— Leading Clinical Trial Recruitment Strategies Center Around Real-World Data. Click here to read the article.
— PIPC Holds 13th Annual Forum. Click here for details.
— More than 40 Leading Organizations Join PIPC Comment Letters on Efforts to Advance Health Equity in Value Assessments. See below for details.
— The German Health Care System and its Impact on Patient Access – Lessons for the U.S. Click here to read more.
— Notice of Upcoming PCORI Funding Announcement – Advancing the Science of Engagement – Cycle 1 2023. Click here for details.
— NCD: Alternatives to QALY-Based Cost-Effectiveness Analysis for Determining the Value of Prescription Drugs and Other Health Interventions. Click here to view the report.
— Health and Human Services’ Civil Rights Division Looks to Overhaul Its Essential IT System. Click here to view 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. Leading Clinical Trial Recruitment Strategies Center Around Real-World Data. As detailed in an article in MedCityNews, large pharmacy and health care retailers, such as Walgreens, CVS, and Walmart are reaching into real-world patient data through clinical trial recruitment. There is a growing movement to break down health data silos and offer a solution to barriers that have plagued the industry for years—chief among them—poor recruitment, which is often cited as the primary reason for clinical trial failure. These retail giants stand to gain access to significant patient data including electronic health records (EHRs), lab results, retail pharmacy data, and insurance claims. The data can provide valuable insights that lead to more informed and inclusive clinical trial design. Click here to read more.
2. PIPC Holds 13th Annual Forum. On December 15, 2022, the Partnership to Improve Patient Care held its 13th Annual Forum highlighting the goals of Inclusion in Health Care Research and Value Assessment. Sara van Geertruyden, Executive Director of PIPC, emphasized the importance of including patients and people with disabilities in research and decision-making processes throughout the health care system and emphasized the discriminatory implications of the QALY and similar metrics. The forum consisted of two panel discussions. The first panel involved a bipartisan, bicameral discussion about priorities for the next Congress, including efforts to extend a ban on use of QALYs across federal programs and support robust engagement of patients and people with disabilities as the administration implements the IRA’s policies related to drug pricing. The second panel included Donna Cryer, founder and CEO of the Global Liver Institute, Kenny Mendez, President and CEO of the Asthma and Allergy Foundation of America (AAFA), and Thayer Roberts, Deputy Director of PIPC. Click here to read the full forum summary.
3. More than 40 Leading Organizations Join PIPC Comment Letters on Efforts to Advance Health Equity in Value Assessments. More than 40 leading groups representing patients, people with disabilities, older adults, and communities of color joined the Partnership to Improve Patient Care (PIPC) in comment letters to the Institute for Clinical and Economic Review (ICER) and the Innovation and Value Initiative (IVI) regarding the methodological flaws of the health technology assessment (HTA) and its implications for health equity. The letter's recommendations underscore the importance of addressing three significant challenges for value assessment: inclusive and representative data, methodologies that value health equity, and engagement of all people impacted directly by the condition being assessed. A true commitment to health equity will require ongoing engagement in this work as new questions arise as part of this process.
4. The German Health Care System and its Impact on Patient Access – Lessons for the U.S. Proposals to control health care cost growth in the U.S. have frequently involved discussion of the evidentiary standards followed in conducting the comparative clinical effectiveness research and/or value assessments that guide policy decision-making. Many other countries, including England and Canada, rely on assessments of a treatment’s cost-effectiveness to set prices. Incorporating use of a one-size fits all metric from another country by attempting to adapt it for use in the U.S. has justly caused concern among stakeholders, particularly patients and persons with disabilities. As public awareness of the quality-adjusted life year's (QALY) — an inherently discriminatory metric that cost effectiveness assessments (CEA) typically rely on — implications for discrimination have grown, U.S. policymakers promoting drug price reform have sought to identify alternatives that rely on comparative clinical effectiveness research (CER) instead of cost-effectiveness. Prominent thought leaders, including President Biden, have mentioned that the U.S. could model the German system's coverage and reimbursement policies. The German system holds important lessons for policy-makers in the U.S. It is important to understand how the German system works, including flaws in the German approach to setting drug prices and its impact on patient access to care, including people with disabilities, older adults, and people with chronic conditions; not just those in “good” health. This issue brief examines the use of CER in Germany with a goal of informing a U.S. audience as to how, moving forward, U.S. policymakers can do better by investing in solutions that keep patients at the forefront of our health care decision making. Click here to read more.
5. Notice of Upcoming PCORI Funding Announcement – Advancing the Science of Engagement – Cycle 1 2023. The Patient-Centered Outcomes Research Institute (PCORI) will release a PCORI Funding Announcement (PFA) on January 10, 2023, seeking to fund studies that build an evidence base on engagement in research. Letters of Intent are due by February 7, 2023, by 5:00 PM (ET). 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 healthcare 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 is inviting interested parties to review the full announcement on their website for details and register for the applicant town hall on January 23, 2023, at noon (ET), where researchers and stakeholders learned more about this PCORI funding initiative.
6. NCD: Alternatives to QALY-Based Cost-Effectiveness Analysis for Determining the Value of Prescription Drugs and Other Health Interventions. On November 28, 2022, the National Council on Disability published its new report supplementing the 2019 report on quality-adjusted life years describing the design and discriminatory impact of QALY-based cost-effectiveness analysis (CEA) – a method of comparing the cost and effectiveness of two or more healthcare interventions and determining if the value of an intervention justifies its cost. As described by NCD, "This brief highlights alternatives to standard CEA: augmented or extended traditional CEA; multi-criteria decision analysis; frameworks for determining value to individual patients; and organizations conducting value assessments or advancing methodologies outside QALY-based CEA. The brief then offers recommendations to avoid the use of QALY.” Click here to view the report.
7. Health and Human Services’ Civil Rights Division Looks to Overhaul Its Essential IT System. As detailed in an article from Nextgov, The Health and Human Services Department’s (HHS) Office of Civil Rights (OCR) filed a request for information on Monday seeking recommendations and solutions to replace its legacy Program Information Management System, or PIMS — the system that controls all of OCR’s activities for complaints, reviews and correspondences about discrimination or rights violations — with a “new, modernized system to manage and track OCR activities and be the system of record." Additionally, HHS is seeking for the new service provider to support all parts of the new system within OCER, such as the migration of more than two terabytes of data to the new system, as well as highly trained and skilled workers — namely, consultants and analysts. Click here to read the article.
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.
On August 31, 2022, California Attorney General Rob Bonta sent letters to hospital CEOs across the state requesting information about how healthcare facilities and other providers are identifying and addressing racial and ethnic disparities in commercial decision-making tools. The request for information is the first step in a DOJ inquiry into whether commercial healthcare algorithms – types of software used by healthcare providers to make decisions that affect access to healthcare for California patients – have discriminatory impacts based on race and ethnicity. Click here to view the Attorney General’s letter. Click here to view a response from advocates urging the Attorney General to include QALYs and similar metrics in his investigation. In March 2022, ICER 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.
On October 6, 2022, the Oregon Health Evidence Review Commission (HERC) Value based Benefits Subcommittee discussed a proposed plan for using QALYs, including proposals to redact the word QALY from the HERC’s deliberations without barring the use of the metric in making decisions. The meeting followed CMS approval of Oregon’s waiver application, which does not bar the use of QALYs in the interim. Click here to read comments submitted by PIPC along with 62 other groups asking CMS to reject the waiver. Click here to view the CMS-approved waiver. At the HERC meeting, the Oregon Health Authority expressed its intent to continue using a prioritized list, shifting it from use under a waiver to use as part of the State Plan Amendment after the waiver expires in 2027. The HERC decided to accept comments on options to either adjust their use of QALYs or to cease mentioning QALYs at meetings and redact their mention from published articles, despite vehement opposition from advocates based on the implications for discrimination and health equity, as well as the potential lack of transparency related to their use to make decisions if simply redacted from studies considered by the HERC. Click here to view the letter from almost 50 advocacy groups asking the HERC to stop using QALYs, to support Oregon’s health equity goals and to lead in developing alternative coverage strategies that do not discriminate. Click here to view testimony from PIPC Chairman Coelho to the Oregon HERC expressing his support for the Americans with Disabilities Act and the implications of QALYs for violating existing federal civil and disability rights laws. Click here to view the options that continue to be under consideration by the HERC to continue using QALYs. Click here to view all of the comments shared with PIPC.
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.
- Canada: A CF advocate has lobbied for years to get the government to fund Trikafta for those that suffer with CF and now she is fighting for the government to develop a rare drug strategy.
- New Zealand: A patient advocate is criticizing the amount of time it has taken Pharmac to announce that they are finally considering funding Trikafta for CF patients.
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.
- Sickle Cell Disease: Research Protocol available. 02/27/2023: Model Analysis Plan. Revised Scoping Document available. Draft Scoping Document available. Public Comments closed.
- Non-Alcoholic Steatohepatitis: Model Analysis Plan available. 2/16/2023: Draft Evidence Report. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. Public comments closed.
- Menopause: Vasomotor Symptoms: Evidence Report and Presentation available. 1/23/2023: Final Evidence Report and Meeting Summary. Draft Evidence Report available. Model Analysis Plan available.
- Alzheimer's Disease: Draft Evidence Report available. Public comments open until 2/2/2023. 3/1/2023: Evidence Report. Model Analysis Plan available. 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: Final Evidence Report and Meeting Summary available. Evidence Presentation available. Draft Evidence Report available. Model Analysis Plan available. Research Protocol available.
- Multiple Sclerosis: CIS, RRMS, and SPMS: Evidence Report available. 1/20/2023: Evidence Presentation. Draft Evidence Report available. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available.
- Gene Therapies for Hemophilia A and B: Final Evidence Report and Meeting Summary available. Evidence Presentation available. Evidence Report available. Draft Evidence Report available. Public comments closed. Model Analysis Plan available. Revised Scoping Document available. Draft Scoping Document available.
- Unsupported Price Increases Occurring in 2020 in California: Final Report available. Protocol available.
- 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
Cycle 1 2023 -- Broad Pragmatic Studies (Standing PFA) Applicant Town Hall
January 13, 2013
Click here to view.
Cycle 1 2023 -- Prevention, Early Identification, and Treatment of Delirium in Older Adults Targeted PFA Applicant Town Hall
January 17, 2023
Click here to view.
Board of Governors Meeting
February 14, 2023
Click here to view.
Medical Journal Articles
Payer–patient Engagement Framework to Strengthen Ethical Formulary Decision-making in Rare Disease Arena in the USA. Click here to view.
The Impact on Cost-Effectiveness of Accounting for Generic Drug Pricing: Four Case Studies. Click here to view.
A Perspective on Life-Cycle Health Technology Assessment and Real-World Evidence for Precision Oncology in Canada. Click here to view.
Identifying And Exploring Bias in Public Opinion on Scarce Resource Allocation During The COVID-19 Pandemic. Click here to view.
Machine Learning Applied to Patient-Reported Outcomes to Classify Physician-Derived Measures of Rheumatoid Arthritis Disease Activity. 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.
Impact of the COVID-19 Pandemic on Healthcare Resource Utilization Across Selected Disease Areas in the USA, 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.
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: Analysis of Requirements for Coverage with Evidence Development (CED) - Topic Refinement. Click here to view.
Technical Brief: Infection Prevention and Control for the Emergency Medical Services and 911 Workforce. Click here to view.
Systematic Review: Management of Infantile Epilepsies. Click here to view.
Systematic Review: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder. Click here to view.
Systematic Review: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
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: 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.