No difference in between-country variability in use of newly approved orphan and non- orphan medicinal products--a pilot study.
BACKGROUND
Regulators and payers have to strike a balance between the needs of the patient and the optimal allocation of resources. Drugs indicated for rare diseases (orphan medicines) are a special group in this context because of their often high per unit costs. Our objective in this pilot study was to determine, for drugs used in an outpatient setting, how utilisation of centrally authorised drugs varies between countries across a selection of EU member states.


METHODS
We randomly selected five orphan medicines and nine other drugs that were centrally authorised in the European Union between January 2000 and November 2006. We compared utilisation of these drugs in six European Union member states: Austria, Denmark, Finland, Portugal, The Netherlands, and Sweden. Utilisation data were expressed as Defined Daily Doses per 1000 persons per year. Variability in use across countries was determined by calculating the relative standard deviation for the utilisation rates of individual drugs across countries.


RESULTS
No association between orphan medicine status and variability in use across countries was found (P = 0.52). Drugs with an orphan medicine status were more expensive and had a higher innovation score than drugs without an orphan medicine status.


CONCLUSIONS
The results show that the variability in use of orphan medicines in the different health care systems of the European Union appears to be comparable to the other newly authorised drugs that were included in the analysis. This means that, although strong heterogeneity in access may exist, this heterogeneity is not specific for drugs with an orphan status.
Stolk
Pieter
P
Division of Pharmacoepidemiology and Pharmacotherapy, Utrecht Institute for pharmaceutical Sciences, Utrecht University, PO Box 80082, 3508 TB Utrecht, The Netherlands. p.stolk@uu.nl
Heemstra
Harald E
HE
Leufkens
Hubert G M
HG
Bloechl-Daum
Brigitte
B
Heerdink
Eibert R
ER
D001317:Austria; D003718:Denmark; D017277:Drug Approval; D004363:Drug Utilization / Q000706:statistics & numerical data*; D005062:European Union; D005387:Finland; D033161:Government Regulation; D006297:Health Services Accessibility / Q000191:economics / Q000331:legislation & jurisprudence; D006801:Humans; D007880:Legislation, Drug; D009426:Netherlands; D009965:Orphan Drug Production / Q000191:economics / Q000331:legislation & jurisprudence / Q000706:statistics & numerical data; D010865:Pilot Projects; D011174:Portugal; D035583:Rare Diseases / Q000188:drug therapy; D013548:Sweden