Research activities in traditional Chinese medicine have, to date, focused on the search for relevant active substances and mechanisms of action. This research approach is shaped partly by the conventional drug development model, which commences with determining the mechanism of disease, followed by the design and synthesis of therapeutically active compounds or molecules, animal and in vitro studies, and finally clinical trials in humans. Demonstration of clinical efficacy in humans using randomised controlled trials may be a better starting point for research into traditional Chinese medicine, given that these therapies are already in common use. An efficacy-driven approach could avoid basic research into therapies that are clinically ineffective, thus sparing precious research resources.