News from Monday, March 20, 2023
Articles
Biden's Victory Lap on Drug Prices Is Premature
(3/18, John Michael O'Brien, Sarasota Herald-Tribune) comments “...Today, pharmaceutical manufacturers bring a new drug to market when the evidence shows it will safely improve health, and they can do that even if the drug treats just a small number of people with one type of disease. But even after the drug is approved, companies continue clinical trials to determine if that drug can help even more people with related illnesses. These studies continue until the patent and other exclusive rights granted by the FDA expire, and that's when generic drugs can enter the market. These new clinical trials often begin three or more years after the drug is first approved and lead to new approved uses eight or more years after launch. However, under the new law, the number of years during which a manufacturer can fully recoup the costs of discovering a new drug will be limited. Additionally, the new law could lead to delays in novel treatments coming to market, especially for people with rarer conditions.” Full
Removing Roadblocks to Kidney Transplant through Health System Change
(3/20, The PCORI Blog) comments “...‘This project is significant both for testing an innovative care delivery model for increasing equitable care for kidney transplants and demonstrating unique ways of leveraging electronic health records to reach patients, provide more equitable care and conduct health equity-related comparative clinical effectiveness research across multiple health systems,’ said Hillary Bracken, a senior program officer in PCORI’S Healthcare Delivery and Disparities Research Department. Importantly, patient and stakeholder engagement has informed the development and design of the proposed STEPS intervention. “ Full
NICE Recommends OXLUMO®? (lumasiran) in England as First Therapy to Treat Underlying Cause of Ultra-Rare Condition Primary Hyperoxaluria Type 1 (PH1)
(3/20, Pharmafile) reports “Alnylam UK Limited, the leading RNA interference (RNAi) therapeutics company, today welcomed a final decision from the National Institute for Health and Care Excellence (NICE) recommending the use of OXLUMO® (lumasiran) on the NHS in England as an option for treating primary hyperoxaluria type 1 (PH1) in people of all ages. Lumasiran is the first treatment made available in England that has the potential to lower harmful oxalate levels, in the majority of patients, that drive the progression of PH1.” Full
Press Releases
AAMC Responds to PCORI’s Enhancing Workforce Development RFI
(3/17, Association of American Medical Colleges Press Release) “...In its comments, the association praised PCORI for establishing a workforce development unit that will identify strategies for equipping current and future PCOR leaders with relevant tools and skills and expressed support of the institute’s work to achieve goals identified through its strategic plan [refer to Washington Highlights, Aug. 27, 2021, Feb. 4, 2022]. ‘Strengthening both the workforce that specializes in this research as well as the systems that support it will produce positive outcomes for patients, caregivers, clinicians, researchers, and health systems,’ the letter stated.” Full
ICER Names Linda Goler Blount, MPH, to Governance Board
(3/20, ICER Press Release) “...‘We are very proud to announce that Linda is joining the ICER Governance Board,’ said Steven Pearson, MD, MSc, President of ICER. ‘Linda has had an extraordinary career at the interface of evidence-based medicine, health policy, and efforts to address health inequity throughout the US healthcare system. She shares the sentiment that ICER’s role as an independent, objective source of evidence is critical to help support more transparent health care policy decisions. Her expertise will strengthen ICER’s ability to help the US health system provide better access to high-value care that all Americans can afford.’” Full
Journals
Causal Inference in Oncology Comparative Effectiveness Research Using Observational Data: Are Instrumental Variables Underutilized?
Marcelo Coca Perraillon, PhD and Ya-Chen Tina Shih, PhD
March 17, 2023, Journal of Clinical Oncology
Reports
Insights Brief: Leveraging Real-World Data to Estimate Race and Ethnicity Representation in Clinical Trial Design
March 2023
Events
Webinar: Applications of Real World Evidence and Insights in Support of Decision-making Throughout the Product Lifecycle to Increase Efficiencies and Reduce Risk
March 21, 2023
10:00AM - 11:00AM