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

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 heterogene...

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Detalles Bibliográficos
Autores principales: Kidwell, Kelley M., Roychoudhury, Satrajit, Wendelberger, Barbara, Scott, John, Moroz, Tara, Yin, Shaoming, Majumder, Madhurima, Zhong, John, Huml, Raymond A., Miller, Veronica
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9077995/
https://www.ncbi.nlm.nih.gov/pubmed/35526036
http://dx.doi.org/10.1186/s13023-022-02342-5

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