1. Deadline TODAY: ICER Seeks Public Input for 2020 Value Assessment Framework - Contact PIPC to Sign On!, click here to read more and see below for details.
2. You're Invited! Value Our Health Briefings on Capitol Hill, see below for details.
3. PIPC Chairman Coelho and Doctors Caucus Co-Founder Dr. Phil Gingrey on PCORI, click here to read the article.
4. NAMI's Andrew Sperling Highlights Concerns with ICER's Review of Treatment-Resistant Depression, click here to read the interview.
5. PIPC Blog: ICER's SMA Study Falls Short on Data Accuracy, click here to read the full post.
6. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
7. ICER Studies: Type 2 Diabetes, Arthritis, Cardiovascular Disease, Duchenne Muscular Dystrophy click here to provide patient input.
8. AACR Briefing on e-Cigarettes and Nicotine Addiction, click here for more information.
9. Applying Real-World Evidence to Beat Cancer, click here to read the article.
10. Submit 2019 Fly-ins, Advocacy Days, and Conferences to Democratic Caucus, click here to submit your events.
11 Upcoming Events and Webinars, see details below.
12. Medical Journal Articles, see details below.
13. AHRQ Effective Program Updates, see details below.
As part of its biannual process of revising its value assessment framework, ICER is seeking public comment on potential changes to improve its process. This is an important opportunity for patients and people with disabilities to tell ICER that one-size-fits-all conceptions of "value" ignore what is important to them. Specifically, the use of QALYs discriminates against older people and people with disabilities, and it has no place in determining the value of treatments. ICER is specifically seeking comment on the QALY and its cousin, the equal-value life year gained (evLYG) metric. PIPC and its partners have developed comments that will highlight for ICER how the use of QALYs and evLYGs devalues their lives, with suggestions on addressing the methodological flaws in its current model. ICER is accepting comments through TODAY, June 10, 2019, and plans to publish draft changes to its framework based on comments received on August 16th. Click here for more information and to submit comments to ICER. Click here to view PIPC's letter, and please contact [email protected] for additional information about the letter being developed by PIPC and others.
2. You're Invited! Value Our Health Briefings on Capitol Hill
Organizations calling on policymakers to Value Our Health are holding two briefings on value assessments on Capitol Hill on June 20. Please join our esteemed panel to learn about value assessments, their potential for discrimination against patients and people with disabilities, and related public policy threats at the federal and state level. Click here to RSVP for a breakfast briefing on the House side (for screen reader accessibility, click here) and click here to RSVP for a lunch briefing on the Senate side (for screen reader accessibility, click here).
3. PIPC Chairman Coelho and Doctors Caucus Co-Founder Dr. Phil Gingrey on PCORI
PIPC Chairman Tony Coelho and Doctors Caucus co-founder Dr. Phil Gingrey both served as health care advocates in Congress, and now they have written an op-ed together in Modern Healthcare about the successes of PCORI and the importance of continuing its work. "If we're to continue to make progress on improving patient care and outcomes, clinicians and patients must have access to more reliable, useful information to help determine which healthcare options are best for them. And in order to ensure that evidence is relevant and accessible to them, patients need a seat at the table in developing it....PCORI has lived up to its name and its mandate to drive patient-centeredness. We both feel strongly that the funding that supports its work should be reauthorized before it expires at the end of fiscal 2019, so the valuable investment in its efforts to date can produce even more useful evidence for healthcare decisionmakers well into the future," the duo wrote. Click here to read the article.
4. NAMI's Andrew Sperling Highlights Concerns with ICER's Review of Treatment-Resistant Depression
Andrew Sperling, the Director of Legislative Advocacy at the National Alliance on Mental Illness (NAMI), was interviewed about ICER's recent study on treatment-resistant depression and how it does not properly value new therapies that can help save lives. "The mental health community is fortunate in that there are many new treatments coming out, and that will likely mean more ICER reviews. There is a new drug for schizophrenia in clinical trials, for example. NAMI will continue to engage with ICER in good faith, but understand that there does not seem to be an interest to change their methods and recognize that QALYs fail to capture what is important to patients. That’s especially troubling with what’s available and what’s not available for treating mental illnesses. Many of these medicines have challenging risk profiles and side effects, so new treatments that address the challenges people with mental illnesses have can be significant breakthroughs for the community that are undervalued in ICER’s current framework," he said. Click here to read the interview.
5. PIPC Blog: ICER's SMA Study Falls Short on Data Accuracy
PIPC published a blog post this week outlining how ICER failed to consider sufficient data in its recent study on SMA and how the study and its use of the QALY hurts patients. From the blog: "ICER’s recently-released final report was disappointing in several ways, including the lack of sufficient data, disregard for outcomes that matter to patients, lack of understanding and oversimplification of the condition, and the inherit weaknesses of the quality-adjusted-life-year (QALY) measure. This is particularly concerning given the real-world consequences we are observing in the U.K. and other countries, which have utilized QALYs to limit patient access. As one mother of a child with SMA living in the U.K. shared: “It’s an abomination of human rights that the drug isn’t available… I've seen children in America who had it when they were first diagnosed and they're walking. To be put in a position where you have to consider moving abroad to keep your son alive is dreadful.” With such clear consequences for children and their families, it is alarming to see ICER ignore patients’ voices and produce such a flawed and short-sighted assessment of these important, breakthrough therapies." Click here to read the full post.
6. 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: PBS is making a woman pay $4,500 per month out of pocket for a breast cancer drug because she does not meet PBS's criteria.
- New Zealand: Breast cancer advocates say that human life is not prioritized by PHARMAC. Click here to read more. Lung cancer patients say similar things. Patients fear that PHARMAC will switch to offering only a less-effective epilepsy drug. Patients are fighting to have PHARMAC cover spinal muscular atrophy drug Spinraza, including through legal action.
- Canada: An Ontario woman living with CF advocates for greater access to lifesaving drugs.
- United Kingdom: NHS has doubled its cataract treatment rationing, ignoring evidence that surgery is effective. Click here to read more.
7. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Cardiovascular Disease, Depression, MS, Duchenne Muscular Dystrophy, Peanut Allergy
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. Please note the following upcoming formal ICER deadlines per their website:
- Arthritis: Revised Scoping Document available. 6/14/2019: Research Protocol. Meeting 10/31/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: Revised Scoping Document available. Meeting 11/14/2019: The New England CEPAC will convene to deliberate on ICER's review of oral semaglutide for the treatment of type 2 diabetes.
- Cardiovascular Disease : Model Analysis Plan available. 7/24/2019: Draft Evidence Report. 9/26/2019 Meeting: Midwest CEPAC to deliberate and vote on ICER's report on evidence presented in ICER's report on additive CVD therapies.
- Depression: Evidence Presentation available. 6/20/2019: Final Evidence Report and Meeting Summary.
- Multiple Sclerosis: Evidence Presentation available. 6/20/2019: Final Evidence Report and Meeting Summary.
- Peanut Allergy: Responses to Public Comments and Evidence Report available. Meeting 6/11/2019: CTAF to review ICER's assessment of treatments for peanut allergy. 7/3/2019: Final Evidence Report.
- Duchenne Muscular Dystrophy: Public comment period OPEN on Draft Evidence Report and Draft Voting Questions through 6/18/2019. Meeting 7/25/2019: New England CEPAC to deliberate and vote on evidence presented in ICER's report on treatments for Duchenne muscular dystrophy.
- Unsupported Price Increase Assessment: 10/8/2019: Final Report.
- Acute Migraine: Open Input Period through 6/25/2019. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments
- Valuing A Cure Project: White Paper available 8/6/2019. Comment period open 8/6/2019-9/3/2019.
8. AACR Briefing on e-Cigarettes and Nicotine Addiction
On June 12, 2019, the American Association for Cancer Research (AACR) will host a Capitol Hill briefing to educate Congressional staff and the public on the potential public health crisis of e-cigarette use and nicotine addiction in youth and young adults. Government officials and public health experts will share their knowledge on regulatory efforts, new data around nicotine addiction, and usage trends. Click here for more information.
9. Applying Real-World Evidence to Beat Cancer
In STAT News, Pfizer's Christopher Boone and COTA's Viraj Narayanan discuss how real-world evidence can be used to better understand cancer and the treatments for it. They highlight FDA's increasing acceptance of real-world evidence, in addition to clinical trials, and how some cancer drugs have newly-accepted indications thanks to real-world evidence. "we’re past the stage of wondering if real-world evidence is valuable, and should now be asking how to apply it. Widespread application and adoption of approaches based on real-world evidence will help us begin to rethink — and outsmart — cancer," they wrote. Click here to read the article.
10. Submit 2019 Fly-ins, Advocacy Days, and Conferences to Democratic Caucus
In the interest of amplified patient and stakeholder engagement, your organization may be interested that the House Majority Leader’s office is compiling a list of fly-ins, advocacy days, and conferences that will be taking place throughout the year. This information will be shared with all House Democratic offices and used for a variety of purposes including scheduling and messaging. Feel free to share any events you have planned. If we hear of similar efforts by the Minority Leader, we hope to share that as well. Please submit your events here.
11. Upcoming Events and Webinars
PCORI at AcademyHealth 2019 Research Meeting
June 2-4, 2019
Click here for details.
2019 NEC Symposium
June 2 - 5, 2019
Click here for details.
Getting Real: The Changing Tide on Real-World Evidence in Drug Development
June 5, 2019
Click here for details.
Advisory Panel on Clinical Effectiveness, and Decision Science Spring 2019 Meeting
June 14, 2019
Click here for details.
PCORI Board of Governors Meeting
June 18, 2019
Click here for details.
What’s in a Number? It Matters in Health Policy Conversations
June 18, 2019
Click here for details.
Understanding FDA’s Real-World Evidence Program: A Presentation of the AMIA Public Policy Committee
June 21, 2019
Click here for details.
A New Path Forward for Using Real World Evidence in Randomized Clinical Trials
June 23, 2019
Click here for details.
Improvements to Rural Health Care Through Patient-Centered Research
June 26, 2019
Click here for details.
PCORI Advisory Panel on Patient Engagement Summer 2019 Meeting
June 27-28, 2019
Click here for details.
Leveraging Randomized Clinical Trials to Generate Real-World Evidence for Regulatory Purposes
July 11-12, 2019
Click here for details.
NVHR Hepatitis C Patient Summit
July 29-30, 2019
Click here for details.
2019 PCORI Annual Meeting
September 18-20, 2019
Click here for details.
FT Pharma Pricing and Value Summit 2019
September 26, 2019
Click here for details.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
12. Medical Journal Articles
Navigating Joint HTA, Procurement, and Fair Pricing: Evidence-Based Insights and Practical Recommendations - A Meeting Report from ISPOR Regional Conference in Warsaw, 2019, click here to view.
Health Technology Assessment as Part of a Broader Process for Priority Setting and Resource Allocation, click here to view.
Improving Quality Measure Maintenance: Navigating the Complexities of Evolving Evidence, click here to view.
A Tool for Empirical Equipoise Assessment in Multigroup Comparative Effectiveness Research, click here to view.
As Health Technology Assessment Evolves So Must its Approach to Patient Involvement, click here to view.
Evaluation of Value-Based Insurance Design for Primary Care, click here to view.
Leveraging Patient/Community Partnerships to Disseminate Patient Centered Outcomes Research in Geriatrics, click here to view.
Enabling Individualised Health in Learning Healthcare Systems, click here to view.
Analysis of Sponsor Hearings on Health Technology Assessment Decision Making, click here to view.
Study Verifies Value of Real-World Evidence, click here to view.
A Framework for Aiding the Translation of Scientific Evidence into Policy: The Experience of a Hospital-Based Technology Assessment Unit, click here to view.
13. AHRQ Effective Program Updates
Systematic Review: Treatment of Depression in Children, click here to view.
AHRQ EPC Program Helps Health Systems Use Evidence, click here to view.
Technical Brief: Pharmacological and Nonpharmacological Treatments for Post-Traumatic Stress Disorder, click here to view.
Systematic Review: Management of Infertility, click here to view.
Key Questions: Cervical Ripening in the Outpatient Setting, click here to view.
Key Questions: Radiation Therapy for Brain Metastases, click here to view.
Protocol: A Rapid Evidence Review of Retention Strategies for Medications for Addiction Treatment (MAT) in Adults with Opioid Use Disorder, click here to view.
Systematic Review: Diagnosis and Treatment of Clinical Alzheimer's-type Dementia (CATD), click here to view.
Systematic Review: Comparative Effectiveness of Analgesics to Reduce Acute Pain in the Prehospital Setting, click here to view.
Systematic Review: Long-Term Drug Therapy and Drug Holidays for Osteoporosis Fracture Prevention, click here to view.
Systematic Review: Telehealth for Acute and Chronic Care Consultations, click here to view.
White Paper: Standardized Library of Asthma Outcome Measures, click here to view.
Research Protocol: Characteristics of Existing Asthma Self-Management Education Packages, click here to view.
Systematic Review: Can Physical Activity Improve the Health of Wheelchair Users?, click here to view.