Replies to post #115093 on Biotech Values

[DewDiligence]

Re: Proposed changes in requirements for Accelerated Approval

The committee debated whether a randomized, two-arm trial rather than a single-arm trial should be required in certain accelerated approval cases. The committee also discussed whether a minimum of two post-marketing trials is necessary and whether accelerated approval should be delayed until the confirmatory trials are actually underway. Ideally, the FDA Office of Oncology would like drug makers to have confirmatory trials underway, with set completion dates, at the time of accelerated approval.

If the rule in bolded text were formally adopted, it would significantly slow down accelerated-approval timelines. However, this seems like a good way of closing a huge loophole in the current way of doing things.

[AlpineBV_Miller]

Pazdur already has gone beyond Congressional rules and requires a confirmatory trial be "substantially under way" before he will allow OOD/ODAC to consider accelerated approval.

For randomized trials, this usually now takes place in a very large single trial with a surrogate interim and OS as the final endpoint. Interestingly, this has resulted in two drug approvals where the 2-in-1 trials never generated prospective survival data (Nexavar and Sutent) because the trials were halted and drug was offered to placebo patients before the survival readout could be made.

For single-arm trials, the requirement is the confirmatory needs to be "substantially" under way. If a company decide to take him to court on this one, he'd lose. But I doubt any of them will.

This means in some way's ODAC was just blustering. More of his grandstanding.

[jq1234]

FDA Officials Talk Tough About Accelerated Approval

To prompt drugmakers to follow through more quickly on post-marketing studies required as part of the controversial acceleted approval program, several FDA officials suggest the agency make use of an existing provision of a 2007 law - issuing fines of up to $10 million. And the FDA may also require post-marketing studies get under way before expedited approval is granted, according to agency officials who floated the possibilities in a new article in the Journal of the National Cancer Institute.

http://www.pharmalot.com/2011/03/fda-officials-talk-tough-about-accelerated-approval/