12/13 March 2024
As reported in "European Commission: Draft Guideline GMP for ATMP open for stakeholder consultation", the European Medicines Agency (EMA) has published a draft guideline on "Good Manufacturing Practice for Advanced Therapy Medicinal Products pursuant to Article 5 of Regulation 1394/2007" for a second public stakeholder consultation. After a first consultation relating to the development of such a GMP for ATMP guideline - which started in July 2015 - there was still an important need for discussion. Further, representatives from local authorities, industry and sciences still expressed concerns with regard to the appropriate form of that guide.
Now, the second round of consultation is finished and the "Summary of the responses to the targeted stakeholder consultation on the development of Guidelines on Good Manufacturing Practice for Advanced Therapy Medicinal Products pursuant to Article 5 of Regulation 1394/2007" shows, that there are still uncertainties whether such a guideline as a stand-alone document will replace the requirements of the Eudralex Volume 4.
Groups like the Alliance for Regenerative (ARM) sees not enough guidance for developers of Advanced Therapy Medicinal Products. Manufacturing companies like Apceth or Lonza submitted a lot of general comments - e.g. "Applying the risk-based approach for ATMPS is highly appreciated as this gives the necessary flexibility for providing ATMPs according to guidelines". There were also detailed wishes for more clearly defined statements in the guidance text, e.g. "Please describe degree of QA system minimally required. E.g. the minimal requirements to ensure the necessary quality and traceability of the ATMPs."
Several organisations like EEBE (European Biopharmaceutical Entreprises) and EFPIA (the European Federation of Pharmaceutical Industries and Associations) recommended to reconsider the decision to adopt the guideline as stand-alone document. Amongst others they stated in their Comments on European Commission Consultation Document,'Good Manufacturing Practice for Advanced Therapy Medicinal Products'
"During the 2015 round of consultation, our comments indicated that either more detail should be added to develop a stand alone guidance, or, alternatively, less detail and more cross-references should be included to develop an annex to the current GMP requirements for medicinal products in the EU, discussing only the specifics for Advanced Therapies. We acknowledge the work that has been done on the draft guideline on GMP for ATMPs, adding further detail from EudraLex Volume 4 into this document to help meet the Commission's stated intent of a standalone document on GMP for ATMPs. However, having had the opportunity to consider the revised, stand alone draft, we have now come to the conclusion that the interests of all developers of Advanced Therapy Medicinal Products would be best served by a specific ATMP Annex to EudraLex Volume 4.
Advantages of an ATMP Annex approach would be:
Summarizing the comments of the two consultations and the discussion of the participants on relevant ECA meetings and conferences, it seems that there is still a need of discusiion.