1. Please Join PIPC For Our 10th Annual Forum!, click here to view the invitation.
2. National Council on Disability Report on QALYs, click here to read the report.
3. Advocates Speak Out Against ICER's Flawed and Discriminatory Methods, click here to read the letters.
4. Patient-Centered Outcomes Research Institute: Healthcare's Savior?, click here to read the article.
5. Banning QALYs Helps People Get Care They Deserve, click here to read the article.
6. International Reference Pricing Would Lead to Fewer New Medicines For People with Disabilities, Report Finds, click here to see the report.
7. PCORI Reauthorization Discussions Advance, see details below.
8. PIPC Urges Policymakers to Protect Patients and People with Disabilities, see below for details.
9. EveryLife Foundation Briefing with the Rare Disease Caucus, click here to learn more and register.
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: Acute Migraine, Type 2 Diabetes, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, 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 Partnership to Improve Patient Care (PIPC) will hold its 10th Annual Forum on December 16, 2019 from 12:00 - 2:00 p.m. at the Reserve Officers Association in Washington, DC. We will be joined by Ana Torres-Davis who will share an overview of the National Council on disability’s important and timely recent report: “Quality Adjusted Life Years and the Devaluation of Life with a Disability.” We will also learn about international healthcare systems from real patients, caregivers, and technical policy experts. RSVP to email@example.com. Click here to view the invitation.
2. National Council on Disability Report on QALYs
The National Council on Disability issued a new report assessing the discriminatory effects of using QALYs to determine treatment coverage. "In an effort to lower their healthcare costs, public and private health insurance providers have utilized the Quality Adjusted Life Year (QALY) to determine the cost-effectiveness of medications and treatment," the Council wrote. "QALYs place a lower value on treatments which extend the lives of people with chronic illnesses and disabilities. In this report, NCD found sufficient evidence of the discriminatory effects of QALYs to warrant concern, including concerns raised by bioethicists, patient rights groups, and disability rights advocates about the limited access to lifesaving medications for chronic illnesses in countries where QALYs are frequently used. In addition, QALY-based programs have been found to violate the Americans with Disabilities Act." In addition, the report calls on Congress to ban the use of QALYs in Medicare and Medicaid and recommended that the administration refrain from modeling US pricing after the pricing in other countries, calling on the administration specifically to rescind its proposed rule to use an international pricing index in Medicare Part B. Click here to read the report.
3. Advocates Speak Out Against ICER's Flawed and Discriminatory Methods
Patient advocates and economists spoke out following an article in the Wall Street Journal that put the spotlight on ICER and its discriminatory value assessments based on QALYs. Sue Peschin of the Alliance for Aging Research, a PIPC member, wrote, "ICER makes value assessments on drugs and other clinical options that rely on deeply flawed cost/QALY thresholds. If we embrace this approach, we are embracing health-care rationing. Studies show that rationing in European countries not only results in access issues, but also translates to higher mortality." Jennifer Bright of the Innovation and Value Initiative wrote that "methods must evolve to provide context-specific insights and better capture what drives value from patients’ perspectives—factors that currently aren’t well represented in the QALY or in clinical data." Click here to read the letters.
4. Patient-Centered Outcomes Research Institute: Healthcare's Savior?
PCORI's successes more than justify its reauthorization, wrote the Bipartisan Policy Center's Bill Hoagland and Anand Parekh. "PCORI-funded studies are producing results that promise to help patients make better-informed treatment decisions that are right for them. They also reduce the burden that many major health conditions, such as cancer, heart disease, opioid abuse, obesity, and arthritis impose on patients, their families, and the healthcare system. In a growing number of cases, that includes cost savings for patients and insurers. By reauthorizing the Patient-Centered Outcomes Research Trust Fund, Congress has an opportunity to reaffirm its support for patient-centered care," the pair wrote in MedPage Today. Click here to read the article.
5. Banning QALYs Helps People Get Care They Deserve
Massachusetts is considering passage of a bill that would ban the use of discriminatory QALYs in coverage determinations. "In Massachusetts, roughly 690,000 patients suffer from some form of rare disease. If the state began relying on QALY assessments, those patients could lose access to lifesaving treatments, such as gene therapies....Widespread use of QALY analyses would cripple drug development. If companies aren't sure whether government programs like Medicaid will cover their medicines, they won't invest billions of dollars in risky research projects in the first place. Misguided value assessments imperil patients. By prohibiting state officials from ever using QALY analyses, Massachusetts lawmakers can protect their constituents and set an example for the rest of the country," wrote Emory University's Kenneth Thorpe about the potential use of QALYs and the proposed ban. Click here to read the article.
6. International Reference Pricing Would Lead to Fewer New Medicines For People with Disabilities
Vital Transformations modeled the impact of a proposal to institute international reference pricing on the biotechnology sector and found that revenue reductions would particularly hurt small, innovative firms that are developing the cures of tomorrow. "Targeted therapies, medicines requiring long-term outcomes, and medicines with lower revenue potential are among the types most likely to be affected by H.R.3’s drastic change to economic incentives. This includes areas where the science is toughest, such as in Alzheimer’s and ALS," the authors wrote. Click here to see the report.
7. PCORI Reauthorization Discussions Advance
Over the summer, the House Energy and Commerce and House Ways and Means Committees marked up legislation to reauthorize the Patient-Centered Outcomes Research Institute (PCORI), for 3 years and for 7 years with limited changes to its underlying priorities, respectively. While PCORI reauthorization was included in legislation to extend various health programs until November 21, 2019, the PCOR trust fund was extended without extending the funding for it. Most recently, Senators Bill Cassidy (R-LA), Mark Warner (D-VA), Shelley Moore Capito (R-WV) and Chris Van Hollen (D-MD) shared a discussion draft of legislation reauthorizing PCORI with full funding for 10 years that proposed additional policy changes for PCORI, and invited stakeholders to provide input on those proposed policy changes. PIPC provided comments on the discussion draft highlighting support for an expert advisory panel for high-impact research, as well as a long-term, fully funded reauthorization that protects PCORI’s patient-centered mission and extends all of the existing funding mechanisms. PIPC also urged consideration of extending the protections in Section 1182 more broadly to cover Medicaid, the CMS Innovation Center, and other reforms that would apply value judgments to national policy decisions. PIPC opposes added payer seats on the PCORI Board Governors and an accompanying shift of the selection of the Methodology Committee to the Board of Governors. Friends of PCORI Reauthorization, a coalition of 184 organizations, has consistently communicated to both House and Senate leaders support for PCORI to remain patient-centered, and specifically support for a 10 year, fully funded reauthorization that continues to direct PCORI to fund comparative clinical effectiveness research. Click here to view PIPC’s comments or click here for information about Friends of PCORI Reauthorization.
8. PIPC Urges Policymakers to Protect Patients and People with Disabilities
PIPC Chairman Tony Coelho expressed concern about policies placing hard-fought existing patient protections under serious threat, especially those relying on policy decisions made in other countries that we know establish prices based on discriminatory cost-effectiveness standards (such as Quality-Adjusted Life Years or QALYs) and similar average metrics. PIPC supports policies to improve care and affordability for patients, adopt and extend safeguards in current statute that prohibit application of discriminatory cost-effectiveness thresholds in Medicare (whether done overtly by Medicare or covertly via “comparative effectiveness analysis” or reference to foreign countries’ use of these thresholds) and codify criteria for patient-centeredness called for in CMMI’s statute. Click here to view prior stakeholder letter on referencing International pricing. Click here to view information on Value Our Health.
9. EveryLife Foundation Briefing with the Rare Disease Caucus
The EveryLife Foundation for Rare Diseases for The Rare Disease Caucus is holding a briefing hosted in partnership with the Rare Disease Congressional Caucus. The briefing will share important information and research on the economic burden of rare diseases. It will be held on December 4th at 11:30 am in Rayburn House Office Building, Room 2075. Click here to learn more and register.
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.
- Australia: Migraine sufferers are being denied treatments to innovative new treatments that can help them keep jobs and live migraine-free.
- Canada: Patients say that they are denied life-changing cystic fibrosis medication due to the government's unwillingness to negotiate.
- New Zealand: Another patient has died due to New Zealand's switching covered epilepsy medication, and now Pharmac has backed down from the switch after a backlash. Click here to read more.
- United Kingdom: Patients desperate for new cystic fibrosis drug call for no delay in bringing it to UK patients. Toddler has to go to Singapore for CAR-T treatment as it is not available on the NHS.
11. ICER's QALY-Based Study Topics: Acute Migraine, Type 2 Diabetes, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis
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:
- Ulcerative Colitis: Revised Scoping Document available. 12/11/2019: Research Protocol.
- Cystic Fibrosis: Revised Scoping Document available. 12/5/2019: Research Protocol.
- Sickle Cell Disease: Model Analysis Plan available. 1/22/2020: Draft Evidence Report and Draft Voting Questions. Meeting 3/26/2020: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for sickle cell disease.
- Arthritis: 11/26/2019: Evidence Report, Revised Voting Questions, and Responses to Public Comments. Meeting 12/9/2019: CTAF to an update to its 2017 rheumatoid arthritis assessment.
- Type 2 Diabetes: 12/9/2019: Final Evidence Report and Meeting Summary. Meeting 11/14/2019: The New England CEPAC convened to deliberate on ICER's review of oral semaglutide for the treatment of type 2 diabetes.
- Acute Migraine: Draft Evidence Report and Draft Voting Questions AVAILABLE, Comment Period OPEN through 12/6/2019. Meeting 1/23/2020: Midwest CEPAC to review ICER's assessment of acute migraine treatments
- 2020 Value Assessment Framework: 1/31/2020: Final Framework Adaptations.
- Non-Alcoholic Steatohepatitis: Draft Scoping Document available. 12/2/2019: Revised Scoping Document.
12. Upcoming Events and Webinars
PCORI Stakeholder Workshop on Maternal Morbidity and Mortality
December 5, 2019
Click here for details.
Advisory Panel on Healthcare Delivery and Disparities Research Winter 2019 Meeting
December 6, 2019
Click here for details.
Webinar: Providing Quality Benefits for Employees with Cardiovascular Disease and Other Chronic Conditions
December 10, 2019
Click here for details.
Webinar: Request for Proposals – Addressing Barriers to Medication Access
December 12, 2019
Click here for details.
PCORI Stakeholder Workshop on Suicide Prevention
December 18, 2019
Click here for details.
EveryLife Foundation’s A Rare Affair fundraiser at the JP Morgan Healthcare Conference
January 12, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
13. Medical Journal Articles
A Review and Synthesis of Frameworks for Engagement in Health Research to Identify Concepts of Knowledge User Engagement, click here to view.
Characteristics of Public Comments Submitted to State Health Technology Assessment Programs in Oregon and Washington, click here to view.
What Is Next for Patient Preferences in Health Technology Assessment? A Systematic Review of the Challenges, click here to view.
Commentary: One Size Does Not Always Fit All in Value Assessment, click here to view.
Engaging the Patient: Patient-Centered Research, click here to view.
Little Consistency In Evidence Cited By Commercial Plans For Specialty Drug Coverage, click here to view.
Ten Ways to Optimise Evidence-Based Policy, click here to view.
Mapping of Health Technology Assessment in China: Situation Analysis and International Comparison, click here to view.
Reflections on the ISPOR Special Task Force on U.S. Value Frameworks: Implications of a Health Economics Approach for Managed Care Pharmacy, click here to view.
Real-World Evidence: Research Reveals a Lack of Racial Diversity in Clinical Trials for Cancer Drugs, click here to view.
14. AHRQ Effective Program Updates
Research Report: Translation of a C. difficile Treatment Clinical Pathway Into Machine-Readable Clinical Decision Support Artifacts Prototyped for Electronic Health Record Integration, click here to view.
OPEN FOR COMMENT THROUGH 12/20/2019: Key Questions: Prehospital Airway Management: A Systematic Review, click here to view.
Research Protocol: Management of Primary Headache During Pregnancy, click here to view.
Research Report: Assessing the Accuracy of Machine-Assisted Abstract Screening With DistillerAI: A User Study, click here to view.
Research Report: Performance and Usability of Machine Learning for Screening in Systematic Reviews: A Comparative Evaluation of Three Tools, click here to view.
OPEN FOR COMMENT THROUGH 11/26/2019: Systematic Review: Interventions for Substance Use Disorders in Adolescents: A Systematic Review, click here to view.
Research Report: Facilitating the Implementation of EPC Reports in Learning Health Systems Engaged in Quality Initiatives: an EPC Pilot Project on ADHD, click here to view.
Research Protocol: Interventions for Dyspnea in Patients with Advanced Cancer, click here to view.
Technical Brief: Treatment for Acute Pain: An Evidence Map, click here to view.
Research Report: Pilot To Promote Entry of Structured Data Into the Systematic Review Data Repository (SRDR), click here to view.