Advanced therapies embrace three major types of medicinal product; Gene therapy, somatic cell therapy and tissue engineered products – see below for a description of these products. The Advanced Therapy Medicinal Products (ATMPs), are expected to have a major impact on public health. These products have the potential to produce revolutionary treatment for a number of diseases such as neurodegenerative conditions (e.g. Alzheimer’s disease), cancer or to treat damaged skin as a result of burns injuries.
Regulation of Advanced Therapies
The regulatory framework for ATMPs was established with the Regulation on Advanced Therapies, which came into force in December 2008. This lays down specific rules for the evaluation, authorization and post-marketing monitoring of ATMPs. The main elements of the Regulation were:
- The establishment of a centralized marketing authorization procedure
- A new, multidisciplinary committee (Committee for Advanced Therapies or CAT)
- A number of special incentives for small and medium sized enterprises
Following the adoption of the Regulation on Advanced Therapies the European Commission and the European Medicines Agency (EMA) published ‘Implementation Plans’ that indicated actions required by the regulatory authorities to establish procedures and develop guidance for applicants in specific areas such as good clinical practice. A number of these have now been developed and can be downloaded using the links below.
It was also necessary to adapt the existing pharmaceutical regulatory legislation to bring it into line with the Advanced Therapies Regulation. Annex 1 of Directive 2001/83/EC sets out what information must be provided for marketing authorization. This was updated by Directive 2009/120/EC, which provided revised definitions and detailed scientific and technical requirements for gene therapy medicinal products and somatic cell therapy medicinal products and established technical requirements for tissue engineered products, as well as for advanced therapy medicinal product containing devices and combined advanced therapy medicinal products.
In addition to its role in assessing marketing authorizations for ATMPs, the CAT also has a number of other functions. These include:
o The provision of scientific advice.
o Recommendations on whether a product qualifies as an ATMP (classification).
o Certification of the early quality and non-clinical data for an ATMP (certification).
Additional information regarding the regulation of advanced therapies can be found on the websites of the EMA and the European Commission DG Enterprise and Industry.
o Implementation Plan for the Regulation on Advanced Therapies
o Directive 2009/120/EC
o Guidance on certification of ATMPs
o Guideline on GCP for ATMPs
- ATMP Implementation Plan.pdf
(15.839 KBkb) Download
- Informal version of Advanced Therapies final Text as discussed by Council on 31 May 2007
(216.09 KBkb) Download
- EBE Position on Advanced Therapies draft Regulation, 7 September 2006
(120.32 KBkb) Download
- EC Proposal of Regulation for Advanced Therapies of 16 November 2005
(372.232 KBkb) Download
- ENVI Final Report on 30 January 2007
(385.108 KBkb) Download
- Adopted amendments (amendments 82 to 156) at EP 1st reading, 25 April 07
(242.191 KBkb) Download
- EBE-EFPIA Press Release on EP adoption of Advanced Therapies Regulation, 25 April 2007
(94.476 KBkb) Download
- EBE-EFPIA Press Release on Council Approval of Advanced Therapies Proposal, 31 May 2007
(332.288 KBkb) Download