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Innovative designs and analyses for small population clinical trials = development strategies and operational engagement for pediatric and rare diseases /

Record Type:	Electronic resources : Monograph/item
Title/Author:	Innovative designs and analyses for small population clinical trials/ by Jingjing Ye, Lei Nie.
Reminder of title:	development strategies and operational engagement for pediatric and rare diseases /
Author:	Ye, Jingjing.
other author:	Nie, Lei.
Published:	Cham :Springer Nature Switzerland : : 2024.,
Description:	xix, 473 p. :ill. (chiefly color), digital ;24 cm.
[NT 15003449]:	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.
Contained By:	Springer Nature eBook
Subject:	Clinical trials. -
Online resource:	https://doi.org/10.1007/978-3-031-60843-8
ISBN:	9783031608438

Innovative designs and analyses for small population clinical trials = development strategies and operational engagement for pediatric and rare diseases /
Ye, Jingjing.

Innovative designs and analyses for small population clinical trialsdevelopment strategies and operational engagement for pediatric and rare diseases /[electronic resource] :by Jingjing Ye, Lei Nie. - Cham :Springer Nature Switzerland :2024. - xix, 473 p. :ill. (chiefly color), digital ;24 cm. - ICSA book series in statistics,2199-0999. - ICSA book series in statistics..

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.

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 investigators, pharmaceutical companies, global regulatory agencies, and customers. The book also highlights the considerations of natural history and patient registry, diagnostic criteria determination, identification of clinically meaningful endpoints, and global regulatory guidance. The authors detail the nuances of clinical trial design as specific to small population disease, drawing from real examples.

ISBN: 9783031608438

Standard No.: 10.1007/978-3-031-60843-8doiSubjects--Topical Terms:

724498
Clinical trials.

LC Class. No.: RM301.27

Dewey Class. No.: 615.10724

Innovative designs and analyses for small population clinical trials = development strategies and operational engagement for pediatric and rare diseases /
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505 0 $a 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.
520 $a 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 investigators, pharmaceutical companies, global regulatory agencies, and customers. The book also highlights the considerations of natural history and patient registry, diagnostic criteria determination, identification of clinically meaningful endpoints, and global regulatory guidance. The authors detail the nuances of clinical trial design as specific to small population disease, drawing from real examples.
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