1. Experts Agree that Foreign Reference Pricing Has the Potential to Limit Access to Needed Treatments for Patients and People with Disabilities, see details below.
2. Rethinking How We Measure a Drug's 'Value,' click here to read the article.
3. Over 50 Leading Patient Groups Join PIPC Letter on ICER’s COVID-19 Treatment Assessments, see details below.
4. SWHR: ICER’s Value Framework is a Work in Progress for Women’s Health, click here to read the blog post.
5. Accounting for our Differences Matters in Value Assessment, click here to read the article.
6. Value Assessment Organizations Should Learn from the Experts on Patient Engagement, click here to read the blog post, the latest entry in PIPC's series The Data Mine.
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, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, High Cholesterol, 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.
Policymakers continue to propose foreign reference pricing as a tactic to lower drug prices within the United States. Though this policy may sound attractive on paper, there are unintended consequences for patients who suffer limited and delayed access to needed treatments. Experts agree this type of policy would be harmful for patients and people with disabilities, and the National Council on Disability made clear in a recent report that importing this type of QALY-based drug pricing would be contrary to United States civil rights law and disability policy and called on the Administration to rescind its International Price Index that would rely on prices set internationally using discriminatory metrics of value. It is imperative that Congress listen to the National Council on Disability and other experts and avoid policies that rely on foreign reference pricing.
2. Rethinking How We Measure a Drug's 'Value'
Writing in STAT News, the Innovation and Value Initiative's (IVI) Patricia Deverka, Louis Garrison, and Samuel Nussbaum say that ICER should rethink how it conducts value assessments, focusing on its study relating to COVID-19. They highlight three primary issues with ICER's analysis of the antiviral Remdesivir: (1) significant uncertainty relating to infection prevalence and the effectiveness of Remdesivir, (2) a lack of transparency in ICER's models, and (3) omitting possible impacts on the health system capacity and health care personnel. Instead, they suggest the following principles for value assessment: (1) Consensus on model inputs, methods, and structures based on public input; (2) Flexibility to accommodate the information needs of stakeholders; (3) Transparency to allow real-time review and updating; and (4) Open-source development of models to enable widespread use and customization. Click here to read the article.
3. Over 50 Leading Patient Groups Join PIPC Letter on ICER’s COVID-19 Treatment Assessments
More than 50 leading groups representing patients and people with disabilities joined the Partnership to Improve Patient Care’s (PIPC) comment letter to the Institute for Clinical and Economic Review (ICER) regarding its assessment of remdesivir and other treatments for the novel coronavirus (COVID-19). The letter offers strong criticism of ICER’s use of flawed methodologies in its assessment, noting that ICER has chosen to completely ignore vast array of stakeholder feedback it has received over the last five years on its framework and processes. The groups reiterate that it’s imperative that discriminatory assessment tools like the quality-adjusted-life-years (QALY) metric are not used in determining the price of treatments, especially during public health emergencies such as COVID-19. “We have consistently raised the red flag that ICER’s value assessments are methodologically flawed and not fit for the purpose of making decisions related to coverage, reimbursement and incentive programs by policymakers and payers,” the letter states. “ The latest assessment from ICER validates our concerns.” Click here to read the letter. Click here to read PIPC's one-pager on the main issues with ICER's report.
4. SWHR: ICER’s Value Framework is a Work in Progress for Women’s Health
The Society for Women's Health Research's (SWHR) Sarah Wells Kocsis writes that treatments may vary widely in terms of value for a given individual, as drugs do not work the same in every person. "ICER takes a population-level approach to value assessment and estimates a health care intervention’s value from two perspectives: long-term 'value for money' and short-term affordability," she writes. "Other frameworks such as the National Comprehensive Cancer Network (NCCN) Evidence Blocks take a patient-level approach by providing patients and clinicians with clinical and economic information about therapies to inform treatment choices. Both approaches to value assessment consider factors such as burden of illness and indirect costs (e.g. productivity), but they incorporate and measure them in different ways. SWHR believes that it is vitally important that all frameworks appropriately capture the value of factors relevant to women, such as caregiver burden and ability to function/work." Click here to read the blog post.
5. Accounting for our Differences Matters in Value Assessment
The Innovation and Value Initiative's (IVI) Jen Bright and former SWHR CEO Dr. Amy Miller write that patients are diverse and value assessment must account for that. "In the ever-accelerating discussion about paying for 'value' we are at a crossroads in the United States," they write. "We all know there is more we can all do to make value assessment methods and models more relevant to real patients. Together, researchers, patients, innovators, regulators, clinicians, payers, and employers should commit to finding and accelerating improved methods and tools. We must work together to both develop and deploy data sets that can help us answer the questions most important to patients. We must continue to think strategically about the most effective way to directly engage patients in research." Click here to read the article.
6. Value Assessment Organizations Should Learn from the Experts on Patient Engagement
Dr. Steven Pearson, founder and president of the Institute for Clinical and Economic Review (ICER), recently wrote that no clear framework exists to engage patients in medical research. In A proposed framework for patient engagement throughout the broader research enterprise, published in the Journal of Comparative Effectiveness Research, Dr. Pearson acknowledges that patient engagement is an important component of medical research, but he fails to recognize years of accomplishments in the field. This misleading premise ignores rigorous patient-centered outcomes research and evolving development of evidence-based frameworks for patient engagement. Click here to read the blog post, the latest entry in PIPC's series The Data Mine.
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.
- Canada: Ottawa urged to approve vital cystic fibrosis treatment.
- New Zealand: With a critical drug not covered, one cancer patient given just two years to live. Farmer with cystic fibrosis fights for access to lifesaving drug. Families affected by forced epilepsy drug switch say that Pharmac's review of the incident is insufficient. Click here to read more. Pharmac switches depression drugs too frequently.
8. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, High Cholesterol
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. All of ICER’s current assessments have been put on hold for 2-3 months in response to COVID-19. We will continue to update below deadlines as we receive more information.
- Ulcerative Colitis: Draft Evidence Report and Draft Voting Questions AVAILABLE. Comment period open through 7/29/2020. Meeting 9/24/2020: CTAF will convene to deliberate and vote on evidence presented in ICER's report on ulcerative colitis therapies.
- Cystic Fibrosis: Evidence Report and Response to Comments AVAILABLE. Meeting POSTPONED: CTAF will convene to deliberate and vote on evidence presented in ICER's report on treatments for cystic fibrosis.
- Sickle Cell Disease: Evidence Report and Responses to Comments AVAILABLE. Meeting POSTPONED: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for sickle cell disease.
- Non-Alcoholic Steatohepatitis: Draft Evidence Report and Voting Questions AVAILABLE, Comment Period OPEN through THIS WEDNESDAY, 6/24/2020. Meeting 8/13/2020: The Midwest CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for NASH.
- Hemophilia A: Research Protocol AVAILABLE. 7/1/2020: Model Analysis Plan.
- Bladder Cancer: Revised Scoping Document AVAILABLE. 7/14/2020: Research Protocol.
- Opioids: Digital Apps: Revised Scoping Document AVAILABLE. 7/14/2020: Research Protocol.
- Opioids: Supervised Injection Centers: Revised Scoping Document AVAILABLE. 7/21/2020: Research Protocol.
- High Cholesterol: Open input period through 6/30/2020.
9. Upcoming Events and Webinars
PCORI Advisory Panel on Rare Disease Spring 2020 Meeting
June 22, 2020
Click here for details.
PCORI Board of Governors Meeting
June 23, 2020
Click here for details.
The Developing HTA Landscape in APAC – Insights and Lessons Learned
June 23, 2020
Click here for details.
PCORI Engagement Awardee Lunch and Learn: Virtual Engagement
June 24, 2020
Click here for details.
2020 PCORI Annual Meeting (A Virtual Event): Accelerating Impact on Care and Patient Outcomes
September 16-17, 2020
Click here for details.
10. Medical Journal Articles
The Impact of Drug Quality Ratings from Health Technology Assessments on the Adoption of New Drugs by Physicians in Germany, click here to view.
Developing a Patient- and Family-Centered Research Agenda for Hospital Medicine: The Improving Hospital Outcomes through Patient Engagement (i-HOPE) Study, click here to view.
Annals Clinical Decision Making: Communicating Risk and Engaging Patients in Shared Decision Making, click here to view.
Comparative Effectiveness Research in Critically Ill Patients: Risks Associated with Mischaracterising Usual Care, click here to view.
Unanticipated Therapeutic Value of the Patient-Centered Outcomes Research Institute (PCORI) Stakeholder Engagement Project for Homebound Older Adults, click here to view.
Impact of a Global Pandemic on Health Technology Assessment, click here to view.
How Are Incremental Cost-Effectiveness, Contextual Considerations, and Other Benefits Viewed in Health Technology Assessment Recommendations in the United States?, click here to view.
The Implementation of Health Technology Assessment Principles in Public Decisions Concerning Orphan Drugs, click here to view.
Decision-Making under Uncertainty: Comparing Regulatory and Health Technology Assessment Reviews of Medicines in the US and Europe, click here to view.
What Types of Real-World Evidence Studies Do U.S. Commercial Health Plans Cite in Their Specialty Drug Coverage Decisions?, click here to view.
Defining the Role of the Public in Health Technology Assessment (HTA) and HTA-Informed Decision-Making Processes, click here to view.
11. AHRQ Effective Program Updates
OPEN FOR COMMENT THROUGH JULY 13: Maternal and Fetal Effects of Mental Health Treatments in Pregnant and Breastfeeding Women: A Systematic Review of Pharmacological Interventions. Click here to view.
Research Protocol: Breast Reconstruction after Mastectomy. Click here to view.
Rapid Evidence Report: Masks for Prevention of COVID-19 in Community and Healthcare Settings: A Living Rapid Review. Click here to view.
OPEN FOR COMMENT THROUGH JULY 6: Systematic Review: Pharmacologic and Nonpharmacologic Treatments for Posttraumatic Stress Disorder: An Update of the PTSD Repository. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 29: Systematic Review: Therapies for Clinically Localized Prostate Cancer. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 29: Systematic Review: Management of Colonic Diverticulitis. Click here to view.
OPEN FOR COMMENT THROUGH JUNE 30: Systematic Review: Management of Primary Headaches in Pregnancy. Click here to view.
Systematic Review: Interventions for Substance Use Disorders in Adolescents. Click here to view.
White Paper: The Evidence Base for Telehealth: Reassurance in the Face of Rapid Expansion During the COVID-19 Pandemic. Click here to view.
White Paper: Standardized Library of Depression Outcome Measures. Click here to view.
Research Protocol: No-Touch Modalities for Disinfection of Hospital/Acute Care Settings: A Rapid Evidence Review. Click here to view.
Research Protocol: Masks for Prevention of COVID-19 in Healthcare and Community Settings. Click here to view.
Systematic Review: Labor Dystocia. Click here to view.