In this week's edition...
— New PIPC Webinar: Understanding Alternative Methods and Metrics for Value Assessment. Click here to RSVP.
— PIPC Recommendations for Enhancing CMS’ Patient Engagement Strategies. Click here to view the recommendations.
— PIPC Submits Comments on ICER MDS Assessment. Click here to read the letter.
— ASAN Names Colin Killick as Executive Director. See details below.
— AHRQ Announces Plans for National Healthcare Extension Service To Disseminate Evidence. Click here to learn more.
— ICHOM Holds Webinar on Value-based Healthcare Model. See details below.
— Health Affairs: Unanswered Questions And Unintended Consequences Of State Prescription Drug Affordability Boards. Click here to read the article.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— PCORI Annual Meeting October 22-23, 2024. See details below.
— Comment on PCORI Systematic Reviews by July 11, 2024. See details below.
— PCORI Engagement Awards opening July 17! Click here to learn more.
— CMS Releases Draft Guidance for Medicare Drug Negotiation Program. See details below.
— Chairman Tony Coelho's Statement on Final Passage of H.R. 485. See details below.
— 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.
— New PIPC Webinar: Understanding Alternative Methods and Metrics for Value Assessment. Click here to RSVP.
— PIPC Recommendations for Enhancing CMS’ Patient Engagement Strategies. Click here to view the recommendations.
— PIPC Submits Comments on ICER MDS Assessment. Click here to read the letter.
— ASAN Names Colin Killick as Executive Director. See details below.
— AHRQ Announces Plans for National Healthcare Extension Service To Disseminate Evidence. Click here to learn more.
— ICHOM Holds Webinar on Value-based Healthcare Model. See details below.
— Health Affairs: Unanswered Questions And Unintended Consequences Of State Prescription Drug Affordability Boards. Click here to read the article.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— PCORI Annual Meeting October 22-23, 2024. See details below.
— Comment on PCORI Systematic Reviews by July 11, 2024. See details below.
— PCORI Engagement Awards opening July 17! Click here to learn more.
— CMS Releases Draft Guidance for Medicare Drug Negotiation Program. See details below.
— Chairman Tony Coelho's Statement on Final Passage of H.R. 485. See details below.
— 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. New PIPC Webinar: Understanding 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 weaknesses. No patient is average, and no measure of value should assume so. Please join PIPC on Monday, June 24 at 2 PM EST to learn more about these new metrics, how they work, and how they are similar and different from some of the methodologies currently used. Click here to RSVP.
2. PIPC Recommendations for Enhancing CMS’ Patient Engagement Strategies. Drawing on robust frameworks from leading organizations including National Health Council (NHC), the Patient-Centered Outcomes Research Institute (PCORI), the PATIENTS Program at the University of Maryland, the Innovation and Value Initiative (IVI), and AcademyHealth, PIPC has published recommendations for CMS that prioritize authentically involving patients and people with disabilities in agency decisions. By synthesizing insights from these frameworks, PIPC advocates best practices to foster meaningful dialogue with patients, caregivers, and people with disabilities across CMS to better capture the insights from those with lived experience to inform CMS decisions. PIPC recommends:
Click here to view the recommendations.
3. PIPC Submits Comments on ICER MDS Assessment. On June 10, PIPC submitted a comment letter on the Institute for Clinical and Economic Review’s (ICER) assessment of anemia in myelodysplastic syndrome (MDS). The letter highlights several flaws in ICER’s model, including its assumption of patient responsiveness to treatment based on health dependance state and exclusion of non-drug costs for ongoing treatment of MDS. PIPC is also concerned with ICER's continued use of the discriminatory QALY and similar measure eyLYG in its assessment. Click here to read the letter.
4. ASAN Names Colin Killick as Executive Director. PIPC congratulates Colin Killick, who will be stepping into the role of Executive Director for the Autistic Self Advocacy Network beginning November 1st. The Board stated, "Colin comes to ASAN with extensive leadership experience in the disability rights movement. For the last five years, he has served as Executive Director of the Disability Policy Consortium, Massachusetts' main statewide disability rights advocacy organization. In that role, he has worked to advance the rights of people with disabilities across a broad variety of contexts, including a successful campaign to grow state housing voucher funding for low-income people with disabilities, overturning discriminatory state Crisis Standards of Care at the height of the COVID-19 public health emergency, and blocking efforts to slash Medicaid Home and Community-Based Services." Avery Outlaw will step into the role of Deputy Executive Director beginning in November, and will continue to serve as Interim ED until the leadership transition takes place. Click here to view the announcement.
5. AHRQ Announces Plans for National Healthcare Extension Service To Disseminate Evidence. AHRQ intends to publish a Notice of Funding Opportunity to create state-based healthcare extension cooperatives to increase the use of patient-centered outcomes research evidence in clinical practice. "Cooperatives will work with Medicaid agencies, managed care organizations, community groups, policy and payment experts and others to improve care delivery. AHRQ will also issue funding opportunities for two related projects: a national coordinating center and a national evaluation center. Working together, these projects will be designed to accelerate the dissemination and implementation of patient-centered outcomes research evidence through improvements in healthcare policy, payment and practice, and to reduce healthcare disparities, especially among people who receive Medicaid, are uninsured or are otherwise medically underserved." Click here to learn more.
6. ICHOM Holds Webinar on Value-based Healthcare Model. The webinar titled "Introducing ICHOM: A Value-based Healthcare Model” provided insights into value-based healthcare. Participants were given an opportunity to better understand ICHOM's mission to globally drive the adoption of value-based healthcare by defining patient-centered outcome measures. ICHOM shared how its sets capture the full impact of medical conditions on patients’ lives, fostering transparency and accountability. ICHOM provides several opportunities to be involved through participation in their initiatives, research collaboration, and implementation of standard sets to enhance patient outcomes and satisfaction. For those implementing ICHOM’s sets, a survey provides an opportunity to be featured on their implementation map, showcasing your organization as a leader in value-based healthcare. Click here for the survey. Click here to view the webinar recording.
7. Health Affairs: Unanswered Questions And Unintended Consequences Of State Prescription Drug Affordability Boards. A column in Health Affairs provides an overview of the unintended consequences and unanswered questions surrounding state PDABs. "...Many uncertainties and gaps remain regarding state prescription drug affordability boards’ assessments of ‘affordability’ and setting of upper payment limits. Across states that have granted PDABs the purported authority to set UPLs, specific selection and review criteria vary and are expected to be further clarified through regulatory rule-making processes. Overall, the singular focus on price-related factors in the absence of statutory requirements to prioritize value-related elements amplifies concerns about potential unintended consequences while providing no assurance that PDABs will achieve their intended goals of improving affordability for patients.” Click here to read the article.
8. 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.
9. PCORI Annual Meeting October 22-23, 2024. PCORI’s two-day event brings together patients, caregivers, researchers and the broader healthcare community to learn about the latest findings from PCORI-funded research and research-related projects as well as to network and share in rich discussions to advance patient-centered comparative clinical effectiveness research. Click here to view information about scholarships provided to those wanting to attend. Click here to learn more.
10. Comment on PCORI Systematic Reviews by July 11, 2024. From May 28 through July 11, 2024, PCORI is seeking comments on three draft reports of audio-based care for managing mental health, diabetes, and other chronic conditions. PCORI is funding the systematic reviews to improve understanding of the evidence base on this topic and to inform the broader healthcare community and our future research investments. Click here to learn more.
11. PCORI Engagement Awards opening July 17! PCORI's Online System will open on July 17 for the submission of Letters of Intent (LOI) for four PCORI Engagement Award Program funding announcements under the Fall 2024 Cycle. These awards are for Capacity Building, Dissemination Initiative, Stakeholder Convening Support, and Building Capacity for Small Organizations to Engage in Patient-Centered Comparative Clinical Effectiveness Research (CER) The PCORI Engagement Award Program is intended to bring more patients, caregivers, clinicians and other healthcare stakeholders into the research process. The goal is to support projects that will build a community better able to participate in patient-centered comparative clinical effectiveness research (CER) and serve as channels to disseminate study results. This is central to PCORI’s mission to fund useful CER that will help patients and those who care for them make better-informed healthcare decisions. Click here to learn more.
12 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.
13. Chairman Tony Coelho's Statement on Final Passage of H.R. 485. In response to the final passage of H.R. 485, PIPC Chairman Tony Coelho is pleased to share this statement.
It is disappointing that a bill to simply extend current law protections to all people with disabilities did not pass the House unanimously and instead passed on a partisan vote. Yet, it is important to note that Republicans and Democrats both stated support for the current law protections against use of QALYs & similar measures that devalue people with disabilities, and they support banning their use more broadly. This is the critical issue for those of us with disabilities. We expect both parties to keep that view and work with the disability community to act on the recommendations of the National Council on Disability, the independent federal agency that identified for policymakers how QALYs & similar measures discriminate and called for a consistent policy across federal programs to address it.
For additional information, you may click here to view the CCD letter supporting H.R. 485, click here to view the statement from supporting organizations and click here to view the letter from supportive organizations. You may view the White House Statement of Administration Policy here, which opposed how the bill would be paid for, while underscoring continued Democratic support for the current law barring QALYs and similar measures.
Emerging Threats in States for Use of Discriminatory Metrics
Several states are considering policies that would reference entities such as the Institute for Clinical and Economic Review (ICER), which calls the discriminatory quality-adjusted life year (QALY) the “gold standard” for value assessment, and others are considering policies to import QALY-based coverage and reimbursement decisions from other countries that restrict access to care. We encourage you to keep track of all state-based threats using this new website tracking state activities. Key issues are highlighted below.
Colorado
On June 14, the Colorado Prescription Drug Affordability Board met to discuss Cosentyx, a treatment for plaque psoriasis and psoriatic arthritis. The Board determined it is unaffordable. The next steps is assessing for an upper payment limit.
On May 23, PIPC submitted a comment letter to the Colorado Prescription Drug Affordability Board (PDAB) on the Board's ongoing process for affordability reviews and establishment of upper payment limits. PIPC's comments come on the heels of additional correspondence to the PDAB urging the Board to reject the use of discriminatory metrics such as quality-adjusted-life-years (QALY) and others that create barriers to access for patients and people with disabilities. The letter notes that, pursuant to the recently-finalized rules governing Section 504 of the Rehabilitation Act, entities that receive federal financial assistance, including state Medicaid programs, are subject to requirements that bar the use of these discriminatory value assessments. The letter also responds to the Board’s discussion about foreign government policies, highlighting the concerns of patients and people with disabilities to such policies as well as the National Council on Disability recommendations against referencing other countries. Click here to read the letter. Click here to view the Board’s conversation about foreign methods and potential to import QALYs (begins at 2:41).
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.
Maryland
The Maryland Prescription Drug Affordability Board’s stakeholder council last met on May 20. Six drugs were selected for cost reviews for drugs treating conditions such as type 2 diabetes, obesity, psoriasis, arthritis and Crohn's disease, eczema and asthma. Written comments sent to the Board are available here. The Stakeholder Council will next meet on June 24 at 2pm - register here to join. Written comments on the Board’s cost reviews may be submitted over the next 60 days to [email protected]. On May 13, PIPC submitted a comment letter to the Maryland Prescription Drug Affordability Board (PDAB) on the Board's ongoing Cost Review Study process. PIPC's comments come on the heels of additional correspondence to the PDAB urging the Board to reject the use of discriminatory metrics such as quality-adjusted-life-years (QALY) and others that create barriers to access for patients and people with disabilities. The letter also notes that, pursuant to the recently-finalized rules governing Section 504 of the Rehabilitation Act, entities that receive federal financial assistance, including state Medicaid programs, are subject to requirements that bar the use of these discriminatory value assessments. Click here to read the letter.
Massachusetts
The Massachusetts Senate Ways and Means Committees was referred the “PACT Act”, reported favorably on October 30, 2023 by the Joint Committee on Health Care Financing. The legislation includes a provision calling for the health policy commission not to base its determinations on measures such as QALYs. The Senate rejected an amendment (#19) pushed by Massachusetts advocates calling for transparency related to the use of cost effectiveness analyses by the commission, as well as increased requirements for stakeholder engagement. The Senate passed the bill on November 15, 2023. Massachusetts advocates will work with the House toward language that more clearly provides for public transparency of the evidence under consideration and opportunities for engagement from patients and people with disabilities.
Michigan
The Michigan legislature is similarly considering legislation SB 483 that would create a Prescription Drug Affordability Board. It includes language related to the use of quality-adjusted life years (QALYs) that was originally developed by the Institute for Clinical and Economic Review (ICER) for other states where it has not precluded boards from referencing QALYs and the equal value of life year gained (evLYG). As drafted, the language limits the use of QALYs only to identifying subpopulations and focuses on discrimination related to life extension, raising concerns that the legislation would allow the PDAB to partner with entities such as PORTAL and ICER that support the use of QALYs and evLYGs to value health care, as seen in other states. The bill has also been subject to critique related to its lack of patient engagement requirements and opportunities. Criteria for Board members would likely exclude patients or people with disabilities and even the stakeholder council does not require a patient perspective. View comments submitted by the Bonnell Foundation: Living with CF here. View NORD comments here. View comments from CANN here.
Oregon
PIPC recently submitted a letter to the Oregon Prescription Drug Affordability Board (PDAB) on the Board's ongoing affordability review activities. The letter expresses concern that the legislative provisions governing the use of quality-adjusted-life-years (QALY) metric and similar measures in legislation that established the board may be interpreted narrowly. PIPC also points out that recently finalized rules governing Section 504 of the Rehabilitation Act clarifies that recipients of federal financial assistance, including Medicaid programs, may not use discriminatory metrics such as QALYs. Click here to read the letter. Click here to view the opinion from Oregon advocate Lorren Sandt. Click here to view the letter from advocates to the legislature requesting pause of the board’s activities and legislative oversight as the board works on its engagement process. Click here to view the drugs under consideration at each meeting, including treatments for obesity, multiple sclerosis, ulcerative colitis, psoriasis, ADHD, HIV, and diabetes. Register here for the next meeting on June 26.
Rhode Island
The Rhode Island Senate passed S2719 on May 21st and the text was referred to House Committee on Health and Human Services. The bill creates a Drug Cost Review Commission. The bill’s definition of “excess costs” outlines a methodology for cost effectiveness analysis that could rely on QALYs and similar measures. The bill specifically mentions referencing the cost effectiveness or value of a drug as part of its analysis, yet does not bar the use of discriminatory measures such as QALYs. The Rhode Island session adjourned, and the PDAB bill did not move forward.
Washington State
At the May 22, 2024 meeting, the Program on Regulation, Therapeutics, and Law (PORTAL) provided information to the Prescription Drug Affordability Board on “Considerations for Conducting Affordability Reviews,” including cost effectiveness. PORTAL is known to be aligned with ICER, which considers QALY-based cost effectiveness to be the gold standard. PORTAL has also in the past referenced the evLYG as a measure to consider in affordability reviews. PIPC is following the PDAB’s implementation in Washington State closely for potential use of discriminatory measures of effectiveness, especially now that its engagement with PORTAL is official and public. Click here for information about the next meeting on July 16.
There are openings on Board’s advisory group! 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
PCORI Board of Governors Meeting
June 17-18, 2024
Click here to view.
PCORI Annual Meeting
October 22-23, 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.
Comparative Effectiveness Research Using Claims Data: Meticulous Methods Don’t Solve Old Problems. Click here to read the article.
1. New PIPC Webinar: Understanding 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 weaknesses. No patient is average, and no measure of value should assume so. Please join PIPC on Monday, June 24 at 2 PM EST to learn more about these new metrics, how they work, and how they are similar and different from some of the methodologies currently used. Click here to RSVP.
2. PIPC Recommendations for Enhancing CMS’ Patient Engagement Strategies. Drawing on robust frameworks from leading organizations including National Health Council (NHC), the Patient-Centered Outcomes Research Institute (PCORI), the PATIENTS Program at the University of Maryland, the Innovation and Value Initiative (IVI), and AcademyHealth, PIPC has published recommendations for CMS that prioritize authentically involving patients and people with disabilities in agency decisions. By synthesizing insights from these frameworks, PIPC advocates best practices to foster meaningful dialogue with patients, caregivers, and people with disabilities across CMS to better capture the insights from those with lived experience to inform CMS decisions. PIPC recommends:
- CMS should develop a formalized process to ensure continuous, robust engagement of patients and people with disabilities at multiple levels.
- Using patient insights, CMS should clearly communicate how it intends to use the input it receives, and how that input is reflected in the final negotiated prices.
- CMS should solicit input from diverse communities to ensure representation of the diversity of the patients and communities affected by the topic.
- CMS should ensure that opportunities for patient engagement are accessible.
- To gauge both successes and challenges, CMS should establish a structured process for continuous review and assessment of its engagement strategy.
- CMS should avoid one-size fits all value metrics.
Click here to view the recommendations.
3. PIPC Submits Comments on ICER MDS Assessment. On June 10, PIPC submitted a comment letter on the Institute for Clinical and Economic Review’s (ICER) assessment of anemia in myelodysplastic syndrome (MDS). The letter highlights several flaws in ICER’s model, including its assumption of patient responsiveness to treatment based on health dependance state and exclusion of non-drug costs for ongoing treatment of MDS. PIPC is also concerned with ICER's continued use of the discriminatory QALY and similar measure eyLYG in its assessment. Click here to read the letter.
4. ASAN Names Colin Killick as Executive Director. PIPC congratulates Colin Killick, who will be stepping into the role of Executive Director for the Autistic Self Advocacy Network beginning November 1st. The Board stated, "Colin comes to ASAN with extensive leadership experience in the disability rights movement. For the last five years, he has served as Executive Director of the Disability Policy Consortium, Massachusetts' main statewide disability rights advocacy organization. In that role, he has worked to advance the rights of people with disabilities across a broad variety of contexts, including a successful campaign to grow state housing voucher funding for low-income people with disabilities, overturning discriminatory state Crisis Standards of Care at the height of the COVID-19 public health emergency, and blocking efforts to slash Medicaid Home and Community-Based Services." Avery Outlaw will step into the role of Deputy Executive Director beginning in November, and will continue to serve as Interim ED until the leadership transition takes place. Click here to view the announcement.
5. AHRQ Announces Plans for National Healthcare Extension Service To Disseminate Evidence. AHRQ intends to publish a Notice of Funding Opportunity to create state-based healthcare extension cooperatives to increase the use of patient-centered outcomes research evidence in clinical practice. "Cooperatives will work with Medicaid agencies, managed care organizations, community groups, policy and payment experts and others to improve care delivery. AHRQ will also issue funding opportunities for two related projects: a national coordinating center and a national evaluation center. Working together, these projects will be designed to accelerate the dissemination and implementation of patient-centered outcomes research evidence through improvements in healthcare policy, payment and practice, and to reduce healthcare disparities, especially among people who receive Medicaid, are uninsured or are otherwise medically underserved." Click here to learn more.
6. ICHOM Holds Webinar on Value-based Healthcare Model. The webinar titled "Introducing ICHOM: A Value-based Healthcare Model” provided insights into value-based healthcare. Participants were given an opportunity to better understand ICHOM's mission to globally drive the adoption of value-based healthcare by defining patient-centered outcome measures. ICHOM shared how its sets capture the full impact of medical conditions on patients’ lives, fostering transparency and accountability. ICHOM provides several opportunities to be involved through participation in their initiatives, research collaboration, and implementation of standard sets to enhance patient outcomes and satisfaction. For those implementing ICHOM’s sets, a survey provides an opportunity to be featured on their implementation map, showcasing your organization as a leader in value-based healthcare. Click here for the survey. Click here to view the webinar recording.
7. Health Affairs: Unanswered Questions And Unintended Consequences Of State Prescription Drug Affordability Boards. A column in Health Affairs provides an overview of the unintended consequences and unanswered questions surrounding state PDABs. "...Many uncertainties and gaps remain regarding state prescription drug affordability boards’ assessments of ‘affordability’ and setting of upper payment limits. Across states that have granted PDABs the purported authority to set UPLs, specific selection and review criteria vary and are expected to be further clarified through regulatory rule-making processes. Overall, the singular focus on price-related factors in the absence of statutory requirements to prioritize value-related elements amplifies concerns about potential unintended consequences while providing no assurance that PDABs will achieve their intended goals of improving affordability for patients.” Click here to read the article.
8. 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.
9. PCORI Annual Meeting October 22-23, 2024. PCORI’s two-day event brings together patients, caregivers, researchers and the broader healthcare community to learn about the latest findings from PCORI-funded research and research-related projects as well as to network and share in rich discussions to advance patient-centered comparative clinical effectiveness research. Click here to view information about scholarships provided to those wanting to attend. Click here to learn more.
10. Comment on PCORI Systematic Reviews by July 11, 2024. From May 28 through July 11, 2024, PCORI is seeking comments on three draft reports of audio-based care for managing mental health, diabetes, and other chronic conditions. PCORI is funding the systematic reviews to improve understanding of the evidence base on this topic and to inform the broader healthcare community and our future research investments. Click here to learn more.
11. PCORI Engagement Awards opening July 17! PCORI's Online System will open on July 17 for the submission of Letters of Intent (LOI) for four PCORI Engagement Award Program funding announcements under the Fall 2024 Cycle. These awards are for Capacity Building, Dissemination Initiative, Stakeholder Convening Support, and Building Capacity for Small Organizations to Engage in Patient-Centered Comparative Clinical Effectiveness Research (CER) The PCORI Engagement Award Program is intended to bring more patients, caregivers, clinicians and other healthcare stakeholders into the research process. The goal is to support projects that will build a community better able to participate in patient-centered comparative clinical effectiveness research (CER) and serve as channels to disseminate study results. This is central to PCORI’s mission to fund useful CER that will help patients and those who care for them make better-informed healthcare decisions. Click here to learn more.
12 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.
13. Chairman Tony Coelho's Statement on Final Passage of H.R. 485. In response to the final passage of H.R. 485, PIPC Chairman Tony Coelho is pleased to share this statement.
It is disappointing that a bill to simply extend current law protections to all people with disabilities did not pass the House unanimously and instead passed on a partisan vote. Yet, it is important to note that Republicans and Democrats both stated support for the current law protections against use of QALYs & similar measures that devalue people with disabilities, and they support banning their use more broadly. This is the critical issue for those of us with disabilities. We expect both parties to keep that view and work with the disability community to act on the recommendations of the National Council on Disability, the independent federal agency that identified for policymakers how QALYs & similar measures discriminate and called for a consistent policy across federal programs to address it.
For additional information, you may click here to view the CCD letter supporting H.R. 485, click here to view the statement from supporting organizations and click here to view the letter from supportive organizations. You may view the White House Statement of Administration Policy here, which opposed how the bill would be paid for, while underscoring continued Democratic support for the current law barring QALYs and similar measures.
Emerging Threats in States for Use of Discriminatory Metrics
Several states are considering policies that would reference entities such as the Institute for Clinical and Economic Review (ICER), which calls the discriminatory quality-adjusted life year (QALY) the “gold standard” for value assessment, and others are considering policies to import QALY-based coverage and reimbursement decisions from other countries that restrict access to care. We encourage you to keep track of all state-based threats using this new website tracking state activities. Key issues are highlighted below.
- Click here to learn more about the potential for Prescription Drug Affordability Boards to discriminate by incorporating the use of QALYS.
- Click here to view the Value Our Health state template legislation that would protect people with disabilities and chronic conditions from the use of QALYs and similar metrics developed by third parties such as ICER in decisions related to reimbursement and coverage, as well as ensure their engagement in decision-making.
- Click here to view a one pager about the flaws in ICER’s methodology.
- Click here to view information from experts on the downside of referencing foreign countries.
- Click here to learn about statutory protections against use of QALYs.
Colorado
On June 14, the Colorado Prescription Drug Affordability Board met to discuss Cosentyx, a treatment for plaque psoriasis and psoriatic arthritis. The Board determined it is unaffordable. The next steps is assessing for an upper payment limit.
On May 23, PIPC submitted a comment letter to the Colorado Prescription Drug Affordability Board (PDAB) on the Board's ongoing process for affordability reviews and establishment of upper payment limits. PIPC's comments come on the heels of additional correspondence to the PDAB urging the Board to reject the use of discriminatory metrics such as quality-adjusted-life-years (QALY) and others that create barriers to access for patients and people with disabilities. The letter notes that, pursuant to the recently-finalized rules governing Section 504 of the Rehabilitation Act, entities that receive federal financial assistance, including state Medicaid programs, are subject to requirements that bar the use of these discriminatory value assessments. The letter also responds to the Board’s discussion about foreign government policies, highlighting the concerns of patients and people with disabilities to such policies as well as the National Council on Disability recommendations against referencing other countries. Click here to read the letter. Click here to view the Board’s conversation about foreign methods and potential to import QALYs (begins at 2:41).
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.
Maryland
The Maryland Prescription Drug Affordability Board’s stakeholder council last met on May 20. Six drugs were selected for cost reviews for drugs treating conditions such as type 2 diabetes, obesity, psoriasis, arthritis and Crohn's disease, eczema and asthma. Written comments sent to the Board are available here. The Stakeholder Council will next meet on June 24 at 2pm - register here to join. Written comments on the Board’s cost reviews may be submitted over the next 60 days to [email protected]. On May 13, PIPC submitted a comment letter to the Maryland Prescription Drug Affordability Board (PDAB) on the Board's ongoing Cost Review Study process. PIPC's comments come on the heels of additional correspondence to the PDAB urging the Board to reject the use of discriminatory metrics such as quality-adjusted-life-years (QALY) and others that create barriers to access for patients and people with disabilities. The letter also notes that, pursuant to the recently-finalized rules governing Section 504 of the Rehabilitation Act, entities that receive federal financial assistance, including state Medicaid programs, are subject to requirements that bar the use of these discriminatory value assessments. Click here to read the letter.
Massachusetts
The Massachusetts Senate Ways and Means Committees was referred the “PACT Act”, reported favorably on October 30, 2023 by the Joint Committee on Health Care Financing. The legislation includes a provision calling for the health policy commission not to base its determinations on measures such as QALYs. The Senate rejected an amendment (#19) pushed by Massachusetts advocates calling for transparency related to the use of cost effectiveness analyses by the commission, as well as increased requirements for stakeholder engagement. The Senate passed the bill on November 15, 2023. Massachusetts advocates will work with the House toward language that more clearly provides for public transparency of the evidence under consideration and opportunities for engagement from patients and people with disabilities.
Michigan
The Michigan legislature is similarly considering legislation SB 483 that would create a Prescription Drug Affordability Board. It includes language related to the use of quality-adjusted life years (QALYs) that was originally developed by the Institute for Clinical and Economic Review (ICER) for other states where it has not precluded boards from referencing QALYs and the equal value of life year gained (evLYG). As drafted, the language limits the use of QALYs only to identifying subpopulations and focuses on discrimination related to life extension, raising concerns that the legislation would allow the PDAB to partner with entities such as PORTAL and ICER that support the use of QALYs and evLYGs to value health care, as seen in other states. The bill has also been subject to critique related to its lack of patient engagement requirements and opportunities. Criteria for Board members would likely exclude patients or people with disabilities and even the stakeholder council does not require a patient perspective. View comments submitted by the Bonnell Foundation: Living with CF here. View NORD comments here. View comments from CANN here.
Oregon
PIPC recently submitted a letter to the Oregon Prescription Drug Affordability Board (PDAB) on the Board's ongoing affordability review activities. The letter expresses concern that the legislative provisions governing the use of quality-adjusted-life-years (QALY) metric and similar measures in legislation that established the board may be interpreted narrowly. PIPC also points out that recently finalized rules governing Section 504 of the Rehabilitation Act clarifies that recipients of federal financial assistance, including Medicaid programs, may not use discriminatory metrics such as QALYs. Click here to read the letter. Click here to view the opinion from Oregon advocate Lorren Sandt. Click here to view the letter from advocates to the legislature requesting pause of the board’s activities and legislative oversight as the board works on its engagement process. Click here to view the drugs under consideration at each meeting, including treatments for obesity, multiple sclerosis, ulcerative colitis, psoriasis, ADHD, HIV, and diabetes. Register here for the next meeting on June 26.
Rhode Island
The Rhode Island Senate passed S2719 on May 21st and the text was referred to House Committee on Health and Human Services. The bill creates a Drug Cost Review Commission. The bill’s definition of “excess costs” outlines a methodology for cost effectiveness analysis that could rely on QALYs and similar measures. The bill specifically mentions referencing the cost effectiveness or value of a drug as part of its analysis, yet does not bar the use of discriminatory measures such as QALYs. The Rhode Island session adjourned, and the PDAB bill did not move forward.
Washington State
At the May 22, 2024 meeting, the Program on Regulation, Therapeutics, and Law (PORTAL) provided information to the Prescription Drug Affordability Board on “Considerations for Conducting Affordability Reviews,” including cost effectiveness. PORTAL is known to be aligned with ICER, which considers QALY-based cost effectiveness to be the gold standard. PORTAL has also in the past referenced the evLYG as a measure to consider in affordability reviews. PIPC is following the PDAB’s implementation in Washington State closely for potential use of discriminatory measures of effectiveness, especially now that its engagement with PORTAL is official and public. Click here for information about the next meeting on July 16.
There are openings on Board’s advisory group! 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.
- Canada: The scientific director of Obesity Canada explains that the lack of coverage for obesity drugs will hurt the country in the long run.
- New Zealand: Blood cancer doctors are urging the government to increase funding for treatments that are not covered under the current system.
- United Kingdom: A treatment for breast cancer could benefit around British patients with a specific type of advanced breast cancer, but there are doubts about whether NICE will approve it on value.
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, Research Protocol available. Public meeting: November 2024
- Transthyretin Amyloidosis Cardiomyopathy: Revised Scoping Document, Research Protocol, Model Analysis Plan available. Public meeting: September 2024
- Myelodysplastic Syndrome: Draft Evidence Report available. Public meeting: July 2024.
- Chronic Obstructive Pulmonary Disease: Evidence Report available. Public meeting: June 2024
- Post-Traumatic Stress Disorder: Evidence Report available. Main review: May 30, 2024.
Upcoming Events and Webinars
PCORI Board of Governors Meeting
June 17-18, 2024
Click here to view.
PCORI Annual Meeting
October 22-23, 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.
Comparative Effectiveness Research Using Claims Data: Meticulous Methods Don’t Solve Old Problems. Click here to read the article.
