Precision medicine is a personalized medical system based on patients' individual biological information, clinical symptoms and signs, forming a new clinical research model and medical practice path. The basic idea of traditional Chinese medicine and the concept of precision medicine share many similarities. The basket trial developed for precision medicine is also suitable for clinical trials and evaluation of the efficacy of traditional Chinese medicine syndrome differentiation and treatment systems. Basket trials are used to evaluate the efficacy of a drug in the treatment of multiple diseases or disease subtypes. It has the advantages of sharing a master protocol, unifying management of subsidiary studies, simplifying the test implementation process, unifying statistical analysis, saving resources, reducing budgets and accelerating the drug evaluation progress. This is similar to the concept of using the "same treatment for different diseases" found in traditional Chinese medicine. This paper introduced the concept and method of basket trials and explored their application and advantages in clinical research into traditional Chinese medicine. This study is expected to provide references for the methodological innovation of clinical research into traditional Chinese medicine.
ObjectiveTo introduce economic evaluation methods for anticancer-drugs with basket trial design, and to provide references for related research and decision-making. MethodsA case analysis was conducted on economic evaluation methods for anticancer-drugs with basket trial design, which was issued by Canadian Agency for Drugs and Technologies in Health (CADTH) in the Economic Guidance Report. Moreover, both the advantages and disadvantages of the methods were analyzed in accordance with the characteristics of basket trials. ResultsPooled analysis and tumor-specific analysis were two methods frequently employed in the case analysis. However, great uncertainties were available in both of them. The uncertainty of the former was mainly reflected in the heterogeneity of the targeted population, while the uncertainty of the latter was mainly shown in the insufficient sample size of the subgroup. ConclusionCurrently, economic evaluation methods for anticancer-drugs with basket trial design are immature. Thus, researchers are required to explore the methods of innovation evaluation with lower uncertainty; reimbursement decision-makers should fully consider the uncertainty of evaluation results and enterprises should collect the real-world data for the demands of evaluation to promote the reasonable allocation of healthcare resources in China.
Precision medicine is an individualized clinical research model established according to gene, environment, lifestyle and other information. As an innovative method of clinical trials, the main scheme design breaks the barriers of traditional randomized controlled trials to the evaluation of targeted therapies in precision medicine and improves the efficiency of clinical research. This paper will systematically introduce the types, concepts and principles of the main scheme design of the new method of precision medicine clinical trial design, and summarize the advantages and limitations of the main scheme design combined with classic cases, aiming at providing scientific and rigorous methodological guidance and clinical practice experience for precision medicine scientific research design.