Real World Evidence on Definitions Under Development: Gottlieb
FDA is developing a guidance document on real world evidence (RWE) consensus definitions that is intended to help advance RWE use in pre- and post-market review processes, FDA commissioner Scott Gottlieb told a National Academy of Sciences 9/19 workshop in Washington, DC. The definitions will relate to how different parts of the agency use or will use RWE in regulatory decision making.
Gottlieb said that improving the use of RWE in clinical and regulatory decisions will require engaging with the healthcare system to change the way clinical information is collected. “Ideally, we’d like to have a system where providers have the right incentives to enter clinically relevant information into [electronic medical records] EMRs at the point of care,” he said. A caveat is that much of the information entered into EMRs are geared to medical billing. Clinically relevant information is often left out and entered into “unstructured notes,” he explained. “We’re unable to learn as much as we could about a product’s profile when that information isn’t accessible. We also need to find a better way to collect information directly from patients. Because an EMR and claims data is really the patient’s perspective filtered via the provider.”
The use of RWE seems much more further along for medical devices than for drugs. Last month, the agency issued a final guidance on the use of RWE in device development. Gottlieb noted that since 2015, the agency approved or cleared more than eight new medical devices and expanded the use of more than six technologies based on RWE. “This includes drug-eluting stents, transcatheter heart valves, and technologies for spinal cord stimulation and esophageal atresia, as well as in IVDs,” he said, adding that the evidence was generated in less time and at a lower cost than in the past. Additionally, device makers are also increasingly addressing post-market study requirements by “leveraging real world data sources,” he said.
Gottlieb told the workshop that the agency is now working on policies to support the use of RWE in approving new indications for already-marketed drugs. “This may be especially relevant in settings like rare diseases or other unmet medical needs, where it can be hard to enroll patients in clinical trials,” he said. “We’re also expanding policies to enable the use of RWE to support post-approval drug study requirements.