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:
Perspectives
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
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author Bhattacharya, I
Manukyan, Z
Chan, P
Harnisch, L
Heatherington, A
author_facet Bhattacharya, I
Manukyan, Z
Chan, P
Harnisch, L
Heatherington, A
author_sort Bhattacharya, I
collection PubMed
description 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.
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spelling pubmed-51025722016-11-14 Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease Bhattacharya, I Manukyan, Z Chan, P Harnisch, L Heatherington, A Clin Pharmacol Ther Perspectives 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. John Wiley and Sons Inc. 2016-08-22 2016-10 /pmc/articles/PMC5102572/ /pubmed/27351288 http://dx.doi.org/10.1002/cpt.417 Text en © 2016 The Authors. Clinical Pharmacology & Therapeutics published by Wiley Periodicals, Inc. on behalf of The American Society for Clinical Pharmacology and Therapeutics This is an open access article under the terms of the Creative Commons Attribution‐NonCommercial‐NoDerivs (http://creativecommons.org/licenses/by-nc-nd/4.0/) License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non‐commercial and no modifications or adaptations are made.
spellingShingle Perspectives
Bhattacharya, I
Manukyan, Z
Chan, P
Harnisch, L
Heatherington, A
Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease
title Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease
title_full Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease
title_fullStr Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease
title_full_unstemmed Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease
title_short Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease
title_sort making every subject count: a case study of drug development path for medication in a pediatric rare disease
topic Perspectives
url 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
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