Rare Diseases, Orphan Drugs, and Orphaned Patients

Table 1.

Numbers of Orphan Drugs Approved by the FDA for Orphan Diseases

Disease Number Approved
Data adapted from Haffner, M. N. Engl. J. Med. ©2006
Cancers 38
Metabolic Diseases 37
Infectious Diseases 23
Neurological Diseases 18
Hematological Diseases 16
Pulmonary Diseases 9
Poisonings 9

This Article

  1. MI August 2006 vol. 6 no. 4 186-191