ABSTRACT
Pharmaceutical regulatory schemes concerned with real world data have been changing remarkably in terms of both drug development and post-marketing.As for drug development, ICH proposed “GCP Renovation” , which includes modernization of E8 Guideline (General Consideration for Clinical Trials) and subsequent renovation of E6 Guideline (Good Clinical Practice). It covers pragmatic clinical trials, randomized controlled trials using patient registry data, and even observational studies using real world data. In the US, “The 21st Century Cures Act” refers to the proposal concerning speeding up the approval review by making clinical trials more efficient. In fact, there are some cases where real world data was used in the approval review of expanding the application. Also, in Japan, Clinical Innovation Network (CIN) plan was announced, and utilizing the natural history data like patient registry as the control group of clinical trials for the approval review is now under consideration.As regards post-marketing surveillance, “Conditional & Accelerated Approval” (October 20, 2017, Yakuseiyakushinhatsu 1020 No. 1) stated that post-marketing confirmation of validity of efficacy and safety using real world data, such as Medical Information Database Network (MID-NET) project and patients registries in CIN plan, is required to get the early approval. Moreover, “The Ordinance on Good Post-Marketing Practice” (October 26, 2017, MHLW Ordinance No. 116) newly provided “Post-marketing Database Study” , which is conducted by use of medical information database like MID-NET, and utilizing real world data for post-marketing safety monitoring began.