EU marketing authorization review of orphan and non-orphan drugs does not differ

• We compare marketing authorisation reviews of orphan and non-orphan drugs.
• Deficits in clinical development plans and outcomes are related to non-approval.
• Similarity exists in orphan and non-orphan review and marketing approval decisions.
• Regulatory standards are equally high for orphan as for non-orphan drugs.

Marketing authorization application dossiers of 17 orphan drugs (ODs) and 51 non-ODs evaluated by the European Medicines Agency (EMA) in the period 2009–2010 were compared. We aimed to identify whether any differences existed between ODs and non-ODs in number and type of deficits brought forward during the EMA review, regarding the clinical development plan, clinical outcome and medical need and studied whether these deficits were similarly associated with marketing approval in the EU. In 71% of the ODs dossiers and 65% of the non-ODs dossiers marketing approval was granted. Differences in deficits were found, but similarities in the way ODs and non-ODs were reviewed and marketing approval decisions were taken, underline that regulatory standards are equally high.