Innovative Methods for Rare Disease Drug Development

ISBN-13: 9780367502102 / Angielski / Twarda / 2020 / 306 str.

Shein-Chung Chow

Opis

Innovative Methods for Rare Diseases Drug Development focuses on biostatistical applications in terms of design and analysis in pharmaceutical research and development from both regulatory and scientific (statistical) perspectives.

Spis treści

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 Approaches for Rare Diseases Drug Development. 10. The n-of-1 Trial Design and Its Application. 11. Two-Stage Adaptive Seamless Trial Design. 12. Master Protocol - Platform Trial Design. 13. Gene Therapy for Rare Diseases. 14. Clinical Development for NASH Program