FDA’s Real World Evidence Guidance Gains Industry Support, but With Slight Modifications

After the U.S. Food and Drug Administration (FDA) took a hard line on the use of real-world evidence in clinical trials, industry leaders are beginning to back the idea, but with some minor changes.

The FDA released a strategic framework for the use of real-world evidence at the end of 2018 that indicated how the regulatory agency plans to use real-world data to improve regulatory decisions. Real-world evidence, also known as real-world data, centers on the collection of information about a drug’s safety and efficacy outside of the structure of a clinical trial. Such data can be gathered from electronic health records, laboratory tests, wearable devices, insurance claims and even social media. For the past couple of months, FDA Commissioner Scott Gottlieb has touted the use of this kind of evidence as a way to improve the making of treatment decisions.

On Tuesday, RAPS reported that since the FDA released its guidelines for the use of real-world evidence, industry leaders have provided some public commentary on the proposal. Andrew Emmett, senior director for Pfizer’s FDA liaison office said in a comment on the proposal that the company believes the “framework is well-crafted and provides a promising roadmap for the future of FDA’s RWE program,” RAPS reported.

In its analysis of the comments, RAPS noted there was a consensus about the need for further discussions regarding the real world evidence plan to “increase opportunities for stakeholder engagement.” The executives who commented, RAPS said, asked the FDA to clarify how it intends to achieve increased opportunities for stakeholders. RAPS reported that multiple company commenters called for the “inclusion of a tentative timetable with specific milestones that support continued stakeholder engagement.” Sanofi was one of these companies. In a comment, Andrew Robertson, Sanofi’s North American head of regulatory science and policy, said the timeline “should account for the planning and negotiation process for the 2022 reauthorization of the Prescription Drug User Fee Act and allow for any learnings from this framework to be considered appropriately.”

Additionally, RAPS said that industry leaders called for the addition of specific scenarios and cases where the use of real-world evidence could be used within the framework.

As BioSpace reported in January, the FDA believes that the use of real-world evidence will provide data that can “complement, augment and expand our understanding of how best to use medical products.” The FDA guidance said real-world evidence has “great utility in postmarket monitoring for the safety of drugs and medical products.”

“Traditional postmarket studies typically require years to design and complete and cost millions of dollars. By encouraging the use of RWD and RWE, we may be able to provide patients and providers with important answers much sooner by potentially identifying a broader range of safety signals more quickly,” Gottlieb said in December.