Marketing-authorisation procedures for advanced-therapy medicinal products
Table of contents
- Procedural advice on the evaluation of ATMPs
- Guideline on the risk-based approach according to Annex I, part IV of Directive/2001/83/EC applied to advanced therapy medicinal products
- Core SmPC, labelling and package leaflet for ATMPs containing genetically modified cells
- Procedural advice on the re-examination of Committee for Medicinal Products for Human Use opinion
- Procedural advice on the consultation of notified bodies in the case of a combined ATMPs
- Dossier requirements
- Archive: Draft procedural advice on the evaluation of ATMPs
The European Medicines Agency (EMA) provides procedural and guidance documents to help companies applying for a marketing authorisation for advanced therapy medicinal products (ATMPs).
For queries specifically on the authorisation of ATMPs, write to advancedtherapies@ema.europa.eu.
In January 2018, EMA updated its procedural advice on the evaluation of ATMPs:
The aim of the update is to clarify the evaluation procedure, to help developers of these medicines navigate the regulatory process in the European Union. The updated guidance:
- reinforces timely and effective interactions between the applicants, EMA and its committees;
- details the roles and responsibilities of the three of EMA's scientific committees involved in the evaluation: the Committee on Advanced Therapies (CAT), the Committee for Medicinal Products for Human Use (CHMP) and the Pharmacovigilance Risk Assessment Committee (PRAC);
- streamlines the processes for adopting the list of questions and list of issues by the committees;
- clarifies in which situations oral explanations might be needed;
- gives developers more time to respond to questions raised by the Committees by allowing longer clock stops.
This guidance concentrates on the initial evaluation of new ATMPs, but its principles also apply to post-authorisation procedures.
For more information on the changes introduced by the updated guidance, see:
The PRAC, the CAT and the CHMP adopted the updated guidance at their December 2017 meetings.
This is part of the joint action plan of the European Commission and EMA to streamline procedures and better address the specific requirements of ATMP developers. For more information, see Advanced therapies: overview.
Guideline on the risk-based approach according to Annex I, part IV of Directive/2001/83/EC applied to advanced therapy medicinal products
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Development of non-substantially manipulated cell-based advanced therapy medicinal products: flexibility introduced via the application of the risk-based approach (PDF/148.81 KB)
First published: 03/07/2017
Last updated: 03/07/2017
EMA/CAT/216556/2017 -
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Guideline on the risk-based approach according to annex I, part IV of Directive 2001/83/EC applied to advanced-therapy medicinal products (PDF/197.79 KB)
Adopted
First published: 08/03/2013
Last updated: 08/03/2013
Legal effective date: 12/02/2013
EMA/CAT/CPWP/686637/2011 -
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Draft guideline on the risk-based approach according to Annex I, part IV of Directive 2001/83/EC applied to advanced-therapy medicinal products (PDF/281.13 KB)
Draft: consultation closed
First published: 24/01/2012
Last updated: 24/01/2012
Consultation dates: 19/01/2012 to 30/06/2012
EMA/CAT/CPWP/686637/2011 -
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Concept paper on the development of a guideline on the risk-based approach according to annex I, part IV of directive 2001/83/EC applied to advanced therapy medicinal products (PDF/41.05 KB)
Draft: consultation closed
First published: 26/01/2010
Last updated: 26/01/2010
Consultation dates: 17/12/2009 to 31/03/2010
EMA/CHMP/CPWP/708420/2009
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Guideline on core SmPC, Labelling and Package Leaflet for advanced therapy medicinal products (ATMPs) containing genetically modified cells (PDF/465.69 KB)
Adopted
First published: 26/10/2022
EMA/CAT/CHMP/158266/2021 -
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ATMP product information template version 1 (DOCX/75.47 KB)
First published: 28/09/2022 -
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Procedural advice on the evaluation of combined advanced therapy medicinal products and the consultation of notified bodies in accordance with Article 9 of Regulation (EC) No. 1394/2007 (PDF/312 KB)
Adopted
First published: 03/03/2011
Last updated: 03/03/2011
EMA/354785/2010 -
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Overview of comments received on procedural advice on the consultation of notified bodies in accordance with Article 9 of Regulation (EC) No. 1394/2007 (PDF/160.27 KB)
First published: 03/03/2011
Last updated: 03/03/2011
EMA/690489/2010 -
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Procedural advice on the consultation of Notified Bodies in accordance with Article 9 of Regulation (EC) No. 1394/2007 (PDF/338.96 KB)
Draft: consultation closed
First published: 29/07/2010
Last updated: 29/07/2010
Consultation dates: 16/07/2010 to 29/10/2010
EMA/354785/2010