Buch, Englisch, 320 Seiten, Format (B × H): 156 mm x 234 mm, Gewicht: 449 g
Buch, Englisch, 320 Seiten, Format (B × H): 156 mm x 234 mm, Gewicht: 449 g
Reihe: Chapman & Hall/CRC Biostatistics Series
ISBN: 978-0-367-50290-4
Verlag: Taylor & Francis Ltd (Sales)
Key Features:
- Reviews critical issues (e.g., endpoint/margin selection, sample size requirements, and complex innovative design).
- Provides better understanding of statistical concepts and methods which may be used in regulatory review and approval.
- Clarifies controversial statistical issues in regulatory review and approval accurately and reliably.
- Makes recommendations to evaluate rare diseases regulatory submissions.
- Proposes innovative study designs and statistical methods for rare diseases drug development, including n-of-1 trial design, adaptive trial design, and master protocols like platform trials.
- Provides insight regarding current regulatory guidance on rare diseases drug development like gene therapy.
Autoren/Hrsg.
Fachgebiete
Weitere Infos & Material
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