Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.

From synthetic data to real-world data, from patient engagement to patient stratification, from combination trials to master protocols and from fully decentralized through hybrid to onsite trials, the world of clinical development is undergoing major changes. Fifty executives share their predictions around clinical trial trends for the year ahead.

FDA review of the company's BLA for a hemophilia gene therapy could be delayed by three months to allow the agency to review a three-year data analysis from an ongoing trial.

During Q3, biopharmas brought in an aggregate $18.1bn in financing and device company fundraising totaled $5.1bn; while in vitro diagnostic firms and research tools players raised $771m.