Pragmatic randomized controlled trials can provide high-quality evidence. However, pragmatic trials need to frequently encounter the missing outcome data due to the challenges of quality assurance and control. The missing outcome could lead to bias which may misguide the conclusions. Thus, it is crucial to handle the missing outcome data appropriately. Our study initially summarized the bias structures and missingness mechanisms, and then reviewed important methods based on the assumption of missing at random. We referred to the multiple imputations and inverse probability of censoring weighting for dealing with missing outcomes. This paper aimed to provide insights on how to choose the statistical methods on missing outcome data.
ObjectiveTo examine statistical performance of different rare-event meta-analyses methods.MethodsUsing Monte-Carlo simulation, we set a variety of scenarios to evaluate the performance of various rare-event meta-analysis methods. The performance measures included absolute percentage error, root mean square error and interval coverage.ResultsAcross different scenarios, the absolute percentage error and root mean square error were similar for Bayesian logistic regression model, generalized mixed linear effects model and continuity correction, but the interval coverage was higher with Bayesian logistic regression model. The statistical performances with Mantel-Haenszel method and Peto method were consistently suboptimal across different scenarios.ConclusionsBayesian logistic regression model may be recommended as a preferred approach for rare-event meta-analysis.
In 2019, the national government issued the document "Implementation Plan for Supporting the Construction of the Boao Lecheng International Medical Tourism Pilot Area", which allowed the use of innovative drugs and medical devices in medical institution of Boao Lecheng. These medical products had been designed to meet urgent clinical requirements and had been approved by regulatory authorities overseas. Through the use of these medical products, real-world data were generated in the routine clinical practice, based on which real-world evidence might be produced for regulatory decision-making by using scientific and rigorous methods. In March 2020, the first medical device product using domestic real-world data was approved, suggesting that the real-world data initiative in Boao Lecheng achieved initial success. This work also provided important experience for promoting the practice of medical device regulatory decision-making based on real-world evidence in China. Here, we shared the preliminary experiences from the study on the first approved medical device product and discussed the issues on developing a real-world data research framework in Boao Lecheng in attempt to offer insights for future studies.
In recent years, the concept of population medicine has emerged as a research field that has important implications for healthcare practice and policy decision-making. It specifically aims to improve overall health of patient populations and safety, quality and efficiency of healthcare system. This paper descried the background, definition and characteristics of population medicine, discussed relationship between population medicine and population health and evidence-based medicine. It also introduced Department of Population Medicine at Harvard Medical School as a world-class model in the field of population medicine, discussed the needs and potential strategies for developing population medicine research in China, and briefly outlined the current development of population medicine in China.