Orphan Drug Designation
To be designated by theMinister of Health, Labor and Welfare, all the followingcriteria must be metaccording to the Article 77-2 of the Pharmaceutical Affairs Law.
[Reference: Quotefrom Notification from Director of Pharmaceutical Affairs Bureau,PABNotification No. 725 dated on August 25, 1993]
1) Treatment of adisease affecting fewer than 50,000 patients in Japan
2) High medicalneeds (no adequate alternative medicines and treatment, orsignificantlyimproved efficacy or safety expected compared to the conventionalproducts)
3) High probabilityof successful development (a strong rationale to use the productand an adequatedevelopment plan)
Rules for the patients for an orphan drug designation wererevised last year, and itwas clarified how to calculate the number of patients in terms of use of the drug by50,000 or lesspatients. If the estimate of users is fewer than 50,000 patients at thepoint of applying for designation, the following new medicinal products can bedesignated as orphan drugs.
[Reference: Quotefrom Notification from Director of Evaluation and LicensingDivision,Pharmaceutical and Food Safety Bureau, ELD NotificationNo.0331007 dated on March 31, 2006]
(1) Vaccine toprevent an infectious disease rarely found in Japan or occurring onlyoverseas,which can affect a limited group of population in Japan, for example,travelersto the epidemic area.
(2) Vaccine toprevent an infectious disease not yet found at the point of applicationbut withthe possibility of a new occurrence or reoccurrence as a result ofgenemutation, for example, to significantly affect life and health of Japanese populations once it occurs, and when it will occur or how epidemic it will beis unknown.
To obtain an orphandrug designation, a sponsor company should submit apredetermined applicationform and an attachment to demonstrate that the newproduct will meet the abovestated criteria to theEvaluationandLicensingDivision,Pharmaceutical and Food Safety Bureau. After application, the Minister of Health,Labor and Welfare may designate it as an orphan drug after hearing opinionsfrom thePharmaceutical Affairs and Food Sanitation Council.
Status of Orphan Drug Designation and Development DOWNROAD