Market uptake of orphan drugs--a European analysis.
OBJECTIVE
Variations in market uptake of an orphan drug have important implications with respect to access to care and inequality of treatment. Therefore, the aim of this study was to quantify both the sales and volume uptake of orphan drugs in Europe and to assess whether a country's gross domestic product (GDP) and/or health technology assessment (HTA) influences the orphan drugs' market uptake.


METHODS
We analysed the numbers of orphan drugs launched and the sales and volume uptake for 17 orphan drugs in 23 European countries from 2001 until the beginning of 2010 using the IMS Health database. Countries were clustered based on GDP and the availability of a formal HTA-organization.


CONCLUSIONS
The uptake of orphan drugs varied across European countries. The highest volumes and contributions of orphan drugs in the first year occurred in countries with a high GDP (and implicitly, a higher budget for healthcare), independently of the existence of an HTA-organization. In contrast, in countries with a low GDP, orphan drugs were less available when there was a formal HTA-organization. There, budgetary restrictions can cause the exclusion of less cost-effective orphan drugs.


CONCLUSIONS
We observed substantial variation in the market uptake of orphan drugs. Such variation may have important implications with respect to access to care and inequality of treatment. The uptake of orphan drugs could be promoted through the clinical added value of orphan drugs (CAVOD) project and various conditional pricing and reimbursement mechanisms.
Research Centre for Pharmaceutical Care and Pharmaco-economics, KU Leuven, Leuven, Belgium.
E
E
Picavet
L
L
Annemans
I
I
Cleemput
D
D
Cassiman
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S
Simoens
D002017:Budgets; D016000:Cluster Analysis; D003132:Commerce / Q000706:statistics & numerical data*; D003362:Cost-Benefit Analysis; D005062:European Union; D059066:Gross Domestic Product / Q000706:statistics & numerical data*; D006297:Health Services Accessibility / Q000191:economics / Q000706:statistics & numerical data; D054625:Healthcare Disparities / Q000191:economics / Q000706:statistics & numerical data; D006801:Humans; D009965:Orphan Drug Production / Q000191:economics / Q000706:statistics & numerical data*; D012051:Reimbursement Mechanisms; D013673:Technology Assessment, Biomedical / Q000458:organization & administration*