Japan has always been a good market for orphan drugs. The Japanese organizations that are involved in the process include the MHLW, PMDA and the National Institute of Biomedical Innovation (NIBIO). Orphan drugs must meet the following requirements – 1. less than 50,000 patients, 2. there is no applicable substitute drug and the drug has high efficacy, and 3. there is a development plan for the drug and the disease.
Japan provides a large number of incentives for orphan drugs in Japan. First, via NIBIO the orphan drug can receive funding to assist in the drug development in Japan. Second, orphan drug applicants can receive guidance from the 3 Japanese organizations discussed above. Third, the PMDA will provide a priority review for orphan drugs. Fourth, applicants can receive tax advantages to reduce development costs in Japan.
Recently, Japan has initiated some new policies to further aid Orphan drug development in Japan. For example, Japan now has more flexibility with respect to the 50,000 patient hurdle. If the drug addresses a specific subset of a disease and those patients are less than 50,000, the drug can still be designated as an orphan. To receive orphan designation as a sub-set of a disease, there must be strong evidence and pharmacological data for that subset.
In addition, a recent notification further addresses the notion of medical need. Instead of just being better than existing drugs or where there is no alternative, if the new drug is very innovative it will be possible to receive orphan status. Also, whereas the old definition said if an alternative drug is not available, which can be vague since almost all drugs have alternatives, the PMDA will now compare the new drug with existing drugs through a side-by-side comparison, clinical studies, research papers, etc. If the new drug shows superiority, it can also be designated as orphan.
To expedite the orphan drug designation (ODD) process, the PMDA will now review the drug for an ODD once non-clinical studies have been done, and the drug is about to do a clinical study – but before it has started the human clinical process. Finally, instead of waiting for the MHLW evaluation, orphan drug designation can now be granted earlier after only the Pharmaceutical Affairs and Food Sanitation review.
These and other orphan drug details should make Japan an even more attractive market than before.
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.