Innovative Methods for Rare Disease Drug Development (Hardcover, 1)

Preface. 1. Introduction. 2. Basic Considerations. 3. Hypotheses Testing for Clinical Evaluation. 4. Endpoint Selection in Clinical Trials. 5. Strategy for Margin Selection. 6. Probability of Inconclusiveness. 7. Probability Monitoring Procedure for Sample Size. 8. Real-World Data and Real-World Evidence. 9. Innovative Approachs for Rare Diseases Drug Development. 10. The n-of-1 Trial Design and its Application. 11. Two-Stage Adaptive Seamless Trial Design. 12. Master Protocols - Platform Trial Design. 13. Gene Therapy for Rare Diseases. 14. Clinical Development for NASH Program