer advocacy and health equity.” While one board member is to have experience with consumer advocacy and health equity, it is not clearly stated that the person represent the patient perspective, nor does the legislation create an advisory process for patients and people with disabilities. The bill was discussed in a public hearing on February 23, 2024.
View the letter opposing the bill from Boomer Esiason Foundation here. You may view comments from International Cancer Advocacy Network here and the Value Our Care Coalition here. Contact information for the committee is here.
Illinois
Legislation was introduced in the Illinois House (HB 4472) on January 17, 2024, creating a Health Care Availability and Access Board, using a model aligned with the NASHP template legislation for creation of Prescription Drug Affordability Boards. An amended version of the legislation was referred to the legislative committees on 2/28/24. The amended version continues to include 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. It was referred to the Health Care Availability & Accessibility Committee in the Illinois General Assembly. The lead sponsors are Representative Nabeela Syed (D) and Representative Emanuel "Chris" Welch (D).
Maryland
The Maryland Prescription Drug Affordability Board’s stakeholder council last met on May 20. Six drugs were selected for cost reviews for drugs treating conditions such as type 2 diabetes, obesity, psoriasis, arthritis and Crohn's disease, eczema and asthma. Written comments sent to the Board are available here. The Stakeholder Council will next meet on June 24 at 2pm - register here to join. Written comments on the Board’s cost reviews may be submitted over the next 60 days to [email protected]. 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.
Massachusetts
The Massachusetts Senate Ways and Means Committees was referred the “PACT Act”, reported favorably on October 30, 2023 by the Joint Committee on Health Care Financing. The legislation includes a provision calling for the health policy commission not to base its determinations on measures such as QALYs. The Senate rejected an amendment (#19) pushed by Massachusetts advocates calling for transparency related to the use of cost effectiveness analyses by the commission, as well as increased requirements for stakeholder engagement. The Senate passed the bill on November 15, 2023. Massachusetts advocates will work with the House toward language that more clearly provides for public transparency of the evidence under consideration and opportunities for engagement from patients and people with disabilities.
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
PIPC recently submitted a letter to the Oregon Prescription Drug Affordability Board (PDAB) on the Board's ongoing affordability review activities. The letter expresses concern that the legislative provisions governing the use of quality-adjusted-life-years (QALY) metric and similar measures in legislation that established the board may be interpreted narrowly. PIPC also points out that recently finalized rules governing Section 504 of the Rehabilitation Act clarifies that recipients of federal financial assistance, including Medicaid programs, may not use discriminatory metrics such as QALYs. 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. Register here for the next meeting on June 26.
Washington State
At its December, 2023 meeting, the Washington State Prescription Drug Affordability Board acknowledged the limitation in its statute on using quality adjusted life years (QALYs) to establish an upper payment limit, yet also raised the possibility of contracting with the Institute for Clinical and Economic Review (ICER) as a consultant to the Board for its methodologies and analysis (1:03:25). At its October, 2023 meeting, the Board discussed partnering the Program on Regulation, Therapeutics, and Law (PORTAL), acknowledging its education of the Colorado Board and collaboration with the National Association of State Health Policy (NASHP) in development of the legislation creating Washington’s PDAB and supporting materials, further underscoring the potential influence of entities that view QALYs and evLYG measures as useful to the affordability review process (47:55). PIPC is following the PDAB’s implementation in Washington State closely for potential use of discriminatory measures of effectiveness.
According to the website, "The Washington State Prescription Drug Affordability Board (Board) is seeking unpaid volunteer members for its Advisory Group. The Advisory Group serves at the direction of the Board. The goal of the Advisory Group is to provide guidance to the Board on the different components of drug affordability in Washington. The Advisory Group members will investigate each drug selected by the Board and will provide a written report to the Board with their findings as to the drug’s affordability. The application is open through July 1, 2024."
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
Ensuring Access Through Collaborative Health
June 11, 2024
Click here to view.
PCORI Board of Governors Meeting
June 17-18, 2024
Click here to view.
AHRQ Effective Program Updates
Rapid Evidence Product: Prevention in Adults of Transmission of Infection With Multidrug-Resistant Organisms. Click here to view.
Systematic Review: ADHD Diagnosis and Treatment in Children and Adolescents. Click here to view.
Research Protocol: Fiber Intake and Laxation Outcomes. Click here to view.
Rapid Evidence Product: Adverse Events Associated With COVID-19 Pharmaceutical Treatments. Click here to view.
Rapid Evidence Product: Computerized Clinical Decision Support To Prevent Medication Errors and Adverse Drug Events. Click here to view.
Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder: 2024 Update of the Evidence Base for the PTSD Trials Standardized Data Repository. Click here to view.
Systematic Review: Healthcare Algorithms on Racial and Ethnic Disparities in Health and Healthcare. Click here to view.
Technical Brief: Measuring Healthcare Organization Characteristics in Cancer Care Delivery Research. Click here to view.
Medical Journal Articles
Sharing Information Across Patient Subgroups to Draw Conclusions from Sparse Treatment Networks. Click here to read the article.
Studying How Patient Engagement Influences Research: A Mixed Methods Study. Click here to read the article.
Principles for Stakeholder Engagement in Observational Health Research. Click here to read the article.
Policy Symposium: Ensuring Equity in Implementation of Ira Drug Price Negotiations: Convening Proceedings Report. Click here to read the report.
Comparative Effectiveness Research Using Claims Data: Meticulous Methods Don’t Solve Old Problems. Click here to read the article.
The Impact of The House Proposed IRA Expansion on the US Biopharma Ecosystem. Click here to view the report.
1. 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.
2. 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, 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.
3. Comment on PCORI Systematic Reviews by July 11, 2024. From May 28 through July 11, 2024, PCORI is seeking comments on three draft reports of audio-based care for managing mental health, diabetes, and other chronic conditions. PCORI is funding the systematic reviews to improve understanding of the evidence base on this topic and to inform the broader healthcare community and our future research investments. Click here to learn more.
4. PCORI Engagement Awards opening July 17! PCORI's Online System will open 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.
5. Join Ensuring Access Through Collaborative Health. The EACH Coalition will hold its next meeting on June 11 at 1pm EST. The coalition responds to states creating Prescription Drug Affordability Boards that are empowered to take actions that could limit patient access to prescription drugs, providing organizational support to engage. Click here to be included in the meeting.
6. Building the Data Capacity for Patient-Centered Outcomes Research. The Office of the Assistant Secretary for Planning and Evaluation at HHS published its annual report on building data capacity for patient-centered outcomes research, The highlights the accomplishments of 36 multi-agency projects that supports the four goals of the strategic plan: Data Capacity for National Health Priorities; Data Standards and Linkages for Longitudinal Research; Technology Solutions to Advance Research; and Person-Centeredness, Inclusion, and Equity. Click here to read the report.
7. CMS Releases Draft Guidance for Medicare Drug Negotiation Program. CMS released guidance for the Medicare Drug Negotiation Program that also discusses the agency’s use of value assessments, as well as its engagement process. Comments are due on July 2, 2024. In the guidance, CMS makes a strong, positive statement of commitment to “learning from, collaborating with, and engaging the public, including patients, consumer advocates, health and data experts, and pharmaceutical supply chain entities in the policy-making process.” The agency also expressed support for collecting real-world data and engaging patients related to its work to identify therapeutic alternatives.
Additionally, the new guidance states that CMS will “review cost-effectiveness measures used in studies relevant to a selected drug to determine whether the measure used is permitted in accordance with section 1194(e)(2), as well as with section 1182(e) of Title XI of the Act.” Yet, the guidance narrowly references the IRA’s statutory language, stating that the Negotiation Program will not use “information that treats extending the life of individuals in these populations as of lower value,” leaving out the ACA language barring similar measures that "discounts the value of a life because of an individual’s disability.”
PIPC looks forward to providing CMS with comments to ensure that the final guidance assures patients and people with disabilities that its implementation of the program will not only be aligned with current law governing the use of value assessment, but also provides concrete steps the agency will take to facilitate meaningful engagement of patients and people with disabilities and rely on high quality sources of evidence. View the guidance here.
8. Chairman Tony Coelho's Statement on Final Passage of H.R. 485. In response to the final passage of H.R. 485, PIPC Chairman Tony Coelho is pleased to share this statement.
It is disappointing that a bill to simply extend current law protections to all people with disabilities did not pass the House unanimously and instead passed on a partisan vote. Yet, it is important to note that Republicans and Democrats both stated support for the current law protections against use of QALYs & similar measures that devalue people with disabilities, and they support banning their use more broadly. This is the critical issue for those of us with disabilities. We expect both parties to keep that view and work with the disability community to act on the recommendations of the National Council on Disability, the independent federal agency that identified for policymakers how QALYs & similar measures discriminate and called for a consistent policy across federal programs to address it.
For additional information, you may click here to view the CCD letter supporting H.R. 485, click here to view the statement from supporting organizations and click here to view the letter from supportive organizations. You may view the White House Statement of Administration Policy here, which opposed how the bill would be paid for, while underscoring continued Democratic support for the current law barring QALYs and similar measures.
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). Click here to register for the next PDAB meeting on June 7.
Connecticut
The Governor’s bill No. 5054 would establish a Prescription Drug Affordability Board with authority to "(1) explore strategies to reduce out-of-pocket drug costs to consumers while supporting innovations in biotechnology and scientific discovery; (2) study the prescription drug supply chain and pharmaceutical pricing strategies to identify opportunities for consumer savings; (3) monitor prescription drug prices in the state; (4) promote innovative strategies for the use of more affordable drugs; and (5) recommend a range of options of prescription drug cost affordability tools to the executive director of the Office of Health Strategy.” The Board is to report on “cost effectiveness evaluations” among the topics to be covered, but is not precluded from referencing evaluations using quality-adjusted life years (QALYs) and similar measures. The board members "shall have an advanced degree and experience or expertise in health care economics, health services research, pharmoeconomics, pharmacology or clinical medicine. At least one such member shall have direct experience with consumer advocacy and health equity.” While one board member is to have experience with consumer advocacy and health equity, it is not clearly stated that the person represent the patient perspective, nor does the legislation create an advisory process for patients and people with disabilities. The bill was discussed in a public hearing on February 23, 2024.
View the letter opposing the bill from Boomer Esiason Foundation here. You may view comments from International Cancer Advocacy Network here and the Value Our Care Coalition here. Contact information for the committee is here.
Illinois
Legislation was introduced in the Illinois House (HB 4472) on January 17, 2024, creating a Health Care Availability and Access Board, using a model aligned with the NASHP template legislation for creation of Prescription Drug Affordability Boards. An amended version of the legislation was referred to the legislative committees on 2/28/24. The amended version continues to include 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. It was referred to the Health Care Availability & Accessibility Committee in the Illinois General Assembly. The lead sponsors are Representative Nabeela Syed (D) and Representative Emanuel "Chris" Welch (D).
Maryland
The Maryland Prescription Drug Affordability Board’s stakeholder council last met on May 20. Six drugs were selected for cost reviews for drugs treating conditions such as type 2 diabetes, obesity, psoriasis, arthritis and Crohn's disease, eczema and asthma. Written comments sent to the Board are available here. The Stakeholder Council will next meet on June 24 at 2pm - register here to join. Written comments on the Board’s cost reviews may be submitted over the next 60 days to [email protected]. 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.
Massachusetts
The Massachusetts Senate Ways and Means Committees was referred the “PACT Act”, reported favorably on October 30, 2023 by the Joint Committee on Health Care Financing. The legislation includes a provision calling for the health policy commission not to base its determinations on measures such as QALYs. The Senate rejected an amendment (#19) pushed by Massachusetts advocates calling for transparency related to the use of cost effectiveness analyses by the commission, as well as increased requirements for stakeholder engagement. The Senate passed the bill on November 15, 2023. Massachusetts advocates will work with the House toward language that more clearly provides for public transparency of the evidence under consideration and opportunities for engagement from patients and people with disabilities.
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
PIPC recently submitted a letter to the Oregon Prescription Drug Affordability Board (PDAB) on the Board's ongoing affordability review activities. The letter expresses concern that the legislative provisions governing the use of quality-adjusted-life-years (QALY) metric and similar measures in legislation that established the board may be interpreted narrowly. PIPC also points out that recently finalized rules governing Section 504 of the Rehabilitation Act clarifies that recipients of federal financial assistance, including Medicaid programs, may not use discriminatory metrics such as QALYs. 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. Register here for the next meeting on June 26.
Washington State
At its December, 2023 meeting, the Washington State Prescription Drug Affordability Board acknowledged the limitation in its statute on using quality adjusted life years (QALYs) to establish an upper payment limit, yet also raised the possibility of contracting with the Institute for Clinical and Economic Review (ICER) as a consultant to the Board for its methodologies and analysis (1:03:25). At its October, 2023 meeting, the Board discussed partnering the Program on Regulation, Therapeutics, and Law (PORTAL), acknowledging its education of the Colorado Board and collaboration with the National Association of State Health Policy (NASHP) in development of the legislation creating Washington’s PDAB and supporting materials, further underscoring the potential influence of entities that view QALYs and evLYG measures as useful to the affordability review process (47:55). PIPC is following the PDAB’s implementation in Washington State closely for potential use of discriminatory measures of effectiveness.
According to the website, "The Washington State Prescription Drug Affordability Board (Board) is seeking unpaid volunteer members for its Advisory Group. The Advisory Group serves at the direction of the Board. The goal of the Advisory Group is to provide guidance to the Board on the different components of drug affordability in Washington. The Advisory Group members will investigate each drug selected by the Board and will provide a written report to the Board with their findings as to the drug’s affordability. The application is open through July 1, 2024."
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: In the past, approximately 200 women with endometrial cancer were forced to pay hundreds of thousands out of pocket for treatment not covered by the PBS.
- Canada: Patients suffering from migraines must pay hundreds out of pocket for an injectable treatment that is not fully covered by provincial drug plans.
- Netherlands: Dozens of MS patients are being turned away for treatment across the country due to stretched resources.
- United Kingdom: NICE's new algorithm could lead to patients being denied life-extending medicines.
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.
- Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease: Draft Scoping Document available. Public meeting: November 2024
- Transthyretin Amyloidosis Cardiomyopathy: Revised Scoping Document, Research Protocol available. Public meeting: September 2024
- Myelodysplastic Syndrome: Draft Evidence Report available. Public meeting: July 2024.
- Chronic Obstructive Pulmonary Disease: Draft Evidence Report available. Public meeting: June 2024
- Post-Traumatic Stress Disorder: Evidence Report available. Main review: May 30, 2024.
Upcoming Events and Webinars
Ensuring Access Through Collaborative Health
June 11, 2024
Click here to view.
PCORI Board of Governors Meeting
June 17-18, 2024
Click here to view.
AHRQ Effective Program Updates
Rapid Evidence Product: Prevention in Adults of Transmission of Infection With Multidrug-Resistant Organisms. Click here to view.
Systematic Review: ADHD Diagnosis and Treatment in Children and Adolescents. Click here to view.
Research Protocol: Fiber Intake and Laxation Outcomes. Click here to view.
Rapid Evidence Product: Adverse Events Associated With COVID-19 Pharmaceutical Treatments. Click here to view.
Rapid Evidence Product: Computerized Clinical Decision Support To Prevent Medication Errors and Adverse Drug Events. Click here to view.
Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder: 2024 Update of the Evidence Base for the PTSD Trials Standardized Data Repository. Click here to view.
Systematic Review: Healthcare Algorithms on Racial and Ethnic Disparities in Health and Healthcare. Click here to view.
Technical Brief: Measuring Healthcare Organization Characteristics in Cancer Care Delivery Research. Click here to view.
Medical Journal Articles
Sharing Information Across Patient Subgroups to Draw Conclusions from Sparse Treatment Networks. Click here to read the article.
Studying How Patient Engagement Influences Research: A Mixed Methods Study. Click here to read the article.
Principles for Stakeholder Engagement in Observational Health Research. Click here to read the article.
Policy Symposium: Ensuring Equity in Implementation of Ira Drug Price Negotiations: Convening Proceedings Report. Click here to read the report.
Comparative Effectiveness Research Using Claims Data: Meticulous Methods Don’t Solve Old Problems. Click here to read the article.
The Impact of The House Proposed IRA Expansion on the US Biopharma Ecosystem. Click here to view the report.
