リアルワールドデータの薬事規制のもとでの活用にむけて

抄録

The environment for pharmaceutical regulations regarding the real world data (RWD) and the real world evidence (RWE) that can be evidenced from RWD is undergoing major changes in both the development and post–marketing surveillance.

In terms of drug development, there is the modernisation of the E8 guideline (general guidelines for clinical trials) at the ICH and the ongoing revision of the E6 guideline (ICH–GCP), known as the ‘GCP Renovation’. It is said that the scope of this will include pragmatic clinical trials, randomised controlled trials using patient registry data, and even research using RWD. This movement was taken up as an issue of speeding up pharmaceutical approval through streamlining clinical trials, etc., in the ‘21st Century Act’ in the United States, and the use of real–world data in the approval process is also being promoted.

Although randomised controlled trials (RCTs) have traditionally been the most appropriate research design for evaluating treatment efficacy and safety, there is also a focus on applications for approval using RWD in areas where development is not progressing and where RCTs are difficult, where there is no evidence from conventional RCTs. This article will outline the current trends in domestic and international government and what are actually using RWD, as well as the issues surrounding the use of RWD.