In This Week’s Issue:
1. Oregon Submits Waiver Renewal to CMS Acknowledging Use of QALYs. See details below.
2. NCD Health Equity Framework Includes Recommendation Against Use of QALYs. Click here to view the framework.
3. Value Our Health: Advancing Value Assessment Principles Centered on Patients and People with Disabilities. See below for details.
4. NCD Recommendation to Include a Comprehensive QALY Ban in BBB Supported by Law and Public Opinion
5. PCORI Board Considers Health Care Cost and Value. Click here to view the PCORI presentation.
6. For Two Years, Australia Failed People with Cystic Fibrosis; It Can’t Happen Again. Click here to view the article.
7. PIPC Submits Comments to CMMI on Listening Session on Incorporating Beneficiary Perspectives into Model Testing, Implementation, and Evaluation. Click here to view the letter.
8. PIPC Submits Comments to PCORI on Proposed Research Agenda. Click here to view the letter.
9. New Targeted PCORI Funding Announcement: Comparative Effectiveness of Interventions Targeting Mental Health in Individuals with Intellectual and Developmental Disabilities. See below for details.
10. PCORI Seeks Nominations for Advisory Panels. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
11. Upcoming opportunity within the Center for Evidence and Practice Improvement at AHRQ, see details below.
12. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
13. Emerging Threats in States for Use of Discriminatory Metrics, see details below.
14. ICER's QALY-Based Study Topics: Alzheimer's Disease, Amyotrophic Lateral Sclerosis (ALS), Beta Thalassemia, COVID-19, Type 2 Diabetes, Hypertrophic Cardiomyopathy, Asthma, Unsupported Price Increase, Fair Access: Coverage Policies. Click here to provide patient input.
15. Upcoming Events and Webinars, see details below.
16. Medical Journal Articles, see details below.
17. AHRQ Effective Program Updates, see details below.
1. Oregon Submits Waiver Renewal to CMS Acknowledging Use of QALYs. See details below.
2. NCD Health Equity Framework Includes Recommendation Against Use of QALYs. Click here to view the framework.
3. Value Our Health: Advancing Value Assessment Principles Centered on Patients and People with Disabilities. See below for details.
4. NCD Recommendation to Include a Comprehensive QALY Ban in BBB Supported by Law and Public Opinion
5. PCORI Board Considers Health Care Cost and Value. Click here to view the PCORI presentation.
6. For Two Years, Australia Failed People with Cystic Fibrosis; It Can’t Happen Again. Click here to view the article.
7. PIPC Submits Comments to CMMI on Listening Session on Incorporating Beneficiary Perspectives into Model Testing, Implementation, and Evaluation. Click here to view the letter.
8. PIPC Submits Comments to PCORI on Proposed Research Agenda. Click here to view the letter.
9. New Targeted PCORI Funding Announcement: Comparative Effectiveness of Interventions Targeting Mental Health in Individuals with Intellectual and Developmental Disabilities. See below for details.
10. PCORI Seeks Nominations for Advisory Panels. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
11. Upcoming opportunity within the Center for Evidence and Practice Improvement at AHRQ, see details below.
12. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
13. Emerging Threats in States for Use of Discriminatory Metrics, see details below.
14. ICER's QALY-Based Study Topics: Alzheimer's Disease, Amyotrophic Lateral Sclerosis (ALS), Beta Thalassemia, COVID-19, Type 2 Diabetes, Hypertrophic Cardiomyopathy, Asthma, Unsupported Price Increase, Fair Access: Coverage Policies. Click here to provide patient input.
15. Upcoming Events and Webinars, see details below.
16. Medical Journal Articles, see details below.
17. AHRQ Effective Program Updates, see details below.
1. Oregon Submits Waiver Renewal to CMS Acknowledging Use of QALYs
On February 18, 2022, Oregon submitted its waiver renewal application to CMS. The state acknowledged the use of QALY-based cost effectiveness studies by the Health Evidence Review Commission (HERC) that developed the prioritized list of services covered under the waiver. While claiming its use of QALYs do not discriminate, the state went on to acknowledge how they are used to prefer treatments deemed to be more cost effective using QALYs and to require use of low cost treatments before accessing those deemed to be less cost effective. The state did request authority to allow exclusion of accelerated approval drugs, but failed to provide information on what "its own rigorous review process” would entail and whether it would rely on QALYs, though the state did reference the Institute for Clinical and Economic Review as a supporting source for its proposal, an entity that uses QALYs and calls them the gold standard. The state did not request renewal of its waiver related to Early Periodic Screening, Diagnostic, and Treatment (EPSDT) services provided to children and adolescents enrolled in the Oregon Health Plan (OHP), thereby allowing them access to medically necessary services in the future. The state also amended its initial waiver renewal draft to eliminate its request to use a closed formulary. Click here to view the schedule for consideration of the waiver renewal. Click here to view the waiver application sent to CMS. Click here to listen to a Podcast with Disability Rights Oregon.
2. NCD Health Equity Framework Includes Recommendation Against Use of QALYs
The National Council on Disability discussed its Health Equity Framework with the public on February 23, 2022. It begins with four core areas that NCD views as foundational for achieving health equity for people with disabilities. It is followed by numerous additional components that were identified by disability community leaders as being of critical importance. The core components are: 1. designating people with disabilities as a Special Medically Underserved Population (SMUP) under the Public Health Services Act; 2. requiring comprehensive disability clinical-care curricula in all US medical, nursing and other healthcare professional schools and requiring disability competency education and training of medical, nursing and other healthcare professionals; 3. requiring the use of accessible medical and diagnostic equipment; and 4. improving data collection concerning healthcare for people with disabilities across the lifespan. Additional components of NCD’s framework include a recommendation to mandate a blanket prohibition on the use of Quality Adjusted Life Years by any federal agency, or recipients of federal financial assistance from the Department of Health and Human Services, both directly and through third-party assessments. Click here to view the framework.
3. Value Our Health: Advancing Value Assessment Principles Centered on Patients and People with Disabilities
Advocates are facing challenges and opportunities at the state and federal level to convince policymakers to advance policies that value outcomes that matter to patients and people with disabilities. Organizations supporting Value Our Health have developed a set of principles supporting value assessment methodologies centered on patients and people with disabilities, opposing summary measures such as the cost-per-quality-adjusted life year or QALY in favor of value frameworks that meaningfully acknowledge diversity and differences among patients and people with disabilities, that are developed using transparent processes and methods and meaningfully engage patients and people with disabilities, therefore capturing costs and benefits that matter to the patient. As states consider legislation that ranges from seeking to bar the use of QALYs to authorizing increased use of QALYs, opportunities exist for advocacy consistent with these principles and the recommendations of entities such as the National Council on Disability, an independent federal agency recommending QALYs be barred across federal programs. Click here to view the Value Our Health principles. Click here to view template legislation for states to consider that would improve the evidence used in Medicaid decisions, require stakeholder engagement and bar state use of QALYs and similar discriminatory metrics. Click here to view the Open Letter from over 90 organizations to state and federal policymakers. Click here for more information about Value Our Health.
4. NCD Recommendation to Include a Comprehensive QALY Ban in BBB Supported by Law and Public Opinion
The National Council on Disability (NCD) has recommended an unambiguous ban on the quality adjusted life year (QALY) within the text of the Build Back Better Act (H.R. 5367), responding to their increasing reference in coverage, reimbursement and incentive programs. The Chairman of the National Council on Disability, Andrés Gallegos, stated, “In our most recent advice to Congress we advise that Congress extend those prohibitions [prohibitions against the use of the QALY in the ACA] throughout federal programs. The Build Back Better Act is a tremendous opportunity to do so and to memorialize the protections with an unambiguous ban on the use of the QALY. We remain hopeful that our advice will be heeded as we all await the final text of that bill.” Click here to view the presentations. Click here to view the NCD’s letter.
The NCD’s recommendations are supported by the law and public opinion. Morning Consult ran a poll on behalf of Partnership to Improve Patient Care focusing on the use of cost-effectiveness assessments to determine the value of coverage and treatment costs. The survey found that Americans want patients and their doctors in charge of health care decision making and are opposed to the use of cost assessments such as Quality-Adjusted Life Year (QALY). PIPC Chairman Tony Coelho published an opinion piece in Morning Consult about the poll concluding that it is time to finally end the ambiguity and prohibit QALYs altogether. DREDF published a paper explains how the use of the QALY, even in tandem with alternative measures such as the Equal Value of Life Years Gained (“evLYG”), violates disability nondiscrimination law. Click here to view the DREDF report. Click here to view the one-pager. Click here to find summaries in both English and Spanish.
5. PCORI Board Considers Health Care Cost and Value
At the last PCORI Board meeting on February 15, 2022, the Board was presented with a framework for activities related to health care cost and value. It included collection of the full range of outcomes, informing the value conversation and supporting policy priorities such as emerging therapies and evidence synthesis. Underlying this framework was a clear focus on stakeholder engagement to center value on what patients value for their care. Click here to view the PCORI presentation.
6. For Two Years, Australia Failed People with Cystic Fibrosis; It Can’t Happen Again.
In a recent piece published in RealClearHealth, cystic fibrosis advocate, Gunnar Esiason, discusses the “bath math” that feeds traditional health technology assessments. He discusses the devastating impacts this has had on CF patients outside the United States, in limiting their access to Trikafta, a life changing CF treatment. He focuses on the persistent challenges of patients in Australia, highlighting just where their HTA is failing patients:
"Where Australia’s arbiter of drug value, the Pharmaceutical Benefits Advisory Committee, and others have gone wrong, is precisely this cost-effectiveness math. Among their errors, they ignore that Trikafta’s price is only temporarily high. Trikafta will go generic and will continue to save lives and avert hospitalization costs in perpetuity at a much lower price. This is demonstrably true of so many drug classes: statins, antibiotics, anti-depressants, cancer treatments. All were expensive at one time and now they are inexpensive generics, still saving lives. If Australians finally gain access to Trikafta, they deserve to take a victory lap. For them, it will represent the end of a hard-won advocacy campaign. But we still owe it to them to question the several years’ delay they have suffered in the name of bad math. That cannot be forgotten." Click here to view the article.
7. PIPC Submits Comments to CMMI on Listening Session on Incorporating Beneficiary Perspectives into Model Testing, Implementation, and Evaluation
In a letter to the Center for Medicare and Medicaid Innovation (CMMI), the Partnership to Improve Patient Care (PIPC) offered comments on CMMI's Listening Session entitled "Incorporating Beneficiary Perspectives into Model Testing, Implementation, and Evaluation." PIPC Chair Tony Coelho applauded CMMI Director Liz Fowler for conducting the listening session, noting that PIPC looks forward to working with the Health Care Payment and Learning Action Network (LAN) to convey the importance of beneficiary perspectives and prioritize best practices that focus on patient-centered outcomes. PIPC also called on CMMI to establish a clear process for engagement with patients and people with disabilities.
"Within CMMI, we have consistently strongly advocated for patient advisory panels to guide the development of alternative payment models and for CMMI to engage with patients and people with disabilities in the development of patient-centeredness criteria to measure the success of alternative payment models, as called for in statute," wrote Chair Coelho. "We are excited for this listening session to be the first of many as CMMI renews its efforts to center its work on those with the most to benefit from its success – patients and people with disabilities."
8. PIPC Submits Comments to PCORI on Proposed Research Agenda
In a letter to the Patient-Centered Outcomes Research Institute (PCORI), the Partnership to Improve Patient Care (PIPC) offered feedback on PCORI's proposed research agenda. In the letter, PIPC Chair Tony Coelho doubled down on his recommendation that PCORI engage in direct outreach to organizations representing patients and people with disabilities in its forthcoming research efforts, noting that PCORI must be more specific and clear with respect to its methodologies. The letter also stresses that PCORI should work with experts that do not view the discriminatory quality-adjusted-life-years (QALY) metric as the "gold standard" for value assessment in health care.
"When health care decisions are made without reliable data on the experience of patients and their preferences, we have a systematic problem," wrote Chair Coelho. "The solution to that problem is a clear and credible source for decision-makers to not only directly access outcomes data meeting criteria for being patient-centered, but to also better understand when – or not – the outcomes data they are relying on is indeed credible and representative of diverse patient perspectives."
9. New Targeted PCORI Funding Announcement: Comparative Effectiveness of Interventions Targeting Mental Health in Individuals with Intellectual and Developmental Disabilities
On Tuesday, January 11, 2022, the Patient-Centered Outcomes Research Institute (PCORI) released the Comparative Effectiveness of Interventions Targeting Mental Health in Individuals with Intellectual and Developmental Disabilities (IDD) Targeted PCORI Funding Announcement (Targeted PFA). This Targeted PFA aims to fund high-quality, rigorous randomized controlled trials or observational, prospective cohort studies that compare the effectiveness of evidence-based approaches (e.g., specific pharmacological and behavioral interventions) that address mental health conditions in individuals with IDD. Proposed studies should compare the effectiveness of pharmacological, behavioral, or other nonpharmacological or combination interventions administered via appropriate delivery modalities (e.g., telehealth, family-based, group, or individual).
Studies focused on pediatric, transitional age, and adult individuals with mild to moderate IDD-related impairment, those with more severe impairment due to IDDs, underrepresented groups, and underserved populations (e.g., individuals in rural settings) are of particular interest.
Here is the funding announcement for more information about this Targeted PFA.
10. PCORI Seeks Nominations for Advisory Panels.
PCORI is currently seeking nominations for appointments to its advisory panels. The advisory panels must include patients or patient advocates. PCORI's staff, board, and methodology committee take advisory panels' recommendations into account when making decisions and determinations. PIPC encourages patients and patient advocates to submit nominations to serve on PCORI's advisory panels, including on the Advisory Panel on Clinical Effectiveness and Decision Science, Advisory Panel on Patient Engagement, and Advisory Panel on Rare Disease. Nominations are open through March 31, 2022. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
11. Upcoming Opportunity Within the Center for Evidence and Practice Improvement at AHRQ
AHRQ will soon be hiring a Senior Staff Service Fellow in CEPI’s Office of the Director to support activities funded by the Patient-Centered Outcomes Research Trust Fund (PCOR TF) under the National Center for Excellence in Primary Care Research (NCEPCR). The Senior Staff Service Fellow will support AHRQ’s mandated roles under the PCOR TF in evidence dissemination and implementation in primary care as well as of training and capacity building for comparative effectiveness research in primary care settings.
The Senior Staff Service Fellow designs, executes and evaluates projects funded by the PCOR TF to increase the uptake of evidence in primary care including by using new models of primary/ambulatory care delivery, advancing health equity and engaging learning health systems. AHRQ is looking for a team player to lead a multidisciplinary team to design, that leads national initiatives to disseminate and implement PCOR findings related to transforming primary/ambulatory care delivery to make care more equitable and patient-centered including a focus on people living with multiple chronic conditions and other at-risk populations.
This Senior Staff Service Fellow will also advise other members of CEPI and AHRQ on dissemination and implementation activities related to the PCOR TF; supports and aligns ongoing primary care projects across all divisions within the Center as well as across AHRQ. Serves as a recognized expert within the areas of primary care, primary care research and implementation science.
12. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
13. 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.
Washington State
The Washington State legislature has introduced SB 5532, a bill establishing a prescription drug affordability board. It does not prohibit the use of metrics such as quality-adjusted life years (QALYs) by the Board as part of its process for conducting an affordability review or in the process for determining which drugs will be made subject to an affordability review, with potential implications for a discriminatory impact in the treatments chosen for payment limits. As described by the National Association of State Health Policy, this legislation is modeled on Canada, which uses QALYs to determine coverage and reimbursement of health care treatments and services. The legislation does bar QALYs as part of the methodology for calculating the “upper payment limit” for drugs selected based on the affordability review. Today, the bill is scheduled for executive session in the House Committee on Appropriates at 11:00 AM Pacific (2 PM EST). Click here to view the bill. Click here to comment on the bill.
Oregon
Recently, the Portland Business Journal published an article on how patient advocates are pushing to overhaul Oregon Medicaid's drug list to ensure people with disabilities are not denied care. The article stated, "The U.S. Department of Health and Human Services in 1992 denied Oregon’s use of the “quality-adjusted life year” metric as discriminatory.” The article also highlighted that, "Disability rights advocates are pushing Oregon’s Medicaid program to change how it decides which treatments to cover in order to ensure that people with disabilities are not discriminated against and denied services. Oregon's waiver application, submitted to CMS on February 18, 2022, did not request to continue the state’s EPSDT waiver or a closed formulary, both issues for which patient and disability advocates expressed opposition. The waiver did defend the state’s use of QALYs and continues to request authority to conduct reviews of drugs approved through FDA’s accelerated pathway to allow for restricted coverage. Click here to read the full article. For original letters on the state’s initial draft, click here to view the DRO letter and click here to view the letter from national disability rights organizations. Click here to view the letter from the Epilepsy Foundation. Click here to view the waiver application as submitted to CMS.
Massachusetts
The Massachusetts House and Senate have each taken the positive step of introducing An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with Disabilities, H.201 and S.753. This bill would enshrine essential patient protections including a ban on the use of the QALY, a requirement for research to meet patient-centeredness criteria, and robust engagement of the patient and disability communities in health care decision making. Also, amendments were introduced to bar the use of QALYs as part of legislation S. 2651 to bar the use of QALYs that were withdrawn. Click here to view Amendment #20.
Maine
Maine is currently considering LD 1636, which would import QALYs from Canada. The bill directly references the prices paid for drugs in five Canadian provinces. Testimony from advocates highlighted concerns that Canada references QALYs and the bill would thereby import the QALY into Maine’s Medicaid program. Before applying for coverage by the provinces, all drugs must complete a Common Drug Review by CADTH, which uses QALYs. In Canada, many individuals living with disabilities are unable to receive the treatments and care they need. The National Council on Disability (NCD) warned in its 2019 report that similar coverage denials and loss of access to care could also be the outcome if the United States if we reference other countries. The NCD also highlighted in its report that Section 504 of the Rehab Act and Section 1557 of the ACA also apply to Medicaid programs because they receive federal financial assistance. Click here to view the bill. Click here to view testimony from CancerCare. Click here to view testimony from Southern Maine Chronic Pain Support Group. Click here to view testimony from the Center for Community Inclusion & Disability Studies.
Maryland
The Maryland legislature has reintroduced the Maryland Nondiscrimination in Health Care Coverage Act, proposing to bar the use of QALYs in state decision-making was reintroduced on February 17, 2022 as H.B. 903. The legislation did not include protections for patients and people with disabilities such as barring the use of discriminatory QALYs. The Board has begun meeting to hear from stakeholders, including hearing a presentation from the Institute for Clinical and Economic Review which relies on QALYs to assess treatment value. Legislation was introduced this year to bar the use of QALYs and implement patient protections that has not advanced in the legislature. On August 3, organizations representing patients and people with disabilities sent a letter urging the Board to avoid policies that would potentially discriminate by relying on discriminatory metrics such as QALYs. Click here to view legislation recently re-introduced in Maryland to bar the use of QALYs and implement patient protections.
14. ICER's QALY-Based Study Topics: Alzheimer's Disease, Amyotrophic Lateral Sclerosis (ALS), Beta Thalassemia, COVID-19, Type 2 Diabetes, Hypertrophic Cardiomyopathy, Asthma, Unsupported Price Increase, Fair Access: Coverage Policies.
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.
15. Upcoming Events and Webinars
Virtual Information Session: The Health Systems Implementation Initiative
March 3, 2022
Click here for details.
PCORI Board of Governors Meeting
March 8. 2022
Click here for details.
16. Medical Journal Articles
“It’s Time to Represent”: Shifting the Paradigm to Improve the Quality of Inputs into Value Assessment Frameworks, click here to view.
Evaluation of Financial Outcomes Under a Value-Based Payment Program for Community Pharmacies, click here to view.
Limited Role of Patient Input in Specialty Drug Coverage Policies, click here to view.
The Importance of Collaboration in Pursuit of Patient-Centered Value Assessment, click here to view.
Toward Better Data Dashboards for US Drug Value Assessments, click here to view.
Prevalence of Avoidable and Bias-Inflicting Methodological Pitfalls in Real-World Studies of Medication Safety and Effectiveness, click here to view.
Association of Co-Pay Elimination with Medication Adherence and Total Cost, click here to view.
The Utility of Patient Engagement in Drug Research and Development, click here to view.
Patient-Powered Research Networks of the Autoimmune Research Collaborative: Rationale, Capacity, and Future Directions, click here to view.
Enhancing Patient Research Partner Engagement: Research in Psoriatic Arthritis, click here to view.
Patient Voices in Value-Based Cancer Care: Priorities for the Biden Administration, click here to view.
The Dollar or Disease Burden: Caps on Healthcare Spending May Save Money, but at What “Cost” to Patients? click here to view.
17. AHRQ Effective Program Updates
Systematic Review: Models of Care That Include Primary Care for Adult Survivors of Childhood Cancer: A Realist Review. Click here to view.
Systematic Review: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Systematic Review: Prehabilitation and Rehabilitation for Major Joint Replacement. Click here to view.
Integrated and Comprehensive Pain Management Programs: Effectiveness and Harms. Click here to view.
Systematic Review: Management of High-Need, High-Cost Patients: A “Best Fit” Framework Synthesis, Realist Review, and Systematic Review. Click here to view.
Research Protocol: Partial Breast Irradiation for Breast Cancer. Click here to view.
Systematic Review: Physical Activity and the Health of Wheelchair Users: A Systematic Review in Multiple Sclerosis, Cerebral Palsy, and Spinal Cord Injury. Click here to view.
Research Report: Developing and Piloting a Tool to Create Dot Plots to Summarize Pooled Data for Multiple Outcomes in Systematic Reviews. Click here to view.
Systematic Review: Malnutrition in Hospitalized Adults. Click here to view.
Research Protocol: Nutrition as Prevention for Improved Cancer Outcomes. Click here to view.
Research Protocol: Telehealth During COVID-19. Click here to view.
Research Protocol: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder: An Update of the PTSD Repository Evidence Base. Click here to view.
On February 18, 2022, Oregon submitted its waiver renewal application to CMS. The state acknowledged the use of QALY-based cost effectiveness studies by the Health Evidence Review Commission (HERC) that developed the prioritized list of services covered under the waiver. While claiming its use of QALYs do not discriminate, the state went on to acknowledge how they are used to prefer treatments deemed to be more cost effective using QALYs and to require use of low cost treatments before accessing those deemed to be less cost effective. The state did request authority to allow exclusion of accelerated approval drugs, but failed to provide information on what "its own rigorous review process” would entail and whether it would rely on QALYs, though the state did reference the Institute for Clinical and Economic Review as a supporting source for its proposal, an entity that uses QALYs and calls them the gold standard. The state did not request renewal of its waiver related to Early Periodic Screening, Diagnostic, and Treatment (EPSDT) services provided to children and adolescents enrolled in the Oregon Health Plan (OHP), thereby allowing them access to medically necessary services in the future. The state also amended its initial waiver renewal draft to eliminate its request to use a closed formulary. Click here to view the schedule for consideration of the waiver renewal. Click here to view the waiver application sent to CMS. Click here to listen to a Podcast with Disability Rights Oregon.
2. NCD Health Equity Framework Includes Recommendation Against Use of QALYs
The National Council on Disability discussed its Health Equity Framework with the public on February 23, 2022. It begins with four core areas that NCD views as foundational for achieving health equity for people with disabilities. It is followed by numerous additional components that were identified by disability community leaders as being of critical importance. The core components are: 1. designating people with disabilities as a Special Medically Underserved Population (SMUP) under the Public Health Services Act; 2. requiring comprehensive disability clinical-care curricula in all US medical, nursing and other healthcare professional schools and requiring disability competency education and training of medical, nursing and other healthcare professionals; 3. requiring the use of accessible medical and diagnostic equipment; and 4. improving data collection concerning healthcare for people with disabilities across the lifespan. Additional components of NCD’s framework include a recommendation to mandate a blanket prohibition on the use of Quality Adjusted Life Years by any federal agency, or recipients of federal financial assistance from the Department of Health and Human Services, both directly and through third-party assessments. Click here to view the framework.
3. Value Our Health: Advancing Value Assessment Principles Centered on Patients and People with Disabilities
Advocates are facing challenges and opportunities at the state and federal level to convince policymakers to advance policies that value outcomes that matter to patients and people with disabilities. Organizations supporting Value Our Health have developed a set of principles supporting value assessment methodologies centered on patients and people with disabilities, opposing summary measures such as the cost-per-quality-adjusted life year or QALY in favor of value frameworks that meaningfully acknowledge diversity and differences among patients and people with disabilities, that are developed using transparent processes and methods and meaningfully engage patients and people with disabilities, therefore capturing costs and benefits that matter to the patient. As states consider legislation that ranges from seeking to bar the use of QALYs to authorizing increased use of QALYs, opportunities exist for advocacy consistent with these principles and the recommendations of entities such as the National Council on Disability, an independent federal agency recommending QALYs be barred across federal programs. Click here to view the Value Our Health principles. Click here to view template legislation for states to consider that would improve the evidence used in Medicaid decisions, require stakeholder engagement and bar state use of QALYs and similar discriminatory metrics. Click here to view the Open Letter from over 90 organizations to state and federal policymakers. Click here for more information about Value Our Health.
4. NCD Recommendation to Include a Comprehensive QALY Ban in BBB Supported by Law and Public Opinion
The National Council on Disability (NCD) has recommended an unambiguous ban on the quality adjusted life year (QALY) within the text of the Build Back Better Act (H.R. 5367), responding to their increasing reference in coverage, reimbursement and incentive programs. The Chairman of the National Council on Disability, Andrés Gallegos, stated, “In our most recent advice to Congress we advise that Congress extend those prohibitions [prohibitions against the use of the QALY in the ACA] throughout federal programs. The Build Back Better Act is a tremendous opportunity to do so and to memorialize the protections with an unambiguous ban on the use of the QALY. We remain hopeful that our advice will be heeded as we all await the final text of that bill.” Click here to view the presentations. Click here to view the NCD’s letter.
The NCD’s recommendations are supported by the law and public opinion. Morning Consult ran a poll on behalf of Partnership to Improve Patient Care focusing on the use of cost-effectiveness assessments to determine the value of coverage and treatment costs. The survey found that Americans want patients and their doctors in charge of health care decision making and are opposed to the use of cost assessments such as Quality-Adjusted Life Year (QALY). PIPC Chairman Tony Coelho published an opinion piece in Morning Consult about the poll concluding that it is time to finally end the ambiguity and prohibit QALYs altogether. DREDF published a paper explains how the use of the QALY, even in tandem with alternative measures such as the Equal Value of Life Years Gained (“evLYG”), violates disability nondiscrimination law. Click here to view the DREDF report. Click here to view the one-pager. Click here to find summaries in both English and Spanish.
5. PCORI Board Considers Health Care Cost and Value
At the last PCORI Board meeting on February 15, 2022, the Board was presented with a framework for activities related to health care cost and value. It included collection of the full range of outcomes, informing the value conversation and supporting policy priorities such as emerging therapies and evidence synthesis. Underlying this framework was a clear focus on stakeholder engagement to center value on what patients value for their care. Click here to view the PCORI presentation.
6. For Two Years, Australia Failed People with Cystic Fibrosis; It Can’t Happen Again.
In a recent piece published in RealClearHealth, cystic fibrosis advocate, Gunnar Esiason, discusses the “bath math” that feeds traditional health technology assessments. He discusses the devastating impacts this has had on CF patients outside the United States, in limiting their access to Trikafta, a life changing CF treatment. He focuses on the persistent challenges of patients in Australia, highlighting just where their HTA is failing patients:
"Where Australia’s arbiter of drug value, the Pharmaceutical Benefits Advisory Committee, and others have gone wrong, is precisely this cost-effectiveness math. Among their errors, they ignore that Trikafta’s price is only temporarily high. Trikafta will go generic and will continue to save lives and avert hospitalization costs in perpetuity at a much lower price. This is demonstrably true of so many drug classes: statins, antibiotics, anti-depressants, cancer treatments. All were expensive at one time and now they are inexpensive generics, still saving lives. If Australians finally gain access to Trikafta, they deserve to take a victory lap. For them, it will represent the end of a hard-won advocacy campaign. But we still owe it to them to question the several years’ delay they have suffered in the name of bad math. That cannot be forgotten." Click here to view the article.
7. PIPC Submits Comments to CMMI on Listening Session on Incorporating Beneficiary Perspectives into Model Testing, Implementation, and Evaluation
In a letter to the Center for Medicare and Medicaid Innovation (CMMI), the Partnership to Improve Patient Care (PIPC) offered comments on CMMI's Listening Session entitled "Incorporating Beneficiary Perspectives into Model Testing, Implementation, and Evaluation." PIPC Chair Tony Coelho applauded CMMI Director Liz Fowler for conducting the listening session, noting that PIPC looks forward to working with the Health Care Payment and Learning Action Network (LAN) to convey the importance of beneficiary perspectives and prioritize best practices that focus on patient-centered outcomes. PIPC also called on CMMI to establish a clear process for engagement with patients and people with disabilities.
"Within CMMI, we have consistently strongly advocated for patient advisory panels to guide the development of alternative payment models and for CMMI to engage with patients and people with disabilities in the development of patient-centeredness criteria to measure the success of alternative payment models, as called for in statute," wrote Chair Coelho. "We are excited for this listening session to be the first of many as CMMI renews its efforts to center its work on those with the most to benefit from its success – patients and people with disabilities."
8. PIPC Submits Comments to PCORI on Proposed Research Agenda
In a letter to the Patient-Centered Outcomes Research Institute (PCORI), the Partnership to Improve Patient Care (PIPC) offered feedback on PCORI's proposed research agenda. In the letter, PIPC Chair Tony Coelho doubled down on his recommendation that PCORI engage in direct outreach to organizations representing patients and people with disabilities in its forthcoming research efforts, noting that PCORI must be more specific and clear with respect to its methodologies. The letter also stresses that PCORI should work with experts that do not view the discriminatory quality-adjusted-life-years (QALY) metric as the "gold standard" for value assessment in health care.
"When health care decisions are made without reliable data on the experience of patients and their preferences, we have a systematic problem," wrote Chair Coelho. "The solution to that problem is a clear and credible source for decision-makers to not only directly access outcomes data meeting criteria for being patient-centered, but to also better understand when – or not – the outcomes data they are relying on is indeed credible and representative of diverse patient perspectives."
9. New Targeted PCORI Funding Announcement: Comparative Effectiveness of Interventions Targeting Mental Health in Individuals with Intellectual and Developmental Disabilities
On Tuesday, January 11, 2022, the Patient-Centered Outcomes Research Institute (PCORI) released the Comparative Effectiveness of Interventions Targeting Mental Health in Individuals with Intellectual and Developmental Disabilities (IDD) Targeted PCORI Funding Announcement (Targeted PFA). This Targeted PFA aims to fund high-quality, rigorous randomized controlled trials or observational, prospective cohort studies that compare the effectiveness of evidence-based approaches (e.g., specific pharmacological and behavioral interventions) that address mental health conditions in individuals with IDD. Proposed studies should compare the effectiveness of pharmacological, behavioral, or other nonpharmacological or combination interventions administered via appropriate delivery modalities (e.g., telehealth, family-based, group, or individual).
Studies focused on pediatric, transitional age, and adult individuals with mild to moderate IDD-related impairment, those with more severe impairment due to IDDs, underrepresented groups, and underserved populations (e.g., individuals in rural settings) are of particular interest.
Here is the funding announcement for more information about this Targeted PFA.
10. PCORI Seeks Nominations for Advisory Panels.
PCORI is currently seeking nominations for appointments to its advisory panels. The advisory panels must include patients or patient advocates. PCORI's staff, board, and methodology committee take advisory panels' recommendations into account when making decisions and determinations. PIPC encourages patients and patient advocates to submit nominations to serve on PCORI's advisory panels, including on the Advisory Panel on Clinical Effectiveness and Decision Science, Advisory Panel on Patient Engagement, and Advisory Panel on Rare Disease. Nominations are open through March 31, 2022. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
11. Upcoming Opportunity Within the Center for Evidence and Practice Improvement at AHRQ
AHRQ will soon be hiring a Senior Staff Service Fellow in CEPI’s Office of the Director to support activities funded by the Patient-Centered Outcomes Research Trust Fund (PCOR TF) under the National Center for Excellence in Primary Care Research (NCEPCR). The Senior Staff Service Fellow will support AHRQ’s mandated roles under the PCOR TF in evidence dissemination and implementation in primary care as well as of training and capacity building for comparative effectiveness research in primary care settings.
The Senior Staff Service Fellow designs, executes and evaluates projects funded by the PCOR TF to increase the uptake of evidence in primary care including by using new models of primary/ambulatory care delivery, advancing health equity and engaging learning health systems. AHRQ is looking for a team player to lead a multidisciplinary team to design, that leads national initiatives to disseminate and implement PCOR findings related to transforming primary/ambulatory care delivery to make care more equitable and patient-centered including a focus on people living with multiple chronic conditions and other at-risk populations.
This Senior Staff Service Fellow will also advise other members of CEPI and AHRQ on dissemination and implementation activities related to the PCOR TF; supports and aligns ongoing primary care projects across all divisions within the Center as well as across AHRQ. Serves as a recognized expert within the areas of primary care, primary care research and implementation science.
12. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- Australia: Patients are reluctantly dipping into their retiree funds to pay for cancer treatments because most treatments are unavailable for use on the PBS. Breast cancer patient is pushing to get Trodelvy, a life-saving drug, approved by PBAC and funded by the PBS. Angelman Syndrome patient's family is desperately raising funds to get her included in a clinical trial to afford a life-saving drug for just one year.
- United Kingdom: Family of a battling cancer patient is pleading for help to fund a trial drug not available under the NHS.
13. 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.
Washington State
The Washington State legislature has introduced SB 5532, a bill establishing a prescription drug affordability board. It does not prohibit the use of metrics such as quality-adjusted life years (QALYs) by the Board as part of its process for conducting an affordability review or in the process for determining which drugs will be made subject to an affordability review, with potential implications for a discriminatory impact in the treatments chosen for payment limits. As described by the National Association of State Health Policy, this legislation is modeled on Canada, which uses QALYs to determine coverage and reimbursement of health care treatments and services. The legislation does bar QALYs as part of the methodology for calculating the “upper payment limit” for drugs selected based on the affordability review. Today, the bill is scheduled for executive session in the House Committee on Appropriates at 11:00 AM Pacific (2 PM EST). Click here to view the bill. Click here to comment on the bill.
Oregon
Recently, the Portland Business Journal published an article on how patient advocates are pushing to overhaul Oregon Medicaid's drug list to ensure people with disabilities are not denied care. The article stated, "The U.S. Department of Health and Human Services in 1992 denied Oregon’s use of the “quality-adjusted life year” metric as discriminatory.” The article also highlighted that, "Disability rights advocates are pushing Oregon’s Medicaid program to change how it decides which treatments to cover in order to ensure that people with disabilities are not discriminated against and denied services. Oregon's waiver application, submitted to CMS on February 18, 2022, did not request to continue the state’s EPSDT waiver or a closed formulary, both issues for which patient and disability advocates expressed opposition. The waiver did defend the state’s use of QALYs and continues to request authority to conduct reviews of drugs approved through FDA’s accelerated pathway to allow for restricted coverage. Click here to read the full article. For original letters on the state’s initial draft, click here to view the DRO letter and click here to view the letter from national disability rights organizations. Click here to view the letter from the Epilepsy Foundation. Click here to view the waiver application as submitted to CMS.
Massachusetts
The Massachusetts House and Senate have each taken the positive step of introducing An Act Advancing Health Care Research and Decision-Making Centered on Patients and People with Disabilities, H.201 and S.753. This bill would enshrine essential patient protections including a ban on the use of the QALY, a requirement for research to meet patient-centeredness criteria, and robust engagement of the patient and disability communities in health care decision making. Also, amendments were introduced to bar the use of QALYs as part of legislation S. 2651 to bar the use of QALYs that were withdrawn. Click here to view Amendment #20.
Maine
Maine is currently considering LD 1636, which would import QALYs from Canada. The bill directly references the prices paid for drugs in five Canadian provinces. Testimony from advocates highlighted concerns that Canada references QALYs and the bill would thereby import the QALY into Maine’s Medicaid program. Before applying for coverage by the provinces, all drugs must complete a Common Drug Review by CADTH, which uses QALYs. In Canada, many individuals living with disabilities are unable to receive the treatments and care they need. The National Council on Disability (NCD) warned in its 2019 report that similar coverage denials and loss of access to care could also be the outcome if the United States if we reference other countries. The NCD also highlighted in its report that Section 504 of the Rehab Act and Section 1557 of the ACA also apply to Medicaid programs because they receive federal financial assistance. Click here to view the bill. Click here to view testimony from CancerCare. Click here to view testimony from Southern Maine Chronic Pain Support Group. Click here to view testimony from the Center for Community Inclusion & Disability Studies.
Maryland
The Maryland legislature has reintroduced the Maryland Nondiscrimination in Health Care Coverage Act, proposing to bar the use of QALYs in state decision-making was reintroduced on February 17, 2022 as H.B. 903. The legislation did not include protections for patients and people with disabilities such as barring the use of discriminatory QALYs. The Board has begun meeting to hear from stakeholders, including hearing a presentation from the Institute for Clinical and Economic Review which relies on QALYs to assess treatment value. Legislation was introduced this year to bar the use of QALYs and implement patient protections that has not advanced in the legislature. On August 3, organizations representing patients and people with disabilities sent a letter urging the Board to avoid policies that would potentially discriminate by relying on discriminatory metrics such as QALYs. Click here to view legislation recently re-introduced in Maryland to bar the use of QALYs and implement patient protections.
14. ICER's QALY-Based Study Topics: Alzheimer's Disease, Amyotrophic Lateral Sclerosis (ALS), Beta Thalassemia, COVID-19, Type 2 Diabetes, Hypertrophic Cardiomyopathy, Asthma, Unsupported Price Increase, Fair Access: Coverage Policies.
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.
- Alzheimer's Disease: Research Protocol available. Draft Scoping Document available. Public Comments closed. 3/23/2022: Model Analysis Plan. 5/10/2022: Model Analysis Plan.
- Amyotrophic Lateral Sclerosis (ALS): Draft Scoping Document available. Public comments closed. 3/4/2022: Revised Scoping Document. 3/11/2022: Research Protocol. 4/28/2022: Model Analysis Plan.
- Beta Thalassemia: Model Analysis Plan available. Research Protocol available. Revised Scoping Document available. 4/13/2022: Draft Evidence Report.
- COVID-19: Draft Evidence Report Available. Public comments open until 3/3/2022. Model Analysis Plan available. Draft Scoping Document and Research Protocol available. 3/28/2022: Evidence Report. 4/12/2022: Evidence Presentation. 5/10/2022: Final Evidence Report and Meeting Summary.
- Type 2 Diabetes: Final Evidence Report and Meeting Summary available. Evidence Presentation available. Evidence Report available. Draft Scoping Document and Research Protocol available.
- Hypertrophic Cardiomyopathy: Final Evidence Report and Meeting Summary available. Evidence Report Available. Evidence Presentation available.
- Unsupported Price Increase: Final Report available.
- Asthma: Final Evidence Report and Meeting Summary available. Evidence Presentation available. Evidence Report available.
- Fair Access: Coverage Policies in 2020: Protocol Available. Final Report available.
15. Upcoming Events and Webinars
Virtual Information Session: The Health Systems Implementation Initiative
March 3, 2022
Click here for details.
PCORI Board of Governors Meeting
March 8. 2022
Click here for details.
16. Medical Journal Articles
“It’s Time to Represent”: Shifting the Paradigm to Improve the Quality of Inputs into Value Assessment Frameworks, click here to view.
Evaluation of Financial Outcomes Under a Value-Based Payment Program for Community Pharmacies, click here to view.
Limited Role of Patient Input in Specialty Drug Coverage Policies, click here to view.
The Importance of Collaboration in Pursuit of Patient-Centered Value Assessment, click here to view.
Toward Better Data Dashboards for US Drug Value Assessments, click here to view.
Prevalence of Avoidable and Bias-Inflicting Methodological Pitfalls in Real-World Studies of Medication Safety and Effectiveness, click here to view.
Association of Co-Pay Elimination with Medication Adherence and Total Cost, click here to view.
The Utility of Patient Engagement in Drug Research and Development, click here to view.
Patient-Powered Research Networks of the Autoimmune Research Collaborative: Rationale, Capacity, and Future Directions, click here to view.
Enhancing Patient Research Partner Engagement: Research in Psoriatic Arthritis, click here to view.
Patient Voices in Value-Based Cancer Care: Priorities for the Biden Administration, click here to view.
The Dollar or Disease Burden: Caps on Healthcare Spending May Save Money, but at What “Cost” to Patients? click here to view.
17. AHRQ Effective Program Updates
Systematic Review: Models of Care That Include Primary Care for Adult Survivors of Childhood Cancer: A Realist Review. Click here to view.
Systematic Review: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Systematic Review: Prehabilitation and Rehabilitation for Major Joint Replacement. Click here to view.
Integrated and Comprehensive Pain Management Programs: Effectiveness and Harms. Click here to view.
Systematic Review: Management of High-Need, High-Cost Patients: A “Best Fit” Framework Synthesis, Realist Review, and Systematic Review. Click here to view.
Research Protocol: Partial Breast Irradiation for Breast Cancer. Click here to view.
Systematic Review: Physical Activity and the Health of Wheelchair Users: A Systematic Review in Multiple Sclerosis, Cerebral Palsy, and Spinal Cord Injury. Click here to view.
Research Report: Developing and Piloting a Tool to Create Dot Plots to Summarize Pooled Data for Multiple Outcomes in Systematic Reviews. Click here to view.
Systematic Review: Malnutrition in Hospitalized Adults. Click here to view.
Research Protocol: Nutrition as Prevention for Improved Cancer Outcomes. Click here to view.
Research Protocol: Telehealth During COVID-19. Click here to view.
Research Protocol: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder: An Update of the PTSD Repository Evidence Base. Click here to view.
