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.

Speakers

Robert Anderson, PhD, Associate VP, Regulatory at Emmes

Dr. Robert Anderson, PhD, is the Associate Vice President of Regulatory Affairs at Emmes with over 25 years of experience in developing regulatory strategies and managing biotherapeutic programs. He is currently responsible for Emmes’ regulatory operations and strategy groups, supporting the Center of Excellence for Cell and Gene Therapy. Prior to joining Emmes, Dr. Anderson held various roles, including Senior Director of Regulatory Global Affairs and Product Reviewer at CBER/FDA. In these positions, he provided consultative support for prophylactic and therapeutic agents, developed strategies for key Agency interactions and interpreted regulatory programs and guidelines.

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Kari Bingham, PharmD, CGT Safety Manager, Pharmacovigilance and Medical Monitoring at Emmes

Kari Bingham PharmD, is the Cell & Gene Therapy Safety Manager for Pharmacovigilance and Medical Monitoring at Emmes. With 6 years of experience as a Safety Monitor, Kari now leads the Safety Monitor team as the Safety Manager for the Cell and Gene Therapy group. In this crucial role, she guides the team through every stage of clinical trials, providing valuable input during protocol development and ensuring study closure. The team’s primary responsibility is to efficiently manage safety events that occur during trials, using their expertise to craft clinical narratives that aid in event assessment. They also work diligently to promptly report these events to sponsors, regulatory authorities, and safety committees. Additionally, Kari brings 13 years of experience in pediatric hematology/oncology and BMT as a clinical pharmacist, further enhancing her expertise in the field.

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