Applied Health Economics and Health Policy

, Volume 8, Issue 5, pp 301–315 | Cite as

Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007

  • Javier Orofino
  • Javier Soto
  • Miguel A. CasadoEmail author
  • Itziar Oyagüez
Original Research Article



Orphan drugs are indicated for the treatment of rare diseases which, in the EU, are defined as those with a prevalence of <5 per 10 000 inhabitants. Characteristically, these diseases negatively affect health-related quality of life and may be life threatening. The EU has passed legislation to encourage pharmaceutical companies to invest in research programmes into rare diseases, with the aim of developing new, safe and effective orphan drugs.


To describe the status of orphan drugs in five countries in the EU (France, Germany, the UK, Italy and Spain), estimate the mean annual cost per patient and indication of these orphan drugs, and determine the associated cost of these drugs in comparison with overall spending on drugs in each country (year 2007 values).


The analysis was limited solely to costs of orphan drugs with sales data available for 2007. The mean annual cost per patient was estimated using recommended regimens for maintenance dose and duration from the summary of product characteristics. Likewise, the ratio between annual costs per patient for treatment of each disease and its prevalence was calculated. Sales data were available for at least one of the countries studied for 38 of the 44 orphan drugs authorized by the European Medicines Agency. Only 21 products had data available for all five countries studied.


Germany was the country with access to the largest number of orphan drugs (36), followed by the UK (34), Spain (28), France (27) and Italy (25). The mean annual cost per patient and indication of the 38 orphan drugs on the market ranged widely from €331 to €337 501. It appears that orphan drugs indicated to treat diseases with a prevalence of <2 per 10 000 inhabitants have higher annual per-patient costs than those indicated to treat diseases with a higher prevalence. The percentage of total drug spending accounted for by orphan drugs in 2007 was 1.7% in France, 2.1% in Germany, 1.0% in the UK, 1.5% in Italy and 2.0% in Spain, with an average overall percentage of 1.7% for these five countries.


In 2007, spending on orphan drugs in five European countries was acceptable in terms of the percentage of these countries’ overall drug expenditure. Mean annual costs per patient of orphan drugs varied widely, with costs being related to the prevalence of the disease for which the product is indicated.


Sunitinib Rare Disease Annual Cost European Medicine Agency Bosentan 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.



The authors thank Mark Riviere, Guy Rousseau and Frommer Vincent for helping us to review the manuscript. No sources of funding were used to conduct this study or prepare this manuscript. Javier Soto is employed by Pfizer Spain, which commercializes some orphan drugs in the Spanish market. The other authors have no conflicts of interest that are directly relevant to the content of this study.

Supplementary material

40258_2012_8050301_MOESM1_ESM.pdf (179 kb)
Supplementary material, approximately 183 KB.


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Copyright information

© Adis Data Information BV 2010

Authors and Affiliations

  • Javier Orofino
    • 1
  • Javier Soto
    • 2
  • Miguel A. Casado
    • 3
    Email author
  • Itziar Oyagüez
    • 3
  1. 1.CellerixMadridSpain
  2. 2.Pfizer Medical UnitMadridSpain
  3. 3.Pharmacoeconomics & Outcomes Research IberiaMadridSpain

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