A bar chart comparing approval rates for non-orphan and orphan new active substances applications submitted to EMA between 2015 and 2020, by therapeutic area. The largest difference is among anti-cancer and immunomodulators where 69% of orphan new active substances were approved and 91% of non-orphan new active substances were approved.

FIGURE 5-5Approval rates for non-orphan and orphan new active substance applications submitted to European Medicines Agency from 2015 to 2020 per therapeutic area

NOTE: Other = other therapeutic areas not described in the top five therapeutic indications list.

SOURCE: CIRS Data Analysis, 2024.

From: 5, FDA and EMA Collaboration

Cover of Regulatory Processes for Rare Disease Drugs in the United States and European Union
Regulatory Processes for Rare Disease Drugs in the United States and European Union: Flexibilities and Collaborative Opportunities.
National Academies of Sciences, Engineering, and Medicine; Health and Medicine Division; Board on Health Sciences Policy; Committee on Processes to Evaluate the Safety and Efficacy of Drugs for Rare Diseases or Conditions in the United States and the European Union; Shore CK, Worku TL, Smith CW, et al., editors.
Washington (DC): National Academies Press (US); 2024 Oct 30.
Copyright 2024 by the National Academy of Sciences. All rights reserved.

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