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Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease

Approximately 50% of rare diseases are evident in children. Fatal disease prognosis and lack of treatments causes 30% of affected children to not live past their fifth birthday. This clear sense of urgency demands innovation and acceleration in drug development. A case study is discussed highlightin...

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Detalles Bibliográficos
Autores principales: Bhattacharya, I, Manukyan, Z, Chan, P, Harnisch, L, Heatherington, A
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5102572/
https://www.ncbi.nlm.nih.gov/pubmed/27351288
http://dx.doi.org/10.1002/cpt.417
Descripción
Sumario:Approximately 50% of rare diseases are evident in children. Fatal disease prognosis and lack of treatments causes 30% of affected children to not live past their fifth birthday. This clear sense of urgency demands innovation and acceleration in drug development. A case study is discussed highlighting the need for data‐rich phase I study design, extensive use of modeling and simulation, use of diverse data sources, and input from collaborators to respond to this urgent call.