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.
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.
Authors: Michael F Drummond; David A Wilson; Panos Kanavos; Peter Ubel; Joan Rovira Journal: Int J Technol Assess Health Care Date: 2007 Impact factor: 2.188
Authors: Francesca Gorini; Michele Santoro; Anna Pierini; Lorena Mezzasalma; Silvia Baldacci; Elena Bargagli; Alessandra Boncristiano; Maurizia Rossana Brunetto; Paolo Cameli; Francesco Cappelli; Giancarlo Castaman; Barbara Coco; Maria Alice Donati; Renzo Guerrini; Silvia Linari; Vittoria Murro; Iacopo Olivotto; Paola Parronchi; Francesca Pochiero; Oliviero Rossi; Barbara Scappini; Andrea Sodi; Alessandro Maria Vannucchi; Alessio Coi Journal: Front Pharmacol Date: 2022-05-16 Impact factor: 5.988