Former FDA Commissioner Offers Strange Idea: Test Efficacy in Post-Marketing

Former FDA Commissioner Offers Strange Idea: Test Efficacy in Post-Marketing

February 16th, 2012 // 2:37 pm @ jmpickett

File this under ‘What was he thinking?’ In an op-ed in The Wall Street Journal, former FDA commish Andy von Eschenbach proposes that the “FDA should approve drugs based on safety and leave efficacy testing for post-market studies.” How would this work? Well, Andy suggests creating pilot programs in which patients would be entered in registries, and the FDA and drugmakers would later determine whether a medicine is effective.

“Take regenerative medicine,” he writes. “If a company can grow cells that repair the retina in a lab, patients who’ve been blinded by macular degeneration shouldn’t have to wait years while the FDA asks the company to complete laborious clinical trials proving efficacy. Instead, after proof of concept and safety testing, the product could be approved for marketing witn every eligible patient entered in a registry so the company and the FDA can establish efficacy through post-market studies.”

In other words, this approach envisions substituting epidemilogical studies for double-blind, randomized clinical trials. This is ironic, because Andy laments that the FDA is losing its status as the gold standard among regulators, which was the rationale for writing his missive. “By empowering the FDA to create new paradigms for evaluating the most promising innovations, Congress can ensure that the FDA serves as a bridge-not a barrier-to cutting-edge technologies,” he intones (here is the op-ed).

Of course, the pharmaceutical industry would benefit, since fewer trials would be required prior to approval, which would lower development costs. Meanwhile, payers – both private and public – would provide coverage for medicines, even though efficacy remains unknown until post-marketing study results are reported. Then again, the industry has a poor track record of initiating post-marketing studies promptly, or even at all. How long such a scenario would exist is unclear.

Imagine, after all, the reaction if studies are not completed on a timely basis or show a lack of efficacy. Meanwhile, registers are ringing and some patients are being treated with medications that do not serve the hoped-for goal. Andy, who is now chairman of the Manhattan Institute’s Project FDA, believes that “breakthrough technologies deserve a breakthrough in the way the FDA evaluates them.” Would this approach amount to a breakthrough? Or a setback?