2021 Volume 11 Issue 3 Pages 213-221
There are orphan drug designation systems in Japan, the United States (US) and Europe (EU), but differences in the criteria for the systems. In Japan, although the Japan Agency for Medical Research and Development (AMED) has initiated the support program for orphan drugs prior to designation since 2017, the program is mainly supported by the development expenses, and it is considered that the needs of companies which promote research and development (R & D) in the early stages of development are not always satisfactory. Therefore, the Regulatory Affairs Committee of the Japan Pharmaceutical Manufacturers Association (JPMA) has been requesting the regulatory authorities for the orphan drug designation system in order to create an environment that can further promote the development of orphan drugs in Japan. In addition, a questionnaire survey was conducted in 2020 on the points considered by industries to be problematic in the current operation of the orphan drug designation system. As a result, it was revealed that there were many cases that did not meet the requirements for designation because the efficacy/safety was not shown to be remarkably high in comparison to existing drugs/treatments at the time of consultation for designation and there were several cases that affected the development plans, such as decreasing the priority of development, delaying the development, and discontinuing the development itself because it was not designated. In addition, the timing of designation was later in Japan than in Europe and the United States, and many products were designated in the later stage of development or immediately before the application for approval. An improved system to promote early R & D with earlier and broader orphan drug designation is desirable.
