Keywords: PDUFA VI; MIDD, Gene therapies
As part of FDA’s PDUFA VI commitments, the Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) are conducting a Model-Informed Drug Development (MIDD) pilot program. Sponsor(s) who apply and are selected will be granted meetings that aim to facilitate the application of MIDD approaches throughout the product development and the regulatory process. Due to their multifaceted and complex mechanisms of action, cell and gene therapies are of particular interest for MIDD approaches, as safety and efficacy assessments incorporate numerous input and data sources. Integration of these various data items into a systematic process and generation of quantitative data to inform regulatory decisions remain challenging. Leveraging insight from quantitative modeling and simulation techniques, such as through the MIDD program, can be crucial for informing decisions for both the sponsor(s) and the regulatory review teams. Here we share some of CBER’s perspectives on the opportunities and challenges for utilizing MIDD approaches for product review, as well as recent applications and exploratory analyses for cell and gene therapies.