1. Chairman's Corner: CBO Confirms Use of Discriminatory Metrics in H.R. 3, see details below.
2. PIPC Congratulates Andy Imparato on Appointment to COVID-19 Health Equity Task Force, click here to view the announcement.
3. Emerging Threats in States for Use of Discriminatory Metrics, see details below.
4. Op-Ed: Washington State Bill Includes Group Known for Discriminatory Practices, click here to read the full op-ed.
5. EveryLife Foundation for Rare Diseases Report on the Economic Burden of Rare Diseases in America and the Rare on the Road Leadership Tour, see details below.
6. Op-Ed: The Biden Administration Needs to Look Beyond ICER for Value Assessment, click here to read the op-ed.
7. Pharmacy Benefit Managers Push for QALYs in Medicare Despite Implications for Discrimination, see details below.
8. IVI Webinar on Principles for Good Value Assessment, click here to register and to learn more.
9. Vaccination Equity and Communication, see details below.
10. PCORI Seeks Nominations for Advisory Panels, click here to learn more, submit a nomination, or apply to be on an advisory panel.
11. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
12. ICER's QALY-Based Study Topics: Atopic Dermatitis, Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma, Alzheimer's Disease, click here to provide patient input.
13. Upcoming Events and Webinars, see details below.
14. Medical Journal Articles, see details below.
15. AHRQ Effective Program Updates, see details below.
In response to a Congressional Budget Office (CBO) working paper acknowledging that it used quality-adjusted life years (QALY) and data from ICER in its work scoring drug pricing legislation, PIPC Chairman Tony Coelho wrote that it is time to lay discriminatory metrics to rest and invest in better solutions. "I am relieved that we are now seeing bipartisan opposition to the use of QALYs, whether from domestic entities such as ICER or foreign countries," he wrote. "Experts such as the National Council on Disability, an independent federal agency, share our concerns. NCD published a long report on how QALYs discriminate and provided recommendations against their use by policymakers, including opposition to importing QALYS from foreign countries. The DNC Platform now includes a statement against the use of QALYs. Senators on both sides of the aisle opposed the use of cost effectiveness based on metrics such as QALYs when creating the Patient-Centered Outcomes Research Institute (PCORI) and barred their use by PCORI and in Medicare in 2010....We don’t need to further entrench the stigma of disability especially during a pandemic." Click here to read Chairman Coelho's blog post.
2. PIPC Congratulates Andy Imparato on Appointment to COVID-19 Health Equity Task Force
Andy Imparato is a disability rights lawyer and the Executive Director of Disability Rights California, where he has spearheaded advocacy on crisis standards of care and vaccine prioritization in the last year. The COVID-19 Health Equity Task Force will provide recommendations for addressing health inequities caused by the COVID-19 pandemic and for preventing such inequities in the future. In receiving this appointment, Andy expressed his gratitude for this opportunity to fight for equity for people with disabilities and other groups who experience discrimination and disparities. Click here to view the announcement.
3. 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 or are considering policies to import QALY-based decisions from other countries. Yet, federal policymakers have emphasized that the use of discriminatory metrics is subject to civil rights laws such as the Americans with Disabilities Act. 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.
Washington State bill SB 5020 was recently introduced for the 2021 session. The legislation states, "In order to determine whether a price increase for a prescription drug is unsupported by new clinical evidence, the state must utilize and rely upon the analyses of prescription drugs prepared annually by the institute for clinical and economic review and published in its annual unsupported price increase report.” Treatments identified by ICER would then be subject to the penalties outlined in the legislation despite ICER’s reliance on quality-adjusted life years (QALYs) which is known to be a discriminatory metric. Federal policymakers on both sides of the aisle have expressed concern that measures that would unlawfully discriminate on the basis of disability or age are subject to section 504 of the Rehabilitation Act, the Americans with Disabilities Act, the Age Discrimination Act, and section 1557 of the Affordable Care Act - including state Medicaid agencies. PIPC, Epilepsy Foundation, AAPD, and AUCD all submitted written testimony.
Last year, advocates applauded the State of Oklahoma for being the first state to pass legislation explicitly barring the use of quality-adjusted life years (QALYs) or any other metric that would devalue lives lived with a disability, whether from within its agency or a third party. Today, Oklahoma will consider separate legislation that would import QALYs from Canada, where the Patented Medicine Prices Review Board relies on a cost-utility analysis model in which health outcomes are expressed as QALYs. The National Council on Disability (NCD) warned in its 2019 report that similar coverage denials and loss of access to care could also be the outcome if the United States if we reference other countries. The NCD also highlighted in its report that Section 504 of the Rehab Act and Section 1557 of the ACA also apply to Medicaid programs because they receive federal financial assistance. Click here to view S.B. 734. Click here to view the NCD report. Click here to view the NCD's recent letter to CMS against importing QALYs. Click here to reach out to the Chair and Members of the Oklahoma Senate Committee on Health and Human Services to express concern that S.B. 734 would import QALYS from Canada and violate the QALY ban in the Nondiscrimination in Health Care Coverage Act passed in 2020.
The legislature in the State of New Mexico has introduced HOUSE BILL 154 which would create a prescription drug affordability board. The proposed law would authorize the board to review the cost of prescription drugs and vote on whether to impose an upper payment limit on purchases and payer reimbursements of prescription drug products in the state, similar to the State of New York. Similar to New York, the bill explicitly authorizes the board to enter into contracts with qualified, independent third parties (such as the Institute for clinical and Economic Review) for services necessary to carry out the powers and duties of the board. New York explicitly references ICER which is known to refer to the quality-adjusted life year (QALY) as the “gold standard” for value assessment. To mitigate states from this kind of discrimination, organizations supporting Value Our Health developed a template for state 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. To provide input for the Monday hearing advocating for the inclusion of protections for people with disabilities and chronic conditions modeled on the bar on use of QALYs in Medicare included in the Affordable Care Act, send an email to SPAC@nmlegis.gov with Name, Entity Representing, Bill Number, and For or Against.
West Virginia is considering introducing legislation very similar to what we have recently seen in Washington State. The bill under consideration would rely on ICER’s unsupported price increase report. We have long been concerned about states referencing ICER due to its reliance on quality-adjusted life years (QALYs) which is known to be a discriminatory metric. Federal policymakers on both sides of the aisle have expressed concern that measures that would unlawfully discriminate on the basis of disability or age are subject to section 504 of the Rehabilitation Act, the Americans with Disabilities Act, the Age Discrimination Act, and section 1557 of the Affordable Care Act - including state Medicaid agencies.
In the past, we have reported that Pennsylvania is considering the establishment of a Health Policy Committee, modeled after Massachusetts. The Pennsylvania State Treasurer is proposing to create a Drug Affordability Review Board and to institute “value-based purchasing” of pharmaceuticals. Interestingly, the Treasurer specifically references VBP arrangements in Oklahoma but does not mention that the Oklahoma state legislature subsequently barred the use of QALYs. We will follow these developments closely to see if Pennsylvania similarly seeks to bar QALYs as part of their considerations.
At a recent meeting of the Vermont Green Mountain Care Board’s (GMCB) pharmaceutical technical advisory group, it came to light that the state is considering legislative initiatives similar to New York that would potentially rely heavily on the work of ICER. GMCB provided a link to a summary of state actions that advanced legislative initiatives from the National Association of State Health Policy (NASHP), including initiatives to reference QALY-based decisions in foreign countries such as Canada and referencing ICER studies. We will follow these developments closely. Click here to learn more about the how patients with cystic fibrosis are impacted by Canadian reimbursement and coverage policies.
We understand that patient and disability advocates are deeply concerned - for a variety of reasons - about the recently approved TennCare waiver. PIPC and other stakeholders sent a letter to CMS opposing the waiver application due to its implications for discrimination as it would likely utilize QALYs to determine cost effectiveness of treatments as part of limited formulary determinations. Unfortunately, CMS approved the waiver. They included a statement that the state should ensure that non-discrimination clauses as provided in 45 CFR 156.125 and 45 CFR 156.225, which prevent discrimination on the basis of a number of factors, including health conditions, are applied to the formulary. But they did not go so far as to explicitly call out that discriminatory measures such as QALYs fall into the category of violating these laws. Instead, they explicitly allow for limited formularies and increased barriers to receiving the right treatment at the right time. We believe that this waiver approval serves as an invitation to incorporate discriminatory cost and clinical effectiveness measures based on averages that will not take into account the unique characteristics and priorities of people with disabilities and serious chronic conditions. Click here to view the waiver approval. Click here to view the stakeholder letter opposing the waiver application. Click here to view Chairman Coelho’s blog stating "it is not enough to have CMS point out anti-discrimination laws such as the Americans with Disabilities Act if the federal government is going to give states explicit authority in an approved waiver to do just that - discriminate."
4. Op-Ed: Washington State Bill Includes Group Known for Discriminatory Practices
Siri Vaeth, the Executive Director of Cystic Fibrosis Research, Inc., wrote that Washington State's SB 2050 includes discriminatory QALY-based studies form ICER to seek to control drug prices. "The intent is good, but the execution is abysmal," she wrote. "The bill relies on a report by a very controversial non-profit, ICER, to determine if drugs are priced too high, and then penalizes the manufacturers accordingly. For those of us whose loved ones live with a rare disease, the inclusion of ICER in this legislation is highly disturbing. ICER is a wolf in sheep’s clothing. It claims to be looking out for patients by policing drug pricing, but the methodology it uses systemically discriminates against people with disabilities and chronic illnesses, while consistently benefitting payers. ICER’s cost-effectiveness analyses rely on a metric called the Quality Adjusted Life year (QALY), which in essence places a value on a person’s life via a QALY score, assigning a person living with a disability or chronic illness a lower value of life than a “healthy” person. Unsurprisingly, this means ICER’s assessments typically find treatments for those with lower scores less valuable, and hence not worth covering." Click here to read the full op-ed.
5. EveryLife Foundation for Rare Diseases Report on the Economic Burden of Rare Diseases in America and the Rare on the Road Leadership Tour
Attend the Rare Disease Congressional Caucus virtual briefing, “Economic Burden of Rare Diseases in America: A Public Health Crisis” on February 25th at 1:00 pm to learn more about the direct medical costs, indirect costs, and non-medical costs of living with a rare disease. Speakers will include Representatives G. K. Butterfield and Gus Bilirakis, Dr. Christopher Austin, NCATS, and Annie Kennedy, EveryLife Foundation for Rare Diseases. Register here.
The Rare on the Road Leadership Tour mobilizes grassroots advocates by bringing the combined expertise of the EveryLife Foundation for Rare Diseases and Global Genes to areas not tapped by other rare disease events, as we work to identify and activate new patients and to ensure the patient voice is larger and louder than ever before. This year, due to the circumstances surrounding COVID-19, the 2021 RARE on the Road Leadership Tour is going virtual with an updated format, which will consist of one interactive webinar and three virtual, state-specific meetings geared toward uniting and activating the rare disease community at the local level. To make this year’s RARE on the Road Leadership Tour as accessible as possible, English language closed caption, in addition to real-time Spanish translation, will be made available in all virtual spaces. Register here.
6. Op-Ed: The Biden Administration Needs to Look Beyond ICER for Value Assessment
The Pioneer Institute's Dr. William Smith wrote in STAT News that as the Biden Administration looks at drug pricing, it must venture beyond ICER and its QALY-based evaluations. "Patient advocates should scrutinize any 'independent review board' the Biden administration commissions because it will have a conflict of interest and a bias against breakthrough therapies," he wrote. "Patients may never receive a new, potentially life-changing therapy because it is rated by a biased review board as not having any 'quantifiable benefit' based on its price tag alone rather than the potentially life-changing impact that it could have for patients. ICER’s sole goal is to reduce drug prices, not evaluate individual patient benefit — which could include quality of life, freedom to work, and time with friends and family, among others. A mathematical equation that defines 'perfect health' one way will never measure that." Click here to read the op-ed.
7. Pharmacy Benefit Managers Push for QALYs in Medicare Despite Implications for Discrimination
In comments to CMS on the “ Most Favored Nation” Interim Final Rule, PBMs commented to CMS that they support reference to QALY-based value assessments that are long-opposed by people with disabilities and serious chronic conditions due to their implications for discrimination. Their comments stated, "Beyond methodological issues related to demonstration design and rollout, PCMA suggests that CMS investigate alternative models that are based on quality and value and focus on treatment outcomes versus cost of treatments. One such alternative that CMS should consider is the Institute for Clinical and Economic Review (ICER) model for value-based price benchmarks which considers a treatment's potential success and the subsequent lifetime health gains when comparing to other less costly therapies.” By contrast, advocates supporting Value Our Health have continued to raise concerns about ICER’s flawed methodology and there is bipartisan opposition to use of QALYs among policymakers. Click here to view information on ICER’s flawed methodology. Click here to view page 31 of the DNC Platform opposing QALYs. Click here to learn about the law barring QALYs in Medicare. Click here to view the PCMA comments.
8. IVI Webinar on Principles for Good Value Assessment
The Innovation and Value Initiative (IVI) will hold a webinar on principles for good value assessment. IVI’s new Principles for Value Assessment in the U.S. will be discussed, along with their practical application for patients, employers, and researchers. IVI's Jen Bright and the Patient Advocate Foundation's Dr. Alan Balch will talk about patient-centricity in action and activities for the year ahead. Join IVI for the webinar on Thursday, February 25 at 2:00 p.m. Eastern. Click here to register and to learn more.
9. Vaccination Equity and Communication
There is critical ongoing work to assure equity in the prioritization of vaccinations, as well as communication to address reluctance and ensure access to information and systems. PIPC Steering member the Association of University Centers on Disabilities (AUCD) aggregated the following resources that we wanted to share:
- Elevated COVID-19 Mortality Risk Among Recipients of Home and Community-Based Services: A Case for Prioritizing Vaccination for This Population
- National Council on Disability letter to Governors Association
- COVID-19 Vaccine Prioritization Dashboard
- Measuring the impact of COVID-19 vaccine misinformation on vaccination intent in the UK and USA
- COVID-19 Vaccination Intent, Perceptions, and Reasons for Not Vaccinating Among Groups Prioritized for Early Vaccination — United States, September and December 2020
- KFF COVID-19 Vaccine Monitor: What Do We Know About Those Who Want to “Wait and See” Before Getting a COVID-19 Vaccine?
10. 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, 2020. Click here to learn more, submit a nomination, or apply to be on an advisory panel.
11. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage?
Other countries are often referenced as examples of how the use of QALYs or similar cost-based thresholds impact access to care.
- Australia: Cancer patient recovers after receiving drug that the government refuses to fund.
- New Zealand: The Diabetes Foundation accuses Pharmac of "penny pinching" and encourages it to cover life-changing drugs for all patients with Type 2 diabetes.
- United Kingdom: Dad runs every street in his city to raise awareness of NHS not covering a life-changing PKU drug. Parents call for NHS to cover a PKU drug so that their children can eat normally.
12. ICER's QALY-Based Study Topics: Atopic Dermatitis, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma, Alzheimer's Disease
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.
- Atopic Dermatitis: Research Protocol available. 3/22/2021: Model Analysis Plan.
- High Cholesterol: Meeting 2/5/2021: The Midwest CEPAC convened to review treatments for high cholesterol. 2/26/2021: Final Evidence Report.
- Anemia in Chronic Kidney Disease: Evidence Report and Response to Public Comments AVAILABLE. Meeting 2/11/2021: CTAF will deliberate and vote on evidence presented in ICER’s report on therapies for anemia in chronic kidney disease.
- Lupus Nephritis: Draft Evidence Report AVAILABLE, Comment Period OPEN through 2/8/2021. Meeting 3/26/2021: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for lupus nephritis.
- Multiple Myeloma: Draft Evidence Report AVAILABLE, Comment Period OPEN through 3/11/2021. Meeting 4/16/2021.
- Alzheimer's Disease: Model Analysis Plan available. : Draft Evidence Report. Meeting 5/7/2020: CTAF will convene virtually to deliberate and vote on evidence presented in ICER's report on Alzheimer's disease.
13. Upcoming Events and Webinars
Challenges in Assessing the Clinical and Economic Value of Future Treatments for Alzheimer’s Disease
February 26, 2021
Click here to view.
Cycle 1 2021 PCORnet Phase 3 Applicant Town Hall
March 2, 2021
Click here to view.
Engagement Awards 2021 Special Cycle -- Applicant Office Hours
March 8, 2021
Click here to view.
14. Medical Journal Articles
Experiences of an HCV Patient Engagement Group: A Seven-Year Journey, click here to view.
Choosing Important Health Outcomes for Comparative Effectiveness Research: 6th Annual Update to a Systematic Review of Core Outcome Sets for Research, click here to view.
Health Technology Assessment With Diminishing Returns to Health: The Generalized Risk-Adjusted Cost-Effectiveness (GRACE) Approach, click here to view.
Comparative Effectiveness Research Needs to Consider Optimal Dosing and Scheduling, click here to view.
Are Patients More Adherent to Newer Drugs?, click here to view.
Improving the Quality of Person-Centred Healthcare from the Patient Perspective: Development of Person-Centred Quality Indicators, click here to view.
Six Ways to Foster Community-Engaged Research During Times of Societal Crises, click here to view.
Improving Comparative Effectiveness Research of Complex Health Interventions: Standards from the Patient-Centered Outcomes Research Institute (PCORI), click here to view.
Improving Access and Quality of Health Care in the United States: Shared Goals Among Patient Advocates, click here to view.
Using Electronic Health Record Data to Identify Comparator Populations for Comparative Effectiveness Research, click here to view.
Pharmaceutical Pricing Benchmarks: Governmental Versus Private Sector, click here to view.
15. AHRQ Effective Program Updates
Systematic Review: Integrating Palliative Care in Ambulatory Care of Noncancer Serious Chronic Illness. Click here to view.
Research Protocol: Management of Infantile Epilepsy. Click here to view.
Systematic Review: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Research Protocol: Improving Rural Health Through Telehealth-Guided Provider-to-Provider Communication. Click here to view.
Systematic Review: Treatments for Acute Pain. Click here to view.
Systematic Review: Acute Treatments for Episodic Migraine. Click here to view.
Research Protocol: Interventional Treatments for Acute and Chronic Pain: Systematic Review. Click here to view.
Research Protocol: Diagnostic Errors in the Emergency Department. Click here to view.
Research Protocol: Maternal and Childhood Outcomes Associated With the Special Supplemental Nutrition Program for Women, Infants and Children (WIC). Click here to view.
White Paper: Outcome Measure Harmonization and Data Infrastructure for Patient-Centered Outcomes Research in Depression. Click here to view.