CER Daily Newsfeed

Contributor: American Patients vs Foreign Governments—A Tale of 2 Value Sets

(2/12, Julie A. Patterson, PharmD, PhD, Tyler D. Wagner, PharmD, PhD, Robert J. Nordyke, PhD, 

Jonathan D. Campbell, PhD, MS, The American Journal of Managed Care) comments “...Value in the US should reflect diverse patient needs, societal benefits, and local treatment contexts, recognizing that both quantitative and qualitative evidence are contributing factors. Cost-effectiveness models are one component of value assessment and should therefore be viewed as a tool, not a rule, to support negotiations between payers and manufacturers in ensuring fair, patient-centered outcomes that enhance health care decision-making and system sustainability. By meaningfully engaging stakeholders, especially patients, we can ensure that assessments capture what truly matters to patients, foster trust, and promote sustainable and equitable access to health care.” Full

Real-World Evidence Supports Switch to Next-Generation BTK Inhibitor for CLL: A Q&A With Margaret Krackeler, MD

(2/12, Rose McNulty, The American Journal of Managed Care) reports “...In a study of 281 patients published in Cancer Medicine, researchers found that 79% remained on zanubrutinib at last follow-up, with notably fewer treatment-limiting and cardiac adverse events (AEs) compared with ibrutinib—outcomes that validated the systemwide formulary switch and offer insights for other health systems navigating similar transitions.” Full

FDA Outlines Evolving Framework for Real-World Evidence and Highlights RCT DUPLICATE Demonstration Project

(2/12, Katie McCool, The Evidence Base) reports “...[CDER’s Dr. Marie Bradley] described the FDA’s RWE program as multifaceted, encompassing internal review pathways, external stakeholder engagement, listening sessions, and demonstration research. Submissions containing RWD are reviewed within the relevant clinical divisions, with consultation from epidemiology and statistics experts as needed. She encouraged sponsors to engage early with the agency, advising that discussions should take place ‘as early as possible, before any sort of solid decisions are made.’ Early dialogue, she suggested, can help identify design or data challenges before substantial resources are committed.” Full