Regular Article
Cost-effectiveness Analysis of Microdose Clinical Trials in Drug Development

https://doi.org/10.2133/dmpk.DMPK-12-RG-044Get rights and content

Summary:

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.

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    This work was performed as part of “Innovative Strategies for Drug Development Using Microdosing Clinical Studies” financed by the New Energy and Industrial Technology Development Organization (NEDO).

    Conflict of interest (COI): Naoe Yamane is an employee of JCL Bioassay Corporation, Hyogo, Japan. The other authors declare no conflict of interest. The survey respondents are researchers who have expertise in microdosing in pharmaceutical industries, universities, contract research organizations and clinical facilities.

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    Copyright © 2013 The Japanese Society for the Study of Xenobiotics. Published by Elsevier Ltd. All rights reserved.