Public Participation in U.S. Food and Drug Administration (FDA) Advisory Committees: A Qualitative Analysis.
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IMPORTANCE: The FDA frequently solicits public input to inform advisory committee meetings reviewing new drug applications. Although intended to enhance transparency and incorporate patient perspectives, the content of these submissions has not been well described. OBJECTIVE: To characterize public comments to FDA advisory committee meetings and examine the values and considerations expressed. DESIGN: Qualitative study using inductive content analysis with an iterative coding framework. Comments were coded by one reviewer and independently double-coded for a random subset; disagreements were resolved through discussion (Light's kappa = 0.81). SETTING: Public comments associated with FDA advisory committee meetings for five new drug applications reviewed between January 2020 and December 2024. PARTICIPANTS: A random sample of 600 public comments from the five products with the highest public engagement: (1) sodium phenylbutyrate-taurursodiol for ALS; (2) teplizumab for type 1 diabetes; (3) midomafetamine (MDMA-assisted therapy) for PTSD; (4) delandistrogene moxeparvovec for Duchenne muscular dystrophy; and (5) eflornithine tablets for pediatric neuroblastoma. MAIN OUTCOME MEASURES: Stakeholder role, stance regarding drug approval, and thematic content. RESULTS: Submissions were most commonly from caregivers or immediate family members (31.5%), individuals without disclosed roles (20.0%), friends or extended family (18.5%), and patients (17.2%). Most comments supported approval (90.2%), with few neutral (7.8%) or opposing (1.3%) views. Four themes emerged: (1) experiential considerations; (2) evidentiary considerations, including clinical trial data and personal observations; (3) regulatory and policy considerations, such as urgency of access; and (4) values-based considerations, such as therapeutic hope and autonomy. CONCLUSIONS AND RELEVANCE: Public comments were overwhelmingly supportive of approval, reflecting experiential, evidentiary, policy, and values-based considerations. Strengthening mechanisms for synthesizing public input may enhance transparency and confidence in the FDA regulatory process.