Industry Lauds FDA RWE Framework, Calls for Tweaks

Regulatory NewsRegulatory News
| 12 February 2019 | By Ana Mulero 

The US Food and Drug Administration (FDA) regulatory framework for the use of real-world evidence (RWE) drew positive reactions from biopharma companies in comments submitted to the docket, although certain recommendations and proposals were also made.

The agency released the framework that describes its RWE program last December and has received 28 comments since then. The comment period remains open, but the submitted comments already offer a look into industry’s take on the framework. Regeneron, Sanofi, Pfizer and Novartis are among those that have provided feedback alongside industry groups PhRMA and BIO.

The commenters commended FDA for posting the 40-page framework and welcomed the agency’s continued efforts to support greater use of RWE in regulatory decision-making.

“We believe the framework is well-crafted and provides a promising roadmap for the future of FDA’s RWE program,” wrote Andrew Emmett, senior director for Pfizer’s FDA liaison office, in the firm’s comment.

They also called on FDA to expand on its RWE framework by providing additional details on specific areas within the agency’s program and addressing certain areas of concerns, such as the current lack of real-world data (RWD) standardization and industry participation in the agency’s RWE pilot projects.

There is consensus in the comments around a need for further discussions about FDA’s RWE plans to increase opportunities for stakeholder engagement. FDA said in its framework it is “making stakeholder engagement a key aspect of its RWE program.” Yet commenters urged FDA to clarify in the framework how it intends to achieve this. Several commenters, including Sanofi, argued for the inclusion of a tentative timetable with specific milestones that support continued stakeholder engagement.

“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,” wrote Sanofi’s North American head of regulatory science and policy Andrew Robertson.

Other recommendations and proposals from the commenters called for adding specific scenarios and RWE use cases, addressing the role of FDA’s new Office of Drug Evaluation Science and RWD derived from foreign studies, discussing the agency’s plan for internal hiring to support advancing RWE expertise and the need for improving RWD infrastructure, as well as establishing an FDA-industry pilot framework.

Commenters also recommended FDA issue guidance on observational study designs and RWE endpoints, among other topics.

Sean Khozin, an acting associate director at FDA’s Oncology Center of Excellence, recently pointed to some of the FDA guidance topics that will soon released under the new RWE framework. These include use of mobile technology and registries.

“Given the breadth of the work laid out in the framework, BIO encourages FDA to communicate specific timing and/or prioritization for the development and release of draft guidances,” wrote Sesquile Ramon, director of science and regulatory affairs at BIO. BIO suggested an updated framework or new updates.



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