ArticleOrphanet journal of rare diseases2009
No difference in between-country variability in use of newly approved orphan and non- orphan medicinal products--a pilot study.
Article in Orphanet journal of rare diseases, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.
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Who cites it
8 citing papers in PubMed.
- Literature Review on Health Emigration in Rare Diseases-A Machine Learning Perspective.International journal of environmental research and public health · 2023Review
- Orphan Drug Use in Patients With Rare Diseases: A Population-Based Cohort Study.Frontiers in pharmacology · 2022Article
- Using 5 consecutive years of NICE guidance to describe the characteristics and influencing factors on the economic evaluation of orphan oncology drugs.Frontiers in public health · 2022Article
- Comparing access to orphan medicinal products in Europe.Orphanet journal of rare diseases · 2019Article
- Determinants of utilisation differences for cancer medicines in Belgium, Scotland and Sweden.The European journal of health economics : HEPAC : health economics in prevention and care · 2017Article
- Why do health technology assessment coverage recommendations for the same drugs differ across settings? Applying a mixed methods framework to systematically compare orphan drug decisions in four European countries.The European journal of health economics : HEPAC : health economics in prevention and care · 2017Article
- Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries.PloS one · 2015Review
- Access to orphan drugs despite poor quality of clinical evidence.British journal of clinical pharmacology · 2011Article
Corrections and comments
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Authors and funding
5 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
backgroundRegulators 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.
methodsWe 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.
resultsNo 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.
conclusionsThe 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.
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