In this week's edition...
— PIPC Submits Comments on 'Cures 2.0.' RFI. See details below.
— FDA’s CBER Hosting Patient Listening Session on Gene Therapy, September 20. See details below.
— Due Wednesday! Request for Information: Strategies for Maximizing Public Engagement in NIH Supported Clinical Research. Click here for more details and to submit a comment.
— PIPC Submits Comments on Medicare Drug Price Negotiation Implementation Guidance. See details below.
— AAAS Seeks Nominations for Working Group on Disability Inclusion and Anti-Ableism. See details below.
— ICHOM Looking for Patient Partners! See details below.
— PCORI Engagement Awards Now Open! Click here to learn more.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— PCORI Annual Meeting October 22-23, 2024. 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.
— PIPC Submits Comments on 'Cures 2.0.' RFI. See details below.
— FDA’s CBER Hosting Patient Listening Session on Gene Therapy, September 20. See details below.
— Due Wednesday! Request for Information: Strategies for Maximizing Public Engagement in NIH Supported Clinical Research. Click here for more details and to submit a comment.
— PIPC Submits Comments on Medicare Drug Price Negotiation Implementation Guidance. See details below.
— AAAS Seeks Nominations for Working Group on Disability Inclusion and Anti-Ableism. See details below.
— ICHOM Looking for Patient Partners! See details below.
— PCORI Engagement Awards Now Open! Click here to learn more.
— Learn More About Alternative Measures for Value Assessment. Click here to learn more.
— PCORI Annual Meeting October 22-23, 2024. 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 We're Reading
1. PIPC Submits Comments on 'Cures 2.0.' RFI. On August 2, the Partnership to Improve Patient Care (PIPC) submitted a comment letter to Reps. Diana DeGette (D-CO) and Larry Bucshon (R-IN) on their recent request for information (RFI) on advancing 21st Century Cures policies.
Recommendations contained in PIPC's letter are as follows:
2. FDA’s CBER Hosting Patient Listening Session on Gene Therapy, September 20. The FDA Center for Biologics Evaluation and Research (CBER) is hosting a public patient listening meeting and opening a docket to better understand patient and care partner perspectives on safety considerations and long-term follow-up for approved gene therapy treatments for rare diseases. FDA states that the objectives of this listening meeting are to hear from patients and care partners on their perspectives on short-term and long-term risks of approved gene therapy products, to learn what types of information patients would find helpful in their decision-making when considering gene therapy, to learn about their considerations and experience with participating in long-term studies after receiving a gene therapy, and to help inform patient-centered protocols for long-term studies that include gene therapy outcomes. The virtual meeting will be held on September 20, 2024 from 11:00 a.m.– 4:30 p.m. ET. Click here for more information and to register!
3. Due Wednesday! Request for Information: Strategies for Maximizing Public Engagement in NIH Supported Clinical Research. The National Institutes of Health (NIH) is seeking stakeholder feedback on ways to ensure that public voices are meaningfully incorporated in NIH supported clinical research studies. "This request for information (RFI) is part of a larger NIH effort to develop a vision and framework for incorporating public voices in all phases and types of clinical research. To accomplish this, NIH asked the Novel and Exceptional Technology and Research Advisory Committee (NExTRAC) to form the ENGAGE Working Group. This Working Group includes patients, advocates, researchers, clinicians, non-profit representatives, and more. For the purposes of this RFI, NIH is specifically seeking input about engaging the public throughout all stages of clinical research and not focused solely on recruitment or participation in clinical research and trials." Responses must be received by no later than August 14, 2024. Click here for more details and to submit a comment.
4. PIPC Submits Comments on Medicare Drug Price Negotiation Implementation Guidance. Nearly 60 organizations and advocates representing the patient and disability communities joined PIPC's comment letter on the Centers for Medicare and Medicaid Services' (CMS) guidance implementing the Medicare Drug Price Negotiation Program. Our comments focus on the agency’s process for engaging patients in its decisions, including determinations related to a treatment’s clinical effectiveness, unmet need and therapeutic alternatives, as well as the agency’s use of value assessments.
While we appreciate that CMS is considering certain recommendations from patients and people with disabilities for improving its engagement, we are concerned that the new guidance does not put forward a concrete plan or process for developing predictable, targeted, and specific tactics for engaging patients and people with disabilities. We are also concerned that the guidance does not capture the limitations on use of quality-adjusted life years (QALYs) and similar measures explicitly described in the Affordable Care Act. Therefore, the letter shares the following recommendations:
5. AAAS Seeks Nominations for Working Group on Disability Inclusion and Anti-Ableism. The American Association for the Advancement of Science is now accepting nominations for the second AAAS Multidisciplinary Working Group (MWG) focused on Disability Inclusion and Anti-Ableism in the science, technology, engineering, mathematics, and medicine (STEMM) enterprise. Nominations will be accepted through Friday, August 23, 2024 by 5:00 PM EST. Click here for additional details on the AAAS working group. Click here to apply.
6. ICHOM Looking for Patient Partners! ICHOM is a global non-profit organization with a mission to unlock the potential of value-based healthcare and ultimately improve patient outcomes worldwide. ICHOM's Patient Partner Alliance is an initiative designed to elevate patients to equal partners in their care, empowering them through shared decision-making. As an Alliance Member, ICHOM is offering a 10% discount to attend the ICHOM 2024 Conference which takes place in Amsterdam on 21-22 October. PIPC members are eligible for a discount! Reach out if you are interested in joining.
7. PCORI Engagement Awards Are Now Open! PCORI's Online System opened on July 17 for the submission of Letters of Intent (LOI) for four PCORI Engagement Award Program funding announcements under the Fall 2024 Cycle. These awards are for Capacity Building, Dissemination Initiative, Stakeholder Convening Support, and Building Capacity for Small Organizations to Engage in Patient-Centered Comparative Clinical Effectiveness Research (CER) The PCORI Engagement Award Program is intended to bring more patients, caregivers, clinicians and other healthcare stakeholders into the research process. The goal is to support projects that will build a community better able to participate in patient-centered comparative clinical effectiveness research (CER) and serve as channels to disseminate study results. This is central to PCORI’s mission to fund useful CER that will help patients and those who care for them make better-informed healthcare decisions. Click here to learn more.
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. 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
The next meeting of the Colorado Prescription Drug Affordability Board is September 6. You may register to participate here.
On May 23, PIPC submitted a comment letter to the Colorado Prescription Drug Affordability Board (PDAB) on the Board's ongoing process for affordability reviews and establishment of upper payment limits. PIPC's comments come on the heels of additional correspondence to the PDAB urging the Board to reject the use of discriminatory metrics such as quality-adjusted-life-years (QALY) and others that create barriers to access for patients and people with disabilities. The letter notes that, pursuant to the recently-finalized rules governing Section 504 of the Rehabilitation Act, entities that receive federal financial assistance, including state Medicaid programs, are subject to requirements that bar the use of these discriminatory value assessments. The letter also responds to the Board’s discussion about foreign government policies, highlighting the concerns of patients and people with disabilities to such policies as well as the National Council on Disability recommendations against referencing other countries. Click here to read the letter. Click here to view the Board’s conversation about foreign methods and potential to import QALYs (begins at 2:41).
Maryland
The Maryland Prescription Drug Affordability Board’s stakeholder council last met on July 22. Click here to register for the Stakeholder Council meeting on August 26. Six Six drugs have been selected for cost reviews for drugs treating conditions such as type 2 diabetes, obesity, psoriasis, arthritis and Crohn's disease, eczema and asthma. On May 13, PIPC submitted a comment letter to the Maryland Prescription Drug Affordability Board (PDAB) on the Board's ongoing Cost Review Study process. PIPC's comments come on the heels of additional correspondence to the PDAB urging the Board to reject the use of discriminatory metrics such as quality-adjusted-life-years (QALY) and others that create barriers to access for patients and people with disabilities. The letter also notes that, pursuant to the recently-finalized rules governing Section 504 of the Rehabilitation Act, entities that receive federal financial assistance, including state Medicaid programs, are subject to requirements that bar the use of these discriminatory value assessments. Click here to read the letter. Click here to learn more about the the Draft Upper Payment Limit Action Plan and comment to [email protected] by August 26.
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
In response to patient advocates’ concerns, the chair of the Oregon Prescription Drug Affordability Board proposed and the board voted in favor of pausing its assessments until 2025. The Board recognized that it does not have an accurate definition of “affordability” and is taking time to review the data collected and how it may be used in its work. The Board will also reconsider its selection of drugs to be assessed. The decision follows a webinar hosted by Caring Ambassadors and Disability Rights Oregon and others on the lack of a robust engagement process and understanding of the Board’s goals and decision-making process, particularly related to whether cost savings would translate to lower patient out-of-pocket costs. Patient advocates applauded the decision. Click here to view the June 26 meeting. Click here to view the webinar educating advocates and advocacy letters to the Board and legislature from patients. Click here to read the letter. Click here to view the opinion from Oregon advocate Lorren Sandt. Click here to view the letter from advocates to the legislature requesting pause of the board’s activities and legislative oversight as the board works on its engagement process. Click here to view the drugs under consideration at each meeting, including treatments for obesity, multiple sclerosis, ulcerative colitis, psoriasis, ADHD, HIV, and diabetes. Register here for the next meeting on August 21.
Washington State
At the May 22, 2024 meeting, the Program on Regulation, Therapeutics, and Law (PORTAL) provided information to the Prescription Drug Affordability Board on “Considerations for Conducting Affordability Reviews,” including cost effectiveness. PORTAL is known to be aligned with ICER, which considers QALY-based cost effectiveness to be the gold standard. PORTAL has also in the past referenced the evLYG as a measure to consider in affordability reviews. PIPC is following the PDAB’s implementation in Washington State closely for potential use of discriminatory measures of effectiveness, especially now that its engagement with PORTAL is official and public. Click here for information about the next meeting on September 18.
International News: What Happens in Countries Using QALYs and Cost-Based Thresholds?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
ICER's QALY-Based Study Topics
The Institute for Clinical Economic Review (ICER) conducts cost effectiveness studies for insurers using the cost-per-QALY methodology. ICER provides guidance on its website for patients and patient advocates to provide direct input related to their experiences with the disease. Click here to provide patient input. Click here to view the topics and deadlines.
Upcoming Events and Webinars
PCORI Board of Governors Meeting
September 10-11, 2024
Click here to view.
PCORI Annual Meeting
October 22-23, 2024
Click here to view.
PCORI Board of Governors Meeting
December 10, 2024
Click here to view.
AHRQ Effective Program Updates
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.
1. PIPC Submits Comments on 'Cures 2.0.' RFI. On August 2, the Partnership to Improve Patient Care (PIPC) submitted a comment letter to Reps. Diana DeGette (D-CO) and Larry Bucshon (R-IN) on their recent request for information (RFI) on advancing 21st Century Cures policies.
Recommendations contained in PIPC's letter are as follows:
- Congress should provide increased oversight of coverage and reimbursement decisions at the state and federal levels to ensure they are using transparent, nondiscriminatory methods and meaningfully engaging patients.
- Congress should pass H.R. 485, the Protecting Health Care for All Patients Act.
- Congress should protect shared decision-making between patients and providers.
2. FDA’s CBER Hosting Patient Listening Session on Gene Therapy, September 20. The FDA Center for Biologics Evaluation and Research (CBER) is hosting a public patient listening meeting and opening a docket to better understand patient and care partner perspectives on safety considerations and long-term follow-up for approved gene therapy treatments for rare diseases. FDA states that the objectives of this listening meeting are to hear from patients and care partners on their perspectives on short-term and long-term risks of approved gene therapy products, to learn what types of information patients would find helpful in their decision-making when considering gene therapy, to learn about their considerations and experience with participating in long-term studies after receiving a gene therapy, and to help inform patient-centered protocols for long-term studies that include gene therapy outcomes. The virtual meeting will be held on September 20, 2024 from 11:00 a.m.– 4:30 p.m. ET. Click here for more information and to register!
3. Due Wednesday! Request for Information: Strategies for Maximizing Public Engagement in NIH Supported Clinical Research. The National Institutes of Health (NIH) is seeking stakeholder feedback on ways to ensure that public voices are meaningfully incorporated in NIH supported clinical research studies. "This request for information (RFI) is part of a larger NIH effort to develop a vision and framework for incorporating public voices in all phases and types of clinical research. To accomplish this, NIH asked the Novel and Exceptional Technology and Research Advisory Committee (NExTRAC) to form the ENGAGE Working Group. This Working Group includes patients, advocates, researchers, clinicians, non-profit representatives, and more. For the purposes of this RFI, NIH is specifically seeking input about engaging the public throughout all stages of clinical research and not focused solely on recruitment or participation in clinical research and trials." Responses must be received by no later than August 14, 2024. Click here for more details and to submit a comment.
4. PIPC Submits Comments on Medicare Drug Price Negotiation Implementation Guidance. Nearly 60 organizations and advocates representing the patient and disability communities joined PIPC's comment letter on the Centers for Medicare and Medicaid Services' (CMS) guidance implementing the Medicare Drug Price Negotiation Program. Our comments focus on the agency’s process for engaging patients in its decisions, including determinations related to a treatment’s clinical effectiveness, unmet need and therapeutic alternatives, as well as the agency’s use of value assessments.
While we appreciate that CMS is considering certain recommendations from patients and people with disabilities for improving its engagement, we are concerned that the new guidance does not put forward a concrete plan or process for developing predictable, targeted, and specific tactics for engaging patients and people with disabilities. We are also concerned that the guidance does not capture the limitations on use of quality-adjusted life years (QALYs) and similar measures explicitly described in the Affordable Care Act. Therefore, the letter shares the following recommendations:
- CMS should avoid one-size fits all value metrics.
- 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.
5. AAAS Seeks Nominations for Working Group on Disability Inclusion and Anti-Ableism. The American Association for the Advancement of Science is now accepting nominations for the second AAAS Multidisciplinary Working Group (MWG) focused on Disability Inclusion and Anti-Ableism in the science, technology, engineering, mathematics, and medicine (STEMM) enterprise. Nominations will be accepted through Friday, August 23, 2024 by 5:00 PM EST. Click here for additional details on the AAAS working group. Click here to apply.
6. ICHOM Looking for Patient Partners! ICHOM is a global non-profit organization with a mission to unlock the potential of value-based healthcare and ultimately improve patient outcomes worldwide. ICHOM's Patient Partner Alliance is an initiative designed to elevate patients to equal partners in their care, empowering them through shared decision-making. As an Alliance Member, ICHOM is offering a 10% discount to attend the ICHOM 2024 Conference which takes place in Amsterdam on 21-22 October. PIPC members are eligible for a discount! Reach out if you are interested in joining.
7. PCORI Engagement Awards Are Now Open! PCORI's Online System opened on July 17 for the submission of Letters of Intent (LOI) for four PCORI Engagement Award Program funding announcements under the Fall 2024 Cycle. These awards are for Capacity Building, Dissemination Initiative, Stakeholder Convening Support, and Building Capacity for Small Organizations to Engage in Patient-Centered Comparative Clinical Effectiveness Research (CER) The PCORI Engagement Award Program is intended to bring more patients, caregivers, clinicians and other healthcare stakeholders into the research process. The goal is to support projects that will build a community better able to participate in patient-centered comparative clinical effectiveness research (CER) and serve as channels to disseminate study results. This is central to PCORI’s mission to fund useful CER that will help patients and those who care for them make better-informed healthcare decisions. Click here to learn more.
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. 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
The next meeting of the Colorado Prescription Drug Affordability Board is September 6. You may register to participate here.
On May 23, PIPC submitted a comment letter to the Colorado Prescription Drug Affordability Board (PDAB) on the Board's ongoing process for affordability reviews and establishment of upper payment limits. PIPC's comments come on the heels of additional correspondence to the PDAB urging the Board to reject the use of discriminatory metrics such as quality-adjusted-life-years (QALY) and others that create barriers to access for patients and people with disabilities. The letter notes that, pursuant to the recently-finalized rules governing Section 504 of the Rehabilitation Act, entities that receive federal financial assistance, including state Medicaid programs, are subject to requirements that bar the use of these discriminatory value assessments. The letter also responds to the Board’s discussion about foreign government policies, highlighting the concerns of patients and people with disabilities to such policies as well as the National Council on Disability recommendations against referencing other countries. Click here to read the letter. Click here to view the Board’s conversation about foreign methods and potential to import QALYs (begins at 2:41).
Maryland
The Maryland Prescription Drug Affordability Board’s stakeholder council last met on July 22. Click here to register for the Stakeholder Council meeting on August 26. Six Six drugs have been selected for cost reviews for drugs treating conditions such as type 2 diabetes, obesity, psoriasis, arthritis and Crohn's disease, eczema and asthma. On May 13, PIPC submitted a comment letter to the Maryland Prescription Drug Affordability Board (PDAB) on the Board's ongoing Cost Review Study process. PIPC's comments come on the heels of additional correspondence to the PDAB urging the Board to reject the use of discriminatory metrics such as quality-adjusted-life-years (QALY) and others that create barriers to access for patients and people with disabilities. The letter also notes that, pursuant to the recently-finalized rules governing Section 504 of the Rehabilitation Act, entities that receive federal financial assistance, including state Medicaid programs, are subject to requirements that bar the use of these discriminatory value assessments. Click here to read the letter. Click here to learn more about the the Draft Upper Payment Limit Action Plan and comment to [email protected] by August 26.
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
In response to patient advocates’ concerns, the chair of the Oregon Prescription Drug Affordability Board proposed and the board voted in favor of pausing its assessments until 2025. The Board recognized that it does not have an accurate definition of “affordability” and is taking time to review the data collected and how it may be used in its work. The Board will also reconsider its selection of drugs to be assessed. The decision follows a webinar hosted by Caring Ambassadors and Disability Rights Oregon and others on the lack of a robust engagement process and understanding of the Board’s goals and decision-making process, particularly related to whether cost savings would translate to lower patient out-of-pocket costs. Patient advocates applauded the decision. Click here to view the June 26 meeting. Click here to view the webinar educating advocates and advocacy letters to the Board and legislature from patients. Click here to read the letter. Click here to view the opinion from Oregon advocate Lorren Sandt. Click here to view the letter from advocates to the legislature requesting pause of the board’s activities and legislative oversight as the board works on its engagement process. Click here to view the drugs under consideration at each meeting, including treatments for obesity, multiple sclerosis, ulcerative colitis, psoriasis, ADHD, HIV, and diabetes. Register here for the next meeting on August 21.
Washington State
At the May 22, 2024 meeting, the Program on Regulation, Therapeutics, and Law (PORTAL) provided information to the Prescription Drug Affordability Board on “Considerations for Conducting Affordability Reviews,” including cost effectiveness. PORTAL is known to be aligned with ICER, which considers QALY-based cost effectiveness to be the gold standard. PORTAL has also in the past referenced the evLYG as a measure to consider in affordability reviews. PIPC is following the PDAB’s implementation in Washington State closely for potential use of discriminatory measures of effectiveness, especially now that its engagement with PORTAL is official and public. Click here for information about the next meeting on September 18.
International News: What Happens in Countries Using QALYs and Cost-Based Thresholds?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- Australia: While the PBS recently approved a Cystic Fibrosis treatment for patients ages 2-5, the lengthy approval process has caused stress and uncertainty for patients and their families.
- New Zealand: The decision to block free access to rare cancer drug means some people diagnosed in the next two months will have to go on to less effective treatments.
- United Kingdom: A breast cancer drug was not recommended by NICE due to arbitrary cost effectiveness restrictions.
ICER's QALY-Based Study Topics
The Institute for Clinical Economic Review (ICER) conducts cost effectiveness studies for insurers using the cost-per-QALY methodology. ICER provides guidance on its website for patients and patient advocates to provide direct input related to their experiences with the disease. Click here to provide patient input. Click here to view the topics and deadlines.
- Acute Pain: Draft Scoping Document available. Public comments due: Aug. 16, 2024.
- Fair Access: Coverage Policies in 2024: Protocol available
- Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease: Draft Scoping Document, Research Protocol, Model Analysis Plan available. Public meeting: November 2024
- Transthyretin Amyloidosis Cardiomyopathy: Draft Evidence Report available. Public meeting: Sept. 2024
- Myelodysplastic Syndrome: Final Evidence Report to be published in August 2024
Upcoming Events and Webinars
PCORI Board of Governors Meeting
September 10-11, 2024
Click here to view.
PCORI Annual Meeting
October 22-23, 2024
Click here to view.
PCORI Board of Governors Meeting
December 10, 2024
Click here to view.
AHRQ Effective Program Updates
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.
