1. Patients and People with Disabilities Remain Concerned about Importing QALYs From Other Countries, see details below.
2. Administration’s Medicaid Initiative Would Invite Closed Formularies, Implicate Use of QALYs, see details below.
3. ICER Releases Revised Value Assessment Framework for 2020, see details below.
4. Webinar on ICER's Sickle Cell Report, click here to view the webinar.
5. Undark: Patients are Rightfully Wary of QALY-based Analyses, click here to read the article.
6. Colorado is Next to Propose Use of the Discriminatory QALY Method to Restrict Coverage, see details below.
7. An Independent Federal Agency Recognizes the Danger of QALYs, click here to read the blog post.
8. New National Health Council Patient Engagement Tool, click here to read more.
9. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
10. 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.
11. Upcoming Events and Webinars, see details below.
12. Medical Journal Articles, see details below.
13. AHRQ Effective Program Updates, see details below.
PIPC Chairman Tony Coelho called on advocates to urge the Administration to follow the recommendations of the National Council on Disability opposing policies that would reference health care prices and policies established in other countries using discriminatory QALYs and similar summary metrics. Chairman Coelho stated, "Is the life of a person with a disability or chronic condition less valuable than the life of a person without one? There is renewed interest among lawmakers to tackle healthcare costs, specifically, the rising cost of prescription drugs. However, the Administration’s proposed International Pricing Index (IPI) model, an initiative proposed in 2018 that would arbitrarily base prices of American medications off the government set prices of medications in foreign countries, is deeply flawed. Yet, we may see some version of it reintroduced soon.” Click here to view the PIPC Chairman Blog. Click here to view a letter from advocates opposing an International Pricing Index. Click here for more information from experts.
2. Administration’s Medicaid Initiative Would Invite Closed Formularies, Implicate Use of QALYs
On January 30, 2020, the administration announced the Healthy Adult Opportunity (HAO) initiative allowing states to carry out demonstrations under section 1115(a)(2) of the Social Security Act (the Act) to provide "cost-effective" coverage using "flexible benefit designs.” Among its provisions, State Medicaid Directors were invited to "adopt a closed formulary in line with Essential Health Benefit (EHB) requirements (with special protections for individuals with HIV and behavioral health conditions)…This means that the open formulary requirements in section 1927 that apply to state plan drug coverage do not apply.” PIPC remains concerned that states will respond to increased opportunities to limit formularies and restrict access to therapies by referencing “cost effectiveness” studies that discriminate against patients and people with disabilities by using quality-adjusted life years (QALYs) and similar summary metrics. Click here to view the letter to State Medicaid Directors. Click here to view stakeholder comments opposing Tennessee’s waiver application. Click here to view a legislative template for states to advance patient-centered research in decision-making.
3. ICER Releases Revised Value Assessment Framework for 2020
On Jan. 31, the Institute for Clinical and Economic Review (ICER) released its final revisions to its value assessment framework. The document describes changes in ICER’s process, including creating a formal process for incorporating new evidence that emerges after the assessment, standardizing cost-effectiveness thresholds across reviews, and increasing the incorporation of real-world evidence.
While the revised framework seeks to formalize a patient engagement program, the program falls short in committing to meaningful incorporation of patient perspectives and preferences into ICER’s reviews. ICER commits to work with patient groups throughout the process, but it does little to ensure that the patient voice is heard. The framework also makes it clear that economic analysis still maintains primacy at ICER and would be counted as more valuable than patient input.
In response to a request for feedback, stakeholders submitted comments on ICER’s draft 2020 value assessment framework last summer, but ICER failed to incorporate their suggestions in several important ways. First and foremost, ICER doubles down on its embrace of the discriminatory QALY measure and its cousin, the equal value of life years gained (evLYG) measure. Stakeholders also called on ICER to meaningfully incorporate patient preferences, but ICER instead opted to ceremonially consider patient input and not commit to patient-centered decision making.
The group did adopt a process to use real-world evidence (RWE) to update assessments of treatments that were approved under an accelerated pathway after the treatment has been on the market for two years. ICER does not, however, commit to using RWE in its base models, choosing to continue to conduct rushed assessments before appropriate data are available.
Unfortunately, ICER’s updated framework does not take a meaningful turn towards patient-centeredness. Under these revised guidelines, ICER will continue to use discriminatory measures to argue for reduced access to life-changing treatments. Click here to view the final value assessment framework. Click here to view PIPC's comments on the framework. Click here to read PIPC's Data Mine blog and see a table comparing PIPC's comments to ICER's final product.
4. Webinar on ICER's Sickle Cell Report
On Jan. 30, 2020 leading sickle cell disease groups and the Partnership to Improve Patient Care (PIPC) held a webinar entitled "The ICER Draft Evidence Report on Sickle Cell Disease: An Overview, Next Steps and Expectations." Interested stakeholders, patients, advocates, caregivers and families received an update on 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 reviewed the contents of the report and provide guidance and information for advocates who may want to provide comments on the report. The presentation also provided information from patient stakeholders on next steps leading up to the ICER in-person meeting on March 26, 2020 and subsequent final report. Click here to view the webinar.
5. Undark: 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.
6. 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.
7. 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.
8. 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.
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.
- 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.
10. 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: Model Analysis Plan available. 4/1/2020: Draft Evidence Report and Draft Voting Questions.
- 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: Final Framework Adaptations AVAILABLE.
- Non-Alcoholic Steatohepatitis: Research Protocol available. 2/5/2020: Model analysis plan.
- Beta Thalassemia: Draft Scoping Document available. 2/4/2020: Revised Scoping Document.
- Hemophilia A: Draft Scoping Document AVAILABLE, comment period open through 2/13/2020.
11. Upcoming Events and Webinars
Driving Evidence-Based Health Plan Coverage by Recognizing Gaps, Updating Practices
February 13, 2020
Click here for details.
Engagement Awards LOI Town Hall: April 2020 Cycle
February 13, 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.
12. 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.
13. AHRQ Effective Program Updates
2019 Year in Review—Accomplishments From the Evidence-based Practice Center Program, click here to view.
Research Protocol: Radiation Therapy for Brain Metastases: A Systematic Review, click here to view.
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.