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Bayesian modeling and simulation to inform rare disease drug development early decision-making: Application to Duchenne muscular dystrophy
Rare disease clinical trials are constrained to small sample sizes and may lack placebo-control, leading to challenges in drug development. This paper proposes a Bayesian model-based framework for early go/no-go decision making in rare disease drug development, using Duchenne muscular dystrophy (DMD...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
Public Library of Science
2022
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9049549/ https://www.ncbi.nlm.nih.gov/pubmed/35482633 http://dx.doi.org/10.1371/journal.pone.0247286 |