In this week's edition...
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— PIPC Submits Letter on Oregon PDAB's Affordability Review Activities. Click here to read the letter.
— PIPC Submits Comments on Maryland PDAB Cost Review Study. Click here to read the letter.
— PIPC Submits Comments on ICER Assessment of COPD. Click here to read the letter.
— CMS Releases Draft Guidance for Medicare Drug Negotiation Program. See details below.
— View PCORI’s April Panel Meetings, Get PCORI Updates. See details below.
— Chairman Tony Coelho's Statement on Final Passage of H.R. 485. See details below.
— Emerging Threats in States for Use of Discriminatory Metrics. See details below.
— What Happens in Countries Using QALYs and Cost-Based Thresholds? See details below.
— ICER's QALY-Based Study Topics. See details below.
— Upcoming Events and Webinars. See details below.
— AHRQ Effective Program Updates. See details below.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— PIPC Submits Letter on Oregon PDAB's Affordability Review Activities. Click here to read the letter.
— PIPC Submits Comments on Maryland PDAB Cost Review Study. Click here to read the letter.
— PIPC Submits Comments on ICER Assessment of COPD. Click here to read the letter.
— CMS Releases Draft Guidance for Medicare Drug Negotiation Program. See details below.
— View PCORI’s April Panel Meetings, Get PCORI Updates. See details below.
— Chairman Tony Coelho's Statement on Final Passage of H.R. 485. See details below.
— Emerging Threats in States for Use of Discriminatory Metrics. See details below.
— What Happens in Countries Using QALYs and Cost-Based Thresholds? See details below.
— ICER's QALY-Based Study Topics. See details below.
— Upcoming Events and Webinars. See details below.
— AHRQ Effective Program Updates. See details below.
What We're Reading
1. 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. PIPC Submits Letter on Oregon PDAB's Affordability Review Activities. 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.
3. PIPC Submits Comments on Maryland PDAB Cost Review Study. 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.
4. PIPC Submits Comments on ICER Assessment of COPD. PIPC recently submitted a comment letter on ICER's assessment of chronic obstructive pulmonary disease (COPD). The letter criticizes ICER for using data sources that do not accurately capture the reality for COPD patients in the U.S. ICER also continues to use the discriminatory QALY and also fails to capture the heterogenous nature of COPD in its assessment.
"If ICER is to take seriously its role of informing health policy decision makers about the value of new therapies, it needs to move away from the assumption that all patients are the same," wrote PIPC Chairman Tony Coelho. "...It is important that ICER accurately capture the value of new treatments, as segments of the patient population still desperately need new options." Click here to read the letter.
5. 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.
6. View PCORI’s April Panel Meetings, Get PCORI Updates. PCORI's multi-stakeholder advisory panels are convening in April 2024 for their Spring 2024 meetings. These research advisory panels are an important way we bring voices from across the healthcare community into our work. They focus on clinical effectiveness and decision science, clinical trials, healthcare delivery and disparities research, patient engagement, and rare diseases. Members of the public are invited to tune in to the meetings via webinar/teleconference. Click here to learn more and register.
PCORI also has evidence updates available for multiple sclerosis, type 2 diabetes, stroke and urinary incontinence. Click here to view. Learn more about upcoming PCORI funding announcements on May 7, 2024 related to rare disease, substance use, pragmatic studies, science of engagement and methods here.
7. 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
Colorado passed legislation in 2021 creating a Prescription Drug Affordability Review Board. Without a bar on using QALYs in the affordability review, Colorado has engaged with entities such as the Program on Regulation, Therapeutics, and Law (PORTAL), an entity that has expressed support for the use of measures including QALYs and the evLYG. Recently, the Colorado Prescription Drug Affordability Board is currently working to determine an upper payment limit for Enbrel, a drug treating arthritis. On Friday April 26, 2024, the Board members engaged in a question and answer session with the Program on Regulation, Therapeutics, and Law (PORTAL) about the process for establishing an upper payment limit. The Board acknowledged that the statute creating the Board prohibited the use of quality-adjusted life years (QALYs) in establishing an upper payment limit. The discussion with PORTAL raised the potential for reference to international prices. One Board member noted that the QALY prohibition would prevent the Board from referencing prices in the United Kingdom. PORTAL referenced Germany as a country not using QALYs. The Board asked about Canada as well, seeming to realize more information is needed, which PORTAL offered to research and provide to the Board. PORTAL has historically supported the use of QALYs and evLYGs, therefore PIPC will be following Board’s methodology closely and its reliance on PORTAL’s recommendations. Click here to view the conversation. The discussion about foreign methods and potential to import QALYs begins at 2:41. Click here to register for the next PDAB meeting on June 7.
As background, concerns about the potential for discrimination were described in a 2023 letter to the Board from 16 patient and disability organizations. For additional background, in 2022 PIPC developed a White Paper on Germany’s process, highlighting its shortcomings for considering outcomes that matter to patients, available here. Additional information about Canada’s reference to QALYs is available here. More information about international methods is here.
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 April 29. Comments provided to the board at the March meeting may be viewed here and include concerns similar to those shared in Colorado related to concerns about health equity and access to care. Drugs potentially referred to the council for review include Biktarvy for HIV, Dupixent for eczema and asthma, Farxiga for chronic kidney disease (CKD), Jardiance or CKD and health failure, Ozempic for obesity, Skyriza for arthritis, psoriasis and Crohn’s disease, Trulicity for disabetes and heart disease, and Vyvance for ADHD. Register for the upcoming stakeholder meeting here. Learn more about the next May 20 Board meeting here. Written comments on the Board's pending decisions, or general comments regarding the Board's work, may be submitted to [email protected] (include in the subject line: May Board Meeting Comments).
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
At the recent Oregon meeting of the Prescription Drug Affordability Board, the board discussed the potential use of an upper payment limit for prescription drugs under review. The Executive Director of the board spoke extensively about concerns related to the board’s work, though did not directly address the concerns shared by patients related to the implications of the board’s work for patient access to care and out-of-pocket costs due to potential unintended consequences. The board discussion indicated the use of a firm to help with engagement. Yet, the survey instrument being used to learn about patient experiences was developed by the board staff and does not seem to have been informed by patient input. Concerns were also shared about the lack of attendance at recent patient listening sessions held in Oregon, at which 2-4 patients are reported to have attended. Nevertheless, September is the expected timeframe for recommendations to the legislature emanating from its affordability reviews despite little engagement from patients and people with disabilities. 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. The April meeting will be posted here.
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
Uncovering the True Cost of Healthcare
May 21, 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. PIPC Submits Letter on Oregon PDAB's Affordability Review Activities. 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.
3. PIPC Submits Comments on Maryland PDAB Cost Review Study. 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.
4. PIPC Submits Comments on ICER Assessment of COPD. PIPC recently submitted a comment letter on ICER's assessment of chronic obstructive pulmonary disease (COPD). The letter criticizes ICER for using data sources that do not accurately capture the reality for COPD patients in the U.S. ICER also continues to use the discriminatory QALY and also fails to capture the heterogenous nature of COPD in its assessment.
"If ICER is to take seriously its role of informing health policy decision makers about the value of new therapies, it needs to move away from the assumption that all patients are the same," wrote PIPC Chairman Tony Coelho. "...It is important that ICER accurately capture the value of new treatments, as segments of the patient population still desperately need new options." Click here to read the letter.
5. 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.
6. View PCORI’s April Panel Meetings, Get PCORI Updates. PCORI's multi-stakeholder advisory panels are convening in April 2024 for their Spring 2024 meetings. These research advisory panels are an important way we bring voices from across the healthcare community into our work. They focus on clinical effectiveness and decision science, clinical trials, healthcare delivery and disparities research, patient engagement, and rare diseases. Members of the public are invited to tune in to the meetings via webinar/teleconference. Click here to learn more and register.
PCORI also has evidence updates available for multiple sclerosis, type 2 diabetes, stroke and urinary incontinence. Click here to view. Learn more about upcoming PCORI funding announcements on May 7, 2024 related to rare disease, substance use, pragmatic studies, science of engagement and methods here.
7. 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
Colorado passed legislation in 2021 creating a Prescription Drug Affordability Review Board. Without a bar on using QALYs in the affordability review, Colorado has engaged with entities such as the Program on Regulation, Therapeutics, and Law (PORTAL), an entity that has expressed support for the use of measures including QALYs and the evLYG. Recently, the Colorado Prescription Drug Affordability Board is currently working to determine an upper payment limit for Enbrel, a drug treating arthritis. On Friday April 26, 2024, the Board members engaged in a question and answer session with the Program on Regulation, Therapeutics, and Law (PORTAL) about the process for establishing an upper payment limit. The Board acknowledged that the statute creating the Board prohibited the use of quality-adjusted life years (QALYs) in establishing an upper payment limit. The discussion with PORTAL raised the potential for reference to international prices. One Board member noted that the QALY prohibition would prevent the Board from referencing prices in the United Kingdom. PORTAL referenced Germany as a country not using QALYs. The Board asked about Canada as well, seeming to realize more information is needed, which PORTAL offered to research and provide to the Board. PORTAL has historically supported the use of QALYs and evLYGs, therefore PIPC will be following Board’s methodology closely and its reliance on PORTAL’s recommendations. Click here to view the conversation. The discussion about foreign methods and potential to import QALYs begins at 2:41. Click here to register for the next PDAB meeting on June 7.
As background, concerns about the potential for discrimination were described in a 2023 letter to the Board from 16 patient and disability organizations. For additional background, in 2022 PIPC developed a White Paper on Germany’s process, highlighting its shortcomings for considering outcomes that matter to patients, available here. Additional information about Canada’s reference to QALYs is available here. More information about international methods is here.
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 April 29. Comments provided to the board at the March meeting may be viewed here and include concerns similar to those shared in Colorado related to concerns about health equity and access to care. Drugs potentially referred to the council for review include Biktarvy for HIV, Dupixent for eczema and asthma, Farxiga for chronic kidney disease (CKD), Jardiance or CKD and health failure, Ozempic for obesity, Skyriza for arthritis, psoriasis and Crohn’s disease, Trulicity for disabetes and heart disease, and Vyvance for ADHD. Register for the upcoming stakeholder meeting here. Learn more about the next May 20 Board meeting here. Written comments on the Board's pending decisions, or general comments regarding the Board's work, may be submitted to [email protected] (include in the subject line: May Board Meeting Comments).
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
At the recent Oregon meeting of the Prescription Drug Affordability Board, the board discussed the potential use of an upper payment limit for prescription drugs under review. The Executive Director of the board spoke extensively about concerns related to the board’s work, though did not directly address the concerns shared by patients related to the implications of the board’s work for patient access to care and out-of-pocket costs due to potential unintended consequences. The board discussion indicated the use of a firm to help with engagement. Yet, the survey instrument being used to learn about patient experiences was developed by the board staff and does not seem to have been informed by patient input. Concerns were also shared about the lack of attendance at recent patient listening sessions held in Oregon, at which 2-4 patients are reported to have attended. Nevertheless, September is the expected timeframe for recommendations to the legislature emanating from its affordability reviews despite little engagement from patients and people with disabilities. 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. The April meeting will be posted here.
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: A cancer patient in Australia is raising awareness about the need for better access to lifesaving medicines.
- Canada: Despite suffering from aggressive brain cancer, patients are being denied access to treatments that have proven to prolong survival and delay recurrence.
- Netherlands: Patients with cystic fibrosis are struggling to gain access to treatment due to arcane, one-size-fits-all qualifications.
- New Zealand: Pharmac's $1.7 billion funding boost will not cover lifesaving medications as patients continue their push for "radical" reform.
- 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
Uncovering the True Cost of Healthcare
May 21, 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.
