Digital health presents both opportunity and threat to Big Pharma. The opportunities lie in streamlining and accelerating R&D and improving patient experience and product outcomes; the threats come from the swath of new competitors vying for a piece of the health care value pie – many with far more experience in consumer-facing services.

US FDA usually recommends using overall survival for pancreatic cancer drugs, but AstraZeneca choose progression-free survival; now ODAC will weigh in on whether the PARP inhibitor olaparib demonstrated a clinically meaningful impact. The firm cites enrollment and design challenges in the gBRCAm population as reasons for using PFS.

Attorneys say Sarepta did not have an obligation to report its appeal, particularly since winning a formal dispute filing with the US FDA is a long shot.