As a novel research model that can address multiple research questions within an overall trial structure, master protocol design shares similarities with the clinical research on syndrome-based traditional Chinese medicine in terms of study design. The sample size estimation in master protocol design is characterized by analyzing the subtrials separately and re-estimation at interim analyses. Specific methods include the combination of Simon’s two-stage design and Bayesian hierarchical design that facilitates information borrowing. By drawing on these methods to estimate dynamically and adjust the sample size for each subtrial in a targeted manner, it is expected to provide a feasible approach for the methodological development of sample size estimation in the field of clinical research on syndrome-based traditional Chinese medicine.
Traditional Chinese medicine (TCM) emphasizes syndrome differentiation and treatment, whereas commonly used evidence-based efficacy evaluation approaches are not aligned with TCM’s individualized diagnostic and therapeutic paradigm. “Average-based” assessments that ignore individual differences not only cannot accurately reflect the true efficacy of TCM interventions but also fail to guide TCM clinical practice. Accordingly, this review systematically summarizes commonly used methods for capturing the heterogeneity of treatment effect and proposes their potential applications in TCM clinical research, aiming to provide efficacy evaluation approaches that better accord with TCM’s diagnostic and therapeutic characteristics and, in turn, to furnish high-quality quantitative evidence for TCM clinical practice.
