CER Daily Newsfeed

Video: Alliance Discussion with Dr. Nakela L. Cook, Executive Director, PCORI

(11/12, ResearchAmerica, YouTube) “We were privileged to host Dr. Nakela Cook, Executive Director of the Patient-Centered Outcomes Research Institute (PCORI) for a virtual fireside chat on Tuesday, Nov. 12, 2025...Dr. Cook shared insights on PCORI’s role in advancing patient-centered research and highlighted both current and upcoming initiatives.” View Video

GetReal Conference 2025: Integrating AI and Real-World Evidence Across the Product Lifecycle

(11/13, Katie McCool, The Evidence Base) reports “...Jan-Willem Versteeg (Utrecht University) explored how AI can be applied not only to clinical data but also to the assessment process itself. ‘I work with AI to abstract data from these reports,’ he explained, describing efforts to compare health technology assessment (HTA) guidance and methods across agencies. Looking forward, he suggested that transforming recommendations into computable rules could enable continuous feedback between assessment and practice: ‘You then can link those to existing clinical databases or clinical databases that will be created with AI, maybe in the future, and then that can be helped to flag and prioritize the work going forward.’” Full

Improving Formulary Access: A Path to Better Care

(11/13, Bioengineer.org) reports “...In addressing the complex issue of affordability, the authors present the idea of value-based insurance design. This approach aligns patient costs with the value of the medications to their health outcomes, thus incentivizing adherence and promoting more judicious use of healthcare resources. Such models have the potential to shift the focus from merely containing costs to actively investing in medications that deliver tangible benefits for patients.” Full

Vinay Prasad, Martin Makary Unveil New FDA Path for Accelerating Custom Gene Editing Therapies

(11/12, Angus Liu, Fierce Biotech) reports “...The new program, called the ‘plausible mechanism pathway,’ is designed to offer a new way to market for personalized therapies, Prasad and Makary said...While the FDA will prioritize rare diseases-especially those that are fatal or associated with severe childhood disabilities-the pathway will also be available for common diseases, particularly those for which there's no alternative treatments or where considerable unmet need exists, the two officials wrote. One of the guiding principles, according to the FDA leaders, is that only diseases for which the biological cause is known will be eligible for the pathway.” Full

FDA Unveils New Path for Approving Drugs for Just One Patient

(11/12, Gerry Smith, Bloomberg) reports “...Historically, every treatment for every disease has needed its own separate study. The advent of Crispr, the Nobel Prize-winning gene-editing technology, has made it theoretically possible for scientists to develop cures for many of the 7,000 rare diseases, a small fraction of which have treatments. But the FDA's previous demands made it seem too expensive and time-consuming for companies to pursue. Makary and Prasad said the FDA's current rules ‘are onerous and unnecessarily demanding, provide unclear patient protection and stifle innovation.’” Subscription Required

FDA’s Plan to Speed Drug Reviews Risks Skepticism From Insurers

(11/13, Nyah Phengsitthy, Bloomberg Law) reports “...‘Are payers going to say, “hey, you got your product through this pathway, that’s awesome, and we’ll pay for it,”’ said Michael Werner, co-lead of Holland & Knight’s health-care and life sciences team. ‘Or are they going to say, “that’s great, but we’re not paying for it until there’s more data?”’ Other studies have indicated that for drugs granted conditional approval, payers are concerned about the potential for clearance to be revoked.” Subscription Required

UK: NICE Backs Roche's Non-Hodgkin Lymphoma Drug Columvi

(11/13, Phil Taylor, PharmaPhorum) reports “...Reimbursement authority NICE published draft guidance today giving a green light to the use of Columvi (glofitamab) in combination with gemcitabine and oxaliplatin chemotherapy as a second-line option for adults with diffuse large B-cell lymphoma (DLBCL) who are ineligible for a stem cell transplant.” Full