Points to Consider No. 13: Materials in ATMP Manufacturing

This document provides key Points to Consider for navigating the complexities of raw materials in the manufacturing of advanced therapy medicinal products (ATMP), aiming to guide stakeholders in establishing robust strategies for raw material management, promoting best practices, and supporting the safe and effective development and manufacturing of ATMPs. The unique nature of ATMPs presents distinct challenges compared to traditional pharmaceuticals, demanding a focused approach to material selection, qualification, and supplier management.

Specifically addressed in this document are the intricacies arising from the fact that ATMP raw materials are often animal- or human-derived, not monographed, not manufactured for good manufacturing practice (GMP) applications (e.g., research-only grade), single-sourced and/or required to be used for aseptic manufacturing.

Very often, ATMP material suppliers and vendors are small, specialized companies with roots in academia that do not have a mature GMP quality system.

These characteristics contribute to a complex regulatory landscape, as regulations for ATMP materials are not globally harmonized. Variations in regulations exist among the U.S. Food and Drug Association (FDA), European Medicines Agency (EMA), Japan Pharmaceutical and Medical Devices Agency (PMDA), and the Pharmaceutical Inspection Co-operation Scheme (PIC/S), as well as individual country-specific laws to which companies must adhere.

This document applies to all process-stream-contacting raw materials, including starting and ancillary materials, excipients, consumables, and container–closure systems for ATMPs both in clinical stage and commercial manufacture. This includes but is not limited to cell-free mRNA therapeutics, viral vector-based in-vivo gene therapies, human cells for autologous or allogeneic cell therapies, or tissue-engineered medicinal products.