title: A benefit/risk approach towards selecting appropriate pharmaceutical dosage forms - an application for paediatric dosage form selection.
abstract: The design and selection of new pharmaceutical dosage forms involves the careful consideration and balancing of a quality target product profile against technical challenges and development feasibility. Paediatric dosage forms present particular complexity due to the diverse patient population, patient compliance challenges and safety considerations of this vulnerable population. This paper presents a structured framework for assessing the comparative benefits and risks of different pharmaceutical design options against pre-determined criteria relating to (1) efficacy, (2) safety and (3) patient access. This benefit/risk framework has then been applied to three hypothetical, but realistic, scenarios for paediatric dosage forms in order to explore its utility in guiding dosage form design and formulation selection. The approach allows a rigorous, systematic and qualitative assessment of the merits and disadvantages of each dosage form option and helps identify mitigating strategies to modify risk. The application of a weighting and scoring system to the criteria depending on the specific case could further refine the analysis and aid decision-making. In this paper, one case study is scored for illustrative purposes. However, it is acknowledged that in real development scenarios, the generation of actual data considering the very specific situation for the patient/product/developer would come into play to drive decisions on the most appropriate dosage form strategy.
authors.affiliation: Global CMC Regulatory Affairs, Merck Sharp & Dohme, Oss, The Netherlands. tom.sam@merck.com
authors.forename: Tom
authors.initials: T
authors.lastname: Sam
authors.forename: Terry B
authors.initials: TB
authors.lastname: Ernest
authors.forename: Jennifer
authors.initials: J
authors.lastname: Walsh
authors.forename: Julie L
authors.initials: JL
authors.lastname: Williams
mesh_terms: D002648:Child; D003657:Decision Making; D004304:Dosage Forms; D006801:Humans; D004364:Pharmaceutical Preparations / Q000008:administration & dosage*; D018570:Risk Assessment
chemical_list: D004304:Dosage Forms; D004364:Pharmaceutical Preparations