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Table 2 ATC Categories of orphan drugs marketed in the U.S., EU, Japan and China

From: The availability and affordability of orphan drugs for rare diseases in China

ATC Category

Orphan drugs of China/U.S.

Orphan drugs of China/EU

Orphan drugs of China/Japan

Orphan drugs of China/U.S. + EU + Japanb

N

%

N

%

N

%

N

%

A-Alimentary tract and metabolism

6/42

14.3

1/12

8.3

6/19

31.6

10/43

23.3

B-Blood and blood forming organs

7/40

17.5

1/3

33.3

4/11

36.4

9/31

29.0

C-Cardiovascular system

6/9

66.7

1/5

20.0

4/7

57.1

6/13

46.2

D-Dermatologicals

1/4

25.0

0/0

-

0/0

-

1/4

25

G-Genito-urinary system and sex hormones

8/10

80.0

0/1

0

1/2

50

7/10

70

H- Systemic hormonal preparations, excluding sex hormones and insulins

15/36

41.6

1/3

33.3

3/7

42.9

9/19

47.3

J- Anti-infectives for systemic use

12/32

37.5

0/2

0

28/43

65.1

32/58

55.2

La- Antineoplastic and immunomodulating agents

53/136

39.0

10/28

35.7

40/66

60.6

55/124

44.4

 L1 for blood cancers

20/45

44.4

6/13

46.2

12/18

66.7

23/45

51.1

 L2 for other cancers

23/43

53.5

4/9

44.4

6/12

50.0

16/35

45.7

M- Musculoskeletal system

5/12

41.7

0/0

-

2/7

28.6

6/12

50.0

N- Nervous system

9/31

29.0

1/6

16.7

3/11

27.3

10/33

30.3

P- Antiparasitic products, insecticides and repellents

4/17

23.5

0/0

-

2/3

66.7

5/12

41.7

R- Respiratory system

2/10

20

0/2

0

1/3

33.3

2/9

22.2

S- Sensory organs

2/8

25

0/0

-

1/4

25

5/12

41.7

V- Various

9/34

26.5

1/3

66.7

1/2

50

8/28

28.6

TOTAL

139/421c

33.0

16/65

24.6

96/185

52.4

165/408

40.4

Coefficient of Variation(C.V.)

-

53.7

-

84.5

-

33.8

-

35.1

  1. N: Number. La: includes L1 and L2 drugs. b: Number of unique orphan drugs approved for marketing in the U.S., EU and Japan. c: Excluding withdrawals of designations and approvals of four orphan drugs