FDA on new criteria for compassionate use and single-patient INDs in oncolog

Thanks to Internet chat rooms, patient advocacy web sites and theseemingly endless top-of-the-news stories about breakthrough new cancer therapies, notably Novartis' recently-approved Gleevec (imatinimab mesylate; Marketletter May 21), cancer patients have become increasingly aware of the promise of investigational therapies. And, they have become more demanding about getting a chance to try one of those drugs, no matter how sick they are or how early the product is in the development process.

That has led to a dilemma for pharmaceutical companies and the US Food and Drug Administration in deciding whether patients should get access to experimental drugs under the compassionate use or single-patient Investigational New Drug program. These programs have existed in some form since the 1960s, but the rules about when to grant an individual patient access to a therapy outside of a trial have been fuzzy and subjective, ultimately decided not by the FDA, but by the pharmaceutical company.

AIDS advocates led the way