1. ICER Releases Cystic Fibrosis Draft Evidence Report: Learn More, see details below.
2. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Sickle Cell Disease, click here to read the letter.
3. Washington State Senate Votes to Give Prescription Drug Affordability Board Power to Use Cost Thresholds Without Patient Representation, see details below.
4. Pioneer Institute: ICER's Methods Are Likely Illegal under the ADA, click here to read the report.
5. Massachusetts Finalizes Value Assessment Regulations, see details below.
6. ICER's Oral Semaglutide Model Exists in an Imaginary World, click here to read the article.
7. International News: What Happens in Countries Using QALYs and Cost-Based Thresholds to Determine Coverage? See below for more.
8. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, click here to provide patient input.
9. Upcoming Events and Webinars, see details below.
10. Medical Journal Articles, see details below.
11. AHRQ Effective Program Updates, see details below.
On February 20, 2020, the Institute for Clinical Economic Review (ICER) released its draft evidence report for cystic fibrosis treatments, concluding that "CFTR modulator therapies plus best supportive care substantially improve patient health outcomes compared to best supportive care” but exceeded "commonly used cost-effectiveness thresholds.” Stakeholders are able to comment until March 18, 2020. To assist advocates, Cystic Fibrosis Research, Inc. invited Ari Ne’eman to conduct a webinar explaining the quality-adjusted life year methodology that is being used by ICER to determine the “cost effectiveness” of new treatments for cystic fibrosis. Click here to view the webinar. Click here to view the draft evidence report.
2. PIPC Submits Comment Letter to ICER on Draft Evidence Report for Sickle Cell Disease
In a letter to the Institute for Clinical and Economic Review (ICER), Partnership to Improve Patient Care (PIPC) Chairman Tony Coelho offered feedback on ICER’s draft evidence report for Sickle Cell Disease (SCD) treatments. Chairman Coelho expressed concerns with ICER’s use of the QALY metric to assess SCD treatments, saying that the QALY is a particularly inappropriate method for evaluating any value accrued from interventions aimed at its alleviation. The letter also criticizes ICER for choosing to use claims data instead of listening to input from patient and clinician stakeholders. “ICER risks doing the SCD community a disservice by using the QALY, a metric highly inappropriate for the assessment of SCD treatments, and by failing to listen to the community about factors like standard of care and outcomes that matter to patients that should be meaningfully incorporated into its model,” wrote Chairman Coelho. Click here to read the letter.
3. Washington State Senate Votes to Give Prescription Drug Affordability Board Power to Use Cost Thresholds Without Patient Representation
On February 18, 2020, the Washington State Senate passed SBB 6088, a bill establishing the prescription drug affordability board (board) that requires the board to identify prescription drugs priced above a certain threshold, with authority to conduct cost reviews of drugs and set upper payment limits for state purchasers. The legislation does not bar the Board from utilizing discriminatory quality-adjusted life years (QALYs) as the metric for determining whether treatments exceed certain thresholds. The Board also does not include representation from patients or people with disabilities, only "five members who have expertise in health care economics or clinical medicine appointed by the governor.” The legislation heads toward state House consideration this week, highlighting the continued challenge that patient and disability advocates face in combatting discrimination in state Medicaid programs. Click here to view the bill summary. Click here to view value assessment principles supported by patients and people with disabilities. Click here to learn more about state activities related to QALYs and here to view a state legislative template supported by Value Our Health.
4. Pioneer Institute: ICER's Methods Are Likely Illegal under the ADA
A new report from the Pioneer Institute found that ICER's methods for value assessment are likely illegal under the Americans with Disabilities Act (ADA) if applied to government programs because they may decrease the availability of effective treatments for people living with a disability and increase the risk of institutionalization of certain people living with a mental disability. Pioneer Institute’s report predicts that a legal challenge to any adoption of ICER’s QALY methodology by state Medicaid systems on ADA grounds seems almost certain. Click here to read the report.
5. Massachusetts Finalizes Value Assessment Regulations
The Massachusetts Health Policy Commission finalized its regulations on how they will evaluate the “value” of medications as a part of the oversight and administration of prescription drug pricing. Advocates sent letters to the Commission and met in person with the commission to advocate for an explicit ban on the use of quality-adjusted life years (QALYs) in the regulation. The Commission ultimately did not include any provisions that would ban or even restrict or limit the use of QALY-based value assessments such as those conducted by the Institute for Clinical and Economic Review (ICER). And they opened the door to international reference pricing from countries that use QALYs. The final regulation did include new language on transparency and public input that may be useful in allowing patients and people with disabilities to make their voices heard in this process. But it was very disappointing that the Commission did nothing to acknowledge that QALYs discriminate, despite significant engagement.
In response to the Commission’s final regulation, PIPC joined patient organizations in Massachusetts in sending out a press statement. We stated, "Massachusetts has always been a leader in both health care and inclusion, and this regulation does not honor that legacy.” We also shared the National Council on Disability report on QALYs and the comment letter that advocates sent to the HPC.
6. ICER's Oral Semaglutide Model Exists in an Imaginary World
Writing in Innovations in Pharmacy, University of Minnesota researcher Paul Langley, Ph.D. writes that ICER's Oral Semaglutide model has little semblance to reality. "While this model differs from previous ICER models, the result is still a framework that constructs a so-called evidence base that fails the demarcation test. It is best described as pseudoscience. The model creates, by assumption, an imaginary world. The claims made for oral semaglutide by ICER should not be taken seriously by health care decision makers. The purpose of this commentary is to point to the limitations of the model with particular reference to the utility metrics employed, the resulting claims for quality adjusted life years (QALYs) and consequent recommendations for price discounting and affordability," Dr. Langley writes. Click here to read the article.
7. 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: Multiple Sclerosis patient's condition is worsening and she wants to be the first Australian to use a life-changing new drug. Parents of child with spinal muscular atrophy desperate to access gene therapy for their daughter before she turns two. Blood cancer is killing Australians, and access to treatment is critical.
- Canada: Crohn's patient struggles with forced transition to different medicine. Family of cystic fibrosis patient worries that without access to a new drug, she may not have much time left.
- New Zealand: Patients forced to pay taxes on medications that are denied to them by the government.
- United Kingdom: Young mother needs access to cystic fibrosis medications to continue to care for her children. NHS will not provide patients with adequate care so they resort to crowdfunding.
8. ICER's QALY-Based Study Topics: Hemophilia A, Beta Thalassemia, Acute Migraine, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments
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. Meeting 6/30/2020: CTAF will convene to deliberate and vote on evidence presented in ICER's report on ulcerative colitis therapies.
- Cystic Fibrosis: Draft Evidence Report and Draft Voting Questions AVAILABLE, comment period OPEN through 3/18/2020. 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: 3/12/2020: Evidence Report and Responses to Comments. 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. 2/25/2020: Final Evidence Report.
- Non-Alcoholic Steatohepatitis: Model analysis plan available. 3/19/2020: Draft Evidence Report. Meeting 5/28/2020: The Midwest CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for NASH.
- Beta Thalassemia: Revised Scoping Document available. 3/11/2020: Research Protocol.
- Hemophilia A: Draft Scoping Document available. 2/25/2020: Revised Scoping Document. Meeting 8/3/2020: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for hemophilia.
- Bladder Cancer: Open input period through 3/10/2020. 3/13/2020: Draft Scoping Document.
- Opioids: Digital Apps: Open input period through 3/10/2020. 3/13/2020: Draft Scoping Document.
- Opioids: Supervised Injection Centers: Open input period, no end date specified.
9. Upcoming Events and Webinars
PCORI Board of Governors Meeting
February 25, 2020
Click here for details.
PCORI Board of Governors Meeting
March 2, 2020
Click here for details.
Webinar: Pharmaceutical Contracting: Focus on Value-Based Contracting
March 3, 2020
Click here for details.
Market Access USA at eyeforpharma Philadelphia
April 15, 2020
Click here for details.
FT US Pharma and Biotech Summit 2020
May 14, 2020
Click here for details.
10. Medical Journal Articles
Physician and Patient Adjustment to Reference Pricing for Drugs, click here to view.
Patient-Centered Outcomes Research and the Injured Patient: A Summary of Application, click here to view.
Does the Use of Health Technology Assessment Have an Impact on the Utilisation of Health Care Resources? Evidence from Two European Countries, click here to view.
Patients, Clinicians and Researchers Working Together to Improve Cardiovascular Health: A Qualitative Study of Barriers and Priorities for Patient-Oriented Research, click here to view.
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.
11. AHRQ Effective Program Updates
Research Protocol: Prehospital Airway Management: A Systematic Review. Click here to view.
OPEN FOR COMMENT THROUGH MARCH 2: Technical Brief: Strategies for Patient, Family, and Caregiver Engagement. Click here to view.
Research Protocol: Pharmacologic and Nonpharmacologic Treatments of Posttraumatic Stress Disorder, click here to view.
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.