Short stories about how US FDA’s conference center changed since COVID-19 forced the end of in-person public events, what the agency did to officially close the books on Makena, and more.

Given the challenges of validating patient-reported outcome instruments specific to very rare diseases, agency is probably going to have to accept the use of unvalidated measures in some cases, Peter Marks tells American Society of Gene and Cell Therapy’s annual meeting.

Drug makers are more enthusiastic than ever about the outlook for developing drugs for Alzheimer's. Scrip reports back on the latest developments at CTAD.

Current accelerated approval provisions are the ‘sweet spot’ for balancing access and efficacy, but more attention needs to be paid by sponsors and the agency to working out regulatory pain points earlier in the development process, CBER director tells gene/cell therapy conference.