
1. National Council on Disability Report on QALYs, click here to read the report.
2. PIPC Submits Comment Letter on ICER Draft Report for Rheumatoid Arthritis, click here to read the letter.
3. AUCD: What is a QALY?, click here to read the article.
4. PCORI Reauthorization Discussions Advance, see details below.
5. Video: Risk of Modeling Other Countries’ Drug Pricing: How a Canadian Child is Forced to Get Sicker to Access Treatment, click here to watch the video.
6. PIPC Urges House to Protect Patients and People with Disabilities in Title 1 of New House Bill, see below for details.
7. EveryLife Foundation Briefing with the Rare Disease Caucus, click here to learn more and register.
8. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
9. ICER Studies: Acute Migraine, Type 2 Diabetes, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, click here to provide patient input.
10. Upcoming Events and Webinars, see details below.
11. Medical Journal Articles, see details below.
12. AHRQ Effective Program Updates, see details below.
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 Forum 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. Click here to read the report.
2. PIPC Submits Comment Letter on ICER Draft Report for Rheumatoid Arthritis
In a comment letter to the Institute for Clinical and Economic Review (ICER), the Partnership to Improve Patient Care (PIPC) offered feedback on ICER’s draft evidence report for rheumatoid arthritis. PIPC Chairman Tony Coelho strongly encouraged ICER to address the shortcomings of its report, specifically criticizing the lack of patient input and use of Quality Adjusted Life Years (QALY)-based methods. He also pointed out that ICER continues to prioritize randomized clinical trial data over real world evidence (RWE), despite the existence of strong RWE on rheumatoid arthritis. “Given the severity of the disease, and the large patient population, we urge you to evaluate these treatments with the perspectives of patients at the center and using methodologies that will improve, not inhibit, the ability of patients to access treatments that they need.” wrote Chairman Coelho. Click here to read the letter.
3. AUCD: What is a QALY?
The AUCD's Nina Erbes wrote about why QALYs pose a danger to people with disabilities. In a blog post explaining how QALYs fail to capture patient experience and can harm the lives of people with disabilities, she cited a presentation from PIPC's Ari Ne'eman and Sara van Geertruyden. "By giving a number value to a year in a person’s life, and saying that someone with a disability has a lower number than someone without a disability, QALYs ignore that people with disabilities can live good and full lives. If someone has a medical condition, a year in their life is not worth less than a year in the life of someone who does not. A person with a disability may live a different life than someone without a disability, but the life of a person with a disability is still just as valuable. QALYs also don’t consider the experiences of people with disabilities. Someone with a disability knows what their own life is like, and an insurance company can’t know exactly how that person feels," she wrote. Click here to read the article.
4. 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.
5. Video: Risk of Modeling Other Countries’ Drug Pricing: How a Canadian Child is Forced to Get Sicker to Access Treatment
Access to treatments is a constant struggle for patients in countries like Canada, where access is limited based on cost effectiveness or assessments of average clinical benefit. We are sharing a video developed in Canada with a mother, Sandy, and her daughter, Laura, who has cystic fibrosis. Sandy and her daughter Laura spoke honestly and freely about their experience with the Canadian health system. It is a heartbreaking story of the requirement to get sicker in order to access a new treatment she needs due to government utilization management for drugs, e.g. fail first policies. As our country considers modeling international drug pricing policies, it demonstrates the inherent risk for patient access to care. Click here to watch the video.
6. 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.
7. 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.
8. 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.
- Canada: As U.S. approves "miracle" new cystic fibrosis drug, Canada's resistance to paying for new drugs comes into sharp relief.
- New Zealand: As Pharmac deliberates, a child with spinal muscular atrophy continues to have his condition deteriorate. Parents of child with SMA wonder why government will not help. Pharmac is believed to have raided a fund meant for rare diseases, leading to more barriers helping patients in need. Pharmac's epilepsy drug switch has led to patients having seizures.
- United Kingdom: UK woman dies weeks before she was slated to try a new cystic fibrosis drug. Condition of a child with cystic fibrosis is deteriorating as they await a coverage decision.
9. ICER Studies: 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: Research Protocol available. 11/21/2019: Model Analysis Plan. 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: Evidence Report and Voting Questions 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.
- 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
- Valuing A Cure Project: 11/15/2019: Revised White Paper.
- 2020 Value Assessment Framework: 12/18/2019: Final Framework Adaptations.
- Non-Alcoholic Steatohepatitis: Draft Scoping Document available. Comment period OPEN through 11/20/2019.
10. Upcoming Events and Webinars
Personalized Medicine Conference
November 13-14, 2019
Click here for details.
Stay, Safe, Side: Seizure First Aid & Priorities for the Epilepsy Community
November 14, 2019
9:30 a.m.
Rayburn House Office Building, Room 2168
RSVP to [email protected].
Webinar: The National Patient-Centered Clinical Research Network (PCORnet)
November 14, 2019
Click here for details.
PCORI Board of Governors Meeting
November 17, 2019
Click here for details.
2019 AUCD Annual Meeting
November 17-20, 2019
Click here for details.
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.
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.
11. Medical Journal Articles
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.
Feasibility of Using Real-World Data to Replicate Clinical Trial Evidence, click here to view.
Variation in US Private Health Plans' Coverage of Orphan Drugs, click here to view.
Incorporating Patient Perspectives and Transparency for Patient-Centered Value Assessment, click here to view.
Accounts from developers of generic health state utility instruments explain why they produce different QALYs: A qualitative study, click here to view.
The Shared Decision-Making Process in the Pharmacological Management of Depression, click here to view.
Training Patients to Review Scientific Reports for the Patient-centered Outcomes Research Institute: An Observational Study, click here to view.
Patient Reported Outcomes Have Arrived: A Practical Overview for Clinicians in Using Patient Reported Outcomes in Oncology, click here to view.
Prioritizing Evidence-Based Interventions for Dissemination and Implementation Investments: AHRQ's Model and Experience, click here to view.
12. AHRQ Effective Program Updates
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.
OPEN FOR COMMENT THROUGH 11/12/2019: Noninvasive Nonpharmacological Treatments for Chronic Pain: A Systematic Review Update, click here to view.
OPEN FOR COMMENT THROUGH 11/12/2019: Systematic Review: Opioid Treatments for Chronic Pain, click here to view.
OPEN FOR COMMENT THROUGH 11/12/2019: Systematic Review: Nonopioid Pharmacologic Treatments for Chronic Pain, click here to view.
Research Report: Pilot To Promote Entry of Structured Data Into the Systematic Review Data Repository (SRDR), click here to view.
Systematic Review: Pharmacologic and Nonpharmacologic Therapies in Adult Patients With Exacerbation of COPD, click here to view.
Research Protocol: Management of Colonic Diverticulitis, click here to view.