Introduction to the Drug Loss Issue
According to news (厚労省 ドラッグ・ロス86品目で「医療上必要性特に高い」医薬品14品目 類薬あり至急の開発不要品目も | ニュース | ミクスOnline) released by MIX Online on April 1, 2025, Japan’s Ministry of Health, Labour and Welfare (MHLW) has completed an evaluation of 86 overseas-approved drugs that remain unapproved in Japan—a problem often referred to as “drug loss.” The review identified 14 drugs as having “particularly high medical necessity.” This initiative is part of the 2024 MHLW Special Research Program aimed at resolving delays in the availability of foreign-approved drugs in Japan.
Breakdown of Drug Categories
The 86 drugs were categorized into four groups based on their necessity:
- Group A (Particularly High Medical Necessity): 14 drugs
- Group B (High Medical Necessity): 41 drugs
- Group C (Low Medical Necessity): 11 drugs
- Group D (No Medical Necessity): 12 drugs
Eight of the 14 Group A drugs are expected to require urgent action, while others are either already in development in Japan or have therapeutic alternatives under development.
Examples of Group A Drugs
Some notable drugs identified as having high medical necessity include:
- Pretomanid (for drug-resistant tuberculosis)
- OmegaVen (for parenteral nutrition-associated cholestasis)
- Artesunate (for severe malaria)
- Avapritinib (for GIST with specific genetic mutations)
- Trifarotene (for acne vulgaris)
- Lefamulin (for community-acquired pneumonia)
- Omadacycline (for bacterial infections)
- Obiltoxaximab (for inhalational anthrax)
Policy Direction and Next Steps
The MHLW will present these 14 drugs to the Expert Committee for Unapproved and Off-label Drugs with High Medical Need. If the committee confirms their priority status, MHLW will request domestic pharmaceutical companies to initiate development. Additionally, 41 drugs in Group B will be evaluated progressively under a new research project funded by the 2024 supplementary budget, to accelerate approval where necessary.
Challenges and Pending Cases
One example of a pending case is brexanolone (for postpartum depression), which remains unevaluated due to delayed feedback from medical societies. This highlights the need for closer coordination between clinical stakeholders and regulatory authorities.
Conclusion
Japan’s structured review and prioritization of unapproved drugs underscore its commitment to improving patient access to essential treatments. By identifying high-need drugs and initiating regulatory pathways for their development, the MHLW is addressing a long-standing issue in Japan’s pharmaceutical landscape—while also fostering collaboration between global innovators and local developers.