Case of Utilization of Registry Data in Drug Development (Additional Indication for Lung Transplantation of Tacrolimus in the US)

Abstract

The utilization of real-world data (RWD)/real-world evidence (RWE) in drug development is useful in designing and planning clinical trials (standard of care, competitor research, inclusion/exclusion criteria, etc.); feasibility studies for clinical trials; approval applications, including external control groups and expanded indications; and for revision of package inserts. Especially in pediatric drug development, intractable diseases, and rare diseases, there are high expectations for utilization, but including these highly expected utilizations, utilization for external control groups, etc., has not progressed due to the issues of data appropriateness and reliability. Under these circumstances, we used registry data from the Scientific Registry of Transplant Recipients (SRTR) in the United States as the main efficacy and safety data, and an additional indication for lung transplantation was approved for Tacrolimus in July 2021 in the United States. Unlike clinical trial data, the reality is that registry data includes inappropriate data from the data reliability perspective. Of course, if these inappropriate data are important data for evaluating the efficacy and safety of the drug, there is a high possibility that the data will become unusable and, as a result, affect the drug evaluation. On the other hand, the SRTR data that we have used for the application at this time had no major issues with the efficacy and safety data and the data reliability, so the U. S. Food and Drug Administration determined that the data quality was with in acceptable limits. It is important for companies to actively and flexibly consider how to utilize RWD/RWE based on an appropriate understanding of the actual situation of RWD/RWE and the appropriateness and reliability of the data to be utilized.