Is the concept of “limited use” approvals falling victim to concerns that the could become a tool for the agency to narrow approved indications and to bar off-label prescribing.”
Janet Woodcock calls it like she sees it, “Given that there is skepticism and controversy, to pick an area where there is a compelling need might be a reasonable thing to do.”
It seems likely that limited use will be limited (at least initially) to anti-infective drugs.
As BioCentury points out, “Restricting the pathway to anti-infectives would allow FDA to address a public health crisis and test drive the concept, but would disappoint patient advocacy organizations and emerging biotech companies that hope the regulatory tool could speed development and approval of new drugs for a variety of conditions.”
And the pathway would be voluntary. Woodcock, “The pathway would be voluntary,” said Woodcock, and would be used to help companies tailor highly streamlined development programs to meet urgent public health needs.
The basic concept is for FDA to allow extremely streamlined development programs for drugs for well-defined subpopulations for which benefits clearly outweigh risks, and to couple expedited approvals with measures intended to discourage inappropriate off-label prescribing.
What measures? And through what authority? BioCentury opines that, “The lack of specifics and distrust of the agency’s intentions have led some critics to assume FDA is seeking a broad expansion of its power over the practice of medicine, and others to accuse the agency of plotting to allow dangerous under-tested drugs on the market.”
There is little controversy about approving drugs based on relatively small studies that demonstrate high levels of efficacy in tightly targeted populations. But FDA’s suggestion that it could work with physicians and payers to limit use of a marketed drug in the absence of documented safety concerns is controversial.
Since the FDA is being attacked from almost every side -- it’s likely they are doing something right.
And anti-infectives are a good place to start.
