AstraZeneca ran that confirmatory study, and it failed. Now, the FDA has to decide whether to pull Iressa off the market. Regardless of what happens, Iressa is finished in the U.S. AstraZeneca has already said it will stop marketing the drug here and, in fact, will be telling doctors about Tarceva, a similar lung cancer drug from OSI Pharmaceuticals (OSIP) and Genentech that does help patients live longer.
The Iressa mess could have been avoided back in 2003 if AstraZeneca had conducted a randomized, controlled study of the drug, such as comparing Iressa with a placebo or best supportive care to see if the drug improved survival. If that study had been conducted and the results had turned out as those released Friday, the FDA probably wouldn't have approved Iressa, and therefore, wouldn't be facing a difficult decision today. AstraZeneca could have put together a controlled study of Iressa; after all, that's exactly what OSI and Genentech did.
My point here is not to be a Monday morning quarterback. Instead, looking ahead, I think that some top FDA officials will use the Iressa situation to argue even more strongly against the use of single-arm, uncontrolled studies as the basis for future drug approvals. While everyone wants to see the FDA work quickly to approve life-saving drugs, we should also be concerned when the agency approves drugs that turn out to be nothing more than expensive placebos, and often with dangerous side-effects, to boot.