Orphans: Regulatory and procedural guidance and forms

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This page lists the guidance documents on orphan designation.

Table of contents


Orphan designation

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Document(s) Language Status First published Last updated Effective Date
Template for registering new active substance on EUTCT (English only)   2018-07-13    
Template for requesting orphan industry user admin role (English only)   2018-07-13    
IRIS quick guide to the portal for orphan industry users (English only)   2018-07-04    
IRIS quick guide to registration (English only)   2018-06-19 2018-07-04  
Procedure for orphan-medicinal-product designation: Guidance for sponsors submitting an application via the current existing submission process until 19 Sept 2018 (English only)   2008-06-23 2018-07-30  
Procedure for orphan medicinal product designation: Guidance for sponsors submitting an application via IRIS secure online portal (English only)   2018-06-22 2018-07-30  
Deadlines for submission of applications for orphan medicinal product designation to the EMA and corresponding COMP timetable for valid applications - 2018-2019 (English only)   2012-05-04 2018-07-25  
Application form for orphan-medicinal-product designation (English only)   2011-11-24 2017-09-26  
Template for sections A to E for the scientific part of the application for orphan designation (English only)   2011-10-14 2018-06-18  
Translations required with the submission of an application for orphan medicinal product designation (English only)   2009-12-09 2014-06-04  
Relevant sources for orphan disease prevalence data (English only)   2012-07-24 2014-12-18  
Examples of prevalence sources previously considered in orphan medicinal product designation procedures (English only)   2014-12-18    
Recommendations on elements required to support medical plausibility and the assumption of significant benefit for an orphan designation (English only) adopted 2004-09-01 2012-03-21  
Points to consider on the calculation and reporting of the prevalence of a condition for orphan designation (English only) adopted 2002-03-22    
Data providers and sources to identify existing authorised medicinal products in the European Union and European Economic Area (English only)   2009-06-11 2015-03-17  
Procedural advice on appeal procedure for orphan-medicinal-product designation or review of orphan-designation criteria at the time of marketing authorisation (English only)   2008-05-04 2013-05-08  

Post-designation

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Document(s) Language Status First published Last updated Effective Date
Post-orphan medicinal product designation procedures: guidance for sponsors to submit an application via the current existing submission process until 19 Sept 2018 (English only)   2015-11-24 2018-07-19  
Post-orphan medicinal product designation procedures - guidance to submit an application via IRIS online portal (English only)   2018-07-19    
Note for guidance on the format and content of the annual report on the state of development of an orphan medicinal product (English only) adopted 2009-02-19 2014-09-10  
Annual report on a designated orphan-medicinal-product (English only)   2011-11-24 2017-09-26  
Fee reductions for designated orphan medicinal products (English only)   2011-02-24 2015-01-14 2013-01-01
Sponsor's report on the maintenance of the designation criteria at the time of marketing authorisation for a designated orphan-medicinal-product (English only)   2015-02-03 2015-07-03  
Reflection paper on benefit-risk assessment methods in the context of the evaluation of marketing authorisation applications of medicinal products for human use (English only) adopted 2008-03-18    
Checklist for sponsors applying for the transfer of orphan medicinal product designation (English only)   2009-01-15 2017-12-01  
Template 1 - Orphan designation transfer form (English only)   2016-04-13 2016-12-13  
Template 2 - Translations of the active ingredient and indication for transfer of orphan-medicinal-product designation (English only)   2008-11-24 2013-07-04  
Template - Orphan designation sponsor’s name and/or address change notification letter (English only)   2015-05-19 2016-12-12 2016-12-12

General

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Document(s) Language Status First published Last updated Effective Date
COMP members interaction with sponsors of applications for orphan designation (English only)   2006-07-12    
Orphan medicinal product designation in the European Union - Leaflet (English only)   2003-11-12 2010-06-08  
Orphan medicines in numbers - The success of ten years of orphan legislation (English only)   2010-05-20    
Orphan drugs and rare diseases at a glance (English only)   2007-07-03    
Note for sponsors of orphan medicinal products regarding enlargement of the European Union (English only)   2006-10-19 2013-02-15  
Amendment, cancellation, waiver or partial waiver of amounts receivable - revenue operations (English only) adopted 2017-06-13   2017-06-09

Standard operating procedures

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