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 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.