Clinical trial innovation barriers

Have you ever wondered why some innovative approaches only get used once or twice? It’s something I’ve often thought about with clinical trials. Take platform trials, which test multiple treatments within a single trial, or adaptive trials, where new treatments can be added as they become available, or protocols can change in response to incoming data (in a predetermined way).

Designs like these are often piloted or used by academic researchers, and sometimes they even show up in FDA guidance, but they rarely get adopted widely by pharmaceutical companies. A new post by Adam Kroetsch explains why.

The recurring theme is uncertainty on what regulators will accept, and since trials are so expensive and slow, with a lot riding on whether a drug application is accepted or rejected, the stakes of failure are enormous. The first company that tries an innovative design carries huge risks. But even a single pilot trial usually isn’t enough to convince companies that a given protocol will be accepted more widely.

He lays out how to get these designs to scale: the FDA should formally recognize modern trial practices so sponsors know in advance what protocols will be accepted; the government should set interoperability standards so trial software can be harmonized; and the NIH should fund more large, phase 3-scale trials and build reusable infrastructure, de-risking innovative designs by making them ready for regulators, repeatable, and adoptable.