The Effects of FDA’s Patient-Focused Drug Development Initiative and Patient Advocacy on New Drug Review

Abstract

This study investigates how a large and historically science-driven federal bureaucracy, the U.S. Food and Drug Administration (FDA), responds to the influences of stakeholders external to the agency in one of its key oversight activities. Does FDA’s behavior in the important process of reviewing new drugs for U.S. marketing approval change in response to pressure from outside actors? Data and qualitative information drawn from FDA published reports, agency-generated datasets, and FDA drug advisory committee materials and transcripts combine to show evidence of strategic FDA behavior. My review of almost 300 FDA drug advisory committee meetings held from 2010 to 2017, and my intensive study of 23 of those meetings in relevant disease states, showed that FDA’s behavior within the new drug review process changed over the time frame of the Patient-Focused Drug Development (PFDD) initiative. PFDD was instituted in 2012 to inform FDA systematically of the views and disease-related experiences of patients and patient advocates in selected disease states. Further, my work showed that FDA did indeed act strategically around a single key drug advisory committee meeting, that being the 2016 meeting to consider the application for marketing approval of Duchenne muscular dystrophy drug eteplirsen. Two of this thesis’ hypotheses were corroborated by evidence of FDA strategic behavior, while a third failed to achieve statistical significance.