1. Opportunity to Comment on Oregon Waiver Renewal Application! See details below.
2. HHS Proposed Notice of Benefit and Payment Parameters Rule on Prohibited Discrimination under Section 1557. See below for details.
3. Upcoming PCORI Funding Announcement to Fund Studies on the Science of Engagement. Click here for more details.
4. NMQF: A Roadmap for Real-World Evidence Generation in Alzheimer’s Disease. Click here to view the white paper.
5. Person-Driven Outcome Measures Learning Collaborative - Applications Open! Click here to learn more and inquire about participation.
6. Speaking Up on ADA with Tony Coelho. Click here to view the one pager.
7. NCD Health Equity Framework Includes Recommendation Against Use of QALYs, Following BBB Recommendation. Click here to view the framework.
8. Value Our Health: Advancing Value Assessment Principles Centered on Patients and People with Disabilities. See below for details.
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, Obesity Management, 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.
Advocates are welcomed to sign a letter calling for CMS to reject Oregon’s waiver renewal application that relies on 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 also requested 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. Click here to view the waiver application sent to CMS. Click here to view and sign (scroll down to sign) a letter opposing Oregon’s use of QALYs by April 8, 2022. Click here to submit your own comments by April 13, 2022.
2. HHS Proposed Notice of Benefit and Payment Parameters Rule on Prohibited Discrimination under Section 1557
In its proposed rule, HHS took steps to refine the Essential Health Benefits nondiscrimination policy, as established under Section 1557 of the ACA, to ensure that benefit designs, and particularly benefit limitations and plan coverage requirements, are based on clinical evidence. HHS called out that benefit designs cannot discriminate based on an individual’s age, expected length of life, present or predicted disability, degree of medical dependency, quality of life, or other health conditions. Consistent with the National Council on Disability’s recommendations in its 2019 report on Quality-Adjusted Life years, PIPC stressed that: (1) plans should not be permitted to reference the discriminatory quality-adjusted-life-years (QALY) metric; (2) research should meet standards of patient-centeredness; and (3) full transparency should be required regarding sources used in decision making. As stated by NCD in its report recommending policymakers avoid the use of QALYs, Section 1557 applies to health insurance programs operated by recipients of federal financial assistance from HHS and therefore covered "health insurance programs should not rely on cost-effectiveness research or reports that gather input from the public on health preferences that do not include the input of people with disabilities and chronic illnesses." Click here to view PIPC’s letter. Click here to view NCD’s recommendation for HHS guidance clarifying that Section 1557 applies to health insurance programs receiving federal financial assistance.
3. Upcoming PCORI Funding Announcement to Fund Studies on the Science of Engagement
The Patient-Centered Outcomes Research Institute (PCORI) intends to release a PCORI Funding Announcement (PFA) on May 3, 2022, seeking to fund studies that build an evidence base on engagement in research. Initial funding will primarily focus on measure development and/or validation, particularly rapid measure development, leveraging opportunities to support teams who may have already begun measure development work. Applicants can also validate or adapt existing measures initially developed for this or different contexts (e.g., community-based participatory research, engagement in health care delivery). A smaller number of early awards will test engagement techniques using a combination of limited existing validated measures of engagement and measures of other research outcomes (e.g., participant recruitment and retention; diversity, equity and inclusion outcomes).
PCORI anticipates multiple funding cycles for this initiative, with the focus of subsequent PFAs to be updated as the evidence base grows. PCORI invites interested participants to review the full preannouncement on the website for details and register for a town hall on May 13, 2022, at 12:00 pm ET where researchers and stakeholders will learn more about this upcoming PFA. Please feel free to share this information with anyone who may be interested.
4. NMQF: A Roadmap for Real-World Evidence Generation in Alzheimer’s Disease
The National Minority Quality Forum (NMQF) released a white paper highlighting how real-world evidence (RWE) is able to include larger patient populations that are historically underrepresented in randomized controlled trials. The paper was drafted in partnership with Biogen about need for and importance of real-world evidence (RWE) as a complement to randomized clinical trial (RCT) data, while building diversity goals for both. NMQF has been at the forefront of highlighting the health equity implications of real-world evidence. Click here to view the newly published roadmap. Click here to view the NMQF paper in 2020 discussing how real-world evidence lacking representation drives health inequity in value assessment.
5. Person-Driven Outcome Measures Learning Collaborative - Applications Open!
The National Committee for Quality Assurance (NCQA) is launching a new learning collaborative for up to 10 organizations to develop and implement reporting methods for Person-Driven Outcome Measures.
With support from The John A. Hartford Foundation and The SCAN Foundation, this learning collaborative will offer organizations guidance and support on collecting and reporting the person-driven outcome measures for use in improving quality in service delivery. These measures are developed to be applicable for all populations with complex support needs. Findings from the learning collaborative will help shape the final reporting and audit guidelines, as well as inform future improvements and refinements to the measures. The learning collaborative will run from April 2022 to July 2023. Click here to learn more and inquire about participation.
6. Speaking Up on ADA with Tony Coelho.
The Disability Garrison podcast, hosted by Holly Carmichael and Michael Murray, seeks to inform listeners about issues that people with disabilities face every day. A series of podcasts were recorded with PIPC Chairman Tony Coelho. Tony spoke at length about his upbringing, disability diagnosis, darkest times, and finding his voice, and how it all ultimately led to his involvement in the Americans with Disabilities Act. Episodes include: Discovering Disability Identity with Tony Coelho (Part 1), Speaking up with Tony Coelho (Part 2), Shaping the ADA with Tony Coelho (Part 3), The Americans with Disabilities Act with Tony Coelho (Part 4). Click here to view entries from the "Because of ADA I Can" Campaign. Click here to view the one pager.
7. NCD Health Equity Framework Includes Recommendation Against Use of QALYs, Following BBB Recommendation
The National Council on Disability (NCD) released a Health Equity Framework in February, 2022. NCD’s framework included 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. This recommendations follows the NCD’s recommendation for 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. Click here to view the framework. Click here to view the NCD’s letter to Congress and here to view NCD’s presentation at the 2021 PIPC forum.
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 explaining 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.
8. 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.
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: Breast cancer patients are being forced to crowd-fund to afford Tucatinib, a lifesaving drug, that was rejected for coverage by the PBS because its cost effectiveness ratio was "unacceptably high."
- United Kingdom: Breast cancer patients are hoping that Olaparib, a lifesaving drug, will be approved for coverage in as they have been suffering from NICE's previous rejection of the drug due to cost-effectiveness reasons.
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.
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. The waiver application is now open for public comment at CMS.
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 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.
14. ICER's QALY-Based Study Topics: Alzheimer's Disease, Amyotrophic Lateral Sclerosis (ALS), Beta Thalassemia, COVID-19, Obesity Management, 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: ICER has decided to extend its timeline for its assessment of the beta-amyloid antibodies for early Alzheimer’s disease, so the timeline is to be determined. Research Protocol available. Draft Scoping Document available. Public Comments closed. 5/10/2022: Model Analysis Plan.
- Amyotrophic Lateral Sclerosis (ALS): Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. Public comments closed. 4/28/2022: Model Analysis Plan.
- Beta Thalassemia: Draft Evidence Report available. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available.
- COVID-19: Evidence Report available. Draft Evidence Report Available. Public comments closed. Model Analysis Plan available. Draft Scoping Document and Research Protocol available. 4/12/2022: Evidence Presentation. 5/10/2022: Final Evidence Report and Meeting Summary.
- Obesity Management: Draft Scoping Document available. Public comments closed. 3/31/2022: Revised Scoping Document. 4/7/2022: Research Protocol.
- Unsupported Price Increases Occurring in 2020 in California: Protocol available. 10/13/2022: Final Report.
- 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
Cycle 2 2022 -- Science of Engagement PFA Applicant Town Hall
May 13, 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.