BioCentury reports that Gottlieb took on critics who say FDA should only consider data from randomized, controlled trials. “For those who’d challenge the suitability of our effort to incorporate real world evidence into our regulatory model, I’d challenge you with the opposite intention: Should a product be marketed based on a data set that speaks to a limited and rigidly constructed circumstance, when the clinical use, and in turn the evidence we might have to evaluate the product, could have been far richer, far more diverse, and more informative?”
RWE is essential to making FDA’s decisions relevant to entire healthcare system, Gottlieb said. He noted that data gathered from routine clinical experience is already being used to make medical, payment and coverage decisions. “We need to close the evidence gap between the information we use to make FDA’s decisions, and the evidence increasingly used by the medical community, by payers, and by others charged with making healthcare decisions.”
In a move that is unusual for a regulator, Gottlieb acknowledged that FDA’s policies on and interpretation of RWE may not be definitive. “FDA needs to think of itself as a curator of information, not just an arbiter, where a single truth standard is secured to a fixed orthodoxy.” He added: “There’s often no single truth standard when it comes to the evidence used to support medical decisions.”
Gottlieb placed RWE in the context of a regulatory life cycle that extends well beyond product approval. “No product is all risky and uncertain one day, and completely safe and effective the next,” he said. “We can’t allow our need for a point of regulatory accountability to prevent us from looking across the line we have to draw, at practical information that’s collected both before and after our point of demarcation, when a product gains a license for initial market entry."
Getting more reliable RWE will require changes in the way clinical data is collected, Gottlieb said, including a shift from electronic health records designed primarily to support billing to records that routinely capture information about what is happening with patients. He also committed FDA to take steps to provide more clarity about the requirements for using RWE in regulatory submissions.