The European Medicines Agency has revised three guidance documents to include information related to orphan medicines. These documents provide guidance to applicants in relation to pre-authorisation and post-authorisation procedures and in relation to applications for marketing authorisation of generic / hybrid medicinal products.
The revision includes questions and answers related to medicines that have been designated as orphans or for indications in which there are already orphan medicines authorised. In the latter case, there is a need for assessment of similarity in comparison with the authorised orphan medicine and, where applicable, the assessment of any of the derogations laid down the Orphan Regulation.
This requirement for similarity and, where applicable, derogation assessment may occur in the framework of the submission of an application for a marketing authorisation of a medicinal product for human use, in the framework of a type-II variation extending the existing therapeutic indication or in the framework of an extension application. All relevant sections of the pre-authorisation and post-authorisation procedural advice have been updated to explain these legal requirements.
Specific questions and answers have also been included in the procedural advice for companies submitting applications for marketing authorisation of generic / hybrid medicinal products. This update addresses the situation where the reference medicinal product is an orphan medicine and the possible need to provide a similarity assessment and, where applicable, a derogation report in the context of a generic / hybrid application.