Premarket and postmarket real-world evidence studies supporting U.S. Food and Drug Administration regulatory decision-making, 2016-2024.

Abstract

There is growing interest in leveraging real-world data, such as electronic health records, administrative claims data, and patient registries, to generate real-world evidence studies that support the U.S. Food and Drug Administration's premarket and postmarket regulatory determinations of effectiveness and/or safety for novel therapeutics. We examined the frequency and characteristics of real-world evidence studies used by the U.S. Food and Drug Administration to support premarket determination