FDA Says Drug Firms Didn't Finish Follow-Ups

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FDA Says Drug Firms Didn't Finish Follow-Ups

http://online.wsj.com/article_email/SB10001424052748703313304576132560915752

074-lMyQjAxMTAxMDAwOTEwNDkyWj.html

Note: The WSJ site might require a subscription ... this article (atypically

for WSJ content I think) seems a fairly balanced account of the issues.

==

At issue is how to make sure the sponsors of drugs that win accelerated

approvals carry out the confirmatory studies ... which I think serves the

patient interest by identifying drugs that might harm instead of help us.

So far, the track record is good: 27 of 49 post-market studies confirming

benefit for drugs that won accelerated approval, but 7 completed studies

haven't confirmed a benefit, and a good number of studies haven't been done.

For Bexxar, I think the issue here is we have arguably a " home run " drug for

a very common lymphoma -- that is not a commercial success (oncologist

don't prescribe it for many reasons too complex to go into here) ... so the

sponsor citing the challenge of enrolling patients seems disingenuous -

even if this challenge might often apply for other drugs, particularly for

the less common indications.

.. So in this instance, there seems to be a very low profit incentive to do

the confirmatory study for a drug that's given once (and uncommonly

prescribed) - which is tragic, in my view, when you consider that it's the

only drug that appears to have genuine curative potential for Follicular

lymphoma - with a substantial fraction of patients in remissions 10+ years

and counting, recently reported at ASH in two studies.

To the questions posed to the Advisory committee*: my hope and expectation

is that FDA won't operate by strict formula - assuming that in some cases

single-arm studies will be the best we can do for the rarer cancers, and

also for the rarer clinical circumstances for common cancers -- such as

relapsed aggressive lymphoma who have failed transplant or are transplant

ineligible. That is, how we can responsibly get to convincing / reasonable

evidence that a drug provides benefit can vary a lot by the indication.

Also, for some agents with unique properties (such as a favorable toxicity

profile), a single arm study might be the only ethical way to test if the

new agent provides clinical benefit.

That said, I think requiring the sponsor to make a good faith effort to

confirm the results of studies submitted to win accelerated approval serves

the patient interest; and I don't expect that this policy would slow down

the evaluation of new drugs for such approvals.

.. The main rate limiting factor to progress, as always, being the very slow

patient enrollment (made worse by the rising number of competing

investigational agents), and sometimes the study design that makes

participation a questionable treatment decision.

Off hand, I can't think of a reason that the data from cooperative group

studies could not be used to satisfy FDA requirements that follow up studies

confirm benefit.

Karl

PS. Perhaps the basis for a letter to FDA?

* See for FDA DRAFT QUESTIONS February 8, 2011

Accelerated Approval & Oncology Experience <http://bit.ly/eqdjhR>

http://bit.ly/eqdjhR

Bcc: undisclosed advisors