title: The use of clinical utility assessments in early clinical development.
abstract: A quickly realizable benefit of model-based drug development is in reducing uncertainty in risk/benefit, comprising individually of safety and effectiveness, two key attributes of a product evaluated for regulatory approval, marketing, and use. In this review, we investigate gaps and opportunities in using fundamental decision analytic principles in drug development and present a quantitative clinical pharmacology framework for the application of such aids for early clinical development decision making. We anticipate that implementation of such emerging tools will enable sufficient scientific understanding of the two attributes to facilitate the early termination of compounds with less than desirable risk/benefit profiles and continuance of compounds with acceptable risk/benefit profiles.
authors.affiliation: Quantitative Clinical Pharmacology, Department of Clinical Pharmacology, Merck Research Laboratories, Merck & Co., Inc., Whitehouse Station, New Jersey, USA.
authors.forename: Anis A
authors.initials: AA
authors.lastname: Khan
authors.forename: Itay
authors.initials: I
authors.lastname: Perlstein
authors.forename: Rajesh
authors.initials: R
authors.lastname: Krishna
mesh_terms: D000465:Algorithms; D003198:Computer Simulation; D003661:Decision Support Techniques; D055808:Drug Discovery / Q000379:methods*; D008962:Models, Theoretical; D011336:Probability; D011364:Professional Practice; D018570:Risk Assessment / Q000379:methods*