Support for advanced-therapy developers
Developers of advanced therapy medicinal products (ATMPs) must be aware of the legislation governing different stages of the medicine development process, including good manufacturing practice (GMP), good clinical practice (GCP) and good laboratory practice (GLP) requirements. The European Medicines Agency (EMA) offers a range of advisory services and incentives to support the development of ATMPs.
EMA provides formal support to developers through:
- Scientific advice and protocol assistance;
- Orphan designation;
- the micro, small and medium-sized enterprise (SME) office;
- Classification of advanced therapy medicinal products (ATMPs);
- Certification of quality and non-clinical data for SMEs.
The legislation provides for scientific and financial incentives to encourage research and development in the area of advanced therapies. Developers of ATMPs can obtain reductions in the fees payable to EMA of:
- 65% fee reduction for a request for scientific advice for ATMPs (90% for SMEs);
- 90% fee reduction for the certification procedure.
The Innovation Task Force provides a forum for informal dialogue between EMA and developers of ATMPs in the early stages of the medicine development process. Registered SMEs may approach the SME office to request a briefing meeting to discuss their planned regulatory strategy.
Information on how to apply for SME incentives are detailed under SME office.
Developers of ATMPs need to be aware of the legislation that is applicable to different stages of the process.
Utilisation of substances of human origin such as blood, tissues and cells in the manufacture of an ATMP requires compliance with relevant legislation (Directive 2002/98/EC and associated implementing directives and Directive 2004/23/EC and associated implementing directives) in relation to procurement, donation and testing.
The European Commission has published a set of GMP guidelines specific to ATMPs:
These guidelines adapt the EU GMP requirements to the specific characteristics of ATMPs and and address the novel and complex manufacturing scenarios utilised for these products. They foster a risk-based approach to the manufacture and testing of such products.
The following annexes of the EU GMP guidelines are also important for ATMP manufacturing:
- Annex 2 - Manufacture of biological active substances and medicinal products for human use;
- Annex 13 - Manufacture of investigational medicinal products;
- Annex 16 - Certification by a qualified person and batch release.
Update: EMA’s CAT, together with the GMDP Inspectors Working Group and the Blood Products Working Party, have prepared a questions-and answers-document on the use of out-of-specification batches of authorised cell or tissue-based ATMPs:
Detailed guidance on specific GCP requirements for ATMPs is available from the European Commission: