Abstract
INTRODUCTION: It remains unclear whether the US clinical trial ecosystem is optimized to evaluate medical interventions efficiently. This study characterizes interventional clinical trials in the USA and examines trial progress over five years. METHODS: Using the Aggregate Analysis of ClinicalTrials.gov (AACT) database, we conducted a cross-sectional study of interventional trials initiated in 2023 and a follow-up cohort tracking five-year completion for trials started in 2018 and registered in ClinicalTrials.gov as of 05/01/2024 by sponsor and therapeutic area. Trials with at least one US site were included. Primary outcomes were enrollment, completion status, intervention type, study arm design, and plan to share individual participant data. RESULTS: Over 7,500 trials met inclusion criteria for each cohort. Most trials started in 2018 (68.4%) and 2023 (62.5%) enrolled fewer than 100 participants. Median enrollment in 2023 was higher for NIH (90) than industry-sponsored (76) trials. Within five years, 60.5% of the 2018 trials were completed, with higher completion among industry (60.9%) than NIH-sponsored (54.3%) trials. In 2023, industry predominantly funded cancer studies and trials testing drug interventions, whereas the NIH prioritized mental health studies and behavioral interventions. More than one-quarter of trials used a single-group design, 72.0% did not plan to share participant-level data, and 72.6% of drug trials were early phase. CONCLUSION: Many clinical trials are small, lack a control group, and are incomplete within five years, with differences by sponsor. Given the urgent need to improve health through rigorous evidence generation, there are likely opportunities to improve the US clinical trial ecosystem.