2023 - Cell and Gene Therapy Clinical Trials’ Regulatory Strategies and Safety Considerations

As the somatic cell and gene therapy platforms continue to advance, so do the US Food and Drug Administration (FDA) and international council for harmonisation of technical requirements for pharmaceuticals for human use (ICH) regulatory guidelines and the competent authority requirements associated with these product classes. Successful preclinical to clinical transition requires careful consideration of these agency guidance documents to minimize the risk of avoidable delays in the development of cell and gene therapy clinical trials in a regulated environment.
Many cell and gene therapy clinical trials come with their own challenges and have unique safety and efficacy considerations before they can be approved for marketing. Early-phase trials emphasize safety and have unique requirements regarding the timing of primary safety assessment. They often require lengthy long-term follow-ups. On the other hand, later phase trials, used to support a marketing application, require statistically significant and clinically meaningful efficacy data. Additionally, the development of novel appropriate endpoints is encouraged to showcase the effectiveness of these therapies.
This webinar will focus on best practices to navigate regulatory communications with the US FDA and how this guidance may inform pharma and biotech companies on the development of manufacturing, non-clinical development plans and investigational approaches.
Join this webinar to get insights on the US FDA guidelines and the managing safety considerations for the efficacy endpoints of these cell and gene therapy products.