Japan Drafts New Program to Enlarge its Orphan Drug Designation Program

On July 10, the Japanese MHLW outlined a plan to increase the number of products allowed to apply for Orphan Drug Designation (ODD) and permit this designation earlier in the drug development process.  Currently, Japan only accepts orphan drugs that are for “state-designated diseases” with under 50,000 patients.  However, the new proposal wants to include other drugs if they are subsets of existing diseases where there are unmet needs.  Also, as part of the definition of orphan drugs, the MHLW states they are drugs where no other therapy is available – but this language is very vague.  The PMDA can oftentimes find an alternative drug for a specific disease although its efficacy may not be as good as the new orphan drug under consideration.  Given this, the PMDA is looking for side-by-side comparisons of the new drug and the alternative therapy via a clinical trial or appropriate medical literature.

In addition, the new orphan drug plan suggests an earlier orphan drug designation process. This can be done if there is already a plan for a clinical trial in place (but not carried out) and non-clinical studies have been completed.   Finally, the plan suggests that orphan drugs receive designation after the Pharmaceutical Affairs and Food Sanitation Council’s review, instead of also waiting for MHLW review,  which occurs less frequently.  Once these orphan drug designation details are finalized, they should be released before year-end.

Written by: Ames Gross – President and Founder, Pacific Bridge Medical (PBM)
Mr. Gross founded PBM in 1988 and has helped hundreds of medical companies with regulatory and business development issues in Asia. He is recognized nationally and internationally as a leader in the Asian medical markets. Mr. Gross has a BA degree, Phi Beta Kappa, from the University of Pennsylvania and an MBA from Columbia University.