1. PIPC and the EveryLife Foundation for Rare Diseases Partner to Release a New White Paper Evaluating the Use of Value Assessments for Cell and Gene Therapies, click here to read the paper.
2. Most Favored Nations Executive Order Released Last Week Would Import Discriminatory QALYs, see details below.
3. PCORI Proposed Principles for the Consideration of the Full Range of Outcomes Data, click here to read the principles and to submit comments.
4. ADA 25 Advancing Leadership Forum on Disability Justice in the Fight for Racial Equity, click here to learn more and register.
5. The Bonnell Foundation Discusses Access Challenges for Cystic Fibrosis Patients Abroad, click here to listen to the podcast.
6. NMQF, Axis Advocacy, Sick Cells, and PIPC Release a White Paper on Value Assessments' Impact on Health Inequities, click here to read the paper.
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, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma, 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.
PIPC and the EveryLife Foundation for Rare Diseases partnered with Health Advnaces to write a new white paper, Value for Whom? Incorporating Patient Perspectives into Value Assessment for Novel Cell and Gene Therapies. The paper explores the topic of the use of traditional cost-effectiveness analyses to determine the “value” of innovative cell and gene therapies determining that there are currently many shortcomings in this process that may have the impact of limiting patient access to needed treatments. As the science of patient input has transformed the clinical trial and regulatory landscape, traditional value assessments have not taken the same steps to incorporate patient data. The paper digs into these issues and explores some new and innovative models that are moving in a better direction and trying to truly determine value to patients. Click here to read the paper.
2. Most Favored Nations Executive Order Released Last Week Would Import Discriminatory QALYs
The Trump administration released the text of an Executive Order allowing the Department of Health and Human Services (HHS) to rely on foreign governments to set prices for drugs covered under Medicare, regardless of the metrics used by those countries or implications for access to care for patients. The Partnership to Improve Patient Care (PIPC) released a statement expressing strong opposition to this concept, as it will have the impact of limiting and delaying access to needed treatments for Americans.
PIPC Chairman Tony Coelho along with leading patient and disability groups have long advocated against this type of discriminatory policy. Chairman Coelho was quoted last week in MedPage Today, saying, "this executive order imports prices based on discriminatory metrics like the quality-adjusted life year, which devalue the lives of seniors, people with disabilities and serious chronic conditions. Other countries use these metrics to ration healthcare. It is dangerous to import foreign pricing policies, and the associated access barriers that come with them." Click here to read the article.
Click here to read a February blog post from Chairman Coelho on the topic. The National Council on Disability (NCD) released a statement upon first news of this Executive Order in July calling out the harms of referencing international pricing -- which relies on the use of discriminatory measures like QALYs. Click here to read the statement.
3. PCORI Proposed Principles for the Consideration of the Full Range of Outcomes Data
At its September 2020 meeting, PCORI’s Board of Governors approved the release of the proposed Principles for the Consideration of the Full Range of Outcomes Data for public comment. This is one component in PCORI’s plan to implement its new statutory mandate to consider the full range of outcomes data in PCORI-funded research.
These principles will serve as a point of reference for PCORI, and as a basis for developing future guidance to potential applicants for PCORI funding on what is included in “the full range of clinical and patient-center outcomes relevant to, and that meet the needs of, patients, clinicians, purchasers, and policy-makers” consistent with its authorizing law. Comment will be open through November 13, 2020. Click here to read the principles and to submit comments.
4. ADA 25 Advancing Leadership Forum on Disability Justice in the Fight for Racial Equity
Join ADA 25 Advancing Leadership for a transformative multisession, multiday, forum centering in-depth, action-oriented conversations on the intersections of racial equity and disability justice. The forum will take place over four days, September 29, October 14, October 22, and October 27. Those interested can sign up for one or multiple sessions. This four-part series cements the connection between racial equity and disability justice. Local and nationally recognized social justice activists will share their stories, strategies, and hopes for the future. Click here to learn more and register.
5. The Bonnell Foundation Discusses Access Challenges for Cystic Fibrosis Patients Abroad
Laura Bonnell, executive director of the Bonnell Foundation and mother of two daughters with Cystic Fibrosis, hosted a podcast interviewing Beth Vanstone of Get Loud for CF, a Canadian mother of a daughter with Cystic Fibrosis. They discussed Beth’s long and arduous process to get access to novel CF therapies that are not covered in Canada for her daughter, Maddy. Laura expressed concern as to what modeling the pricing policies of Canada and other countries that rely on the QALY could mean for American patients in the context of the Most Favored Nations Executive Order. They also hit on a wide range of topics impacting CF families around the world. Click here to listen to the podcast.
6. NMQF, Axis Advocacy, Sick Cells, and PIPC Release a White Paper on Value Assessments' Impact on Health Inequities
National Minority Quality Forum, Axis Advocacy, Sick Cells, and Partnership to Improve Patient Care released a white paper investigating the limitations of applying traditional value assessments to communities of color. The paper finds that the pervasive issues that concern the patient and disability communities about value assessments, like over reliance on averages and the use of the Quality-Adjusted Life Year, become even more concerning when assessing treatments for conditions that disproportionately impact communities of color. If relied on in coverage decisions, these assessments could have the effect of exacerbating health inequities. Click here to read the paper.
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: Canadian teen fights for reform to drug access processes. Woman with cystic fibrosis talks of "heartbreaking" fight to get government to provide access to life-changing medication.
- United Kingdom: UK cystic fibrosis sufferer only alive because drug manufacturer gave her life-saving medicine on compassionate grounds, as government continues to refuse to fund it.
8. ICER's QALY-Based Study Topics: Hemophilia A, Sickle Cell Disease, Ulcerative Colitis, Cystic Fibrosis, Bladder Cancer, Opioid Treatments, High Cholesterol, Anemia in Chronic Kidney Disease, Lupus Nephritis, Multiple Myeloma
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.
- Ulcerative Colitis: Revised Voting Questions and Evidence Report AVAILABLE. Meeting 9/24/2020: CTAF will convene to deliberate and vote on evidence presented in ICER's report on ulcerative colitis therapies.
- Cystic Fibrosis: CTAF convened 8/27/2020 to deliberate and vote on evidence presented in ICER's report on treatments for cystic fibrosis. 9/23/2020: Final Evidence Report.
- 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.
- Hemophilia A: Draft Evidence Report AVAILABLE. Comment period OPEN through THIS WEDNESDAY, 9/23/2020. Meeting 10/30/2020: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for hemophilia.
- Bladder Cancer: Draft Evidence Report AVAILABLE. Comment period OPEN through 10/15/2020. Meeting 11/20/2020: Midwest CEPAC will convene virtually to deliberate and vote on evidence presented in ICER's report on digital therapeutics for bladder cancer.
- Opioids: Digital Apps: Draft Evidence Report AVAILABLE. Meeting 11/18/2020: Midwest CEPAC will convene virtually to deliberate and vote on evidence presented in ICER's report on digital therapeutics for opioid use disorder (OUD).
- Opioids: Supervised Injection Centers: Model Analysis Plan available. 9/24/2020: Draft Evidence Report.
- High Cholesterol: Research Protocol available. 9/24/2020: Model Analysis Plan.
- Anemia in Chronic Kidney Disease: Research Protocol available. 10/15/2020: Model Analysis Plan.
- Unsupported Price Increase Assessment: 1/8/2021: Final Assessment and Report.
- Lupus Nephritis: Draft Scoping Document AVAILABLE, Comment Period OPEN through TODAY, 9/21/2020, Meeting 3/26/2021: New England CEPAC will convene to deliberate and vote on evidence presented in ICER's report on treatments for lupus nephritis.
- Multiple Myeloma: 9/22/2020: Draft Scoping Document.
- Service Dogs for PTSD: Open Input Period through 10/2/2020.
9. Upcoming Events and Webinars
SWHR Virtual Panel on Uterine Fibroids
September 22, 2020
Click here for details.
National Health Council 2020 Virtual Science of Patient Engagement Symposium
September 29-30, 2020
Click here for details.
PCORI Webinar Series: Considering the Full Range of Outcomes in PCORI Research -- Patients, Caregivers and Consumers
October 5, 2020
Click here for details.
PCORI: New Evidence to Inform Decisions
October 16, 2020
Click here for details.
10. Medical Journal Articles
Replication of Randomized Clinical Trial Results Using Real-World Data: Paving the Way for Effectiveness Decisions, click here to view.
Improving Transparency to Build Trust in Real-World Secondary Data Studies for Hypothesis Testing—Why, What, and How: Recommendations and a Road Map from the Real-World Evidence Transparency Initiative, click here to view.
Measuring and Improving Quality in the US: Where Are We Today?, click here to view.
Potential Impact of Missing Outcome Data on Treatment Effects in Systematic Reviews: Imputation Study, click here to view.
Advancing Community-Engaged Research: Increasing Trustworthiness Within Community-Academic Partnerships, click here to view.
Patient and Other Stakeholder Engagement in Patient-Centered Outcomes Research Institute Funded Studies of Patients with Kidney Diseases, click here to view.
Patient-Reported Outcomes: Central to the Management of COVID-19, click here to view.
How to Include Patient-Reported Outcome Measures in Clinical Trials, click here to view.
Evaluation of the Use of Cancer Registry Data for Comparative Effectiveness Research, click here to view.
Outcome-Based Payment Schemes: What Outcomes Do Patients with Cancer Value?, click here to view.
11. AHRQ Effective Program Updates
Systematic Review: Therapies for Clinically Localized Prostate Cancer. Click here to view.
Research Report: Developing Consistent and Useful Quality Improvement Study Data Extraction for Health Systems. Click here to view.
OPEN FOR COMMENT THROUGH 9/28/2020: Systematic Review: Acute Treatments for Episodic Migraine. Click here to view.
OPEN FOR COMMENT THROUGH 9/28/2020: Systematic Review: Treatments for Acute Pain. Click here to view.
Technical Brief: Strategies for Patient, Family, and Caregiver Engagement. Click here to view.
Research Protocol: Interventions To Decrease Hospital Length of Stay. Click here to view.
Rapid Evidence Product: Retention Strategies for Medications for Addiction Treatment in Adults With Opioid Use Disorder. Click here to view.
OPEN FOR COMMENT THROUGH 9/28/2020: Systematic Review: Cervical Ripening in the Outpatient Setting. Click here to view.