東華大學圖書館 |

語系: 繁體中文

說明(常見問題)

回圖書館首頁

手機版館藏查詢

登入

回首頁

切換: 標籤 | MARC模式 | ISBD

FindBook

Google Book

Amazon

博客來

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

紀錄類型:	書目-電子資源 : Monograph/item
正題名/作者:	Innovative designs and analyses for small population clinical trials/ by Jingjing Ye, Lei Nie.
其他題名:	development strategies and operational engagement for pediatric and rare diseases /
作者:	Ye, Jingjing.
其他作者:	Nie, Lei.
出版者:	Cham :Springer Nature Switzerland : : 2024.,
面頁冊數:	xix, 473 p. :ill. (chiefly color), digital ;24 cm.
內容註:	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
標題:	Clinical trials. -
電子資源:	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 /
LDR:02364nmm a2200337 a 4500 001 2381106
003 DE-He213
005 20241120115510.0
006 m d
007 cr nn 008maaau
008 250418s2024 sz s 0 eng d
020 $a 9783031608438 $q (electronic bk.)
020 $a 9783031608421 $q (paper)
024 7 $a 10.1007/978-3-031-60843-8 $2 doi
035 $a 978-3-031-60843-8
040 $a GP $c GP
041 0 $a eng
050 4 $a RM301.27
072 7 $a PBT $2 bicssc
072 7 $a MED090000 $2 bisacsh
072 7 $a PBT $2 thema
082 0 4 $a 615.10724 $2 23
090 $a RM301.27 $b .Y37 2024
100 1 $a Ye, Jingjing. $3 3735030
245 1 0 $a Innovative designs and analyses for small population clinical trials $h [electronic resource] : $b development strategies and operational engagement for pediatric and rare diseases / $c by Jingjing Ye, Lei Nie.
260 $a Cham : $b Springer Nature Switzerland : $b Imprint: Springer, $c 2024.
300 $a xix, 473 p. : $b ill. (chiefly color), digital ; $c 24 cm.
490 1 $a ICSA book series in statistics, $x 2199-0999
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.
650 0 $a Clinical trials. $3 724498
650 0 $a Drugs $x Testing. $3 646233
650 0 $a Pediatric pharmacology $x Research. $3 3735032
650 0 $a Rare diseases $x Treatment $x Research. $3 3735033
650 1 4 $a Biostatistics. $3 1002712
650 2 4 $a Clinical Research. $3 3531315
650 2 4 $a Statistics in Engineering, Physics, Computer Science, Chemistry and Earth Sciences. $3 3591853
650 2 4 $a Applied Statistics. $3 3300946
650 2 4 $a Data Science. $3 3538937
650 2 4 $a Machine Learning. $3 3382522
700 1 $a Nie, Lei. $3 3735031
710 2 $a SpringerLink (Online service) $3 836513
773 0 $t Springer Nature eBook
830 0 $a ICSA book series in statistics. $3 2153476
856 4 0 $u https://doi.org/10.1007/978-3-031-60843-8
950 $a Mathematics and Statistics (SpringerNature-11649)