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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