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Table 2 Clinical trials conducted for ultra-orphan drugs and orphan drugs

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

 

RCT (number of drugs)

Dose-response trial (number of drugs)

Extension trials (number of drugs)

Japan

Outside of Japan

Japan

Outside of Japan

Parallel-group trial and traditional 3 + 3 design

Forced titration trial

Optional titration trial

Parallel-group trial and traditional 3 + 3 design

Forced titration trial

Optional titration trial

All ultra-orphan drug

3/43 (7%)

21/43 (49%)

5/43 (12%)

4/43 (3%)

13/43 (30%)

16/43 (37%)

2/43 (5%)

8/43 (19%)

16/43 (37%)

Metabolic drug

1/16 (6%)

11/16 (69%)

1/16 (6%)

1/16 (6%)

6/16 (38%)

7/16 (44%)

1/16 (6%)

4/16 (25%)

10/16 (63%)

Anticancer drug

1/11 (9%)

4/11 (36%)

4/11 (36%)

0/11 (0%)

4/11 (36%)

6/11 (55%)

0/11 (0%)

2/11 (18%)

1/11 (9%)

Biological drug

1/8 (13%)

2/8 (25%)

0/8 (0%)

2/8 (25%)

2/8 (25%)

1/8 (13%)

1/8 (13%)

1/8 (13%)

2/8 (25%)

Others

0/8 (0%)

4/8 (50%)

0/8 (0%)

1/8 (13%)

1/8 (13%)

2/8 (25%)

0/8 (0%)

1/8 (13%)

3/8 (38%)

All orphan drugs other than ultra-orphan drugs

30/88 (34%)

41/88 (47%)

33/88 (38%)

1/88 (1%)

31/88 (35%)

30/88 (34%)

0/88 (0%)

13/88 (15%)

8/88 (9%)

Metabolic drug

8/12 (7%)

3/12 (25%)

5/12 (42%)

0/12 (0%)

7/12 (58%)

3/12 (25%)

0/12 (0%)

3/12 (25%)

2/12 (17%)

Anticancer drug

7/33 (21%)

9/33 (27%)

18/33 (55%)

1/33 (3%)

5/33 (15%)

11/33 (33%)

0/33 (0%)

5/33 (15%)

2/33 (6%)

Biological drug

3/7 (43%)

1/7 (14%)

2/7 (29%)

0/7 (0%)

3/7 (43%)

0/7 (0%)

0/7 (0%)

1/7 (14%)

0/7 (0%)

Others

12/36 (33%)

28/36 (85%)

18/36 (50%)

0/36 (0%)

16/36 (44%)

17/36 (47%)

0/36 (0%)

4/36 (11%)

4/36 (11%)

  1. Classification of clinical trials of the ultra-orphan drugs and orphan drugs other than ultra-orphan drugs for each therapeutic category. Others: drugs other than metabolic drugs, anticancer drugs and biological drugs