Abstract
The evaluation of quality, safety, and efficacy of medicinal products by the European Medicines Evaluation Agency (EMEA) via the centralized procedure is the only available regulatory procedure for obtaining marketing authorization for gene therapy (GT) medicinal products in the European Union. The responsibility for the authorization of clinical trials remains with the national competent authorities (NCA) acting in a harmonized framework from the scientific viewpoint. With the entry into force of a new directive on good clinical practice implementation in clinical trials as of 1 May 2004, procedural aspects will also be harmonized at EU level. Scientifically sound development of medicinal products is the key for the successful registration of dossiers and for contributing to the promotion and protection of public health. The objective of this paper is to introduce the EMEA regulatory processes and scientific activities relevant to GT medicinal products.
Highlights
The objective of this paper is to introduce the European Medicines Evaluation Agency (EMEA) regulatory processes and scientific activities relevant to gene therapy (GT) medicinal products
Whilst the EMEA has no remit for the authorization of the conduct of clinical trials in the European Union (EU), it is agreed that a scientifically sound development is necessary in order to demonstrate the efficacy of the product and to exclude any major safety problem incompatible with the safe use of the product
The outcome of the procedure is a Committee for Proprietary Medicinal Products (CPMP) scientific opinion, which is either favourable or unfavourable. This is communicated to the European Commission, which in turn is responsible for converting CPMP opinions into legally binding decisions
Summary
Marisa Papaluca Amati,∗† Francesco Pignatti, Alexis Nolte, Nirosha Amerasinghe, Daniel Gustafsson, Isabelle Moulon, and Patrick Le Courtois. The European Agency for the Evaluation of Medicinal Products, 7 Westferry Circus, Canary Wharf, London E14 4HB, UK. The evaluation of quality, safety, and efficacy of medicinal products by the European Medicines Evaluation Agency (EMEA) via the centralized procedure is the only available regulatory procedure for obtaining marketing authorization for gene therapy (GT) medicinal products in the European Union. The responsibility for the authorization of clinical trials remains with the national competent authorities (NCA) acting in a harmonized framework from the scientific viewpoint. With the entry into force of a new directive on good clinical practice implementation in clinical trials as of 1 May 2004, procedural aspects will be harmonized at EU level. The objective of this paper is to introduce the EMEA regulatory processes and scientific activities relevant to GT medicinal products
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