European Medicines Agency releases revised guidance on advanced-therapy classification for public consultation

30 June 2014

The European Medicines Agency's Committee for Advanced Therapies (CAT) has revised a Draft reflection paper on classification of advanced-therapy medicinal products to reflect the current thinking of the committee on how ATMPs should be classified.

The Agency has released the paper today for public consultation. Comments should be sent to [e-mail] by 31 October 2014.

Based on examples, the revised paper clarifies the cases in which medicines can be classified as ATMPs and those in which they cannot. It also discusses some borderline cases and areas where scientific knowledge is limited or evolving rapidly.

The paper clarifies in particular:

• what constitutes a substantial manipulation of cells or tissues;
• what is considered as a non-homologous use of cells or tissues (or use for a different essential function);
• when medicines based on recombinant viral vectors are considered as vaccines against infectious disease and not as gene-therapy medicinal products;
• the criteria for classification as combined ATMPs, i.e. products that incorporate an active substance and one or more medical devices.

ATMPs are medicines that are made from tissues, genes or cells and may offer groundbreaking new treatment opportunities for many diseases and injuries. So far, the CAT has issued over 100 recommendations for the classification of innovative medicines as ATMPs.

ATMP classification, which is non-mandatory and free of charge, gives access to incentives specific for ATMPs, such as fee reduction for scientific advice, and services offered by the Agency, such as the certification of quality and non-clinical data of ATMPs. The classification also helps sponsors to identify the applicable regulatory framework for their medicine and the development path and the scientific or regulatory guidance to be followed.

ATMP classification, along with other pre-authorisation services offered by the Agency such as Innovation Task Force briefing meetings, often constitutes the first contact between the Agency and ATMP developers. It is often used as a first step to initiate a tailored dialogue on product development plan with the Agency.