Leveraging Real-World Insights to Inform Long-term Follow-up for Cell and Gene Therapies: Opportunities for Pharmacoepidemiologists

Background: Cell and gene therapies (CGTs) have revolutionized the treatment of rare, chronic and life limiting diseases, including lymphoma, sickle cell disease, and hemophilia B. Approved CGTs have increased substantially and there are currently hundreds of CGTs under development. CGTs are frequently administered only once but have direct and indirect lifelong impacts on the health and well-being of patients. The proliferation of CGTs has brought to light unique pharmacoepidemiologic research challenges. Required patient monitoring underscores the breadth and depth of real-world data needed to assess the long-term benefit-risk CGTs.

Objectives: The objective of the symposium is to highlight the challenges in long-term safety and effectiveness studies for CGTs and discuss potential solutions. Pharmacoepidemiologists looking for diverse perspectives on what is required to assess the benefit-risk of CGTs and how to conduct scientifically robust and operationally feasible studies in this field will benefit from attending.

Description: The symposium will highlight the perspective and/or experience from regulators, academics, and industry professionals with input from 4 ISPE Special Interest Groups (i.e., CGT, Database, Rare Disease and Cancer) from planning to executing long-term follow-up studies in CGT. The proposed structure is as follows:

▪ Introduction (Jamie Geier, 5 min)
▪ Regulatory agency requirements and guidance on the post-authorization safety and effectiveness evaluations for CGT/innovative therapy (Quazi Ataher, 10 min)
▪ Study design options/considerations (i.e., fit for purpose strategies based on the research question and patient population) (Emily Bratton, 15 min)
▪ Data considerations (i.e., registries, tokenization, patient privacy) (Rosa Gini, 15 min)
▪ Feasibility assessments (i.e., good practices) (Kui Huang, 15 min)
▪ Challenges and potential opportunities (key takeaways incorporating academic, regulatory, industry and patient perspectives) (Peter Mol, 10 min)
▪ Open discussion and Q&A (Led by Jamie Geier, 20 min)

o Use case examples relevant to each topic will be shared to concretize lessons learned.