1. Discrimination and Health Care: How it Impacts You, click here to register for the webinar.
2. COMMENTS DUE TOMORROW: Amplifying the Voices of Alzheimer’s, see details below.
3. New York Times Story on the Experience of One Patient with Sickle Cell Disease, click here to read the article.
4. PIPC Chairman Applauds NCD Efforts to Combat Discriminatory Health Care Policies, click here to read the letter.
5. National Health Council and Tufts Launch New Patient-Centered Value Assessment Initiative, click here to learn more.
6. States Rely on Template Legislation that References ICER and Discriminatory Metrics, see details below.
7. Emerging Threats in States like CO and ME for Use of Discriminatory Metrics, see details below.
8. IVI to Hold Second Methods Summit, click here to learn more.
9. Vaccination Equity and Communication, see details below.
10. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
11. ICER's QALY-Based Study Topics: Hypertrophic Cardiomyopathy, Myasthenia Gravis, Atopic Dermatitis, Lupus Nephritis, Alzheimer's Disease, Asthma, click here to provide patient input.
12. Upcoming Events and Webinars, see details below.
13. Medical Journal Articles, see details below.
14. AHRQ Effective Program Updates, see details below.
The Bonnell Foundation: Living with Cystic Fibrosis will hold a webinar on Thursday, June 10 at 4pm Eastern to discuss the ways that policymakers and payers make decisions about whether or to cover a given treatment. The system being proposed to state and federal policymakers was put forth by a self-appointed group that uses Quality Adjusted Life Years (QALY). The process for valuing treatments using QALYs puts a lower value on lives lived with a disability or chronic disease. The webinar will also cover the existing civil and disability rights laws that have created precedent against using QALYs in public health programs, as well as threats at the state and federal level for incorporating the use of QALYs. Click here to register.
2. COMMENTS DUE TOMORROW: Amplifying the Voices of Alzheimer’s
As treatment for Alzheimer’s Disease undergoes scrutiny from the Institute for Clinical and Economic Review, the Alliance for Aging Research has developed a new report entitled “Assessing Value of Therapies in Alzheimer’s Disease: Considerations to create a practical approach to value.” Comments on the report are due tomorrow, June 2. Click here to view the report.
Additionally, the Alliance for Patient Access (AfPA) has shared a toolkit for advocates to provide input. They have also provided specific suggestions for activities that advocates can engage in to provide input. Click here to view the toolkit. Click here to view the action page.
3. New York Times Story on the Experience of One Patient with Sickle Cell Disease
The New York Times ran a story on the experience of one woman, Lisa Craig, living with sickle cell disease (SCD) and her struggle to get her doctor to listen to her. As she waits for gene therapies that may successfully treat SCD, she has worked with several doctors to get them to take her pain seriously and to treat her properly. As the Times notes, "Many sickle cell patients feel frustrated that doctors don’t believe patients know what works. Often, that’s narcotic doses much higher than the average person requires. Yet asking for specific medications can fuel distrust, compounded by many doctors’ lack of familiarity with sickle cell." Click here to read the article.
4. PIPC Chairman Applauds NCD Efforts to Combat Discriminatory Health Care Policies
Partnership to Improve Patient Care (PIPC) Chair Tony Coelho penned a letter to National Council on Disability (NCD) Chair Andrés Gallegos applauding the Council's incredible work and recommendations that seek to improve the lives of people living with disabilities and push back against discrimination in health care.
The letter specifically calls out applauds NCD's steady drumbeat of concern about the use of discriminatory metrics in our healthcare system, including concerns about the use of the quality-adjusted life years (QALY) metric in federal health programs, whether in the form of an International Pricing Index, Most Favored Nation policy, or the recently introduced H.R. 3. "We believe that more work needs to be done to provide a full picture and understanding of the inherent bias of discriminatory metrics used in health care decision-making, and how they promote the stigma that disabled lives, especially in communities of color, do not have a quality of life worth saving," wrote Chair Coelho.
Click here to read the letter.
5. National Health Council and Tufts Launch New Patient-Centered Value Assessment Initiative
The National Health Council (NHC), in partnership with the Center for the Evaluation of Value and Risk in Health (CEVR) at Tufts Medical Center announced a new partnership to develop good–practice recommendations to assist researchers in developing more patient-centered value assessments. The project is part of a collaboration between two Value Assessment Centers of Excellence: the Patient-Driven Values in Healthcare Evaluation (PAVE) and Tufts Center for Enhanced Value Assessment (CEVA). NHC says that many researchers do not include patient input, or make use of patient-provided information (e.g., experiences, perspectives, patient-reported outcomes, desired outcomes, goals, etc.) in value assessment, even when data do exist. As a result, most value assessment research is methodologically sound according to current guidelines but does not reflect the lived experiences of patients. This initiative aims to change that. Click here to learn more.
6. States Rely on Template Legislation that References ICER and Discriminatory Metrics
We are seeing many states reference pieces of template legislation put forward by the National Association of Health Policy (NASHP). Though the intent of these bills is to lower healthcare costs, several implicate the discriminatory Quality-Adjusted Life Year (QALY) and others rely on the Institute for Clinical and Economic Review (ICER) as the sole source for evidence. The three primary bills are:
- Canadian Reference Pricing -- This piece of template legislation directly references the prices paid for drugs in five Canadian provinces. Before applying for coverage by the provinces, all drugs must complete a Common Drug Review by CADTH, which uses QALYs. The result of this is that in Canada is that many individuals living with disabilities are unable to receive the treatments and care they need. 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.
- Unsupported Price Increase -- This piece of template legislation relies on one report by ICER to determine whether a price increase on certain therapies were supported by additional evidence. This is concerning as it codifies reference to an independent entity lacking oversight and accountability as a sole source of information. ICER is the sole arbiter as to what evidence it uses to develop this report, and patients have consistently expressed concern with ICER’s lack of transparency and omission of real-world evidence. ICER also has the ability to change the inputs and methodology of the report without oversight. Though the 2020 report does not use the QALY, it is possible that future reports will.
- Drug Pricing Review Board -- This piece of template legislation establishes a drug review board or commission. The goal of the board is to allow the state to review and evaluate the reimbursement rate and/or coverage for pharmaceuticals. Most have fairly broad parameters of how they can assess a drug’s value, and specifically permit the state to rely on third-party research or contract directly with a third-party for the purpose of fulfilling its duties. As has happened in other states such as New York and Massachusetts, without patient protections, these bills allow the new state board or commission to reference value assessments that rely on QALY and similar metrics. It is imperative that these boards has patient and disability representation and that a QALY ban is included in the legislation.
7. Emerging State Bills Reference the QALY and Value Assessments
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. Other states are taking positive steps to ban the use of the QALY and other discriminatory metrics.
The Colorado state legislature has introduced SB 21-175 creating a prescription drug affordability review board. In some states these types of boards and commissions have led to referencing value assessments based on QALYs from the Institute for Clinical and Economic Review (ICER). In Massachusetts, the Health Policy Commission went so far as to contract with ICER. An amendment to SB 21-75 was adopted that prohibits the use of QALYs in the section of the bill that determines an upper payment limit. A similar amendment in the sections establishing the commission’s authority to perform value assessments for treatments would prevent QALYs from being used to target treatments for people with disabilities and older adults. The bill passed the Senate and but could be amended in the House to bar QALYs in section 10-16-1306, which would then prevent the medications subject to the UPL from unfairly representing treatments for people with disabilities that are devalued under the QALY metric (consistent with language in the Value Our Health legislative template).
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.
The New Jersey state legislature has introduced A 2418 creating a prescription drug affordability review board. In some states these types of boards and commissions have led to referencing value assessments based on QALYs from the Institute for Clinical and Economic Review (ICER). A robust QALY ban would mitigate this issue and provide important protections for patients and people with disabilities.
SB844 establishes a Prescription Drug Affordability Board in the Department of Consumer and Business Services to review prices for prescription drug products meeting specified cost criteria. It is based on the NASHP template legislation and states, "The board may enter into a contract with a qualified, independent third party for any service necessary to carry out the powers and duties of the board.” Based on past experience, the expectation is that the third party would likely be an entity such as the Institute for Clinical and Economic Review (ICER) which continues to call QALY’s the gold standard for assessing cost effectiveness of new treatments. A robust QALY ban would address these shortcomings. The Oregon Medicaid and EPSDT waiver is up for renewal this year, providing an opportunity to address the state’s possible use of QALYs.
CA AB 1130, the California Health Care Quality and Affordability Act, is being considered in California to create an Office of Health Care Affordability. Similar to the Massachusetts Health Policy Commission, the legislation would authorize reviewing health costs and "require the office to set priority standards for various health care metrics.” In Massachusetts, the Health Policy Commission went so far as to contract with ICER. We understand the legislation is likely to pass the Appropriations Committee this session, and amendments to protect patients from discrimination has not been considered. It was originally proposed by Governor Gavin Newsom in the California Budget for 2021-22 which included a proposal for a new Office of Health Care Affordability to be housed within the Office of Statewide Health Planning and Development (OSHPD) that would seek to promote “cost efficient” care. It is anticipated that this office could advance as an independent bill, or as part of the California budget in mid-June. The Value Our Health template language has not been introduced or passed in California to protect patients from the use of QALYs and discriminatory considerations of cost effectiveness.
8. IVI to Hold Second Methods Summit
The Innovation and Value Initiative (IVI) announced that it will hold a second methods summit to drive patient-centered value assessment. The multi-stakeholder event is intended to drive consensus on priority patient inputs, methods, and research that more fully represent the patient perspective in comparative effectiveness research and economic evaluations of health care interventions. The three-part series will take place in Fall 2021. Overall, the goal of IVI’s summit is to define an action agenda for patient-centered outcomes research and cost-effectiveness research by exploring patient outcomes, beyond traditional clinical factors, that represent important impacts to patients and must be accounted for in economic analyses. Click here 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. 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.
- New Zealand: Cystic fibrosis patient spends $25k to go to UK for a life-changing drug as it is not available in New Zealand. Cancer sufferer "angry" with Pharmac, noting that people have to sell their homes or go abroad to afford medication due to Pharmac's underfunding. Patients call new Pharmac funding "disgraceful." Man may be alive today if Pharmac had not switched his epilepsy medication.
- United Kingdom: NICE rejects life-prolonging prostate cancer drug, which could benefit 4,000 men per year.
11. ICER's QALY-Based Study Topics: Hypertrophic Myocardiopathy, Myasthenia Gravis, Atopic Dermatitis, Lupus Nephritis, Alzheimer's Disease, Asthma
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.
- Hypertrophic Cardiomyopathy: 5/14/2021: Research Protocol available. 7/2/2021: Model Analysis Plan. Meeting 10/22/2021: CTAF will deliberate and vote on evidence presented in ICER’s report on therapies for obstructive hypertrophic cardiomyopathy.
- Myasthenia Gravis: Research Protocol available. 6/7/2021: Model Analysis Plan. Meeting 9/24/2021: New England CEPAC will deliberate and vote on evidence presented in ICER’s report on therapies for myasthenia gravis.
- Atopic Dermatitis: Draft Evidence Report AVAILABLE, Comment Period OPEN through 6/11/2021. Meeting 7/23/2021: An assessment of treatments for atopic dermatitis by the New England CEPAC.
- Hereditary Angioedema: 7/27/2021: Final Updated Assessment with RWE Update.
- Unsupported Price Increase: 11/16/2021: Final Report.
- Asthma: Draft Scoping Document available. 6/7/2021: Revised Scoping Dcoument. Meeting 11/19/2021: New England CEPAC will deliberate and vote on evidence presented in ICER’s report on therapies for severe asthma.
- Alzheimer's Disease: Draft Evidence Report AVAILABLE, comment period OPEN through TOMORROW, 6/2/2021. Meeting 7/15/2021: CTAF will deliberate and vote on evidence presented in ICER’s report on Alzheimer’s disease.
- Fair Access: Coverage Policies in 2020: Protocol Available. 10/20/2021: Final Report.
12. Upcoming Events and Webinars
Engagement Awardee Lunch and Learn Engagement in Practice: Success Stories Across PCORI-funded Projects
June 9, 2021
Click here to view.
ISPOR Summit 2021
June 10, 2021
Click here to view.
Building Data Capacity for Patient Centered Outcomes Research: A Comprehensive Ecosystem for PCOR - Workshop 3
June 14, 2021
Click here to view.
PCORI Board of Governors Meeting
June 15, 2021
Click here to view.
PCORI Advisory Panel on Patient Engagement Summer 2021 Meeting
July 15-16, 2021
Click here to view.
13. Medical Journal Articles
The Utility of Patient Engagement in Drug Research and Development, click here to view.
Patient-Powered Research Networks of the Autoimmune Research Collaborative: Rationale, Capacity, and Future Directions, click here to view.
Enhancing Patient Research Partner Engagement: Research in Psoriatic Arthritis, click here to view.
Patient Voices in Value-Based Cancer Care: Priorities for the Biden Administration, click here to view.
The Dollar or Disease Burden: Caps on Healthcare Spending May Save Money, but at What “Cost” to Patients?, click here to view.
Embedding Clinical Trials Within Routine Health-Care Delivery: Challenges and Opportunities, click here to view.
Approval and Coverage of Cancer Drugs in England, Canada, and the US, click here to view.
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.
14. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH JUNE 18, 2021: Systematic Review: Models of Care that Include Primary Care for Adult Survivors of Childhood Cancer. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 15, 2021: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 21, 2021: Systematic Review: Integrated Pain Management Programs. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 7, 2021: Systematic Review: Prehabilitation and Rehabilitation for Major Joint Replacement. Click here to view.
Systematic Review: Safety of Vaccines Used for Routine Immunization in the United States: An Update. Click here to view.
Research Protocol: Evaluation of Mental Health Applications. Click here to view.
Maternal, Fetal, and Child Outcomes of Mental Health Treatments in Women: A Systematic Review of Perinatal Pharmacologic Interventions. Click here to view.
Rapid Evidence Product: Improving Pain Management in the Context of America's Opioid and Substance Use Disorders Crisis. Click here to view.
Systematic Review: Cervical Ripening in the Outpatient Setting. Click here to view.
Surveillance Report: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.