Japan Further Loosens Drug Regulations with Respect to Rare Diseases and Conditional Approval

As mentioned in our newsletter throughout the last year, Japan is trying to reduce their drug lag – where many innovative Western drugs have still not been registered in Japan. On October 23, one new notification and one revised notification were promulgated. For the new notification, rare disease drug applications can now be submitted without clinical trial data on Japanese patients if three benchmarks occur. First, overseas data that supports the drug’s evaluation has already been completed, and the drug has been approved overseas on that basis. Second, if the number of Japanese patients is very small, and doing a trial in Japan would be extremely difficult. And third, if the benefit to the Japanese patient is considered higher than the risk of not providing the medication. However, if the drug will affect Japanese patients differently due to ethnic characteristics, the MHLW may still prefer some local Japanese trials.

In addition, on the same day, the MHLW revised its regulation on conditional approvals.  If delaying the conditional drug approval will lead to fast-progressing diseases or the death of the patient, then local clinical studies may not be needed.  Also, if confirmatory trials to make sure that the efficacy and safety of the drug are done after conditional approval, the types of patients do not need to exactly match the scope of the conditional approval.   In other words, there is more flexibility with respect to the patients and their disease progression, etc. Finally, Japanese patients may not be required to be involved for conditional approval, since overseas clinical trials already in progress may suffice.

Written by: Ames Gross – President and Founder, Pacific Bridge Medical (PBM)