A recent article from BusinessWeek, "The FDA's Cancer Chief Speaks Out", gives a fascinating insight into the FDA approval process. It takes the form of a Q&A with Richard Pazdur, who runs the FDA's cancer division. In it he touches on one of the all time greatest challenges facing biotech
companies with a drug under review (it relates to cancer but could be applicable to most disease areas) – when to cut a drug from further development:
The FDA is often criticized for being too conservative when it comes to cancer drug reviews, particularly by biotech companies. But these companies also lack regulatory experience. How would you apportion the blame, if any?
One of the things we have seen is a reluctance, sometimes, of smaller companies to make really critical decisions regarding their drugs, whether to curtail the development of a drug. Large companies, because they have a portfolio of drugs, generally if a drug fails to meet specified goals they'll look to abandon that drug—i.e., cut their losses. Whereas as a smaller company, if you only have one drug, then sometimes that is not an option.
There is considerable discussion in the drug industry about looking at how a drug performs in subgroups of trial participants, even if it has failed to meet its goal in the larger trial. What is your view of this approach?
One of the problems we've had is people coming to us after a drug fails because they've invested millions and millions of dollars into a drug; and then it's, "How can we salvage this?" In other words, failing your primary endpoint and then trying to salvage a trial by looking at subgroups of patients. That's akin to shooting an arrow and having it land on a wall and then drawing a target around it. It's an attempt to resurrect a trial that has failed.
Clearly, a small biotech (with investors to satisfy) may have a lead program supporting the lions share of its valuation. And if such a program fails, what should a company do? It's a really difficult call, but to play devil's advocate, is throwing more investment dollars at it going to solve something that inherently doesn't work? Maybe that's the time to cut losses and move on; this is the high stakes game of biotech.