On April 1, 2015, Japan’s Ministry of Health, Labor and Welfare (MHLW) notified stakeholders of an updated criterion for orphan drug designation. This change will likely apply to the orphan medical device designation system as well.
Drugs that treat “designated intractable diseases” are now eligible for orphan drug designation. This is now defined as diseases that affect approximately 0.1% of the Japanese population — and the government has clarified that a Japanese patient population as large as 180,000 will be considered eligible. Prior to this change, orphan drug designation was only allowed for drugs that treated diseases affecting 50,000 or fewer Japanese patients.
The change means that a variety of treatments will now qualify for orphan drug designation in Japan. This includes ulcerative colitis, which affects approximately 144,000 Japanese, and Parkinson’s disease, which affects 110,000 Japanese.
Aside from the change to the patient population criterion, other requirements for orphan drug designation remain the same. Japan offers a variety of incentives for companies researching and developing orphan drugs, such as preferential tax treatment, subsidy payments for drugs, and priority review for marketing authorization.