P238: Ensuring Success in MRCTs with Optimal Sample Sizes for Global Drug Development
Poster Presenter
Angela Hu
VP, Biostatistics
Parexel United States
Objectives
To provide US-based sponsors with strategies for determining optimal sample sizes in Multi-Regional Clinical Trials (MRCTs) that include Asian populations, ensuring compliance with both FDA and Asian regulatory requirements.
Method
We developed SAS/R programs for sample size calculation and simulation, incorporating region-specific regulatory requirements from FDA, PMDA, and NMPA. A visualization tool was created to illustrate sample size impacts across regions.
Results
We will explore the complexities of determining optimal sample sizes that not only meet US regulatory standards but also address the specific requirements of various Asian regulatory bodies. This includes navigating the challenges of demonstrating consistent efficacy and safety across diverse populations, with a particular emphasis on Asian subgroups. Our discussion will cover:
> Strategies for balancing FDA expectations with those of Asian regulators, such as the PMDA in Japan or NMPA in China.
> Statistical approaches to sample size estimation that account for potential regional differences in treatment effects.
> Methods for ensuring adequate representation of Asian populations while maintaining overall trial efficiency.
> Case studies illustrating successful MRCTs, highlighting key learnings and best practices.
Conclusion
This presentation aims to provide US-based sponsors with practical insights and methodologies for designing and implementing MRCTs that effectively include Asian populations. By optimizing sample sizes and trial designs, sponsors can enhance the global applicability of their clinical data, streamline the drug development process, and improve the likelihood of successful regulatory approvals across multiple regions.
