title: Cost-effectiveness analysis of microdose clinical trials in drug development.
abstract: Microdose (MD) clinical trials have been introduced to obtain human pharmacokinetic data early in drug development. Here we assessed the cost-effectiveness of microdose integrated drug development in a hypothetical model, as there was no such quantitative research that weighed the additional effectiveness against the additional time and/or cost. First, we calculated the cost and effectiveness (i.e., success rate) of 3 types of MD integrated drug development strategies: liquid chromatography-tandem mass spectrometry, accelerator mass spectrometry, and positron emission tomography. Then, we analyzed the cost-effectiveness of 9 hypothetical scenarios where 100 drug candidates entering into a non-clinical toxicity study were selected by different methods as the conventional scenario without MD. In the base-case, where 70 drug candidates were selected without MD and 30 selected evenly by one of the three MD methods, incremental cost-effectiveness ratio per one additional drug approved was JPY 12.7 billion (US$ 0.159 billion), whereas the average cost-effectiveness ratio of the conventional strategy was JPY 24.4 billion, which we set as a threshold. Integrating MD in the conventional drug development was cost-effective in this model. This quantitative analytical model which allows various modifications according to each company's conditions, would be helpful for guiding decisions early in clinical development.
authors.affiliation: Pharmaceutical Regulatory Science, Graduate School of Pharmaceutical Sciences, The University of Tokyo, Tokyo, Japan.
authors.forename: Naoe
authors.initials: N
authors.lastname: Yamane
authors.forename: Ataru
authors.initials: A
authors.lastname: Igarashi
authors.forename: Makiko
authors.initials: M
authors.lastname: Kusama
authors.forename: Kazuya
authors.initials: K
authors.lastname: Maeda
authors.forename: Toshihiko
authors.initials: T
authors.lastname: Ikeda
authors.forename: Yuichi
authors.initials: Y
authors.lastname: Sugiyama
mesh_terms: D002853:Chromatography, Liquid / Q000379:methods; D002986:Clinical Trials as Topic / Q000191:economics*; D017321:Clinical Trials, Phase I as Topic / Q000191:economics; D003362:Cost-Benefit Analysis; D055808:Drug Discovery / Q000191:economics*; D006801:Humans; D004364:Pharmaceutical Preparations / Q000008:administration & dosage* / Q000032:analysis / Q000097:blood; D049268:Positron-Emission Tomography / Q000379:methods; D053719:Tandem Mass Spectrometry / Q000379:methods
chemical_list: D004364:Pharmaceutical Preparations