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Table 1 Classification of drugs examined

From: Points to consider: efficacy and safety evaluations in the clinical development of ultra-orphan drugs

  Number of drugs (%)
  Ultra-orphan drugs n = 43 Orphan drugs other than ultra-orphan drugs n = 88
Therapeutic category
 Metabolic drug 16 (37%) 12 (14%)
 Anticancer drug 11 (26%) 33 (38%)
 Biological drug 8 (19%) 7 (8%)
 Cardiovascular drugs - 10 (11%)
 Central nervous system drugs 1 (2%) 6 (7%)
 Sensory organ agents - 5 (6%)
 Hormones 1 (2%) 4 (4%)
 Peripheral nervous system drugs 1 (2%) 3 (3%)
 Digestive organ agents 1 (2%) 2 (2%)
 Others 4 (10%) 6 (7%)
Drugs with new ingredients
  31 (72%) 50 (57%)
Approved in other countries outside Japan
  36 (84%) 63 (72%)
  1. Classification of therapeutic category, drugs with new ingredients and drugs that had already been approved in countries outside Japan of the 43 ultra-orphan drugs and 88 orphan drugs other than ultra-orphan drugs approved in Japan in the period from January 2001 to December 2014. The classification of drug’s therapeutic category was based on the standard commodity numbers used in Japan