Orphans: Regulatory and procedural guidance and forms

The European Medicines Agency (EMA) makes guidance documents on orphan designation available to stakeholders.

For more information, see: 

• Orphan designation: Overview

Submission guidance

Regulatory Procedure Management (RPM) for the Product Lifecycle Management (PLM) - Frequently asked questions

Quick interactive guide to IRIS registration process

Summary:

Quick interactive guide to IRIS registration process: This presentation is interactive. Please follow the instructions and click on the appropriate icon to be directed to the relevant section.

Template for registering new active substance on EUTCT

Template for requesting orphan industry user admin role

IRIS guide for applicants - How to create, submit and manage IRIS applications, for industry and individual applicants

Reference Number: EMA/444925/2018 Version 3.14

IRIS guide to registration and RPIs

Reference Number: EMA/444925/2018 Version 2.19 Summary:

Preliminary requirements for all IRIS submissions, including substance and Research Product Identifier registration.

Orphan designation

Statement on the amended policy on orphan designations for inherited retinal dystrophies

Reference Number: EMADOC-628903358-34665

Procedural advice for orphan medicinal product designation: Guidance for sponsors

Reference Number: EMA/420706/2018 Rev.19

Deadlines for submission of applications for orphan medicinal product designation to the EMA and corresponding COMP timetable for valid applications - 2022-2023

Reference Number: EMADOC-628903358-2283

Template for sections A to E for the scientific part of the application for orphan designation

Template - Translations required with the submission of an application for orphan medicinal product designation

Points to consider on the estimation and reporting of the prevalence of a condition for orphan designation

Adopted Reference Number: EMA/COMP/436/01 Rev. 1

Procedural advice on appeal procedure for orphan-medicinal-product designation or review of orphan-designation criteria at the time of marketing authorisation

Reference Number: EMEA/2677/01 Rev. 3

Post-designation

Procedural advice for post-orphan medicinal product designation activities: Guidance for sponsors

Reference Number: EMA/469917/2018 Rev.16

Sponsor's report on the maintenance of the designation criteria at the time of marketing authorisation for a designated orphan-medicinal-product

Checklist for sponsors applying for the transfer of Orphan Medicinal Product (OMP) designation

Reference Number: EMA/41277/2007 Rev.15

Template - Translations required with the submission of an application for transfer of orphan medicinal product designation

General

COMP members interaction with sponsors of orphan designation applications

Reference Number: EMEA/COMP/150409/2006 Rev. 1

Business process description

Business process description for orphan status management

Reference Number: EMA/367793/2024 Legal effective date: 25/10/2024

• 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