Industry and patient groups take issue with draft guidance’s skeptical tone on utility and reliability of externally controlled studies, with some saying the guidance ignores special considerations inherent in studying rare diseases. Recommendations on access to patient-level data for external control arms also are problematic and likely infeasible, stakeholders say.

Private Company Edition: Alexis Borisy and his Curie.Bio co-founders will invest $5m-$7m to help companies get to lead optimization within 12-18 months. Also, Feng Zhang-founded Aera launched with $193m, Hexagon Bio raised a $77.3m series B round and Halda emerged with $76m.

In an exclusive interview with Scrip, Chugai's CEO says the now 20-year-old strategic partnership with Roche has been one of the keys to Chugai’s successful growth and has allowed smooth market expansion for both firms, as well as enabling R&D investment by the Japanese firm. A flexible, platform-based approach to R&D, rather than focusing on specific target diseases or technologies, has also benefitted Chugai, Osamu Okuda noted.

Can the decentralized global healthcare ecosystem to turn real-world evidence from a buzzword to a useful approach to fit-for-purpose evidence generation?