Abstract
IMPORTANCE: Populations participating in clinical trials should benefit from resulting interventions, according to ethical guidance. Yet, exploratory studies show low- and middle-income countries (LMICs) participating in trials for US Food and Drug Administration (FDA) approvals lack timely access to the medicines they test, raising exploitation concerns. OBJECTIVE: To determine whether and when medicines receive market authorization (ie, become physically accessible) in the countries where they are tested for FDA approval, as well as trends over time by country income and geographic region. DESIGN AND SETTING: This cross-sectional retrospective analysis of phase 2 and 3 trials supporting FDA approval of novel medicines from 2015 to 2018 used data from the Drugs@FDA database, ClinicalTrials.gov website, and drug regulatory agency websites. Data were analyzed from November 2022 to January 2025. MAIN OUTCOMES AND MEASURES: Proportion of medicines receiving market authorization (physically accessible) where tested for FDA approval, median time to authorization, and proportion of countries with physical access to all medicines that they tested within 1 to 5 years of FDA approval by country, income group, and geographic region. RESULTS: The FDA approved 172 medicines between 2015 and 2018 based on 885 trials spanning 89 countries (median [IQR] of 16 [7-26] countries per medicine). Among the 77 countries publicly reporting marketing authorizations, 11 (14%) had physical access to all of the medicines they tested by 5 years after FDA approval. Of the 144 medicines tested outside of the US, 34 (24%) were physically accessible in all countries where they were tested. High-income countries had greater physical access, with 45 of 142 (32%) tested medicines physically accessible, than upper-middle-income (19 of 87 medicines [22%]) or lower-middle-income countries (7 of 55 medicines [13%]) within 5 years of FDA approval (P < .001). Among tested medicines accessible at the 5-year time point, physical access was lowest in Africa (11 of 40 [28%]) and highest in Western Europe (104 of 127 [82%]) (P < .001). Physical access improved over time for high-income countries (2015-2016 vs 2017-2018 FDA approvals: 13 of 58 [22%] vs 32 of 84 [38%]; P < .001), with no statistically significant improvements for upper-middle-income (2015-2016 vs 2017-2018 FDA approvals: 7 of 38 [18%] vs 12 of 49 [24%]; P = .08) or lower-middle-income countries (2015-2016 vs 2017-2018 FDA approvals: 4 of 29 [14%] vs 3 of 26 [12%]; P = .48). CONCLUSIONS AND RELEVANCE: Results of this cross-sectional study showed that most countries involved in trials for FDA approvals do not gain timely physical access to the medicines that they help evaluate, particularly LMICs, with no improvements over time for LMICs.