Japan’s Initiative for Streamlined Orphan Drug Designation

According to the news article (厚労省・薬事検討会が議論開始  オーファン指定の基準明確化、早期化で「3割増」と試算 | ニュース | ミクスOnline (mixonline.jp)) released by MixOnline on 11th of July, 2023, the Ministry of Health, Labour and Welfare’s “Study Group on the Drug Regulatory System to Enhance Drug Discovery and Ensure Stable Supply” initiated discussions aimed at clarifying the criteria for designating. The committee broadly agreed to clarify the criteria and accelerate the designation of orphan drugs. The MHLW also estimates that clarifying the requirements for the designation would increase the number of orphan-designated drugs by about 30%. On the other hand, it was also proposed that priority review, one of the preferential measures for orphan designation, shouldn’t be applied to items subject to the relaxed requirements until the PMDA’s review system is fully expanded. The study group will summarize ideas by the end of the year or the end of the fiscal year (the end of March). The committee intends to implement those items that can be put into operation before the summary is announced and to make improvements through notices, Q&A, and any other means.