Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease. Issue 4 (22nd August 2016)

• Record Type: Journal Article
• Authors: Bhattacharya, I; Manukyan, Z; Chan, P; Harnisch, L; Heatherington, A
• Is Part Of: Clinical pharmacology & therapeutics. Volume 100:Issue 4(2016:Oct.)
• Page Start: 330
• Page End: 332
• Publication Date: 2016-08-22
• Subjects: Pharmacology -- Periodicals; Therapeutics -- Periodicals; 615.5
• DOI: 10.1002/cpt.417
• Languages: English
• ISSNs: 0009-9236

Abstract

Abstract : 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.