— Efficiency Versus Health Equity in Health Technology Decisions. Click here to read the article.
— Taking Action to Address Health Equity in Oncology. Click here to read the study.
— PIPC Submits Comments on ICER's Assessment of Obesity Management Treatments. Click here to read the letter.
— Let the Work Begin – Is the Shift Towards Patient-Centered Healthcare Research Actually Starting? 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.
— Beryl Institute: The Impact of Bias on Health Equity and the Human Experience. Click here to purchase a copy of the paper.
— When Is Obesity Care Worth It? Click here to read the article.
— PCORI Approves $258 Million to Fund 26 New Research Studies. Click here to view the release.
— Scaling the Patient Perspective in Health Technology Assessments. 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. Efficiency Versus Health Equity in Health Technology Decisions. As detailed in a featured article from ISPOR, "health technology assessment (HTA)-related decisions have almost exclusively been driven by quantitative evidence for a new technology's efficiency, namely its comparative- and cost-effectiveness, as well as its predicted budget impact." HTAs have been used for decades to inform policy makers and assist them with healthcare resource allocation but the article notes that "considering efficiency alone has a potential to negatively affect fairness, or equity, in terms of healthcare resource distribution." Throughout the article, ISPOR discusses the challenge of quantifying fairness, the underutilization of cost-effectiveness analyses, and the tradeoffs between efficiency and fairness. Notably, gaps in access to healthcare resources will continue to grow if the inclusion of equity in HTA is not adopted. Click here to read the article.
2. Taking Action to Address Health Equity in Oncology. In the last decade, a study from AJMC explains, "biopharmaceutical companies have discovered and developed numerous new treatment options that have improved outcomes and prolonged survival for many patients with cancer. Ontada has partnered with many of these life science firms to conduct studies on these novel therapies, and with clinicians by providing a suite of technology solutions and education designed to enable the efficient, effective, evidence-based practice of medicine." The article notes that health equity is not a new problem, as people of color and those with lower incomes have been disproportionately impacted, specifically by COVID-19. Collaborating to foster health equity, identifying social determinants of health (SDOH) correlated with patient access and outcomes, among other efforts have led Ontada to lead initiatives "to build a comprehensive and expansive view of patients as well as their care and outcomes." Click here to read the study.
3. PIPC Submits Comments on ICER's Assessment of Obesity Management Treatments. In a letter to the Institute for Clinical and Economic Review (ICER), the Partnership to Improve Patient Care (PIPC) provided feedback on ICER's assessment of treatments for obesity management. PIPC offered criticism of ICER's model for assuming the burden of obesity is limited solely to cardiovascular disease (CVD) risk, as well as for ignoring the benefits of treatment on physical function, which can have a significant impact on a patient’s quality of life. PIPC is also disappointed for ICER's reliance on the quality-adjust-life-years (QALY) which has discriminatory implications. "The prevalence of obesity in the United States is growing, and it is important that appropriate interventions are available to individuals that address obesity and limit risk of associated diseases," wrote PIPC Chair Tony Coelho. "PIPC encourages ICER to make changes in its model to ensure it is representative of a real-world population, including using real world evidence where possible and recognizing the broad implications of obesity beyond risk of CVD."
4. Let the Work Begin – Is the Shift Towards Patient-Centered Healthcare Research Actually Starting? As detailed in an article from the Innovation and Value Initiative (IVI), "Patient engagement in healthcare research is having a moment. Conferences, public policies, and thought leaders have finally begun to echo what so many of us have said for decades – patients, with all their rich complexities, must be at the center of healthcare research as full partners. And, healthcare research and value assessment must find ways to reduce health inequity, not exacerbate it." Recognizing this urgency to new "scientific approaches, the IVI and Sick Cells are collaborating on numerous initiatives to ensure that we ask the research questions that matter the most to people living with serious health conditions." This work builds off of IVI's report from its 2021 Methods Summit. Click here to read the article.
5. 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.
6. Beryl Institute: The Impact of Bias on Health Equity and the Human Experience. The Beryl Institute recently published a paper that identifies what bias looks like in healthcare settings and acknowledges its serious and lasting consequences on patient outcomes and overall experience. In this paper, healthcare leaders share what they have learned about addressing bias and the immediate need to begin rebuilding trust with those they serve. Patients and families share their lived experiences of being exposed to bias and how it has impacted their overall approach to healthcare and what needs to change in the future. The paper offers tangible ways in which organizations, leaders and the community-at-large, can create a path to health equity through intentional action to address bias. Click here to purchase a copy of the paper. Click here to view the patient reflections accompanying the paper.
7. When is Obesity Care Worth It? A recent article from ConscienHealth questions ICER's recent draft analysis on effectiveness and value for the current options in obesity medicine, along with two new studies that looks at the value for money in obesity care and the worth of medicines. The article noted that "this broad question of value may well be impossible to answer in an unqualified way. The simple reason is that obesity is heterogeneous and the response to therapy is extremely variable. With earlier medicines (and really just about any treatment) the response can range from nil to life changing. So, the value is different for different people in different circumstances." The article details that in the ICER analysis, it deems semaglutide (Wegovy) less cost effective than another medicine on the market, despite being less effective and expensive. The ICER study found that semaglutide cleared a threshold of $150,000 per QALY over a 30-year time period for cost effectiveness and stated that the QALY thresholds "provide a very crude benchmark for cost effectiveness and thus, some people argue that they QALYs should not be used for this purpose." Click here to read the article.
8. PCORI Approves $258 Million to Fund 26 New Research Studies. The Patient-Centered Outcomes Research Institute (PCORI) approved funding awards totaling $258 million to support 26 new research studies on July 26, 2022. These studies aim to fill evidence gaps and improve health care decision making for a range of high-burden conditions among adults and children, including asthma, bone fractures, migraines, intellectual and developmental disabilities, and sinus infections. Five comparative clinical effectiveness research (CER) studies focus on improving mental health and another four CER studies focus on heart conditions, which have both continued to worsen in the U.S. amid the COVID-19 pandemic. Additionally, PCORI announced about $3.9 million for two projects that aim to promote wider, faster adoption of useful findings from completed PCORI-funded CER studies into typical care practices. Click here to view the release.
9. Scaling the Patient Perspective in Health Technology Assessments. As detailed in an article in Health Affairs, payers have begun to adopt formalized health technology assessments (HTAs) "in an effort to allocate health care resources most efficiently to the highest value health interventions." The authors note that an HTA "estimates the value of health interventions by estimating their net clinical and economic benefits." The problem with taking the HTA approach is that "current standards for the conduct of HTAs may not accurately represent the value to patients of specific interventions because such standards do not measure benefits and costs from the patient perspective. A core pillar of patient-centered research and care is the appropriate selection and use of patient input—for example, around which clinical and economic outcomes to elevate—in designing studies that generate outcomes that matter to patients. Many research teams and disciplines have begun to adopt patient-important outcomes and to account for patient perspectives in decisions about treatment options." 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.
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.
The Colorado Department of Insurance staff on the Prescription Drug Affordability Board briefed stakeholders on its draft Affordability Review rule on August 10, 2022. They will provide a briefing on the draft Upper Payment Limit rule on September 21. Click here to register. Colorado’s law creating the Board included a bar on use of QALYs in establishing the upper payment limit, though it has not been referenced in its materials. Click here to register for the next meeting of the full Board on August 26.
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 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 received a clinical recommendation from the Pharmacology and Therapeutics Advisory Committee to fund Trikafta for people six and older and Pharmac is admitting that it would save money in the long run. However, MS patients are still anxiously awaiting for Pharmac to provide funding the life saving drug, but negotiations on the price has not yet finished. Pharmac is claiming that it wants to fund Trikafta but that its current budget is not sufficient enough to provide funding for MS 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): Evidence Report available. Evidence Presentation available. 9/19/2022: Final Evidence Report and Meeting Summary. Draft Evidence Report available. Public comments closed. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available.
- Menopause: Vasomotor Symptoms: 8/23/2022: Model Analysis Plan. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. Public comments closed.
- 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: 8/31/2022: Evidence Report. Draft Evidence Report available. Public comments closed. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available.
- 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: Model Analysis Plan available. 9/13/2022: Draft Evidence Report. 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 Engagement Awards October 2022 Cycle -- Applicant Office Hours (Two)
September 7, 2022
Click here to view.
PCORI Board of Governors Meeting
September 19-20, 2022
Click here to view
Medical Journal Articles
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
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.