Accelerated Approval: FDA Meeting Could Be A Sign Of Things To Come

Pharmaceutical companies and biotechs, beware. FDA will be holding a meeting of its Oncologic Drugs Advisory Committee on Nov. 8 to discuss post-approval study commitments by sponsors for drugs granted accelerated approval.

For those of you who don't already know, the agency can grant AA to certain cutting edge drugs that point to a clinical benefit based on accepted surrogate endpoints. In exchange for conditional approval, drug companies must conduct a Phase IV postmarket study to confirm the earlier results.

This meeting is a must for drug developers and as close as you'll get to must-see TV at FDA.

Right now, there are essentially two camps at FDA. One group feels you should get drugs out to patients for serious and life-threatening illnesses as quickly as possible, despite sometimes questionable efficacy, and let the docs decide. The second camp believes drugs must show "reasonable" efficacy before you can put novel treatments out on the market.

In other words, one believes physicians should be the gatekeepers, while the other group believes FDA is the one and only gatekeeper. All of this is occuring under the perception that FDA is getting more conservative in general due to Vioxx, COX-2s, antipsychotics etc. There are flaws with both philosophies, but we won't go there today. The bottom line is accelerated approval is being increasingly used - with decreasing reliability - by the pharmaceutical and biotech industries to keep development costs down and get products to the market faster.

The single biggest problem right now with AA is that drug companies have not been doing the promised post-approval study. ODAC will discuss the best ways for sponsors to keep their word and follow through with study follow-ups. Postmarket trials for Johnson & Johnson's Doxil, MedImmune's Ethyol, SkyePharma's DepoCyt and Pfizer's Celebrex, among others, will be discussed by the panel.

More importantly, though, this meeting could be a marker for where FDA is headed with accelerated approval. Will there be a crackdown on Phase IV? How does ODAC feel about AA? Which individual panel members appear to be the most disheartened by the lack of postmarket follow-up commitments?

The answers to these questions will be critical for current and future accelerated approvals. With ODAC recommending approval for GlaxoSmithKline's leukemia drug Arranon and Celgene's MDS therapy Revlimid, it looked like a big win for AA. However, there has already been a serious setback for one of those decisions (more on that in my next post).

The November meeting is not the end-all, be-all. But it will serve as an important reference point for accelerated approvals. So mark your calendar, because this is one meeting you won't want to miss.

Speaking of meetings, for more insight into AA, FDA's priorities and where the Medicare drug benefit is headed in 2006, you should make sure and attend the Windhover FDA/CMS Summit Nov. 29-Dec. 1 in Washington, D.C. The speaker lineup is exceptional and the topics for discussion are sure to provide solutions and opportunities for a wide range of health care stakeholders. For more on the meeting, go to http://www.windhover.com/fda-cms.