Cost-effectiveness analysis of microdose clinical trials in drug development

Drug Metab Pharmacokinet. 2013;28(3):187-95. doi: 10.2133/dmpk.dmpk-12-rg-044. Epub 2012 Sep 11.


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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Chromatography, Liquid / methods
  • Clinical Trials as Topic / economics*
  • Clinical Trials, Phase I as Topic / economics
  • Cost-Benefit Analysis
  • Drug Discovery / economics*
  • Humans
  • Pharmaceutical Preparations / administration & dosage*
  • Pharmaceutical Preparations / analysis
  • Pharmaceutical Preparations / blood
  • Positron-Emission Tomography / methods
  • Tandem Mass Spectrometry / methods


  • Pharmaceutical Preparations