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This analysis aims to understand the factors leading to the discordance in orphan drug approvals between the FDA and EMA from 2011 to 2023.

July 8, 2026PLoS MedicineOpen Access

Discordance in orphan drug approvals between the U.S. Food and Drug Administration and the European Medicines Agency: A retrospective observational analysis

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This analysis aims to understand the factors leading to the discordance in orphan drug approvals between the FDA and EMA from 2011 to 2023.
Collected data on FDA orphan drug approvals from 2011 to 2023.
Used descriptive statistics to analyze trends and discordance between agencies.
Employed logistic regression to assess factors influencing regulatory outcomes.
Only 29% of FDA orphan approvals received corresponding EMA marketing authorization with orphan designation.
Cases from 2017-2023 showed lower odds (OR 0.66; 95% CI [0.48,0.92]; p = 0.013) of EMA marketing authorization compared to 2011-2016.
Small and medium-sized company approvals had significantly lower odds of receiving EMA orphan designations compared to large companies.

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Retrospective analysis investigates regulatory outcome discordance in orphan drug approvals, suggesting implications for therapy access in Europe.

Ding et al. (2026) studied this question.

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Retrospective analysis investigates regulatory outcome discordance in orphan drug approvals, suggesting implications for therapy access in Europe.