— Resources, Roles, and Returning Value: Lessons from Patient- and Participant-Driven Research Communities. Click here to read the blog.
— Learning Drives Innovation in New PCORI Initiatives. Click here to learn more about the initiatives.
— Senators Want Answers on Disability Related Barriers to Health Care. Click here to read the article.
— New 'Science of Engagement' PCORI Funding Announcement Opens July 12. See details below.
— Don’t Let Payer Investments in QALYs Undermine California’s Health Equity Goals. Click here to read the blog.
— PCORI Approves Strategic Plan at Board of Governors Meeting. See details below.
— A Blueprint to Advance Patient-Centered Core Impact Sets. Click here to read the article.
— Person-Driven Outcome Measures Help Achieve Equitable Outcomes. Click here to read the article.
— Emerging Threats in States for Use of Discriminatory Metrics. See details below.
— What Happens in Countries Using QALYs and Cost-Based Thresholds? See details below.
— ICER's QALY-Based Study Topics. See details below.
— Upcoming Events and Webinars. See details below.
— AHRQ Effective Program Updates. See details below.
1. Resources, Roles, and Returning Value: Lessons from Patient- and Participant-Driven Research Communities
As Patient and Participant-Driven Research Communities (PDRCs) have become widespread, PCORI embarked on an initiative to understand the patient and participant-driven research landscape more fully and organize a learning network of patient and participant-driven research communities as a venue for them to share knowledge, coordinate activities, and develop resources. In partnership with NORC and AcademyHealth, PCORI assembled a Learning Network of eight PDRCs to create a forum for peer-to-peer learning. Three workgroups were subsequently formed to address challenges identified by learning network members with the goals of providing forums for sharing learnings and identifying generalizable lessons that could be shared broadly. Ultimately, 3 priority gap areas were identified: improving representation, enhancing digital engagement, and supporting sustainability. Click here to read the blog.
2. Learning Drives Innovation in New PCORI Initiatives. Our efforts to engage patients and stakeholders across the healthcare community, and focus on outcomes of interest to patients and those who care for them, are how we conduct Research Done Differently®. Patient-centered outcomes research (PCOR) inherently entails innovation, and innovation inherently entails learning, as experimentation generates evidence that leads to new questions and approaches through a "virtuous cycle" of learning. Three current PCORI initiatives provide excellent examples of this approach to innovation and iterative learning:
- Learning Health Systems 2.0 (in collaboration with the Agency for Healthcare Research and Quality [AHRQ])
- Health Systems Implementation Initiative
- Science of Engagement Initiative
3. Senators Want Answers on Disability Related Barriers to Health Care. An article in Disability Scoop covers a letter from federal lawmakers calling on the Government Accountability Office (GAO) to study the "barriers facing Americans with disabilities when they seek access to medical treatment services." The letter, signed by five Democratic Senators, states that “we write to request that the Government Accountability Office (GAO) conduct a study on barriers people with disabilities experience in accessing healthcare — including reproductive healthcare — in the United States,” the senators wrote. “Even though discrimination based on disability is prohibited by law, people with disabilities continue to face issues with accessibility when it comes to getting the healthcare they need, when they need it. The lack of accessible healthcare contributes to and exacerbates health disparities experienced by people with disabilities.”
4. New 'Science of Engagement' PCORI Funding Announcement Opens July 12. On July 12, 2022, PCORI's Online System will open for the submission of Letters of Intent (LOI) for the Science of Engagement PCORI Funding Announcement (PFA). LOIs will be due August 9, by 5 pm ET. Applications will be due November 15, by 5 pm ET. PCORI seeks to fund studies that build an evidence base on engagement in research, including:
- Measures to capture structure/context, process, and outcomes of engagement in research;
- Techniques that lead to effective engagement in research;
- How these techniques should be modified and resourced for different contexts, settings, and communities to ensure equity in engagement and research; and
- How engagement supports successful research, thereby advancing PCORI’s National Priorities for Health.
5. Chairman's Corner: Don’t Let Payer Investments in QALYs Undermine California’s Health Equity Goals. PIPC Chair Tony Coelho published a new blog that outlines concerns about the use of ICER's discriminatory value assessment metrics in California. In March 2022, ICER received a grant from the California Health Care Foundation, including support for its work to develop value assessments relying on QALYs. As part of this grant announcement, ICER recognized proudly that policymakers are now “regularly referencing ICER’s health-benefit price benchmarks” and made no reference to the potential for discrimination from use of an inherently discriminatory metric to drive health care decisions.
As Chair Coelho astutely points out, California’s diversity is among its greatest strengths, yet ICER has been strongly critiqued for its reliance on average metrics that fail to represent diverse populations. ICER touts how its value assessments can be leveraged to reduce costs, but in reality, its value assessments developed for use by payers will benefit payers at the expense of access to care for patients and people with disabilities — not their benefit.
"I urge California policymakers not to ignore the recommendations of so many disability rights leaders on this issue and to be skeptical about the ability of ICER to make fair health care recommendations on this vital issue," wrote Chair Coelho. "Reducing people to a one-size-fits-all standard of health care is not the equitable way to reduce health costs."
6. PCORI Approves Strategic Plan at Board of Governors Meeting. On June 14, the Patient-Centered Outcomes Research Institute (PCORI) approved its Strategic Plan (PDF) during its Board of governors meeting. The plan provides a roadmap for PCORI’s future funding of patient-centered comparative clinical effectiveness research (CER) as well as stakeholder engagement, dissemination and implementation of research results, and investments to enhance the nation’s CER infrastructure. The plan centers on five National Priorities for Health and incorporates a Research Agenda adopted by PCORI’s Board of Governors earlier this year. PCORI also approved new research funding opportunities for studies on reducing maternal health inequities, as well as research using telehealth to improve management of multiple chronic conditions. Click here to watch PCORI Executive Director Nakela L. Cook, M.D., discuss the PCORI Strategic Plan and share the "long-term goals that the organization aims to achieve through its implementation."
7. A Blueprint to Advance Patient-Centered Core Impact Sets. An article in Health Affairs covers a proposed solution from the National Health Council to advance patient-centeredness in the value assessments. "Stakeholders assessing the impacts of diseases and treatments on patients could benefit from having a resource to guide them on what patients report as most important. Thus, the National Health Council is offering a solution: patient-centered core impact sets (PC-CIS), a patient-prioritized list of the impacts a disease and its treatments have on patients, careers, and families...Unfortunately, PC-CIS do not currently exist. Thus, the NHC is spearheading a multi-stakeholder effort to create a PC-CIS blueprint and toolkit, a ‘how-to’ manual that can be used to develop a PC-CIS for a specific disease, related diseases, or population(s).”
8. Person-Driven Outcome Measures Help Achieve Equitable Outcomes. Sarita Mohanty and Terry Fulmer discuss the benefits of person-driven outcome measures in a Health Affairs article. "Ideally, person-driven outcome measures should be part of every patient’s care plan, but this approach is imperative for patients with complex care needs. For these measures to be successful, the patient must be an equal partner in both the development and assessment of the care they receive. The changes proposed by this system of measurement constitute a bold, fresh way of approaching and measuring care."
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.
ICER has received a grant from the California Health Care Foundation to develop annual unsupported price increase reports as well as a policymaker guide for using its research to determine “fair” access and pricing to drugs. Advocates are concerned of the implications of this grant given ICER’s reliance on QALYs. Simultaneously, the California legislature is considering AB 1130, which would establish the Office of Health Care Affordability to develop policies for lowering health care costs for consumers, set and enforce cost targets, and create a state strategy for controlling the cost of health care. This bill does not currently contain a ban on the use of the QALY.
On April 27, 2022, SB 8901 was introduced in the New York Senate, which would import QALYs from Canada by directly referencing the QALY-based prices paid for prescription drugs in four Canadian provinces. It has passed out of the Finance Committee and been referred to the Rules Committee. Click here to view the bill. Click here to learn more about state efforts to import discriminatory QALYs from Canada.
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. Click here to view letters from Disability Rights Oregon and Paul Terdal. Click here to view the letter from Patients Rights Action Fund.
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.
International News: What Happens in Countries Using QALYs and Cost-Based Thresholds?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- New Zealand: A community fundraising attempt is helping a nine-year-old epilepsy patient afford a very expensive drug that Pharmac does not fund.
- United Kingdom: Leukemia patient is appealing to the public to help raise money for the potential need for CAR-T therapy, not funded by the NHS, while she waits for a bone marrow transplant that is funded by the NHS
ICER's QALY-Based Study Topics
The Institute for Clinical Economic Review (ICER) conducts cost effectiveness studies for insurers using the cost-per-QALY methodology. ICER provides guidance on its website for patients and patient advocates to provide direct input related to their experiences with the disease. Click here to provide patient input. Click here to view the topics and deadlines.
- Amyotrophic Lateral Sclerosis (ALS): Draft Evidence Report available. Public comments open until 7/13/2022. 8/4/2022: Evidence Report. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available.
- Menopause: Vasomotor Symptoms: Revised Scoping Document available. Draft Scoping Document available. Public comments closed. 7/7/2022: Research Protocol. 8/23/2022: Model Analysis Plan.
- Alzheimer's Disease: 10/5/2022: Model Analysis Plan. Research Protocol available. Revised Scoping Document available.
- Beta Thalassemia: Evidence Report available. Evidence Presentation available. 7/19/2022: Final Evidence Report and Meeting Summary. Draft Evidence Report available. Public comments closed. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available.
- COVID-19: Final Evidence Report and Meeting Summary available. Evidence Presentation and Report available. Draft Evidence Report Available. Public comments closed. Model Analysis Plan available. Draft Scoping Document and Research Protocol available.
- Obesity Management: Model Analysis Plan available. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. Public comments closed. 7/13/2022: Draft Evidence Report. 8/31/2022: Evidence Report.
- Multiple Sclerosis: CIS, RRMS, and SPMS: Research Protocol available. 8/17/2022: Model Analysis Plan. Revised Scoping Document available. Draft Scoping Document available. Public comments closed.
- Gene Therapies for Hemophilia A and B: Revised Scoping Document available. 7/28/2022: Model Analysis Plan. Draft Scoping Document available. Public comments closed.
- 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.
- Asthma: Final Evidence Report and Meeting Summary available. Evidence Presentation available. Evidence Report available.
Upcoming Events and Webinars
Science of Engagement PFA Applicant Town Hall
July 19, 2022
Click here for details.
Using Real-World Evidence on Therapy Adherence to Demonstrate Value
July 20, 2022
Click here for details.
Board of Governors Meeting
July 26, 2022
Click here for details.
Medical Journal Articles
Can Alternative Payment Models and Value-Based Insurance Design Alter the Course of Diabetes in the United States? Click here to view.
A Pragmatic Guide to Assessing Real Option Value for Medical Technologies, click here to view.
Stakeholder Engagement Infrastructure to Support Multicenter Research Networks: Advances from the Clinical Research Networks Participating in PCORnet, click here to view.
Use of Pragmatic and Explanatory Trial Designs in Acute Care Research: Lessons from COVID-19, click here to view.
Impact of the COVID-19 Pandemic on Healthcare Resource Utilization Across Selected Disease Areas in the USA, click here to view.
Total Cost of Care Differences in National Comprehensive Cancer Center (NCCN) Concordant and Non-Concordant Breast Cancer Patients, click here to view.
The Role Of Prices In Excess US Health Spending, click here to view.
Application of Quantitative Bias Analysis for Unmeasured Confounding in Cost–Effectiveness Modelling, click here to view.
Seriously Ill Individuals—A Canary in the Coal Mine for Medicare’s Transition to Accountable Health Care, click here to view.
Perceptions and Use of Telehealth Among Mental Health, Primary, and Specialty Care Clinicians During the COVID-19 Pandemic, click here to view.
Effect of Electronic Symptom Monitoring on Patient-Reported Outcomes Among Patients With Metastatic Cancer: A Randomized Clinical Trial, click here to view.
- Systematic Review: Schedule of Visits and Televisits for Routine Antenatal Care. Click here to view.
- Systematic Review: Telehealth for Women's Preventive Services. 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: Maternal and Child Outcomes Associated With the Special Supplemental Nutrition Program for Women, Infants, and Children (WIC). Click here to view.
- Research Report: Research Gaps in Women’s Health: 2022. Click here to view.
- 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.
- Integrated and Comprehensive Pain Management Programs: Effectiveness and Harms. Click here to view.