Real-world evidence supports drug development

On January 7th, the State Drug Administration issued the Guiding Principles for Real-World Evidence Support drug research and development and review (Trial), which define the relevant definitions of real-world research, the source and applicability of real-world data, the basic design of real-world research, and the evaluation of real-world evidence to guide the evaluation of the effectiveness and safety of drugs using real-world evidence.According to the Guiding Principles, real-world research is the process of collecting data related to the health of the subjects (real-world data) or aggregated data derived from these data in a real-world environment for pre-set clinical problems, and obtaining clinical evidence (real-world evidence) of drug use and potential benefits-risks through analysis. Real-world evidence can be used to support drug regulatory decisions, including providing evidence of effectiveness and safety for the registration and listing of new drugs, providing evidence for changes in the instructions for listed drugs, and providing evidence for post-market requirements or re-evaluation of drugs.The Guiding Principles comb through the potential sources of real-world data in the country, including, but not limited to, health information systems, health insurance systems, disease registration systems, and proactively collected data on patient health in forward-looking research designs, with a view to developing ideas for real-world data to be used in drug development. The Guiding Principles make it clear that the applicability of real-world data is assessed mainly through data relevance and reliability, that the bid should be analyzed on the basis of applicable real-world data to form effective real-world evidence, and that real-world evidence evaluation should follow two main principles, one is whether it can support the clinical questions that need to be answered, and the other is whether the existing real-world data can be scientifically designed, rigorously organized and reasonably analyzed to obtain the required real-world evidence.Linkcontrolled trials are generally considered to be the gold standard for evaluating drug safety and effective, and are widely used in drug clinical studies. But randomized controlled trials have their limitations. Strict entry criteria prevent the trial population from adequately representing the target population, the standard interventions used are not fully consistent with clinical practice, and the limited sample size and short follow-up time lead to inadequate detection of rare adverse events. In addition, traditional randomized controlled trials are difficult to implement in certain disease areas, such as certain rare diseases that lack effective treatment and major life-threatening diseases, and traditional randomized controlled trials often require high time costs.Therefore, how to use real-world evidence to evaluate the effectiveness and safety of drugs in the field of drug development and regulation has become a common and challenging issue for relevant regulatory agencies, the pharmaceutical industry and academia around the world. The United States, the European Union, Japan and other countries and regions have been using real-world data to evaluate the safety of medical products, such as the United States in 2008 launched the sentinel program, the use of existing electronic medical health data to achieve active monitoring of the safety of medical products after the market. (Health Journal)