Sample size determination for a specific region in a multiregional trial
Journal
Journal of Biopharmaceutical Statistics
Journal Volume
20
Journal Issue
4
Pages
870-885
Date Issued
2010
Author(s)
Abstract
Recently, geotherapeutics have attracted much attention from sponsors as well as regulatory authorities. A bridging study defined by the International Conference on Harmonisation (ICH) E5 is usually conducted in the new region after the test product has been approved for commercial marketing in the original region due to its proven efficacy and safety. However, extensive duplication of clinical evaluation in the new region not only requires valuable development resources but also delays availability of the test product to the needed patients in the new regions. To shorten the drug lag or the time lag for approval, simultaneous drug development, submission, and approval in the world may be desirable. On September 28, 2007, the Ministry of Health, Labour and Welfare (MHLW) in Japan published the Basic Principles on Global Clinical Trials guidance related to the planning and implementation of global clinical studies. The 11th question and answer for the ICH E5 guideline also discuss the concept of a multiregional trial. Both guidelines have established a framework on how to demonstrate the efficacy of a drug in all participating regions while also evaluating the possibility of applying the overall trial results to each region by conducting a multiregional trial. In this paper, we focus on a specific region and establish statistical criteria for consistency between the region of interest and overall results. More specifically, four criteria are considered. Two criteria are to assess whether the treatment effect in the region of interest is as large as that of the other regions or of the regions overall, while the other two criteria are to assess the consistency of the treatment effect of the specific region with other regions or the regions overall. Sample size required for the region of interest can also be evaluated based on these four criteria. Copyright ? Taylor & Francis Group, LLC.
Subjects
Bridging Study; Consistent Trend; Multiregional Trial
SDGs
Other Subjects
drug; low density lipoprotein cholesterol; article; atherosclerosis; clinical study; coronary artery disease; drug approval; drug efficacy; drug safety; geographic distribution; health care planning; human; hypercholesterolemia; mathematical computing; placebo effect; practice guideline; priority journal; probability; sample size; statistical model; theoretical model; Algorithms; Anticholesteremic Agents; Atherosclerosis; Ethnic Groups; Humans; Hypercholesterolemia; Internationality; Models, Statistical; Multicenter Studies as Topic; Probability; Randomized Controlled Trials as Topic; Sample Size
Type
journal article