How The FDA Is Helping To Operationalize Patient Voice In Clinical Trials

By Alysia Drummond, inSeption Group

While sponsors have increasingly attempted to incorporate patient experience data (PED) into their submissions, these data are frequently excluded from the labeling. In many cases, the FDA cited insufficient rigor or a high potential for bias as the reason for exclusion. In response, and particularly since the passage of the 21st Century Cures Act in 2016, the FDA has taken steps to clarify how it expects patient-focused drug development (PFDD) to be conducted. This includes a series of guidance documents that define more clearly the agency’s expectations for methodological rigor and how sponsors can achieve it.

In addition to the PFDD guidance documents the FDA is developing, the agency offers several mechanisms to assist drug developers in implementing patient voice. Among these is the patient voice-focused portal, CDER Patient-Focused Drug Development | FDA. The portal provides a range of resources, including a single-page, quick reference to help companies establish an early patient-focused mindset, as well as a COA compendium, condition-specific meeting reports, detailed guidance documents, and even a podcast.3All of these resources are foundational and intended to be a starting point; each drug developer still needs to determine how the FDA’s guidance applies specifically to their program.