Table 1.
Drugs used in the analysis with corresponding indication and prevalence.
| Drug | Indication simplified | Prevalence per 10,000 |
|---|---|---|
| Adcetris | Hodgkin disease | 1.00 |
| Adempas | Pulmonary hypertension | 2.00 |
| Afinitor* | Renal cell carcinoma | 4.20 |
| Aldurazyme* | Mucopolysaccharidosis I | 0.03 |
| Alprolix** | Haemophilia B | 0.20 |
| Arzerra | Chronic lymphocytic leukaemia | 3.50 |
| Atriance | Precursor T-Cell lymphoblastic leukaemia-lymphoma | 1.10 |
| Blincyto | Precursor cell lymphoblastic leukaemia-lymphoma | 1.00 |
| Bosulif | Chronic myeloid leukaemia | 1.60 |
| Bronchitol | Cystic fibrosis | 1.30 |
| Busilvex* | Conditioning treatment prior to haematopoietic-progenitor-cell transplantation | 0.70 |
| Carbaglu | N-acetylglutamate synthetase deficiency | 0.00 |
| Cayston | Gram negative bacterial lung infection in cystic fibrosis | 1.30 |
| Ceplene | Acute myeloid leukaemia | 0.70 |
| Cerdelga | Gaucher disease type 1 | 0.30 |
| Coagadex** | Hereditary factor X deficiency | 0.09 |
| Cometriq | Metastatic medullary thyroid carcinoma | 0.70 |
| Cresemba | Mucormycosis | 0.06 |
| Cyramza* | Stomach neoplasms (Gastric cancer) | 3.00 |
| Cystadane | Homocystinuria | 0.17 |
| Dacogen | Acute myeloid leukaemia | 1.00 |
| Darzalex** | Plasma cell myeloma | 1.75 |
| Defitelio** | Severe hepatic veno-occlusive disease | 0.40 |
| Deltyba | Tuberculosis | 2.00 |
| Diacomit | Severe myoclonic epilepsy in infancy | 0.40 |
| Elaprase | Mucopolysaccharidosis II | 0.02 |
| Esbriet | Idiopathic pulmonary fibrosis | 3.00 |
| Evoltra | Precursor cell lymphoblastic leukaemia-lymphoma | 0.40 |
| Exjade | Iron overload | 2.70 |
| Fabrazyme* | Fabry disease | 0.03 |
| Farydak | Multiple myeloma | 3.20 |
| Firazyr | Hereditary angioedemas | 2.50 |
| Firdapse | Lambert-Eaton myasthenic syndrome | 0.10 |
| Galafold** | Fabry disease | 1.00 |
| Gazyvaro | Chronic lymphocytic leukaemia (follicular lymphoma) | 2.40 |
| Gliolan | Malignant glioma | 3.70 |
| Glivec* | Chronic myeloid leukaemia | 0.90 |
| Glybera | Familial lipoprotein lipase deficiency | 0.02 |
| Granupas | Tuberculosis | 2.00 |
| Hetlioz** | Non-24-hour sleep-wake disorder | 1.85 |
| Holoclar** | Limbal stem cell deficiency | 0.30 |
| Iclusig | Chronic myeloid leukaemia | 0.80 |
| Idelvion | Haemophilia B | 0.10 |
| Ilaris* | Cryopirin associated syndromes | 0.05 |
| Imbruvica | Mantle cell lymphoma | 0.17 |
| Imnovid | Multiple myeloma | 2.20 |
| Increlex | Laron syndrome | 2.00 |
| Inovelon | Epilepsy | 1.50 |
| Jakavi* | Chronic idiopathic myelofibrosis | 0.50 |
| Kalydeco | Cystic fibrosis | 1.20 |
| Kanuma | Lysosomal acid lipase deficiency | 0.20 |
| Ketoconazole HRA | Endogenous Cushing’s syndrome | 0.90 |
| Kolbam | Inborn errors in primary bile acid synthesis | 0.07 |
| Kuvan | Phenylketonurias | 1.70 |
| Kyprolis | Multiple myeloma | 1.30 |
| Lenvima | Differentiated thyroid carcinoma | 0.60 |
| Litak* | Hairy cell leukaemia | 2.40 |
| Lynparza | Ovarian cancer | 2.90 |
| Lysodren* | Adrenal cortex neoplasms | 0.10 |
| Mepact | Osteosarcoma | 0.50 |
| Mozobil | Hematopoietic stem cell transplantation for multiple myeloma, lymphoma | 1.00 |
| Myozyme | Glycogen storage disease type II | 0.14 |
| Naglazyme* | Mucopolysaccharidosis VI | 0.02 |
| Nexavar | Renal cell carcinoma | 3.01 |
| Nexobrid | Deep partial- and full-thickness thermal burns | 1.00 |
| Nplate | Thrombocytopenic idiopathic purpura | 1.00 |
| Ofev | Idiopathic pulmonary fibrosis | 3.00 |
| Opsumit | Pulmonary arterial hypertension | 1.80 |
| Orfadin* | Tyrosinemias | 0.10 |
| Orphacol | Digestive system diseases, inborn errors of metabolism | 0.06 |
| Pedea* | Patent ductus arteriosus | 2.13 |
| Peyona | Primary apnoea | 0.85 |
| Photobarr* | Barrett oesophagus | 3.60 |
| Plenadren | Adrenal insufficiency | 4.50 |
| Prialt* | Pain injections, spinal | 1.54 |
| Procysbi | Nephropathic cystinosis | 0.10 |
| Raxone | Leber’s hereditary optic neuropathy | 1.00 |
| Ravicti** | Carbamoyl-phosphate synthase-1 deficiency | 0.14 |
| Replagal* | Fabry disease | 0.02 |
| Revatio* | Pulmonary hypertension | 1.00 |
| Revestive | Short-bowel syndrome | 0.20 |
| Revlimid | Multiple myeloma | 1.30 |
| Revolade* | Idiopathic thrombocytopenic purpura | 2.50 |
| Savene | Extravasation of diagnostic and therapeutic materials | 0.03 |
| Scenesse** | Erythropoietic protoporphyria | 0.19 |
| Signifor | Cushing’s disease | 1.20 |
| Siklos | Sickle cell anaemia | 0.50 |
| Sirturo | Pulmonary multidrug resistant tuberculosis | 2.00 |
| Soliris | Paroxysmal nocturnal haemoglobinuria | 0.10 |
| Somavert | Acromegaly | 0.60 |
| Sprycel | Chronic myelogenous leukaemia, BCR-ABL positive | 0.89 |
| Strensiq | Hypophosphatasia | 0.10 |
| Strimvelis | Adenosine deaminase deficiency | 0.02 |
| Sutent* | Malignant gastrointestinal stromal tumours | 0.30 |
| Sylvant | Multicentric Castlemans disease who are human immunodeficiency virus (HIV) negative and human herpesvirus-8 (HHV-8) negative | 0.99 |
| Tasigna | Chronic myelogenous leukaemia, BCR-ABL positive | 1.00 |
| Tepadina | Conditioning treatment prior to allogeneic or autologous haematopoietic progenitor cell transplantation (HPCT) in haematological diseases | 0.60 |
| Thalidomide-Celgene | Multiple myeloma | 1.20 |
| Thelin | Pulmonary arterial hypertension | 0.50 |
| Tobi Podhaler | Pseudomonas aeruginosa lung infection in cystic fibrosis | 1.30 |
| Torisel | Renal cell carcinoma | 3.50 |
| Tracleer* | Pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension | 0.95 |
| Translarna** | Duchenne muscular dystrophy | 0.30 |
| Trisenox* | Acute promyelocytic leukaemia | 0.80 |
| Unituxin** | Neuroblastoma | 1.10 |
| Uptravi | Pulmonary arterial hypertension | 1.80 |
| Ventavis* | Primary pulmonary hypertension | 2.20 |
| Vidaza | Myelodysplastic syndromes | 2.05 |
| Vimizim | Mucopolysaccharidosis, type IVA (Morquio A syndrome) | 1.40 |
| Volibris | Pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension | 2.00 |
| Votubia | Renal angiomyolipoma with tuberous sclerosis complex | 1.00 |
| VPRIV | Gaucher disease | 0.30 |
| Vyndaqel | Transthyretin amyloidosis | 2.90 |
| Wakix** | Narcolepsy | 5.00 |
| Wilzin* | Hepatolenticular degeneration | 0.60 |
| Xagrid | Essential thrombocythaemia | 2.50 |
| Xaluprine | Acute lymphoblastic leukaemia | 1.20 |
| Xyrem* | Narcolepsy | 5.00 |
| Yondelis | Sarcoma | 0.61 |
| Zavesca | Gaucher disease | 0.60 |
*Orphan designation withdrawn or expired.
** No prices available in the countries in scope; probably not commercially available.