Observational studies as a tool for introducing innovative oncology approaches into clinical practice: A review

Randomized clinical trials (RCTs) are the “gold standard” of evidence-based medicine. However, it is no less evident that real practice presents many new and important questions that are difficult to answer in the RCT. To what extent does the real patient meet the criteria for inclusion in the study? What are the efficacy and safety of the drug in comorbid and elderly patients? How deviations from the treatment regimen affect safety, etc. The introduction of innovative oncology approaches is disproportionately faster than in other fields. That is why a balanced analysis of the effects of innovation in real-world practice is relevant. Regulatory authorities are aware of this and demonstrate a willingness to consider and actively support real-world evidence research as part of drug applications, which is a powerful incentive for developing this area. The purpose of the article is to analyze the regulatory approaches in the United States and Europe to real-world studies when considering drug applications and to discuss the experience of organizing an observational study of the effectiveness of individual drugs for the treatment of oncological diseases in Moscow as a unique example of interaction between regulatory authorities and the expert community of oncologists.