— Over 40 Leading Patient & Disability Groups Call 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.
— A Step Backward — No Voice for Stakeholders in Updated Reconciliation Provisions. Click here to read the blog.
— Scaling the Patient Perspective in Health Technology Assessments. Click here to read the article.
— Disability and Patient Advocacy Groups Raise Concerns Over CHCF's Decision to Fund ICER. Click here to read the letter.
— DOJ and HHS Issue New Guidance on Nondiscrimination in Telehealth. Click here to view the press release.
— New ISPOR Report Published, Call for Comments, Upcoming Events, and More! See details below.
— PCORI Engagement Award Program Offers New Funding to Build Capacity for Small Organizations to Engage in PCOR/CER. Click here to view the blog.
— 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. Over 40 Leading Patient & Disability Groups Call on Congress to Ensure Drug Price Negotiations Don't Discriminate. 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 to ensure HHS factors in the concerns of patients and other affected stakeholders in any drug price negotiation policy. Specifically, the letter calls on Congress to ensure that the Department of Health and Human Services (HHS) establishes a decision-making process that is informed by meaningful input from patients and other affected stakeholders related to the Inflation Reduction Act's drug price negotiation process, including 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. The letter also urges 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.
The Inflation Reduction Act passed the Senate yesterday, August 7, and will move on to a House vote later this week. Click here to read the letter. Click here to read PIPC Chairman Tony Coelho’s opinion highlighting the omitted language on affected stakeholder input.
2. 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.
3. 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.
4. 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.
5. Chairman's Corner: A Step Backward — No Voice for Stakeholders in Updated Reconciliation Provisions. The Inflation Reduction Act's provision on drug pricing negotiation did not include the provisions recommended by the National Council on Disability extending the Affordable Care Act’s ban on use of QALYs in Medicare across federal programs or an unambiguous QALY ban. The legislation also omitted an earlier provision to allow affected stakeholders to provide input in the negotiation provisions outlined in Section 1194.
In response, PIPC Chair Tony Coelho published a new blog that criticizes the updated reconciliation package's provisions for being unresponsive to "calls from the disability community to unambiguously bar the use of discriminatory value metrics like the quality-adjusted life year (QALY) across federal programs." Additionally, he raised that the new provisions also fail to provide opportunities for affected stakeholders to provide input. He added, that the National Council on Disability urged Congress not to rely on QALYs and later strongly encouraged the “inclusion of an unambiguous ban on the QALY within the text of the Build Back Better Act (H.R. 5367) in such a way as to convey unequivocal application to the drug price negotiation process” and further recommended extending the QALY ban in Medicare across federal programs as part of their Health Equity Framework." Click here to read the full blog piece. Click here to view the new legislative text. Click here to view the NCD letter to Congress. Click here to view the NCD Health Equity Framework, recommendation #8.
6. 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.
7. Disability and Patient Advocacy Groups Raise Concerns Over CHCF's Decision to Fund ICER. On July 19th, PIPC joined the Cystic Fibrosis Research Institute (CFRI), Disability Rights California, DREDF, the Coelho Center, and Familia Unida, among others, on a letter to the California Health Care Foundation (CHCF) to (1) applaud the Foundation on its focus on fair access to health care and (2) raise concerns about the equity implications of the Foundation's recent announcement to fund ICER. The letter explains that ICER uses "discriminatory and flawed comparative effectiveness metrics such as the QALY to assess the value of medicines." The undersigned organizations are urging the Foundation to "ensure that ICER does not use discriminatory, biased value assessments or comparative effectiveness measures as they complete CHCF-funded work" and "to assure Californians that the work it is funding will not be used against patients and people with disabilities to drive health policy." Click here to view the letter.
Additionally, PIPC Chair Tony Coelho published a blog, on June 21, that outlines concerns about the use of ICER's discriminatory value assessment metrics in California. He noted that, as part of CHCF's funding 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. He also urged policymakers in California to not ignore the recommendations of the many disability and patient advocacy groups on this issue. Click here to view the blog.
8. DOJ and HHS Issue New Guidance on Nondiscrimination in Telehealth. On Friday, July 29, the Department of Justice (DOJ) and the Department of Health and Human Services (HHS) issued joint guidance on nondiscrimination in telehealth. This guidance aims to provide clarity on how federal nondiscrimination laws require accessibility for people with disabilities and others in health care provided via telehealth. Nondiscrimination laws, including Sec. 504 of the Rehabilitation Act of 1973, Title VI of the Civil Rights Act of 1964 and Section 1557 of the Patient Protection and Affordable Care Act, require that telehealth be accessible to people with disabilities and limited English proficient persons. They work together to eradicate discrimination and protect access to health care access for all, especially those with disabilities. Assistant Attorney General Kristen Clarke of the Justice Department's Civil Rights Division stated, "the DOG will vigorously enforce the ADA and other civil rights laws to ensure that health care providers offering telehealth services are doing so free from discrimination." Click here to view the press release.
9. New ISPOR Report Published, Call for Comments, Upcoming Events, and More! ISPOR is preparing to respond to call for comments on a draft guidance document by the U.S. Food and Drug Administration: “Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments.” We ask that members who would like to provide input please do so by August 18th using the following link: Submit Comments. The ISPOR Machine Learning Methods in HEOR Task Force published a new ISPOR Report, titled “Machine Learning Methods in Health Economics and Outcomes Research—the PALISADE Checklist: an ISPOR Task Force Good Practices Report,” in the July 2022 issue of Value in Health. Click here to access the article and view the press release here.
10. PCORI Engagement Award Program Offers New Funding to Build Capacity for Small Organizations to Engage in PCOR/CER. The October 2022 Cycle of the Eugene Washington PCORI Engagement Award Program is now open. This funding cycle has a new funding opportunity for small groups: Building Capacity for Small Organizations to Engage in Patient-Centered Outcomes Research (PCOR)/Comparative clinical Effectiveness Research (CER). This cycle also offers funding for three other Engagement Award (EA) opportunities: Capacity Building, Dissemination Initiative, and Stakeholder Convening Support. Learn about all four funding opportunities below. Since 2014, the EA Program has awarded over $140 million to more than 680 projects. All of these projects help build communities ready to take part in PCOR, which is a type of CER. Click here to view the blog.
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. Click here to view the letter to CHCF from advocates. Click here to view PIPC Chairman Tony Coelho's opinion.
The Colorado Department of Insurance staff on the Prescription Drug Affordability Board will brief stakeholders on its draft Affordability Review rule on August 10, 2022. Click here to register. 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. Click here to view letters from Disability Rights Oregon and Paul Terdal. Click here to view the letter from Patients Rights Action Fund. On June 8, 2022, CMS extended the current Oregon waiver through September 30, 2022, providing additional time to review the demonstration extension application. Click here to view the letter from CMS.
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. These bills did not advance in 2022 and are expected to be raised again in 2023.
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: SMA patient's quality of life is in danger because Pharmac is refusing to fund Spinraza and Risdoplan, two life-changing medications. MS patient advocates are delivering a private petition to the Parliament to beg them to green light a proven treatment for MS that has garnered remarkable results. MS patients are calling on the Ministry of Health to approve and fund stem cell treatment for MS after years of demands. The Health Minister is acknowledging that the treatment shows positive results but is saying that it's up to the drug-buying agency Pharmac to decide which drugs are funded.
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. 08/19/2022: Evidence Presentation. Draft Evidence Report available. Public comments closed. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available.
- Menopause: Vasomotor Symptoms: Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. Public comments closed. 8/23/2022: Model Analysis Plan.
- 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: Draft Evidence Report available. Public comments open until 8/8/2022. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. 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: 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
Letter of Intent Deadline for Science of Engagement PCORI Funding Announcement
August 9, 2022
Click here to view.
Engagement Awards: Building Capacity for Small Organizations to Engage in PCOR/CER (October 2022 Cycle) Applicant Office Hours (One)
August 11, 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.