World health dilemmas: Orphan and rare diseases, orphan drugs and orphan patients

Table 4 Examples of orphan-designated drug products with marketing approvals in the United States for both common and rare disease indication

Drug product name	Orphan indications
Adalimumab	Paediatric Crohn's disease
Aldesleukin	Primary immunodeficiency disease
Allopurinol	Ex vivo preservation of kidneys for transplants
Aminosidine	Tuberculosis and Mycobacterium avium
Azathioprine	Graft-vs-host disease
Aztreonam	Improvement of symptoms in bronchiectasis
Bevacizumab	Ovarian, stomach and pancreatic cancer
Bleomycin sulphate	Pancreatic cancer
Cetuximab	Pancreatic cancer
Cisplatin liposomal	Osteogenic sarcoma metastatic to the lung
Colchicine	Behcet's Syndrome
L-Cycloserine	Gaucher's disease
Cyclosporine	Prophylaxis and treatment of GVH disease
Cyclosporine A implant	Prevention of rejection in cornea transplant
Cyclosporine liposomal	Bronchiolitis obliterans
Doxorubicin	Hepatocellular carcinoma
Doxorubicin HCl liposomal	Soft tissue sarcomas
Doxorubicin nanoparticles	Hepatocellular carcinoma
Eflornithine HCl	Pneumocystis carinii pneumonia in AIDS
Epoetin alpha	Myelodysplastic syndrome
Erlotinib HCl	Malignant gliomas
Etidronate disodium	Degenerative metabolic bone disease
Everolimus	Gastroenteropancreatic tumors
Histrelin	Acute intermittent and other porphyries
Immuneglobulin	Juvenile rheumatoid arthritis
Infliximab	Chronic sarcoidosis
Interferon alfa-2a	Esophageal carcinoma
Peginterferon alfa-2a	Chronic myelogenous leukemia
Interferon alfa-2b	Ovarian carcinoma, brain tumors
Peginterferon alfa-2b	Chronic delta hepatitis
Metronidazole (topical)	Perioral dermatitis
Metronidazole	Pouchitis
N-acetylcysteine	Acute liver failure
Paclitaxel	Pancreatic cancer
Paclitaxel aqueous gel	Esophageal and brain cancer
Paclitaxel micellar	Ovarian cancer
Paclitaxel protein-bound	Stage IIB to IV melanoma
Sorafenib	Stage IIB through stage IV melanoma
Ribavirin	Haemorrhagic fever with renal syndrome
Rituximab	Immune thrombocytopenic purpura
Thiotepa	Haematopoietic stem cell transplantation
Tranexamic acid	Hereditary angioneurotic edema
Urofollitropin	Initiation and re-initiation of spermatogenesis
Ursodiol	Cystic fibrosis liver disease

GVH: Graft versus host. Adapted from ref[43-45].