— Register Today! PIPC Forum on December 13 from 12-2pm. Click here to register for the event.
— 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.
— Chairman's Corner: Chairman Coelho Testifies in Oregon Against Use of QALYs. Click here to read the testimony.
— PIPC Chairman Blog: Aligning Health Technology Assessment with Efforts to Advance Health Equity. Click here to view the report.
— PIPC Submits Comments to ICER on Menopause Treatment Assessment. Click here to view the letter.
— Oregon Advocates Comment Opposes Options to Use QALYs by HERC. See details below.
— PCORI Seeks Nominations for Advisory Panels. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
— Sick Cells Reports: Medicaid Access to Sickle Cell Treatments and Finding Equity in Value. Click here to view the report.
— Experts Discuss Innovations in Care Delivery, Technology Driving Integration of Patient-Centric Value Frameworks. Click here to read the article.
— PIPC Joins Over 80 Organizations Urging HHS Civil Rights Office to Ban QALYs Across Federal Programs. 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. Register Today! PIPC Forum on December 13 from 12-2pm. Please save the date to join the Partnership to Improve Patient Care (PIPC) for a discussion about the importance of inclusion in research and value assessment as health technology assessment organizations, payers and policymakers take steps to advance health equity. We will hear from advocates with first-hand experience working with HTA organizations to improve the inclusiveness of data used to determine the value of treatments, the methodologies for assessing value to eliminate bias and the engagement of people that are historically omitted from medical research. We will also hear from political and policy leaders about their work to advance policies that ensure the values of patients and people with disabilities are central to the research and value assessments driving coverage and reimbursement decisions in federal programs such as Medicare and Medicaid. Click here to view the Save the Date and to RSVP to join at the Reserve Officers Association.
2. 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.
3. 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.
4. Chairman's Corner: Chairman Coelho Testifies in Oregon Against Use of QALYs. On November 17, 2022, PIPC Chairman Tony Coelho delivered a testimony to Oregon's Health Evidence Review Commission (HERC) advocating against the commission's use of the QALY. In his testimony he stated that when HHS first reviewed the Oregon waiver application in 1992, the waiver was rejected because of its use of the QALY which would violate the Americans with Disabilities Act (ADA). He voiced that "there is no need for QALY-based cost effectiveness analyses to understand the clinical impact of different treatments on the same condition," noting that ICER includes this metric in several of its value assessment reports. ICER has been criticized by its supporters for the use of the measure as it has "discriminatory implications" and "does not accurately capture the quality of life for patients." Coelho further condemned HERC's use of the measure "behind closed doors" and strongly urged the commission to end its use of the harmful metric. Click here to read the full testimony.
5. PIPC Chairman Blog: Aligning Health Technology Assessment with Efforts to Advance Health Equity. Today, PIPC Chairman Tony Coelho published a blog about the report from the Global Liver Institute, National Minority Quality Forum, Preparedness & Treatment Equity Coalition and the Partnership to Improve Patient Care (PIPC) entitled Aligning Health Technology Assessment with Efforts to Advance Health Equity. Chairman Coelho highlighted that the report is timely for several reasons. First, organizations such as ICER and IVI that conduct value assessments are currently engaged in their own processes for addressing the historical biases inherent in HTA methodologies. Second, CMS has made health equity a pillar of its strategic plan and is undergoing efforts to implement a process of establishing a “fair price” for prescription drugs as required by the Inflation Reduction Act, alongside CMMI responding to an Executive Order calling for ideas on new health care payment and delivery models that would “lower drug costs and promote access to innovative drug therapies.” If used, flawed HTA will work against the agencies’ health equity goals by entrenching its inherent bias into decisions impacting care access. Third, the report is timely as states such as Oregon continue to rely on flawed sources of evidence to make decisions about access to care for Medicaid beneficiaries and the HHS Office for Civil Rights considers policies addressing the use of discriminatory metrics in value assessment in HHS programs, including Medicaid. Click here to view the Chairman Blog.
The new report provides recommendations that may assist HTA organizations, health systems, payers, and policymakers that want to center their value assessment work on health equity. The report states, "In the United States, value-based health care is a concept that has gained strong support among policymakers and payers. Despite significant investment in value assessment, the process and metrics historically used to value therapeutic interventions have worked against health equity. High value care must also be equitable care.” The report provides recommendations to address data gaps, to test and validate new methods to eliminate bias, and to meaningfully engage diverse stakeholders in the patient and disability communities. Click here to view the report.
6. PIPC Submits Comments to ICER on Menopause Treatment Assessment. In a letter to the Institute for Clinical and Economic Review (ICER), the Partnership to Improve Patient Care (PIPC) submitted feedback on ICER's assessment of treatments for vasomotor symptoms associated with menopause. The letter criticizes ICER for using flawed patient-reported outcomes tools to generate utility values that are not representative of the patient experience. PIPC also takes issue with the model for being overly simplistic and conducted too early without the full range of data. "We continue to be concerned about ICER’s reliance on quality-adjusted life years (QALYs) to value treatments, a method that perpetuates not only devaluation of people living with disabilities and chronic conditions but also over-reliance on simplistic modeling and flawed utilities," wrote PIPC Chair Tony Coelho. "PIPC urges ICER to pause this assessment until more full data is available and to amend its methodologies more broadly to better reflect the patient experience."
7. Oregon Advocates Comment Opposes Options to Use QALYs by HERC. On November 1, 2022, letters (option 1; option 2) with 37 signatures were provided to the Oregon Health Evidence Review Commission (HERC) related to its proposed guide for use of quality-adjusted life years (QALYs) in HERC’s meeting materials, processes and decisions, particularly related to the prioritized list of services for coverage under Medicaid. Patients and people with disabilities, as they commented in the past, stated the use of QALYs has no place in health care decisions due to their inherent discriminatory algorithms that drive health inequity. Advocates provided separate letters on Options 1 and 2 in order to meet the restrictive 1000-word limit on comments required by the HERC. Advocates clearly stated that the alternatives in front of the HERC do not sufficiently address the shortcomings of QALYs. Oregonians, Paul Terdal and Caring Ambassadors, sent separate comments as well. Click here to view all of the comments shared with PIPC.
8. PCORI Seeks Nominations for Advisory Panels. PCORI is currently seeking nominations for appointments to its advisory panels. The advisory panels must include patients or patient advocates. PCORI's staff, board, and methodology committee take advisory panels' recommendations into account when making decisions and determinations. PIPC encourages patients and patient advocates to submit nominations to serve on PCORI's advisory panels, including on the Advisory Panel on Clinical Effectiveness and Decision Science, Advisory Panel on Patient Engagement, and Advisory Panel on Rare Disease. Nominations are open through March 31, 2023. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
9. Sick Cells Reports: Medicaid Access to Sickle Cell Treatments and Finding Equity in Value. First, in a report finding that Medicaid payers require prior authorization and/or step therapy at least 50% of the time for several sickle cell treatments, most survey respondents reported using evidence of comparative clinical effectiveness and established clinical benefits when making decisions about whether SCD therapies would be subject to step therapy. A state Medicaid program that specifically commented that they considered assessments from ICER when determining PDL placement and/or utilization management techniques despite patient concerns that ICER used a definition of value that did not account adequately for nonmedical and indirect costs, lacked appropriate comparators, and left out the patient perspective in its value-based price metric. Following this Medicaid report, Sicks Cells and the Innovation and Value Initiative published a report discussing the potential implications of flawed or incomplete analyses for health and racial equity in future decision-making and identifies recommendations for improvements in key focus areas to advance racial equity in health technology assessment. The report references the ICER value assessment of sickle cell treatments as a constructive example. The report states, "When decisions affecting coverage and access to potentially beneficial therapies are based on their estimated value, several vital questions arise, including, 'Value for whom?' and whether clinical and other data inputs represent real-world patient communities and lived experience. These decisions bear significant consequences for equity and disparities in outcomes. As the use of HTA gains increasing traction in the U.S., it is especially important to understand and explore how racism is built into the current structures of the HTA process, and how that widens or contributes to existing health and racial inequalities.” Click here to view the report on Medicaid Coverage. Click here to view the Health Equity report.
10. Experts Discuss Innovations in Care Delivery, Technology Driving Integration of Patient-Centric Value Frameworks. On October 12, 2022, two expert stakeholders from Avalere Health discussed the utility of patient-centric value frameworks for equitable health care delivery, in additional to challenges and strategies regarding its implementation at the Academy of Managed Care Pharmacy (AMCP) Nexus 2022 meeting. Notably, the experts note that "patient preferences and consideration of SDOH and other external factors are key to achieving value-based care delivery, but the lack of data on these variables or even a mechanism to define their impact remains a major limitation to the integration of these frameworks." Click here to read the article.
11. Advocates Urge HHS Civil Rights Office to Ban QALYs Across Federal Programs. PIPC joined over 80 organizations in signing a letter to the HHS Office for Civil Rights commenting on the proposed rule implementing Section 1557 of the Affordable Care Act. The groups support a comprehensive proposed rule seeking to strengthen civil rights protections in federally funded health programs and HHS programs and agreed that the ability to access needed health care fully and free from discrimination is critical and requires action to support and strengthen existing nondiscrimination laws. Their comments focused on the agency’s request for comments on the extent, scope and nature of value assessment methods that discriminate on the basis of race, color, national origin, sex, age, or disability; the use of clinical algorithms in health care decision-making; and nondiscrimination requirements and enforcement. A letter from the American Association of People with Disabilities to HHS stated, "QALYs assign lower values to the improvements that a treatment can bring to the length and quality of life of people with disabilities, making it more likely that drugs that help disabled people to maintain their function, quality of life, and independence will “not make the cut” for being included in an insurer’s formulary, or that access to the drug will involve layers of utilization management.” A letter from the Consortium for Constituents with Disabilities stated, "When making life or death health care decisions, the government should not rely on instruments that are based on surveys of the general public. These instruments will reinforce the devaluation and discrimination that Congress has intended to address through civil rights and antidiscrimination statutes."
Among the recommendations, the groups urged the Office for Civil Rights to advance a rulemaking that codifies a ban on the use of methods for calculating value that penalize individuals or groups of individuals on the basis of race, color, national origin, sex, age, or disability as part of utilization management, formulary design, price negotiations, alternative payment models and other incentive-based programs impacting access to care and affordability of care. The letter also urged HHS to learn from the pandemic and codify regulations that health care providers may not rely on clinical algorithms that function to discriminate against providing care to people based on race, color, national origin, sex, age, and disability and to ensure state Medicaid programs are meeting the ADA’s requirements for accessibility to the information on which they make decisions and communicating that information in a manner that does not disadvantage people with disabilities or people with limited access to technology. Click here to view the sign-on letter. Click here to view the AAPD letter. Click here to view the CCD letter. Click here to view the letter from 71 organizations representing complex conditions.
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: Diabetes patients are being denied coverage of life-saving drugs and their physicians are pressing for coverage so they can stop recommending patients try cheaper but more dangerous alternatives first. A CF patient and her mother are lobbying the government to address the continuing challenges to the CF and rare disease communities, while many patients are unable to access Trikafta, a life-saving drug.
- New Zealand: A bowel cancer patient is paying unreasonably high prices for life-extending drugs and is being forced to raise funds for her treatment as the government still fails to cover the drugs.
- United Kingdom: An MS patient is raising money in order to have treatment, which is available on the NHS but only in very specific medical circumstances. Another MS patient is also struggling to pay for his medication, which is not available on the NHS and he is traveling to London to hand deliver a petition with more than 14,000 signatures to the government to fund the drugs.
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: Draft Scoping Document available. Public Comments open until 12/15/2022. 12/23/2022: Revised Scoping Document. 1/6/2023: Research Protocol.
- Non-Alcoholic Steatohepatitis: 12/22/2022: Model Analysis Plan. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available. Public comments closed.
- Menopause: Vasomotor Symptoms: 12/21/2022: Evidence Report. Draft Evidence Report available. Public Comments closed. Model Analysis Plan available. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available.
- Alzheimer's Disease: 12/22/2022: Draft 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: 12/21/2022: Evidence Report. Draft Evidence Report. Public comments closed. Model Analysis Plan. Research Protocol available. Revised Scoping Document available. Draft Scoping Document available.
- Gene Therapies for Hemophilia A and B: Evidence Presentation available. 12/19/2022: Final Evidence Report and Meeting Summary. 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
PIPC Forum 2022
December 13, 2022
Click here to view.
Board of Governors Meeting
December 13, 2022
Click here to view.
Board of Governors Meeting
February 14, 2023
Click here to view.
Medical Journal Articles
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.