Innovative Designs and Analyses for Small Population Clinical Trials

Jingjing Ye is Senior Director, Head of Data Science of Operational Excellence (DSOE) in Global Statistics and Data Science (GSDS). She leads a team to promote innovations in various organizations within BeiGene. She has been active in the scientific community, serving as an FDA representative between industry leaders, academic institutions, and the FDA on various scientific working groups, including pediatric, external controls in oncology, simulation in adaptive designs, and quantitative imaging biomarkers. She currently leads several cross-disciplinary working groups, including ASA Biopharmaceutical (BIOP) Section Pediatric Working Group, ASA-DIA Master Protocol and DIA Data Sciences.
Lei Nie is the Associate Division Director for DB II, in the US Food & Drug Administraion. He is an elected fellow of the American Statistical Association and has authored a total of 99 peer-reviewed journal publications. Passionate about multidisciplinary collaboration and the important work performed at the FDA, he always looks for ways to improve efficiencies and to maintain a good work-family balance.

1. Introduction.- 2. The Case Study of NCI-COG Pediatric Match Trials.- 3. The Case Study of Non-oncology (Lonafarnib).- 4. Natural History and Patient Registry.- 5. Diagnostic Criteria and Endpoints.- 6. Enrichment Designs.- 7. Pediatric Extrapolation.- 8. Cross-over Designs.- 9. Adaptive Designs.- 10. Master Protocols.- 11. Other Designs.- 12. Modeling and Simulations.- 13. Frequentist Approaches.- 14. Bayesian Approaches.- 15. Operation and Other Considerations.- 16. Case Studies.