Scott Gottlieb has fired a shot across the FDA's statistical-confidence bow. The former FDA Deputy Commissioner, using the Wall Street Journal as his command post, charges the regulator with "steadily disregarding" the provisions of the FDA Modernization Act that give it "broad discretion" to reduce the quantity and rigor of clinical data needed to approve drugs targeting grave illnesses.
The U.S. Congress, says Gottlieb, now a fellow at the American Enterprise Institute, should force the regulator into speeding the availability of such drugs. It should reaffirm the provisions of the Modernization Act and mandate that the agency approve drugs for severe conditions on the basis of one study or, at least, a "more lenient statistical orthodoxy" than the current norm of two randomized placebo-controlled trials. The Act has given the FDA the power to do so since 1997, but the agency more often chooses to reserve that power.
"While Congress may not want to get into the business of establishing the FDA's analytical methods, it can call on the agency to convene an advisory panel to cultivate principles that are more permissive when it comes to very bad diseases," Gottlieb writes. And Congress also needs to improve FDA's review process to make it more inclusive. "The science embedded in the most novel drugs is increasingly complex, requiring collaboration across many disciplines, including clinical medicine, pharmacology and statistical modeling."
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