The master protocol platform trial is a trial in which multiple treatments or different combinations of treatments are consistently evaluated in the context of a single disease, possibly within several sub-studies for different disease subtypes, allowing multiple treatments to enter or exit the trial platform based on a certain decision algorithm. Compared with the traditional clinical trial design, the master protocol platform trial as a scientific innovative model of clinical trial can accelerate drug evaluation to deal with the contradiction between the awaited evaluation of numerous drugs and lack of effective therapy for diseases. By using the large-scale randomized controlled clinical trial for COVID-19 inpatients (RECOVERY) led by the University of Oxford in the UK in 2020 as an illustration, this paper introduced the concept, design principle, and basic trial process of the master protocol platform trial to provide methodological references for clinical research of drugs.
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.
Master protocol with adaptive design is a new complex innovative trial design that combines an adaptive treatment strategy and master protocol. It is more flexible and adjustable. In the complex clinical trial environment, the dynamics emphasized in this design are consistent with the idea of traditional Chinese medicine (TCM) syndrome differentiation and treatment. In this study, we summarized its concept, characteristics and advantages, and we also discussed its application in TCM clinical research. We hope this paper can provide more thinking and suggestions for TCM clinical trials.
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.