Orphans: Regulatory and procedural guidance and forms

  • Email
  • Help

This page lists the guidance documents on orphan designation.

Table of contents

Orphan designation

Back to top

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 version 1.3 (English only)   2018-06-19 2018-08-28  
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  
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  


Back to top

Document(s) Language Status First published Last updated Effective Date
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


Back to top

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

Back to top

Related links