Clinical Trial Designs Based on Sequential Conditional Probability Ratio Tests and Reverse Stochastic Curtailing

Biometrics ◽  
1998 ◽  
Vol 54 (2) ◽  
pp. 682 ◽  
Author(s):  
Ming Tan ◽  
Xiaoping Xiong ◽  
Michael H. Kutner
2013 ◽  
Vol 8 (5) ◽  
pp. 437-442 ◽  
Author(s):  
Zoe Moodie ◽  
Holly Janes ◽  
Yunda Huang

2021 ◽  
Vol 16 ◽  
Author(s):  
Erica Winter ◽  
Scott Schliebner

: Characterized by small, highly heterogeneous patient populations, rare disease trials magnify the challenges often encountered in traditional clinical trials. In recent years, there have been increased efforts by stakeholders to improve drug development in rare diseases through novel approaches to clinical trial designs and statistical analyses. We highlight and discuss some of the current and emerging approaches aimed at overcoming challenges in rare disease clinical trials, with a focus on the ultimate stakeholder, the patient.


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