NOT YOUR FATHER'S FDA

Sweeping Changes in Store

It happens in small steps, but every day we learn another bit about the changes the Hamburg/Sharfstein team are bringing to an FDA saddled in dysfunction, corruption, and incapacity. Most recently, they have announced a program that would bring much more transparency to decisons on NDAs and applications for new indications that could unearth material on drugs and devices that are normally not surfaced except in litigation. Read about it in the New York Times:

http://www.nytimes.com/2009/06/02/health/policy/02fda.html?emc=tnt&tntemail1=y

After years of having to put up with an FDA leadership that wanted mostly to hide under rocks or their jockey shorts of their benefactors, it is almost difficult to believe we are seeing this much change. I still wonder how many dead-enders there are who will offer pockets of resistance--and maybe more--as reform goes on. Old and corrupt regimes die hard, as the last eight years have taught us, even at their most grotesque.

One interesting tidbit I didn't know. After the scam around the CLASS study was uncovered, and JAMA learned that it had been given the only part of the data set, the journal explicitly disowned the article. Neverthless, detailers continued to use it to sell Celebrex. I never get tired of quoting what Bob Temple said at the time: That the hype from the JAMA article will always have greater impact than our labeling does.

YOU ARE AT FDA!

Test Drive This! Be an FDA Reviewer!

Below part of an assignment I am giving my students. It would be great to hear the thoughts of people here, both about what is and what should be....

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You are an FDA reviewer and need to balance the benefits and risks of a potential new drug.. What are the most important criteria or data that you think you need in order to make an informed, correct decision?

The circumstances listed below are just to help spark (but not limit) your thinking. Circle and comment upon those you think morst important.

--Your sister-in-law works for the company, and her job is uncertain.

--The company has always had an excellent track record, especially for drugs of this kind.

--The two major phase 3 trials (A and B) submitted for NDA approval were: A with 400 subjects enrolled over six months, and B with 400 S's . enrolled for one year. It is anticipated that many thousands, perhaps millions, of people will eventually use the drug for a chronic condition (thus, over many years).

--The studies submitted within the proposal showed a 37% efficacy (mild to moderate improvement in the condition) relative to 29% for the placebo (sugar pill). Adverse events (potential side effects) were about equal. Thus 63% of subjects showed no improvement (and no evident harm) in the timeframe of the submitted studies.

--The drug is primarily to be used for osteoarthritis.

--The drug is primarily to be used for breast cancer.

--Several large studies show that a competing drug (made by another company) in the same class, and for the same condition, is twice as effective as the drug submitted.

--In one of the trials submitted, the data suggested the drug might increase the risk for heart attacks and strokes, but the “signal” is just under being statistically significant within the trials submitted.

--Same situation as above, and you know that other drugs already approved in the same have shown statistically significant increased risk of heart attacks and strokes.

--Same situation as above two, and your supervisor says you have 48 hours to complete your review because of the PDUFA deadline. So far, most of the other reviewers have supported approving the NDA.. Either point relevant? Either point likely to make a difference in the "real world"?