title: Making Every Subject Count: A Case Study of Drug Development Path for Medication in a Pediatric Rare Disease.
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.
authors.affiliation: Quantitative Clinical Sciences, Pharmatherapeutics, Pfizer Inc, Cambridge, Massachusetts, USA. neil.bhattacharya@pfizer.com.
authors.forename: I
authors.initials: I
authors.lastname: Bhattacharya
authors.affiliation: Quantitative Clinical Sciences, Pharmatherapeutics, Pfizer Inc, Cambridge, Massachusetts, USA.
authors.forename: Z
authors.initials: Z
authors.lastname: Manukyan
authors.affiliation: Pharmacometrics, Pfizer Inc, Sandwich, United Kingdom.
authors.forename: P
authors.initials: P
authors.lastname: Chan
authors.affiliation: Pharmacometrics, Pfizer Inc, Sandwich, United Kingdom.
authors.forename: L
authors.initials: L
authors.lastname: Harnisch
authors.affiliation: Quantitative Clinical Sciences, Pharmatherapeutics, Pfizer Inc, Cambridge, Massachusetts, USA.
authors.forename: A
authors.initials: A
authors.lastname: Heatherington
mesh_terms: D002648:Child; D017321:Clinical Trials, Phase I as Topic / Q000379:methods*; D003198:Computer Simulation; D055808:Drug Discovery / Q000379:methods*; D006801:Humans; D000070516:Intersectoral Collaboration; D008954:Models, Biological; D035583:Rare Diseases / Q000188:drug therapy*; D012107:Research Design