1. PCORI Announces Engagement Award on Consideration of the Full Range of Outcomes Data, see details below.
2. It is Time to Let Go of the QALY’s Legacy of Discrimination. Click here to read the article.
3. Value Assessment in the News, see details below.
4. Announcing the Disability Equity Collaborative. Click here to visit the new website.
5. Is the QALY Fit for Purpose? Click here to read the journal.
6. PCORI Approves $254 Million to Fund 28 New Research Studies Addressing Urgent Health Problems. Click here to read the press release.
7. 2021 PCORI Annual Meeting Scheduled for Nov. 17-19, click here for details.
8. Public Comment on PCORI Proposed National Priorities for Health, see details below.
9. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
10. Emerging Threats in States for Use of Discriminatory Metrics, see details below.
11. IVI to Hold Second Methods Summit, click here to learn more.
12. ICER's QALY-Based Study Topics: Hypertrophic Cardiomyopathy, Myasthenia Gravis, Atopic Dermatitis, Alzheimer's Disease, Asthma, Type 2 Diabetes, click here to provide patient input.
12. Open Position: Epilepsy Foundation Is Hiring an Advocacy Coordinator. Click here to apply.
13. Upcoming Events and Webinars, see details below.
14. Medical Journal Articles, see details below.
15. AHRQ Effective Program Updates, see details below
As a component of PCORI’s reauthorization, a provision was included requiring PCORI-funded research to consider the full range of outcomes data, including potential burdens and economic impacts. PCORI has adopted principles for collecting this data. As PCORI moves forward in this process, it is accepting applications for Engagement Awards to fund convenings focused on defining appropriate and relevant burdens and economic impact data, identifying sources of and gaps in data, and opportunities to advance the rigor and standards when conducting research that considers a full range of outcomes data. PCORI will play an important role in defining what factors on cost and economic burden are important to the individuals our healthcare system serves and how to collect this data in a rigorous and patient-centric manner. Organizations wishing to apply for these Engagement Awards may do so here.
2. It is Time to Let Go of the QALY's Legacy of Discrimination
In a piece published by The Center for Dignity in Healthcare for People with Disabilities, authors Kelly Israel of Autistic Self Advocacy Network and Sara van Geertruyden of PIPC explain the Quality-Adjusted Life Year (QALY), how it is used, and its discriminatory implications. The authors explain the politics of the QALY as well as some of the challenges we face as we try to move beyond this discriminatory metric. Ultimately they conclude that investment is needed to develop methods to assess values of treatments that do not discriminate and sustained advocacy is needed to remind states and other entities of their obligations of nondiscrimination under the ADA and other disability rights laws. Click here to read the full article.
3. Value Assessment in the News
Federal policymakers are considering value assessment options, from domestic reference pricing to foreign reference pricing. As policymakers consider alternatives to QALYs and the evLYG which is derived from a QALY, it is important that policymakers to understand value assessment, how discriminatory metrics are used in value assessment and how innovators like PCORI and IVI are developing new methods for assessing cost and economic data related to care and treatment. For more information about evLYG, please view this PIPC Chairman blog and this Twitter thread that explains the evLYG method in more detail. We urge you to share widely the concerns from the National Council on Disability related to QALYs and evLYG (p. 61) and CBO’s use of QALYs to score H.R. 3 as a reminder that patients and people with disabilities oppose the use of this metric and metrics related to it like evLYG. Click here to view principles for value assessment centered on patients and people with disabilities.
4. Announcing the Disability Equity Collaborative
The University of Colorado Anschutz has recently formalized the Disability Equity Collaborative (DEC) with support from an engagement award from PCORI. DEC is the nation’s leading organization providing evidence-based knowledge and practical solutions and tools to address accessible healthcare for persons with disabilities. DEC and its partners are committed to developing meaningful, practical solutions to ensure access to equitable, high quality health care for all people. Learn more about DEC here and sign up to receive more information from DEC.
5. AJMC: Is the QALY Fit for Purpose?
The American Journal of Managed Care (AJMC) published a journal article that examines some of the flaws of the quality-adjusted-life-years metric. "We argue that the QALY does not currently pass these 3 tests to an acceptable standard," the authors write. "We argue for methods to achieve incremental improvement in both the scientific and ethical standards used in constructing measures such as the QALY and for an end to the inertia in improving a measurement system that is widely considered inadequate." Click here to read the journal.
6. PCORI Approves $254 Million to Fund 28 New Research Studies Addressing Urgent Health Problems
On July 27th, the PCORI Board of Governors approved $254 million in funding for 28 new research studies aimed at some of the most urgent health problems.
- Four studies approved for a total of $33 million will assess interventions to improve care during pregnancy and after childbirth to reduce the higher rates of health problems among Black women and Latinas and their infants.
- Five studies totaling $58 million will assess interventions to reduce suicide among adolescents and young adults, which is the second leading cause of death among U.S. youth ages 15-24.
- Several awards totaling $135 million were the first made under a new PCORI funding opportunity to support large multiphase comparative clinical effectiveness research (CER) studies designed to fill critical gaps in evidence.
- Two awards totaling $12 million will support studies assessing ways to deliver mental health care to young people with intellectual and developmental disabilities (IDD), who have higher rates of need and use of emergency care.
Details of these and other studies approved for funding by PCORI’s Board today are on the website. Click here to read the press release.
7. 2021 PCORI Annual Meeting Scheduled for Nov. 17-19
PCORI announced that its 2021 annual meeting will be held virtually on Wednesday, November 17 through Friday, November 19. The event serves as a report to the nation on PCORI’s progress in funding initiatives to determine which care approaches work best, for whom, and under what circumstances. Click here for details.
8. Public Comment on PCORI Proposed National Priorities for Health
In 2020, PCORI started an initiative to identify new priorities to direct its work in the coming years. PCORI's Board of Governors approved posting for public comment of five proposed National Priorities for Health developed in consultation with stakeholders, advisors, and experts. Public comment on the priorities began June 28 and will run through August 27. The five priorities are:
- Increase Evidence for Existing Interventions and Emerging Innovations in Health
- Enhance Infrastructure to Accelerate Patient-Centered Outcomes Research
- Advance the Science of Dissemination, Implementation, and Health Communication
- Achieve Health Equity
- Accelerate Progress Toward an Integrated Learning Health System
Click here to read more and to submit comments.
9. 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: CF patients are pleading with the Health Minister to get the federal government to subsidize Trikafta, a life-saving drug.
- New Zealand: Cystic fibrosis patients are distraught over recommended restrictive criteria by CADTH for Trikafta, a life-saving drug. SMA patient's mother is delighted as Human Rights Commissioner joins their corner to call on Pharmac to review Spinraza refusal. SMA teen is being forced to move to Australia because Pharmac refuses to fund Spinraza. Former Ministry of Health Deputy Director-General is calling for more reforms so that life-saving drugs can be funded in a timely manner.
- United Kingdom: PKU patient's father is running 237 miles to raise awareness and fund Kuvan, a life-saving drug, because the NHS refuses.
10. 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. Other states are taking positive steps to ban the use of the QALY and other discriminatory metrics.
The Maryland legislature passed legislation earlier this year creating a Prescription Drug Affordability Board. The legislation did not include protections for patients and people with disabilities such as barring the use of discriminatory QALYs. The Board has begun meeting to hear from stakeholders, including hearing a presentation from the Institute for Clinical and Economic Review which relies on QALYs to assess treatment value. Legislation was introduced this year to bar the use of QALYs and implement patient protections that has not advanced in the legislature. On August 3, organizations representing patients and people with disabilities sent a letter urging the Board to avoid policies that would potentially discriminate by relying on discriminatory metrics such as QALYs.
The Governor of Colorado has signed into law SB 21-175, which creates a prescription drug affordability review board. SB 21-75 did include an amendment that prohibits the use of QALYs in the section of the bill that determines an upper payment limit, but it does not include similar protections 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. 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. In order to mitigate this, it is important that the Board has representation from patients and people with disabilities. The Governors office is currently accepting applications for both the Colorado Prescription Drug Affordability Review Board and the Colorado Prescription Drug Affordability Advisory Council.
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.
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. This language 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 will be advanced as part of the California budget. 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. Patient groups sent a letter to a CA State Assembly member and a letter to committee and subcommittee chairs urging them to avoid policies that would potentially discriminate by relying on discriminatory metrics such as QALYs.
11. 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.
12. Open Position: Epilepsy Foundation Is Hiring an Advocacy Coordinator
The Epilepsy Foundation is seeking an Advocacy Coordinator to join our team. This position will support the Foundation’s Advocacy Team in advancing policy that overcomes the challenges of living with epilepsy and accelerates therapies to stop seizures, find cures and save lives. Policy focus areas are access to care, disability rights, public health & education, and research and innovation. Click here to view the full position description and directions on how to apply.
13. ICER's QALY-Based Study Topics: Hypertrophic Myocardiopathy, Myasthenia Gravis, Atopic Dermatitis, Alzheimer's Disease, Asthma, Type 2 Diabetes
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.
- Type 2 Diabetes: Draft Scoping Document and Research Protocol available. 11/09/21: Draft Evidence Report. Meeting 1/20/2022: Deliberation and vote on evidence presented in ICER's report on therapies for Type 2 Diabetes
- Hypertrophic Cardiomyopathy: Model Analysis Plan available. 8/18/2021: Draft Evidence Report. Meeting 10/22/2021: CTAF will deliberate and vote on evidence presented in ICER’s report on therapies for obstructive hypertrophic cardiomyopathy.
- Myasthenia Gravis: Draft Evidence Report available - Comments open until 8/18/2021. 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: Meeting with New England CEPAC was held 7/23/2021. Final Evidence Report available 8/17/2021.
- Hereditary Angioedema: RWE Update: Final updated assessment released 8/23/2021.
- Unsupported Price Increase: 11/16/2021: Final Report.
- Asthma: Model Analysis Plan available. 9/16/2021: Draft Evidence Report. Meeting 11/19/2021: New England CEPAC will deliberate and vote on evidence presented in ICER’s report on therapies for severe asthma.
- Fair Access: Coverage Policies in 2020: Protocol Available. 10/20/2021: Final Report.
14. Upcoming Events and Webinars
PCORI Wikipedia Edit-a-thon
August 18, 2021
Click here to view.
Whistleblowers of America: Workplace Promise Institute 2021 Conference
September 9-10, 2021
Click here to view.
PCORI Annual Meeting
November 17-19, 2021
Click here to view.
15. Medical Journal Articles
The Importance of Collaboration in Pursuit of Patient-Centered Value Assessment. Click here to view.
Toward Better Data Dashboards for US Drug Value Assessments, click here to view.
Prevalence of Avoidable and Bias-Inflicting Methodological Pitfalls in Real-World Studies of Medication Safety and Effectiveness, click here to view.
Association of Co-Pay Elimination With Medication Adherence and Total Cost, click here to view.
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.
15. AHRQ Effective Program Updates
Research Protocol: Creating Efficiencies in the Extraction of Data From Randomized Trials: A Prospective Evaluation of a Machine Learning and Text Mining Tool. Click here to view.
Research Protocol: Schedule of Visits and Use of Telemedicine for Routine Antenatal Care. Click here to view.
Systematic Review: Breast Reconstruction After Mastectomy, click here to view.
Surveillance Report: Living Systematic Review on Cannabis and Other Plant-Based Treatments for Chronic Pain. Click here to view.
Systematic Review: Prehospital Airway Management. Click here to view.
Systematic Review: Radiation Therapy for Brain Metastases. 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.