Craigslist Bethesda: Item wanted–a spine. Any spine. Call 888-463-6332 M. Hamburg

What is it about Bethesda? How is that bright, well-intentioned medical and scientific professionals enter into the FDA, and somehow become transf0rmed into brainless puppets spewing the same pseudostatistical rationalizations as their forebears? Perhaps there is something in the water (call the EPA), or in the food supply (call the FDA…oh, never mind). In any event, it is remarkable that some of the decision makers are even ambulatory, given the complete lack of visible means of support for their spinal cords.

The most recent evidence, tafamidis. The FDA today turned down Pfizer‘s application for tafamidis in the treatment of the rare, and devastating disease, Transthyretin Familial Amyloid Polyneuropathy. Just a few weeks ago, NP enthusiastically, and naively, welcomed the decision by a FDA AC to support the provisional approval of  tafamidis on the basis of surrogate endpoints, provided that Phase IV testing be subsequently completed.

As NP commented at the time: “This could be a huge precedent. Given the immense challenges, particularly the time required, associated with confirming impact on disease progression, the process of having to do so prior to drug approval mandates enormously expensive and slow Phase III testing. NIR believes strongly that changes in specific functional measures and biomarkers may prove to be equally useful predictors in other neurodegenerative disorders, including Alzheimer’s and Parkinson’s….the FDA’s AC move in this direction is a sign of where the regulatory process must move eventually, and the sooner, the better.”

We added: ” If they do not accept the AC’s recommendation, that would be an indication that their public statements regarding improvements in the regulatory environment constitute insincere posturing. We will know soon enough.”

And now we know. Business as usual. The company has been instructed to run another clinical trial, which based on past trials, will take 2-3 years. Never mind the surrogates, never mind the Advisory Committee. Which brings to mind a question: If you are going to ignore the advice from your Advisors, why bother with them in the first place? It’s not as if their vote had been a close call, it was 13-4. Many  of them are people with clinical experience in treating TTR. We doubt that can be said of anyone in an executive role with the Agency. But it almost seems like the AC’s purpose is to give cover to the Agency in going in the direction it wants to go, and if the AC prefers another course, ignore them. After all, they’re just experts.

Most probably, the Agency was afraid of the very thing that we welcomed: A Precedent. They did not want to be asked–why did you say yes to Pfizer, and not to us?For us it looked like an opportunity, to them, a slippery slope.

It’s always easier to say no, after all, there aren’t that many TTR patients, they probably don’t have a lobbyist, and you can always fall back on the old standby rationalization, that you are protecting the public from ineffective and/or unsafe therapeutics. But to be blunt about it, the Agency only protected itself and its Standard Operating Procedure. No one else benefits from this decision.

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