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Innovative Designs and Analyses for Small Population Clinical Trials Development Strategies and Operational Engagement for Pediatric and Rare Diseases /

This book discusses a multidisciplinary and multi-stakeholder collaborative approach to small population clinical trials. It provides and illustrates a systematic framework that depends upon a collaborative infrastructure between the patient community, patient advocacy groups, researchers and invest...

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Bibliographic Details
Main Authors: Ye, Jingjing (Author), Nie, Lei (Author)
Corporate Author: SpringerLink (Online service)
Format: Electronic eBook
Language:English
Published: Cham : Springer Nature Switzerland : Imprint: Springer, 2024.
Edition:1st ed. 2024.
Series:ICSA Book Series in Statistics,
Subjects:
Biometry.
Clinical medicine > Research.
Statistics .
Artificial intelligence > Data processing.
Machine learning.
Biostatistics.
Clinical Research.
Statistics in Engineering, Physics, Computer Science, Chemistry and Earth Sciences.
Applied Statistics.
Data Science.
Machine Learning.
Online Access:https://doi.org/10.1007/978-3-031-60843-8
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Table of Contents:
  • 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.

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