Application of Bayesian methods to accelerate rare disease drug development: scopes and hurdles

Application of Bayesian methods to accelerate rare disease drug development: scopes and hurdles

Abstract Background Design and analysis of clinical trials for rare and ultra-rare disease pose unique challenges to the practitioners. Meeting conventional power requirements is infeasible for diseases where sample sizes are inherently very small. Moreover, rare disease populations are generally he...

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Bibliographic Details
Main Authors: Kelley M. Kidwell, Satrajit Roychoudhury, Barbara Wendelberger, John Scott, Tara Moroz, Shaoming Yin, Madhurima Majumder, John Zhong, Raymond A. Huml, Veronica Miller
Format: Article
Language:English
Published: BMC 2022-05-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Small sample
Clinical trial
Prior distribution
External control
Meta-analytic predictive approach
SMART
Online Access:https://doi.org/10.1186/s13023-022-02342-5

Internet

https://doi.org/10.1186/s13023-022-02342-5

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