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EU Market Authorization Procedure

The final stage in the development of a gene therapy product after conducting clinical trials is the market application. The European Medicines Agency (EMA) (formerly known as EMEA) is a European agency for the evaluation of medicinal products, including gene therapy medicinal products. The legislative framework for market authorization of human gene therapy products and other medicinal products is initially based on Regulation (EC) No 726/2004 laying down Community procedures for the authorisation and supervision of medicinal products for human and veterinary use and establishing a European Medicines Agency. If the application concerns a product that contains a GMO, the market authorisation dossier submitted directly to the EMA should also contain the environmental risk assessment in accordance with the principles set out in Annex II to Directive 2001/18/EC. Regulation (EC) 726/2004 also describes that the competent authorities of 2001/18/EC must be consulted. In addition, the regulation indicates that an application must be accompanied by a written consent to the deliberate release into the environment of GMOs for research and development purposes provided for in part B of Directive 2001/18/EC. Thus, there is a clear connection between market authorisation of a gene therapy product and the environmental risk assessment based on Directive 2001/18/EC for clinical trials.

Regulation (EC) No 726/2004 and Directive 2001/83/EC were amended by Regulation (EC) No 1394/2007 of the European Parliament and of the Council of 13 November 2007 on advanced therapy medicinal products (ATMPs). As briefly mentioned above, ATMPs are any of the following medicinal products for human use:

  • a gene therapy medicinal product as defined in Part IV of Annex I to Directive 2001/83/EC;
  • a somatic cell therapy medicinal product as defined in Part IV of Annex I to Directive 2001/83/EC, or
  • a tissue engineered product that contains or consists of engineered cells or tissues, and is presented as having properties for, or is used in or administered to human beings with a view to regenerating, repairing or replacing a human tissue.

Committee for Advanced Therapies (CAT)
In accordance of this ATMP regulation the Committee for Advanced Therapies (CAT) has been established. The CAT is a multidisciplinary committee, gathering together experts in Europe to assess the quality, safety and efficacy of ATMPs and follow scientific developments in the field. The main responsibility of the CAT is to prepare a draft opinion on each ATMP application submitted to the European Medicines Agency, before the Committee for Medicinal Products for Human Use (CHMP) adopts a final opinion on the granting, variation, suspension or revocation of a marketing authorisation for the medicine concerned. At the request of the EMA Executive Director or of the European Commission, an opinion is also drawn up on any scientific matter relating to ATMPs. The CAT is composed of different members, including five members or co-opted members of the CHMP. These members are appointed by the CHMP itself.

Committee for Medicinal Products for Human Use (CHMP)
The CHMP is the committee at the European Medicines Agency that is responsible for preparing opinions on questions concerning medicines for human use. Assessments conducted by the CHMP are based on purely scientific criteria and determine whether or not the medicines concerned meet the necessary quality, safety and efficacy requirements (in accordance with EU legislation, particularly Directive 2001/83/EC). These processes ensure that medicines have a positive risk-benefit balance in favour of patients/users of these products once they reach the marketplace.

Gene Therapy Working Party (GTWP)
The CAT Gene Therapy Working Party (GTWP) was a multidisciplinary group of European experts that provided recommendations to the CAT on all matters relating directly or indirectly to gene therapy. It was established following the discontinuation of the Gene Therapy Working Party of the CHMP. The GTWP was replaced by ad-hoc drafting groups in September 2012.