ABSTRACT
In recent years, the use of complementary and alternative medicine including botanical drug product and traditional Chinese (herbal) medicine (TCM) for treating critical and/or life-threatening diseases in humans has received much attention. In pharmaceutical/clinical development of a given TCM, one of the major criticisms is the lack of objectively scientific evidence (documents) of clinical safety and efficacy. Unlike the Western medicines (WM), TCM often consists of multiple components (active ingredients) whose pharmacological activities are often unknown or are not fully characterized or understood. Thus, standard methods for WM clinical trials may not be appropriately applied directly to TCM clinical trials. In this article, some statistical considerations including selection of study design, preparation of matching placebo, development of study endpoint, validation of an instrument, calibration of the validated instrument, and power calculation for sample size estimation are discussed. These considerations have an impact on effectively and scientifically evaluation of clinical safety and efficacy of TCM in clinical trials. In addition, some practical issues regarding testing for consistency in raw materials, stability of drug substance, and animal studies are also discussed.
