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Table 2 Number of diseases and medicinal products per category

From: Essential list of medicinal products for rare diseases: recommendations from the IRDiRC Rare Disease Treatment Access Working Group

 

Number

Diseases

Total

134

Metabolic/inborn errors

40

Neurologic

20

Hematologic

24

Inflammatory

23

Endocrine

11

Pulmonary

5

Immunologic

2

Miscellaneous

9

Medicinal products

Total medicinal products (MP)

204

Drugs

125

Biologics

79

MP for metobolic diseases

51

MP for neurologic diseases

34

MP for hematologic diseases

43

MP for inflammatory diseases

28

MP for endocrine diseases

15

MP for pulmonary diseases

22

MP for immunologic diseases

3

MP for miscellaneous

9

MP used to treat multiple diseases

6

MP approved by the FDA

139

MP approved by the EMA

160

MP approved by the NMPA

51

MP on the WHO list of essential medicines (2019)

25

  1. Summary of the total number of listed diseases, the number of diseases per category, the number of total medicinal products, the number of drugs and biologics, the number of medicinal products per disease category, the number of medicinal products treating multiple diseases, and the number of medicinal products approved in each jurisdiction