Table 3. Key differences in the procedures for orphan designation in the EU and US^*.

Items	EU	US
Terminology	Orphan medicinal product designation	Orphan drug designation
Application to	Committee for Orphan Medicinal Products	Office of Orphan Products Development
Timetable	Timetable for submission and assessment published by EMA	Any time; no defined timetable
Prevalence criteria	Disease or condition affects < 5 in 10,000 persons in the EU	Disease or condition affects < 200,000 persons in the US
Dossier	Sections A-E according to ENTR/6283/00	Nine parts according to 21 CFR 316.20
Key aspects of the application	Medical plausibility Prevalence Justification of significant benefit or why other methods are not satisfactory	Scientific rationale Prevalence
Sponsor established in territory	Proof of establishment in EU	Not required
Translations	Translations of product name and proposed orphan indication into all official languages of the EU plus Icelandic and Norwegian	Not required

^*Adapted from references of (2,4,8–10).