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.
This paper explores the methodological characteristics and key considerations of umbrella trials. By allocating different treatment strategies based on patients' molecular features, umbrella trials significantly enhance screening efficiency and can quickly identify ineffective therapies. Through the analysis of patient allocation strategies, statistical model selection, and error control methods, we can better utilize this design to accelerate drug development and achieve more efficient personalized treatment. However, despite significant progress in methodology and practice, umbrella trials still face multiple challenges during implementation, including trial design, sample size calculation, patient recruitment, informed consent, and resource allocation. Addressing these challenges in the future will help further optimize the application of umbrella trials. This study aims to provide thoughts and inspirations for researchers conducting umbrella trials and promote the steady development of this field.