Orphans: Regulatory and procedural guidance and forms
This page lists the guidance documents on orphan designation.
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Quick interactive guide to IRIS registration process (PDF/971 KB)
First published: 25/08/2020
Last updated: 10/03/2021 -
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Template for registering new active substance on EUTCT (XLSX/33.76 KB)
First published: 13/07/2018
Last updated: 10/01/2023 -
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Template for requesting orphan industry user admin role (DOC/40 KB)
First published: 13/07/2018
Last updated: 13/07/2018 -
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IRIS guide for applicants - How to create and submit scientific applications, for industry and individual applicants (PDF/4.12 MB) (updated)
First published: 04/07/2018
Last updated: 28/11/2023
EMA/444925/2018 Version 3 -
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IRIS guide to registration and RPIs (PDF/1.32 MB)
First published: 19/06/2018
Last updated: 25/05/2023
Version 2.15
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Statement on the amended policy on orphan designations for inherited retinal dystrophies (PDF/157.01 KB)
First published: 25/01/2023
EMADOC-628903358-34665 -
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Procedural advice for orphan medicinal product designation: Guidance for sponsors (PDF/301.22 KB)
First published: 22/06/2018
Last updated: 20/10/2023
EMA/420706/2018 Rev 13 -
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Deadlines for submission of applications for orphan medicinal product designation to the EMA and corresponding COMP timetable for valid applications - 2022-2023 (PDF/108.66 KB)
First published: 04/05/2012
Last updated: 25/04/2022
EMADOC-628903358-2283 -
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Template for sections A to E for the scientific part of the application for orphan designation (DOCX/35.31 KB)
First published: 14/10/2011
Last updated: 20/04/2023 -
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Template - Translations required with the submission of an application for orphan medicinal product designation (DOC/71 KB)
First published: 10/12/2009
Last updated: 06/10/2022 -
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Points to consider on the estimation and reporting of the prevalence of a condition for orphan designation (PDF/148.52 KB)
Adopted
First published: 22/03/2002
Last updated: 21/06/2019
EMA/COMP/436/01 Rev. 1 -
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Procedural advice on appeal procedure for orphan-medicinal-product designation or review of orphan-designation criteria at the time of marketing authorisation (PDF/129.47 KB)
First published: 04/05/2008
Last updated: 16/12/2020
EMEA/2677/01 Rev. 3
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Procedural advice for post-orphan medicinal product designation activities: Guidance for sponsors (PDF/368.53 KB)
First published: 19/07/2018
Last updated: 20/10/2023
EMA/469917/2018 Rev.14 -
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Sponsor's report on the maintenance of the designation criteria at the time of marketing authorisation for a designated orphan-medicinal-product (DOC/1.19 MB)
First published: 03/02/2015
Last updated: 13/03/2020 -
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Checklist for sponsors applying for the transfer of Orphan Medicinal Product (OMP) designation (PDF/160.29 KB)
First published: 15/01/2009
Last updated: 09/06/2022
EMA/41277/2007 Rev.15 -
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Template - Translations required with the submission of an application for transfer of orphan medicinal product designation (DOC/65.5 KB)
First published: 25/11/2008
Last updated: 06/10/2022
- Guideline on the format and content of applications for designation as orphan medicinal products and on the transfer of designations from one sponsor to another
- Regulation (EC) No 141/2000 of the European Parliament and the Council of 16 December 1999 on orphan products
- Commission Regulation (EC) No 847/2000 of 27 April 2000 laying down the provisions for implementation of the criteria for designation of a medicinal product as an orphan medicinal product and definitions of the concepts 'similar medicinal product' and'clinical superiority'
- Commission Regulation (EU) 2018/781 of 29 May 2018 amending Regulation (EC) No 847/2000 as regards the definition of the concept 'similar medicinal product'
- Commission notice on the application of Articles 3, 5 and 7 of Regulation (EC) No 141/2000 on orphan medicinal products (2016/C 424/03)
- Guideline on aspects of the application of Article 8(1) and (3) of Regulation (EC) No 141/2000: Assessing similarity of medicinal products versus authorised orphan medicinal products benefiting from market exclusivity and applying derogations from that market exclusivity
- Guideline on aspects of the application of Article 8(2) of Regulation (EC) No 141/2000 of the European Parliament and the Council: Review of the period of market exclusivity of orphan medicinal products
- Register of designated orphan medicinal products
- Meeting dates for the Committee for Orphan Medicinal Products (COMP)
- FDA - Office of Orphan Products Development
- Japanese National Institute for Biomedical Innovation: Services to promote development of medicinal products for rare diseases