Risk-based approach according to Annex I, part IV of Directive 2001/83/EC applied to Advanced Therapy Medicinal Products - Scientific guideline

This document provides guidance on the application of the risk-based approach in the preparation of a marketing authorisation application. The concept of a 'Risk-based approach' has been introduced to the legislation with the revision of Annex 1, part IV of Directive 2001/83/EC as amended by Directive 2009/120 EC.

Keywords: Advanced therapy medicinal products (ATMPs), risk-based approach, risk, risk factor

Current effective version

Guideline on the risk-based approach according to annex I, part IV of Directive 2001/83/EC applied to advanced-therapy medicinal products

Adopted Reference Number: EMA/CAT/CPWP/686637/2011 Legal effective date: 12/02/2013 Summary:

The clinical use of Advanced Therapy Medicinal Products (ATMPs) in humans may be associated with specific risks to the patient and to third parties. These risks are determined by various risk factors, which are related to the quality, biological activity and application of the ATMP.

Document history - First version (current)

Guideline on the risk-based approach according to annex I, part IV of Directive 2001/83/EC applied to advanced-therapy medicinal products

Adopted Reference Number: EMA/CAT/CPWP/686637/2011 Legal effective date: 12/02/2013 Summary:

The clinical use of Advanced Therapy Medicinal Products (ATMPs) in humans may be associated with specific risks to the patient and to third parties. These risks are determined by various risk factors, which are related to the quality, biological activity and application of the ATMP.

Draft guideline on the risk-based approach according to Annex I, part IV of Directive 2001/83/EC applied to advanced-therapy medicinal products

Draft: consultation closed Consultation dates: 19/01/2012 to 30/06/2012 Reference Number: EMA/CAT/CPWP/686637/2011 Summary:

This guideline describes the intention of the risk-based approach and details its methodological application. The methodology is based on the identification of risks and associated risk factors of an advanced-therapy medicinal product and the establishment of a specific profile for each risk. With the use of the identified risk profile the applicant shall justify the extent of data presented in the various sections of the marketing-authorisation application dossier.

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

Draft: consultation closed Consultation dates: 17/12/2009 to 31/03/2010 Reference Number: EMA/CHMP/CPWP/708420/2009