Challenges to Efficiently Developing Drugs and Solving Unmet Needs Using RWD

Abstract

The application of Real-World Data (RWD) can be broadly divided into two categories: those areas where the formal principles of drug development can be applied and those where they are difficult to apply, such as in the case of orphan diseases. We will discuss the latter topic in this article. In Japan, the recognition of the possibility of using RWD in pharmaceutical applications has increased in recent years. This is largely due to the efforts of Dr. Takeda’s Special Research of Health Labour Sciences Research Grant in 2015 on the use of patient registries for drug development, which was intensified under the Clinical Innovation Network initiative of the Ministry of Health, Labour and Welfare (MHLW). The MHLW’s notifications on the usage of registries in 2021 have further solidified their place in the industry. RWD utilization extends beyond just serving as the external control group in single arm clinical trials. The possibility of diversification of the composition policy of the data package and greater involvement from pharmacoepidemiologists in the development and approval review may change in the near future. These events align with previous cases of regulatory approval reviews in Japan and globally, and they are not limited to mere theoretical possibility. It is crucial to shift the discussion to addressing RWD in terms of developing and evaluating drugs in areas that tend to be neglected from therapeutic development, rather than exploiting RWD, using examples of drugs that cannot be developed with current principle. It is essential to consider measures to resolve unmet needs.