In order to facilitate the development of new drugs, Japan’s Ministry of Health, Labor, and Welfare (MHLW) is seeking an increased budget for its Orphan Drug Designation program. In order to obtain this designation in Japan, orphan drugs need to fulfill certain criteria, such as the number of patients with the targeted disease must be fewer than 50,000 patients. Compared to the US and EU, the number of products granted orphan drug designation is significantly smaller in Japan. This is because the US and EU health authorities designate a broader scope of products as orphan drugs, and the required level of efficacy and safety for these designated products is lower than that required in Japan.
As Japanese industry officials advocate for the issuance of the orphan drug status for a broader scope of products and designation in earlier stages of drug development, the MHLW will request additional funding from the Ministry of Finance in an effort to increase resources at the Pharmaceuticals and Medical Devices Agency (PMDA). A company with a drug that receives an Orphan Drug Designation may obtain R&D funding from the National Institutes of Biomedical Innovation, Health, and Nutrition — as well as other benefits such as priority review eligibility and extension of the data protection period in Japan.