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
Procedure for orphan-medicinal-product designation: Guidance for sponsors (English only)   2008-06-23 2015-06-25  
Deadlines for submission of applications for orphan-medicinal-product designation to the European Medicines Agency 2017/2018 (English only)   2012-05-04 2017-03-19  
Application form for orphan-medicinal-product designation (English only)   2011-11-24 2017-09-26  
Common European Medicines Agency / Food and Drug Administration application form for orphan-medicinal-product-designation (English only)   2008-11-24 2017-09-26  
Template for sections A to E for the scientific part of the application for orphan designation (English only)   2011-10-14 2014-09-10  
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  


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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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