Abstract
Pharmaceutical companies incorporate different features into the trials for new drug applications (NDAs) to render them efficient, making use of their experience. The objective of this analysis was to examine the associations between outcome and features related to study design and clinical development experience in commercially sponsored clinical trials.We collected data of phase 2 and phase 3 trials of all the drugs that obtained approval for depression, schizophrenia, asthma, hypertension, and diabetes in Japan from 1970 to 2011. In total, 145 trials from 90 test drugs were eligible for our study. We calculated the effect size, the standard mean of differences between test drug and comparator therapeutic effects, as the objective variable for use in our analysis. A linear mixed effect model with nested and crossed random effects was used in the analysis including variety of therapeutic area, test drugs and clinical trials.The analysis showed that trial features including sample size, subjective endpoints and active comparator of the same mode of action were negatively associated with effect size. In addition, sponsors’ domestic clinical development experience with similar drugs seemed to have a positive association, but prior development experience in foreign countries did not. The accumulation of skills and knowledge within sponsors, and accumulated experience in domestic professionals who implement clinical trials under study contracts with sponsors would be of great importance for yielding clear outcomes.This study provides additional evidence with respect to possible sizes and directions of the influence of study design features that must be considered when planning and implementing trials for new drug applications, and when retrospectively comparing outcomes from trials with different designs and environments.Electronic supplementary materialThe online version of this article (doi:10.1186/2193-1801-3-740) contains supplementary material, which is available to authorized users.
Highlights
It has been suggested that recent stagnation in drug development is related to both a decrease in the probability of success, and an increase in the costs of research and development (Pammolli et al 2011)
Data were extracted from common technical documents posted on the Japanese regulatory agency site, from the interview forms, which are unique to Japan, from pharmaceutical company’s websites, and from publications on clinical trials conducted for New drug application (NDA) (Additional file 1: Table S1)
Distribution of effect size In total, 145 trials from 90 test drugs were eligible for inclusion in our study
Summary
It has been suggested that recent stagnation in drug development is related to both a decrease in the probability of success, and an increase in the costs of research and development (Pammolli et al 2011). Pharmaceutical companies are usually interested in generating evidence to support clinical efficacy and safety at the minimum cost and burden, which often depends on their experience, and their choice of strategy. In this global development era, global companies can choose the region in which they initiate clinical development and submit NDAs first, after assessing the likelihood of success, costs, and future profits. Previous studies have not sufficiently investigated such aspects of experience
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