— Senate Republicans Urge HHS to Ban the Use of QALYs and Other Discriminatory Comparative Effectiveness Research. Click here to read the letter.
— GAO Makes Appointments to PCORI Governing Board, New Chair and Vice Chair Named. Click here to read the press release.
— CDER Official Reflects on a Decade of Patient-Focused Drug Development. Click here to read the article.
— Accelerating Patient-Centered Outcomes Research through Synthetic Health Data Generation. Click here to read the article.
— Exploring Equity in Health Care Value. Click here to read the article.
— PCORI Funding Opportunity Addresses Telehealth to Treat Patients with Multiple Chronic Conditions. Click here to learn more.
— Patient Insights & Current Vs. Next-Gen Patient Engagement. Click here to read the article.
— Over 40 Leading Patient & Disability Groups Called on Congress to Ensure Drug Price Negotiations Don't Discriminate. Click here to read the letter.
— 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. Senate Republicans Urge HHS to Ban the Use of QALYs and Other Discriminatory Comparative Effectiveness Research. On September 14 a group of Senate Republicans sent a letter to Secretary Becerra and Administrator Brooks LaSure about the implementation of the Medicare negotiation provision in the Inflation Reduction Act expressing concerns about the potential implications to patient access. The letter specifically noted the fact that the CBO used the QALY when modeling the bill, made clear the QALY is a discriminatory metric, and requested HHS commit to excluding any research including the QALY and lay out a concrete plan to enforce the legislation’s ban on the use of discriminatory comparative effectiveness research. The letter also called for HHS to lay out a plan for robust engagement with affected stakeholders – including patients, people with disabilities, and caregivers – throughout the process noting that, “the exclusion of patient voices from policymaking poses potentially catastrophic consequences, particularly for individuals living with rare diseases or with disabilities.”
2. GAO Makes Appointments to PCORI Governing Board, New Chair and Vice Chair Named. On September 22, 2022, Gene L. Dodaro, Comptroller General of the United States and head of the U.S. Government Accountability Office (GAO), announced the appointment of six new members to the Governing Board of the Patient-Centered Outcomes Research Institute (PCORI). Dodaro also reappointed two members and designated the Governing Board’s new chair and vice chair. Dodaro said, “The individuals named today bring strong credentials and experience to the PCORI Governing Board’s mission of funding and evaluating patient-centered research, with the goal of helping patients make better health-care decisions." Click here to read the press release.
3. CDER Official Reflects on a Decade of Patient-Focused Drug Development. Dr. Theresa Mullin, Ph.D., associate director for strategic initiatives within the Food and Drug Administration (FDA) Center for Drug Evaluation and Research (CDER), said that patient-focused drug development is "a systematic approach to help ensure that patients' experiences and their perspectives and needs and priorities are captured and meaningfully incorporated into drug development and benefit-risk assessment." Using the patient-focused allows FDA staff and industry to involve patients and their caregivers in drug development, clinical trials, and regulatory decisions and identify what outcomes are important to them, according to Dr. Mullin. Click here to read the article.
4. Accelerating Patient-Centered Outcomes Research through Synthetic Health Data Generation. An article in HealthITbuzz discussed the the ONC's Synthetic Health Data Project and its aim to produce high-quality synthetic health data and more varied and plentiful synthetic health records to support Patient-Centered Outcomes Research (PCOR) for patients with complex care needs. Click here to read the article.
5. Exploring Equity in Health Care Value. This article from the Innovation and Value Initiative (IVI), published in STAT News, highlights how IVI is improving both the science and the practice of value assessment to make healthcare decision-making more transparent, equitable and representative of patients and their families. IVI has been engaging early with patient communities and thought leaders to consider issues and opportunities to improve health equity in value assessment. The article notes that to address these challenges, IVI launched a multistakeholder Equity Initiative this past April, which is "focused on defining measurable and actionable changes to value assessment frameworks that realign processes for greater transparency, representation, and prioritize data generation and economic health development methods to support health equity." Click here to read the article.
6. PCORI Funding Opportunity Addresses Telehealth to Treat Patients with Multiple Chronic Conditions. The COVID-19 pandemic spurred a fast expansion of the use of telehealth to care for patients remotely. An upcoming Targeted PCORI Funding Announcement will fund studies seeking to determine the comparative clinical effectiveness of different approaches to telehealth to best treat patients with multiple chronic conditions, particularly among vulnerable populations. Up to $50 million is available under this funding opportunity, which is open now. Letters of Intent (LOIs) are due October 4, 2022, and applications are due January 10, 2023. Click here to learn more.
7. Patient Insights & Current Vs. Next-Gen Patient Engagement. As detailed in an article from the Clinical Leader, the Food and Drug Administration (FDA) and European Medicine Agency (EMA), along with the pharmaceutical industry, are prioritizing patient engagement in their agendas. Certain guidelines proposed including patient perspectives but the guidelines often lack theoretical development and empirical guidance. "The result is often that patient engagement turns into an ethical token of limited value, and the risk is that industry decisions are made based on flawed insights." This article details certain critiques of "some of the existing guidelines for patient engagement, with particular focus on how they enter the research field lacking the rigor of empirical research standards. Finally, it concludes with recommendations for patient engagement within boundaries that would not overstep onto research but instead complement it with diverse forms of interaction with patients." Click here to read the article.
8. Over 40 Leading Patient & Disability Groups Called on Congress to Ensure Drug Price Negotiations Don't Discriminate. As the Inflation Reduction Act is being implemented, patients and people with disabilities have asked that the Department of Health and Human Services (HHS) to establish a decision-making process that is informed by meaningful input from patients and other affected stakeholders related to its drug price negotiation process, including any process the Secretary uses for determining the “fair price” for selected drugs under Medicare in instances when evidence of a drug’s value is being considered. More than 40 leading groups representing patients, people with disabilities, and older Americans joined the Partnership to Improve Patient Care (PIPC) in sending a letter to Congressional Democratic leadership emphasizing this point prior to the bill's passage. The letter also urged lawmakers to recognize and communicate to HHS that current safeguards against devaluing the lives of individuals with disabilities, older adults, and other subpopulations experiencing health disparities, as well as protections regarding the use of comparative clinical effectiveness research in Medicare, under the existing provisions of Section 1182 of the Affordable Care Act must be adhered to as part of the process of negotiation outlined in the legislation. Click here to read the letter. Click here to read PIPC Chairman Tony Coelho’s opinion highlighting the omitted language on affected stakeholder input.
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. Click here to view the letter to CHCF from advocates.
On September 22, 2022, the Nevada Patient Protection Committee received a presentation highlighting efforts have been undertaken in other states with the goal of lower drug prices including establishing a Prescription Drug Affordability Board, relying on ICER’s unsupported price increase report, and referencing the prices of Canadian drugs, risking reliance on metrics that discriminate with potential implications for access to care, similar to the experience of patients and people with disabilities in Canada. The Committee meeting did not include participation from patients and people with disabilities, and the Committee did not discuss the discriminatory implications of the QALY. The next meeting is on October 19, 2022. Click here for more information and to register to participate the week prior to the meeting. The PPC allows testimony to be submitted and opportunities to comment during the meeting
On October 6, 2022, the Oregon Health Evidence Review Commission (HERC) Value based Benefits Subcommittee will discuss a proposed plan for using QALYs. Click here to register. The meeting follows Oregon’s waiver application, submitted to CMS on February 18, 2022, defending the state’s use of QALYs and continuing 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 current waiver was extended through September 30, 2022, providing additional time to review the demonstration extension application. Click here to view the letter from CMS. Click here to view letters from Disability Rights Oregon and Paul Terdal. Click here to view the letter from Patients Rights Action Fund.
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: Pharmac recently informed CF advocates that it will not fund Trikafta, a life-saving drug, which is shattering patients' hopes for access. to the treatment. A CF patient's family was considering moving to New Zealand where the drug was funded until the recent decision. Some families are now becoming "medical refugees" due to a lack of funding as they have moved overseas to one of the thirty other countries where Trikafta is available. Also, diabetes patients are expected to continue to be greatly impacted by type 1 diabetes if the government does not make access to new insulin delivery technology a priority.
- United Kingdom: MS patients are continuing to suffer as Fampridine remains out of reach two years after approval by NHS. An MS patient is being forced to receive treatment of HSCT in Mexico because it is not offered in his country.
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): Final Evidence Report and Meeting Summary available. Evidence Presentation available. Draft Evidence Report available. Public comments closed. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available.
- Menopause: Vasomotor Symptoms: Model Analysis Plan available. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. 10/11/2022: Draft Evidence Report.
- Alzheimer's Disease: 10/5/2022: Model Analysis Plan. Research Protocol available. Revised Scoping Document available.
- Beta Thalassemia: Final Evidence Report and Meeting Summary available. Evidence Presentation available. 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: Evidence Report available. Evidence Presentation available. 10/17/2022: Final Evidence Report and Meeting Summary. Draft Evidence Report available. Model Analysis Plan available. Research Protocol available.
- Multiple Sclerosis: CIS, RRMS, and SPMS: Model Analysis Plan. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. 10/17/2022: Draft Evidence Report.
- Gene Therapies for Hemophilia A and B: Draft Evidence Report available. Public comments open until 10/11/2022. Model Analysis Plan available. Revised Scoping Document available. 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
PCORI Annual Meeting
October 26-27, 2022
Click here to view.
Patient-Centered Pain Care Using Artificial Intelligence and Mobile Health Tools: A Randomized Comparative Effectiveness Trial. Click here to view.
The Use of Generic Patient-Reported Outcome Measures in Emergency Department Surveys: Discriminant Validity Evidence for the Veterans RAND 12-Item Health Survey and the EQ-5D. Click here to view.
Challenges and Opportunities for Advancing Patient-Centered Clinical Decision Support: Findings from a Horizontal Scan, click here to view.
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.
AHRQ Effective Program Updates
Research Report: Health System Panel to Inform and Encourage Use of Evidence Reports. 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.