1. PIPC Joins Over 60 Organizations Urging CMS to Reject QALY's in OR Waiver Renewal Application. Click here to read the letter.
2. GAO Calls for Nominations to PCORI Board of Governors. Click here to read more.
3. PCORI Board of Governors Meeting - Transcript Released. Click here to view the transcript.
4. HHS Proposed Notice of Benefit and Payment Parameters Rule on Prohibited Discrimination under Section 1557. See below for details.
5. Upcoming PCORI Funding Announcement to Fund Studies on the Science of Engagement. Click here for more details.
6. Person-Driven Outcome Measures Learning Collaborative - Applications Open! Click here to learn more and inquire about participation.
7. Speaking Up on ADA with Tony Coelho. Click here to view the one pager.
8. NCD Health Equity Framework Includes Recommendation Against Use of QALYs, Following BBB Recommendation. Click here to view the framework.
9. Value Our Health: Advancing Value Assessment Principles Centered on Patients and People with Disabilities. See below for details.
10. New Targeted PCORI Funding Announcement: Comparative Effectiveness of Interventions Targeting Mental Health in Individuals with Intellectual and Developmental Disabilities. See below for details.
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.
The Partnership to Improve Patient Care (PIPC), along with 62 leading advocacy groups representing patients and people with disabilities, submitted a comment letter to the Centers for Medicare and Medicaid Services (CMS) on Oregon's 1115(a) Demonstration Waiver renewal application. The letter expresses concern that Oregon's application would further entrench health inequalities and deny access to care for those with disabilities and chronic conditions through the use of the quality-adjusted-life-year (QALY) metric. The state's defense of its use of the discriminatory QALY, as well as its efforts to exclude FDA-approved drugs from coverage, are inconsistent with Oregon's stated goals of health equity. "We urge CMS to reject Oregon’s waiver renewal application, in accordance with its decision in 1992," the letter states. "A consistent policy across Medicare and Medicaid barring the use of QALYs will serve to improve health equity, access to medically necessary care, and compliance with disability rights laws." Click here to view the letter.
2. GAO Calls for Nominations to PCORI Board of Governors
The US Government Accountability Office has issued a call for nominations to fill seats on the PCORI's Board of Governors that will open when some members’ terms end in September. Board members represent a wide range of healthcare stakeholders, including clinicians, health plans, patients, and healthcare consumers. Nominate a candidate to join this diverse leadership body by May 10. Click here to read more. Click here to view the notice on the federal register.
3. PCORI Board of Governors Meeting - Transcript Released
At its March 8, 2022, meeting, the PCORI Board of Governors approved several slates of funding awards totaling about $71.5 million to support 12 new comparative clinical effectiveness research (CER) and methods studies, and three projects to facilitate implementation of PCORI-funded research findings in the treatment of urinary incontinence, care of patients with sickle cell disease, and mental health care.
The Board also approved development of two new PCORI Funding Announcements (PFAs)—one on health systems strategies to reduce disparities in hypertension management and control and the other on building an evidence base to stakeholder engagement in patient-centered CER. In addition, the Board adopted a new Research Agenda, which will help in outlining how PCORI will focus its patient-centered CER funding and other activities in the years ahead.
The Board also approved PCORI's provision of $25 million to support the second phase of the Learning Health Systems Training Program and received an overview about PCORI's work on maternal morbidity and mortality—a priority area for PCORI funding—from Executive Director Nakela L. Cook, MD, MPH. Click here to see the full transcript.
4. 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.
5. 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.
6. 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.
7. 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.
8. 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.
9. 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.
10. 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.
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.
- Canada: Rare disease advocates are lobbying the government to help save lives by implementing its own rare disease strategy.
- New Zealand: Only one of two brothers with CF is receiving Trikafta, the life-saving drug, while the other brother continues to suffer without government funding. Parents of CF patients are speaking up and fighting for Trikafta to be publicly funded. CF advocate is arguing for the government to dramatically increase Pharmac's budget so that they can cover Trikafta.
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.
Oregon's waiver application, submitted to CMS on February 18, 2022 defended 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. Disability and patient stakeholders have submitted comments to CMS asking that it reject Oregon’s use of the discriminatory QALY. Click here to read comments submitted by PIPC along with 62 other groups.
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. Governor Charlie Baker filed his health care bill, SB 2774, An Act Investing in the Future of our Health, on March 17, 2022. The bill expands the Health Policy Commission’s authority to determine the value of a drug beyond Medicaid to private payers. As written, the bill allows for use of cost-effectiveness assessments and analysis by a third-party, and it does not include a QALY ban. The bill requires disclosure of methods used by third parties and disclosures of potential limitations of that research. In a hearing on SB 2774 on April 11, patient advocates testified that the bill as written has the potential to discriminate and must include a QALY ban.
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 Presentation and Report available. Draft Evidence Report Available. Public comments closed. Model Analysis Plan available. Draft Scoping Document and Research Protocol available. 5/10/2022: Final Evidence Report and Meeting Summary.
- Obesity Management: Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. Public comments closed. 5/25/2022: Model Analysis Plan. 7/13/2022: Draft Evidence Report.
- 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
Advisory Panel on Healthcare Delivery and Disparities Research Spring 2022 Meeting
May 11, 2022
Click here for details.
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: Treatments for Acute Pain. Click here to view.
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.