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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