International – Cell and gene therapy : Regulatory Focus, July issue

Feature articles during July focused on global regulatory strategy for cell and gene therapy, with articles on US and EU regulations and guidances and the development and manufacture of the therapies. Also included were articles on recasting the corrective and preventive action (CAPA) process as a continuous improvement process, a military-civilian perspective on real-world evidence (RWE) to support regulatory decision making, and regulatory reporting in multinational trials during COVID-19.

Advanced therapy medicinal products (ATMPs), including cell therapies, gene therapies, and tissue-engineered products, are highly complex treatments that differ from traditional medicines, both in how they are made and administered and in the benefits they may provide. Regulations for these products were established relatively recently and are still evolving in many jurisdictions globally. The novelty of these products, the inherent complexities of cell and gene therapy products, and the lack of experience with such products pose many challenges for developers.

In part one of this two-part series, this month’s expert authors address these challenges and offer hands-on, practical advice and guidance on regulation and production of ATMPs. If there is one clear, take-home message to developers, it is that early and frequent collaboration with regulatory agencies, during both the approval and development phases, is paramount. It saves time and money, and it reduces the risk of a negative impact on the trajectory of a clinical trial. Part 2 of the series will cover upstream manufacturing and process controls for biologics, the ATMP regulatory landscape in China, EU GMP requirements for autologous cell therapies and parenteral biologics, and regulatory challenges and opportunities in the US…