Statistical issues in regulatory reviews of CBER products
*Shiowjen Lee, FDA 


Center for Biologics Evaluation and Research (CBER) regulates blood products (both derivatives/components and devices maintaining safe blood supply), preventive vaccines (e.g., flu vaccines and childhood vaccines), tissue, cellular and gene therapies and therapeutic cancer vaccines. CBER products are different from those regulated by CDER with respect to working mechanism and manufacturing processes. These factors subsequently contribute challenges to the study design and statsitical inferences of clinical trial data. Although the statistical principles may seem to be similar to those of products that are regulated by other FDA Centers, there are unique statistical issues for CBER products. The objective of this presentation is to share experiences of statsitical issues commonly encountered in CBER reviews. Recommendations and considerations will be provided to aid sponsors in their preparation of submissions.