To help relieve uncertainty among sponsors about its role in regulatory decisions, the FDA will publish guidance on its application of real-world evidence to drug approvals and post-market evaluations, FDA Commissioner Scott Gottlieb told the National Academy of Sciences.
Gottlieb advocated looking beyond the fully randomized, prospective, placebo-controlled trial that, he said, has long held sway at the top of the evidence hierarchy.
The forthcoming guidance will describe RWE and its potential for satisfying FDA’s pre- and post-market requirements, Gottlieb said. The agency already draws on practical data to infer how medical products function in the marketplace, he said. And the FDA relies on RWE in making decisions on safety — something spelled out in existing guidance on evaluation or emerging risks, he said.
To advance the RWE initiative, the FDA will work with healthcare providers to change the way it collects clinical information, including via electronic medical records, Gottlieb said.