Trade Groups Say FDA’s PDUFA Proposal Balances Innovation With Safety

The FDA’s goals for the next version of PDUFA strike a balance between fostering innovation via real-world data usage while still preserving safety standards, PhRMA and BIO officials concluded after reviewing the agency’s proposal.

Under a draft of the FDA’s benchmark goals for the next five-year cycle of PDUFA, the agency is taking a walk-before-you-run approach to study design, explained PhRMA Vice President Jeff Francer during a briefing on the issue Wednesday. The performance goals were issued by the agency Friday (DID, July 18).

As an example, Francer noted that the agency will conduct public workshops on real-world evidence before running pilot studies. And over the five-year period expected to begin in October 2017, the agency has pledged to issue more guidance on using real-world evidence to make manufacturers less reliant on clinical trial data.

“The ultimate benefit could be we can take high-quality electronic medical records and maybe even payer data and use them to show safety and efficacy,” Francer said. This may potentially “decrease the reliance on extremely costly” randomized clinical trials, but “that is much further down the road.”

The agency also intends to convene experts to determine how drugmakers can leverage data culled from its Sentinel Initiative and other sources, such as electronic health records, to determine the effectiveness and safety of therapies.

Kay Holcombe of BIO noted that the agency already has leveraged real-world data for both postmarket safety studies and drug approvals.

No official estimates have been made on much time and money industry might save by using real-world evidence, but it could shave off as many as three years of development time and save tens of millions of dollars, she said. — José Vasquez

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