— CMS Releases Draft Guidance for Medicare Drug Negotiation Program. See details below.
— Colorado PDAB Discusses Reference to Pricing in Foreign Countries. See details below.
— Growing Economic Consensus That How We Value Medicines Must Change. Click here to view the opinion.
— HHS’ Finalizes the Regulations on Section 504 of the Rehab Act Clarifying Rules for Nondiscrimination. Click here to view PIPC’s overview of the rule.
— Efforts to Collect Disability Data Ramp Up. See details below.
— PIPC Submits Comments on ICER Assessment of PTSD. Click here to read PIPC's letter.
— Chicago Sun Times: Prescription Drug Affordability Boards Do More Harm Than Good. Click here to view the opinion.
— 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.
— Colorado PDAB Discusses Reference to Pricing in Foreign Countries. See details below.
— Growing Economic Consensus That How We Value Medicines Must Change. Click here to view the opinion.
— HHS’ Finalizes the Regulations on Section 504 of the Rehab Act Clarifying Rules for Nondiscrimination. Click here to view PIPC’s overview of the rule.
— Efforts to Collect Disability Data Ramp Up. See details below.
— PIPC Submits Comments on ICER Assessment of PTSD. Click here to read PIPC's letter.
— Chicago Sun Times: Prescription Drug Affordability Boards Do More Harm Than Good. Click here to view the opinion.
— 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. CMS Releases Draft Guidance for Medicare Drug Negotiation Program.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.
2. Colorado PDAB Discusses Reference to Pricing in Foreign Countries. 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.
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
3. Growing Economic Consensus That How We Value Medicines Must Change. Richard Xie, a senior health economist at RA Capital and former director of research at the Innovation and Value Initiative, penned an opinion calling for the field of health economists to break from tradition. He stated, "The U.S. system is not perfect — payers and middlemen drive up out-of-pocket costs so some people aren’t actually able to afford appropriate care despite years of paying premiums, for example.” He argued against European-style cost effectiveness research, stating "Unfortunately for patients in these countries, however, value is estimated by conventional CEAs that ignore key factors reflecting how medicines benefit patients and society, and how the marketplace works in practice (including how branded medicines eventually become inexpensive generics).” Click here to view the opinion.
4. HHS’ Finalizes the Regulations on Section 504 of the Rehab Act Clarifying Rules for Nondiscrimination. The U.S. Department of Health and Human Services (HHS) announced the final rule governing Section 504 of the Rehabilitation Act, which protects the rights of people with disabilities in programs and activities receiving federal financial assistance. In response to the proposed rule, last year the Partnership to Improve Patient Care (PIPC) joined 100 organizations and individuals on a letter providing the agency with a series of recommendations. As described in the final rule, the new regulations would bar health care decisions made using measures that discount gains in life expectancy, which would likely include the quality-adjusted life year (QALYs) and the combined use of QALYs and equal value of life years gained (evLYG). HHS’ rule represents a critical step forward to protecting patients and people with disabilities and sends a strong message that we need better solutions for U.S. decision-making that don’t rely on the biased, outdated standards used by payers in the U.S. and other countries. PIPC expressed appreciation for the agency’s broad interpretation of what constitutes the discriminatory use of value assessment in its description of the rule. Click here to view PIPC’s overview of the rule.
5. Efforts to Collect Disability Data Ramp Up. Disability researchers and advocates continue to push for improved mechanisms for collecting data on people with disabilities to inform better care models. In response, the Centers for Medicare and Medicaid Innovation (CMMI) is advancing the option to collect disability data as part of the collection of Sociodemographic Data Elements in at least one of their innovation models, the Enhancing Oncology Model. Also, the Joint Commission, as a part of their new Health Equity Certificate, requires documentation of disability status and processes to provide disability accommodations. The Health Resources and Services Administration (HRSA) is not yet compliant with the collection of disability status as required by Section 4302 of the Affordable Care Act. The Disability Equity Collaborative has developed a toolkit below for health care organizations to use in their implementation of documenting disability status and accommodations in the electronic health questions. Click here to access the toolkit and share it with your colleagues.
6. PIPC Submits Comments on ICER Assessment of PTSD. PIPC recently submitted a comment letter on ICER's assessment of treatments for post-traumatic stress disorder (PTSD). The letter highlights several key flaws in ICER's model, including its use of discriminatory cost effectiveness models like the quality-adjusted-life-years (QALY) and equal value of life year gained (evLYG). ICER's assessment also falls short by failing to account for heterogeneity and instead focusing its reports on the "average" patient. Click here to read PIPC's letter.
7. Chicago Sun Times: Prescription Drug Affordability Boards Do More Harm Than Good. Jen Laws, President & CEO of the Community Access National Network, wrote an opinion for the Chicago Sun Times raising concerns about the impact of prescription drug affordability boards on equitable access to medications and the landscape of health care financing in favor of insurers, not patients. He stated, "Prescription drug affordability boards simply aren’t the catchy answer some Illinois legislators are being sold on. An upper limit payment is a direct threat to health equity efforts — leaving behind needy families and communities in favor of those who might be able to afford to go out of state or pay cash for a medication.” Click here to view the opinion.
8. 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.
9. 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).
Maine
A bill introduced in the Maine legislature, LD 1829, would create a Prescription Drug Affordability Board with 6 members. The board is not required to include patients or people with disabilities. The 12-member advisory council includes a consumer representative appointed by the Governor, but does not require patient and disability participation. The board would be authorized to establish upper payment limits and to reference Medicare prices, yet the process for assessing affordability and establishing payment limits does not bar the use of quality-adjusted life years (QALYs) and similar measures. The bill received a “divided report” on February 1, 2024, in the Committee on Health Coverage, Insurance and Financial Services with 7 legislators favoring passage and 4 legislators opposing passage. Click here to view the bill. View Laura Bonnell’s testimony here. Contact information for Committee members and staff is here.
Maryland
The next meeting of the Maryland Prescription Drug Affordability Board’s stakeholder council is 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.
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.
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. The next meeting is May 15.
Vermont
On January 4, 2024, the Vermont Senate Health and Welfare Committee raised S. 98, legislation that would direct the Green Mountain Care Board to conduct an affordability review using cost-effectiveness studies. The language limiting the use of quality-adjusted life years (QALYs) is modeled on language used in other states to allow for use of cost effectiveness measures such as the equal value of life year gained (evLYG). As drafted, the language limits the use of QALYs only to identifying subpopulations and only focuses on discrimination related to life extension, raising concerns that the state could partner with entities such as PORTAL and ICER that support the use of QALYs and evLYGs to value health care.
ICER acknowledges that it developed the language for use in Washington State that is now being used across states. While the legislative language is touted as restricting QALYs, it is not modeled on the Affordable Care Act’s ban on use of QALYs and similar measures in Medicare, which comprehensively bans discriminatory measures. The Value Our Health template for state legislatures includes a provision banning use of discriminatory measures that is modeled on the ACA language (see section (d)). Advocates that want to weigh in may reach out to the committee assistant Kiki Carasi-Schwartz at [email protected].
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.
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
2024 Science of Patient Engagement Symposium
May 8-9, 2024
Click here to view.
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.
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.
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.
1. 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.
2. Colorado PDAB Discusses Reference to Pricing in Foreign Countries. 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.
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
3. Growing Economic Consensus That How We Value Medicines Must Change. Richard Xie, a senior health economist at RA Capital and former director of research at the Innovation and Value Initiative, penned an opinion calling for the field of health economists to break from tradition. He stated, "The U.S. system is not perfect — payers and middlemen drive up out-of-pocket costs so some people aren’t actually able to afford appropriate care despite years of paying premiums, for example.” He argued against European-style cost effectiveness research, stating "Unfortunately for patients in these countries, however, value is estimated by conventional CEAs that ignore key factors reflecting how medicines benefit patients and society, and how the marketplace works in practice (including how branded medicines eventually become inexpensive generics).” Click here to view the opinion.
4. HHS’ Finalizes the Regulations on Section 504 of the Rehab Act Clarifying Rules for Nondiscrimination. The U.S. Department of Health and Human Services (HHS) announced the final rule governing Section 504 of the Rehabilitation Act, which protects the rights of people with disabilities in programs and activities receiving federal financial assistance. In response to the proposed rule, last year the Partnership to Improve Patient Care (PIPC) joined 100 organizations and individuals on a letter providing the agency with a series of recommendations. As described in the final rule, the new regulations would bar health care decisions made using measures that discount gains in life expectancy, which would likely include the quality-adjusted life year (QALYs) and the combined use of QALYs and equal value of life years gained (evLYG). HHS’ rule represents a critical step forward to protecting patients and people with disabilities and sends a strong message that we need better solutions for U.S. decision-making that don’t rely on the biased, outdated standards used by payers in the U.S. and other countries. PIPC expressed appreciation for the agency’s broad interpretation of what constitutes the discriminatory use of value assessment in its description of the rule. Click here to view PIPC’s overview of the rule.
5. Efforts to Collect Disability Data Ramp Up. Disability researchers and advocates continue to push for improved mechanisms for collecting data on people with disabilities to inform better care models. In response, the Centers for Medicare and Medicaid Innovation (CMMI) is advancing the option to collect disability data as part of the collection of Sociodemographic Data Elements in at least one of their innovation models, the Enhancing Oncology Model. Also, the Joint Commission, as a part of their new Health Equity Certificate, requires documentation of disability status and processes to provide disability accommodations. The Health Resources and Services Administration (HRSA) is not yet compliant with the collection of disability status as required by Section 4302 of the Affordable Care Act. The Disability Equity Collaborative has developed a toolkit below for health care organizations to use in their implementation of documenting disability status and accommodations in the electronic health questions. Click here to access the toolkit and share it with your colleagues.
6. PIPC Submits Comments on ICER Assessment of PTSD. PIPC recently submitted a comment letter on ICER's assessment of treatments for post-traumatic stress disorder (PTSD). The letter highlights several key flaws in ICER's model, including its use of discriminatory cost effectiveness models like the quality-adjusted-life-years (QALY) and equal value of life year gained (evLYG). ICER's assessment also falls short by failing to account for heterogeneity and instead focusing its reports on the "average" patient. Click here to read PIPC's letter.
7. Chicago Sun Times: Prescription Drug Affordability Boards Do More Harm Than Good. Jen Laws, President & CEO of the Community Access National Network, wrote an opinion for the Chicago Sun Times raising concerns about the impact of prescription drug affordability boards on equitable access to medications and the landscape of health care financing in favor of insurers, not patients. He stated, "Prescription drug affordability boards simply aren’t the catchy answer some Illinois legislators are being sold on. An upper limit payment is a direct threat to health equity efforts — leaving behind needy families and communities in favor of those who might be able to afford to go out of state or pay cash for a medication.” Click here to view the opinion.
8. 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.
9. 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).
Maine
A bill introduced in the Maine legislature, LD 1829, would create a Prescription Drug Affordability Board with 6 members. The board is not required to include patients or people with disabilities. The 12-member advisory council includes a consumer representative appointed by the Governor, but does not require patient and disability participation. The board would be authorized to establish upper payment limits and to reference Medicare prices, yet the process for assessing affordability and establishing payment limits does not bar the use of quality-adjusted life years (QALYs) and similar measures. The bill received a “divided report” on February 1, 2024, in the Committee on Health Coverage, Insurance and Financial Services with 7 legislators favoring passage and 4 legislators opposing passage. Click here to view the bill. View Laura Bonnell’s testimony here. Contact information for Committee members and staff is here.
Maryland
The next meeting of the Maryland Prescription Drug Affordability Board’s stakeholder council is 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.
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.
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. The next meeting is May 15.
Vermont
On January 4, 2024, the Vermont Senate Health and Welfare Committee raised S. 98, legislation that would direct the Green Mountain Care Board to conduct an affordability review using cost-effectiveness studies. The language limiting the use of quality-adjusted life years (QALYs) is modeled on language used in other states to allow for use of cost effectiveness measures such as the equal value of life year gained (evLYG). As drafted, the language limits the use of QALYs only to identifying subpopulations and only focuses on discrimination related to life extension, raising concerns that the state could partner with entities such as PORTAL and ICER that support the use of QALYs and evLYGs to value health care.
ICER acknowledges that it developed the language for use in Washington State that is now being used across states. While the legislative language is touted as restricting QALYs, it is not modeled on the Affordable Care Act’s ban on use of QALYs and similar measures in Medicare, which comprehensively bans discriminatory measures. The Value Our Health template for state legislatures includes a provision banning use of discriminatory measures that is modeled on the ACA language (see section (d)). Advocates that want to weigh in may reach out to the committee assistant Kiki Carasi-Schwartz at [email protected].
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.
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.
- Netherlands: Patients with cystic fibrosis are struggling to gain access to treatment due to arcane, one-size-fits-all qualifications.
- New Zealand: A recent funding boost to Pharmac will not cover the costs of new cancer medicines despite increased warnings from patients and advocates about the need for more options.
- United Kingdom: While some treatments for cystic fibrosis have been made available through the NHS, patients are still waiting for other medications to be approved that could further help patients and their families.
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.
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- Transthyretin Amyloidosis Cardiomyopathy: Revised Scoping Document, Research Protocol available. Public meeting: September 2024
- Myelodysplastic Syndrome: Revised Scoping Document, Model Analysis Plan, Research Protocol available. Public meeting: July 2024.
- Chronic Obstructive Pulmonary Disease: Draft Evidence Report available. Public meeting: June 2024
- Post-Traumatic Stress Disorder: Revised Scoping Document, Research Protocol, Model Analysis Plan available. Main review: May 30, 2024.
Upcoming Events and Webinars
2024 Science of Patient Engagement Symposium
May 8-9, 2024
Click here to view.
Uncovering the True Cost of Healthcare
May 21, 2024
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PCORI Board of Governors Meeting
June 17-18, 2024
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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.
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.
