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Table 2 Overview of the drugs included in the study

From: No difference in between-country variability in use of newly approved orphan and non- orphan medicinal products - a pilot study

Active substance Market authorisation date Indication DDD Innovation score Cost per DDD ranking RSD for utilisation Number of countries with > 0 utilisation
With orphan status        
imatinib 11 Nov 2001 Chronic myeloid leukaemia, acute lymphoblastic leukaemia, myelodysplastic/myeloproliferative diseases, advanced hypereosinophilic syndrome, chronic eosinophilic leukaemia, metastatic malignant gastrointestinal stromal tumours, dermatofibrosarcoma protuberans 400 mgb 12 12 7.2% 5
bosentan 15 May 2002 Pulmonary arterial hypertension 0.25 g 6c 14 37.1% 6
zinc acetate dehydrate 13 Oct 2004 Wilson's disease. 0.15 g 15 6 44.9% 4
nitisinone 21 Feb 2005 Hereditary tyrosinemia type 1 20 mg 15c 13 112.1% 4
sodium oxybate 13 Oct 2005 Cataplexy in narcolepsy patients. 7.5 g 10c 11 127.6% 3
Without orphan status        
levetiracetam 29 Sep 2000 Epilepsy 1.5 g 8 5 29.1% 6
telmisartan/hydrochlorothiazide 19 Apr 2002 Essential hypertension. 65 mga 9c 2 110.0% 4
emtricitabine 24 Oct 2003 HIV-1 infected adults and children in combination with other antiretroviral agents. 0.2 g 9c 7 142.5% 5
adefovir dipivoxil 6 Mar 2003 Chronic Hepatitis B 10 mg 12 9 89.2% 5
oxybutinin 26 Feb 2004 Symptoms of urge incontinence and/or increased urinary frequency. 15 mg 2 3 90.5% 6
pregabalin 6 Jul 2004 Peripheral and central neuropathic pain 0.3 g 10c 4 68.9% 6
efalizumab 20 Sep 2004 Moderate to severe chronic plaque psoriasis 10 mg 8c 10 53.5% 5
abacavir/lamuvidine 17 Dec 2004 Combination therapy for Human Immunodeficiency Virus (HIV). 0.9 ga 9c 8 109.9% 5
desloratidine 21 Sep 2000 Allergic rhinitis and chronic idiopathic urticaria 5 mg 3 1 41.1% 6
  1. a Combination preparation, DDD determined according to WHO guidelines. b DDD determined by authors based on literature, not WHO-CC. c Innovativeness score determined by authors based on algorithm by Motola et al.
  2. RSD: Relative Standard Deviation, DDD: Defined Daily Dose