scholarly journals A Bayesian adaptive design for clinical trials in rare diseases

2017 ◽  
Vol 113 ◽  
pp. 136-153 ◽  
Author(s):  
S. Faye Williamson ◽  
Peter Jacko ◽  
Sofía S. Villar ◽  
Thomas Jaki
Keyword(s):  
Clinical Trials ◽  
Rare Diseases ◽  
Adaptive Design ◽  
Bayesian Adaptive Design

A Bayesian adaptive design for two-stage clinical trials with survival data

2009 ◽  
Vol 15 (4) ◽  
pp. 468-492 ◽  
Author(s):  
Uttam Bandyopadhyay ◽  
Atanu Biswas ◽  
Rahul Bhattacharya
Keyword(s):  
Clinical Trials ◽  
Survival Data ◽  
Adaptive Design ◽  
Two Stage ◽  
Bayesian Adaptive Design

scholarly journals A Bayesian Adaptive Design for Combination of Three Drugs in Cancer Phase I Clinical Trials

2016 ◽  
Vol 6 (1) ◽  
pp. 1-11 ◽  
Author(s):  
Mourad Tighiouart ◽  
Quanlin Li ◽  
Steven Piantadosi ◽  
Andre Rogatko
Keyword(s):  
Clinical Trials ◽  
Phase I ◽  
Adaptive Design ◽  
Phase I Clinical Trials ◽  
Bayesian Adaptive Design

Bayesian adaptive design for clinical trials in Duchenne muscular dystrophy

2021 ◽  
Author(s):  
Stephen L. Lake ◽  
Melanie A. Quintana ◽  
Kristine Broglio ◽  
Jennifer Panagoulias ◽  
Scott M. Berry ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Duchenne Muscular Dystrophy ◽  
Muscular Dystrophy ◽  
Adaptive Design ◽  
Bayesian Adaptive Design

scholarly journals Participant-funded clinical trials on rare diseases

2020 ◽  
Vol 93 (4) ◽  
pp. 267.e1-267.e9
Author(s):  
Rafael Dal-Ré ◽  
Francesc Palau ◽  
Encarna Guillén-Navarro ◽  
Carmen Ayuso
Keyword(s):  
Clinical Trials ◽  
Rare Diseases

Current Advances in Clinical Trials for Rare Disease Populations: Spotlight on the Patient

2021 ◽  
Vol 16 ◽  
Author(s):  
Erica Winter ◽  
Scott Schliebner
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Drug Development ◽  
Rare Disease ◽  
Rare Diseases ◽  
Statistical Analyses ◽  
Clinical Trial Designs ◽  
Novel Approaches

: 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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