12/13 March 2024
In the recent past, ATMPs have increasingly moved from research and development to clinical trial phases or to the status of approved medicinal product. As a result, this product group has increasingly moved into the focus of regulatory authorities and GMP inspectors. In the near past, you could therefore read about regulatory developments on several occasions, both in the USA and in Europe, for example through the Stakeholder Consultation on the Draft Guideline GCP for ATMP. The results of the consultation are also available on the website of the European Commission.
Due to the complexity and heterogeneity of the group of ATMPs, there was an urgent need for further regulatory support to complement the classical GCP guidelines, e.g. the ICH guidelines. Special challenges, based on the special properties such as small batch sizes, short shelf life, etc., often make it necessary to adapt existing specifications for ATMPs. Usually, regulatory information was lacking for this purpose. This gap is to be closed by the now available final guideline, which deals with topics such as design of clinical studies, non-clinical studies, quality of investigational ATMPs, the safety of clinical trials, traceability, retained samples, safety reports and monitoring.
It is interesting to note that this document also addresses a topic that recently attracted the attention of the ATMP community, namely the handling of OOS products, and the extent to which they can be released for use on patients despite their OOS status. In the summer, the EMA published a document entitled "Questions and answers on the use of out-of-specification batches of authorised cell/tissue-based advanced therapy medicinal products".
Further details on the GCP requirements can be found directly in the new guideline "Good Clinical Practice specific to Advanced Therapy Medicinal Products".
For all who are interested in the manufacture of ATMPs, please refer to the Annex 2 of the PIC/S GMP Guidelines currently published for comments, consisting of two parts, one of which is dedicated to ATMPs. The ATMP Interest Group of the ECA Foundation also provides the opportunity to comment here. The corresponding information can be found in the member s'area of the group website. Membership is free of charge.