1. Sickle Cell Webinar and Survey, see details below.
2. Patients are Rightfully Wary of QALY-based Analyses, click here to read the article.
3. Don't Legalize Discrimination against People with Disabilities, click here to read the article.
4. ICER Discriminates against People with Rare Diseases, click here to read the article.
5. Colorado is Next to Propose Use of the Discriminatory QALY Method to Restrict Coverage, see details below.
6. An Independent Federal Agency Recognizes the Danger of QALYs, click here to read the blog post.
7. Friends of PCORI Reauthorization Congratulates PCORI's New Executive Director, click here to read the press release.
8. BPC Highlights Bipartisanship Driving PCORI’s Recent Reauthorization, see details below.
9. New National Health Council Patient Engagement Tool, click here to read more.
10. Patient-Reported Outcome Measures for Oncology Care Call for Public Comment -- Comment by January 29, click here to read more and to comment.
11. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
12. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, Arthritis, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, click here to provide patient input.
13. Upcoming Events and Webinars, see details below.
14. Medical Journal Articles, see details below.
15. AHRQ Effective Program Updates, see details below.
We are excited to invite you to join the second webinar on: The ICER Draft Evidence Report on Sickle Cell Disease: An Overview, Next Steps and Expectations. The webinar will be hosted by leading sickle cell disease groups and the Partnership to Improve Patient Care.
We welcome interested stakeholders, patients, advocates, caregivers and families to join a webinar about the progress of the ICER assessment to-date. ICER’s draft evidence report on sickle cell disease was just released on January 23, and the webinar will review the contents of the report and provide guidance and information for advocates who may want to provide comments on the report. You will hear updates from patient stakeholders on next steps leading up to the ICER in-person meeting on March 26, 2020 and subsequent final report.
January 30, 2020
RSVP to firstname.lastname@example.org
Sick Cells, the Sickle Cell Disease Association of America (SCDAA) and a multi-disciplinary task force are working with ICER to inform the evaluation of the clinical and economic impact of new treatments for SCD. We are asking patients and caregivers to complete the “My Life with Sickle Cell Disease” survey that will be live on Thursday, January 16th. The results of the survey will impact the ICER review in several ways:
- It will allow ICER to more accurately measure the impact SCD has on day-to-day activities.
- It will give caregivers a voice, which is often missing from clinical trial data.
- It will help ICER put a price tag on some of the expenses that result from SCD. Specifically, the survey will capture information about out-of-pocket costs and how much is spent out of cash or bank accounts to help manage SCD.
There are important areas of ICER’s report that may lack data, so completing the survey can help inform these critical pieces of the report. Survey closes on January 31st. Click here to take the survey.
2. Patients are Rightfully Wary of QALY-based Analyses
In an article highlighting the experience of two young men with Duchenne Muscular Dystrophy, PIPC Chairman Tony Coelho and Ari Ne'eman discuss the dangers of QALYs and why they do not serve patients well. "When you engage in that process of [evaluating drugs on the same measure], you lose some significant nuance in terms of the amount of benefit that’s being delivered," said Ne'eman. Chairman Coelho added, "I worry that more focus is being given to what is most cost-effective for the ‘average patient’ than creating a system that works for each individual patient." Click here to read the article.
3. Don't Legalize Discrimination against People with Disabilities
Writing in the Tennessean, the Partnership to Fight Chronic Disease's Kenneth Thorpe says that QALY-based "value assessments" are effectively legalized discrimination against people with disabilities. "The continued use of QALY analyses is a moral atrocity. Just because people are blind, or need a wheelchair, or live with an incurable disease or genetic disorder, that doesn't mean their lives are less valuable or they are less worthy of treatment. But that's precisely the premise behind QALY analyses, which are still considered legitimate by many policy-makers. It's time we ban the use of these unethical assessments and treat disabled Americans with the dignity they deserve," he writes. Click here to read the article.
4. ICER Discriminates against People with Rare Diseases
ICER's work could undermine treatments to help individuals living with rare diseases and treats them as less than people in perfect health, writes Randy Rutta. "This metric effectively discriminates against the elderly, persons with chronic conditions and other sicknesses, and people living with disabilities. ICER’s position is that these individuals aren't in perfect health to begin with. So, the group doesn't count an additional 12 months of life expectancy as a full QALY. For example, our hypothetical treatment for a genetic disease might still extend a 90-year-old infirm patient's life by one year, but ICER might count the gain as only 0.5 QALY, due to the perceived lower quality of his/her life. As a result, that same $30,000 treatment would cost $60,000 on a QALY-adjusted basis — potentially blocking access and affordability for patients," he says. Click here to read the article.
5. Colorado is Next to Propose Use of the Discriminatory QALY Method to Restrict Coverage
On December 12, 2019, the Colorado Department of Health CarePolicy and Financing released a report entitled “Reducing Prescription Drug Costs in Colorado.” The report proposes to reduce prescription drug costs in Colorado, including by “monitoring new ways to price prescription drugs, including QALY pricing methodologies.” The report specifically references ICER, stating, "A nonprofit in Boston called the Institute for Clinical and Economic Review (ICER) is using a calculation that factors in a dollar amount associated with being healthy in order to estimate how a drug should be priced...some drug manufacturers have moved toward aligning with ICER’s QALY-based dollar estimate when evaluating the price of certain newer drugs...Countries like Canada, Britain, Ireland and the Netherlands have used these types of calculations to leverage drug prices with manufacturers and to determine which drugs their government-funded health programs should cover...use of cost-per-QALY reporting such as that conducted by ICER may help payers leverage bigger discounts from drug makers, determine limitations to coverage for certain drugs, or indicate preferential coverage of alternative treatments with better estimated value. QALY pricing methodology is also an opportunity that could be explored by a newly-created Affordability Board.” PIPC has significant concerns that the QALY methodology is discriminatory, and shares the National Council on Disability’s opposition to using QALYs and referencing international prices that use QALYs. Click here to view the Colorado report. Click here to view our concerns about ICER and here to learn about QALYs. Click here for additional information about international care access challenges.
6. An Independent Federal Agency Recognizes the Danger of QALYs
PIPC published a new Data Mine blog post exploring the recent report from the National Council on Disability that finds that QALYs are discriminatory and should not be used to make coverage decisions. From the blog post: "Unfortunately, QALY-based analyses are already on our shores. While Medicare does not currently use a QALY-based analysis to inform coverage and reimbursement decisions, plenty of private insurers and states do. In light of this, NCD recommended that insurance programs jointly run by the Federal Government and the States, such as Medicaid, should not rely on cost- effectiveness research or reports that gather input from the public on health preferences that do not include the input of people with disabilities and chronic illnesses." Click here to read the blog post.
7. Friends of PCORI Reauthorization Congratulates PCORI's New Executive Director
With PCORI recently reauthorized for ten years, Dr. Nakela Cook will begin as PCORI's Executive Director in mid-April. Friends of PCORI Reauthorization co-chair and PIPC Chairman the Honorable Tony Coelho said “I want to congratulate Dr. Nakela Cook on being selected to serve as PCORI’s next Executive Director. With a newly minted 10-year vote of confidence from Congress, PCORI has a tremendous opportunity ahead to continue driving an efficient and informed health system that is truly patient-centered and responsive to the individual characteristics, needs, and priorities of patients and people with disabilities. We are excited to work with Dr. Cook in fulfilling PCORI’s statutory promise of amplifying the voices of patients and people with disabilities in determining research priorities.” Click here to read the press release.
8. BPC Highlights Bipartisanship Driving PCORI’s Recent Reauthorization
The Bipartisan Policy Center (BPC) recently announced it was pleased to see bicameral, bipartisan passage of certain health provisions, including a 10-year reauthorization of the Patient-Centered Outcomes Research Institute (PCORI), which is key to promoting patient access to evidence-based treatments and high-value care. Click here to view the BPC announcement. Click here to view the press release from PIPC Chairman Tony Coelho and Friends of PCORI Reauthorization co-chair the Honorable Dr. Phil Gingrey. Click here to read BPC's newsletter.
9. New National Health Council Patient Engagement Tool
The National Health Council released a new contracting principles template and fair market value calculator to aid in patient engagement efforts.
This project aims to support sustainable agreements between stakeholders and the patient and caregiver community as part of medical product development by:
- Developing, for U.S. use initially (adding other countries in the future), a fair-market value (FMV) calculator for compensation of patients and patient groups involved in “patient engagement activities” taking place between patient organizations and/or unaffiliated patients and private companies. The final product will be a calculator that companies can use and further customize for their own needs. Also, building blocks, such as engagement activities and principles, will be included.
- Adapting, for use in the U.S., the Conflict-of-Interest Principles and Privacy Principles (with examples) developed by the E.U. Patient Focused Medicines Development (PFMD) initiative to create standardization across all activities.
- Adapting, for use in the U.S., the contract templates developed by PFMD to simplify and standardize the process for patient and patient groups.
Click here to read more.
10. Patient-Reported Outcome Measures for Oncology Care Call for Public Comment -- Comment by January 29
The Pacific Business Group on Health is calling for public comment on its Patient-Reported Outcome Measures for Oncology Care, which it is cooperatively developing with the Centers for Medicare and Medicaid Services (CMS). From PBGH: "The aims for this project are to enhance patient-centered cancer quality measurement by 1) fully developing and testing patient-reported outcome-performance measures (PRO-PMs) regarding health-related quality of life (HRQOL) and pain for patients with breast, colon and lung cancer, and 2) preparing documentation for successful submission of the measures to National Quality Forum (NQF) and CMS." The comment period closes January 29, 2020. Click here to read more and to comment.
11. 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: Alberta Care will not cover the cost of a patient's lifesaving cancer treatment, forcing her to pay out of pocket. Since Health Canada won't cover a lifesaving spinal muscular atrophy treatment, one family is desperately trying to access the drug through fundraising or a lottery.
- New Zealand: Couple cycle to Parliament with a coffin in tow to raise awareness over Pharmac's failure to provide access to lifesaving drugs. Click here, here, here, and here to read more. One cancer patient who received a lifesaving drug through a compassionate care program is pushing Pharmac to fund the drug to help others. Pharmac is finally seeking proposals for new medicines for diabetes patients after neglecting them for years. Click here to read more.
- United Kingdom: UK child cancer free after seeking CAR-T Cell therapy in Singapore when NHS would not allow him access to it. NHS neglects Lyme Disease sufferers.
12. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, 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: Research Protocol available. 2/3/2020: Model Analysis Plan.
- Cystic Fibrosis: Model Analysis Plan available. 2/20/2020: Draft Evidence Report and Draft Voting Questions. Meeting 4/30/2020: CTAF will convene to deliberate and vote on evidence presented in ICER's report on treatments for cystic fibrosis.
- Sickle Cell Disease: Draft Evidence Report and Draft Voting Questions AVAILABLE, comment period open though 2/20/2020. 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.
- Acute Migraine: Midwest CEPAC met 1/23/2020 to review ICER's assessment of acute migraine treatments. 2/13/2020: Final Evidence Report.
- 2020 Value Assessment Framework: 1/31/2020: Final Framework Adaptations.
- Non-Alcoholic Steatohepatitis: Research Protocol available. 2/5/2020: Model analysis plan.
- Beta Thalassemia: Draft Scoping Document AVAILABLE, comment period OPEN through TODAY, 1/27/2020. 2/4/2020: Revised Scoping Document.
- Hemophilia A: Draft Scoping Document AVAILABLE, comment period open through 2/13/2020.
13. Upcoming Events and Webinars
Patient Registries, Real World Evidence and HEOR
January 27-28, 2020
Click here for details.
PCORI Board of Governors Meeting
January 28, 2020
Click here for details.
PCORI Advisory Panel on Patient Engagement Winter 2020 Meeting
January 31, 2020
Click here for details.
PCORI Board of Governors Meeting
February 25, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
14. Medical Journal Articles
HTA Around the World: Broadening Our Understanding of Cross-Country Differences, click here to view.
Early Returns From the Era of Precision Medicine, click here to view.
Who Assigns Value in Value-Based Insurance Design?, click here to view.
Does the Institute for Clinical and Economic Review Revise Its Findings in Response to Industry Comments?, click here to view.
The Patient-Centered Outcomes Research Network Antibiotics and Childhood Growth Study: Implementing Patient Data Linkage, click here to view.
Viewpoint: Adding Patient-Reported Outcomes to Medicare's Oncology Value-Based Payment Model, click here to view.
Beyond Involvement: Multiple Methods and Purposes of Shared Decision Making, click here to view.
Behavioral Economic Insights to Improve Medication Adherence in Adults with Chronic Conditions: A Scoping Review, click here to view.
The Need for Clinical Judgement in the Application of Evidence-Based Medicine, click here to view.
Building Capacity in Evidence-Based Medicine in Low-Income and Middle-Income Countries: Problems and Potential Solutions, click here to view.
A Review and Synthesis of Frameworks for Engagement in Health Research to Identify Concepts of Knowledge User Engagement, click here to view.
15. AHRQ Effective Program Updates
Research Protocol: Cervical Ripening in the Outpatient Setting, click here to view.
Research Protocol: Treatments for Acute Episodic Migraine, click here to view.
Research Protocol: Mixed Methods Review: Integrating Palliative Care With Chronic Disease Management in Ambulatory Care, click here to view.
Online Training for SRDR+ Now Available at the Evidence Synthesis Academy, click here to read more.
Research Protocol: Treatments for Acute Pain: A Systematic Review, click here to view.
Research Report: Registries for Evaluating Patient Outcomes: A User's Guide, click here to view.
Research Protocol: Management of High-Need, High-Cost Patients: A Realist and Systematic Review, click here to view.
Systematic Review: Achieving Health Equity in Preventive Services, click here to view.
Research Protocol: Strategies for Patient, Family, and Caregiver Engagement, click here to view.
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.
Research Protocol: Management of Primary Headache During Pregnancy, click here to view.