Global spending on orphan drugs in France, Germany, the UK, Italy and Spain during 2007.
BACKGROUND
Orphan drugs are indicated for the treatment of rare diseases which, in the EU, are defined as those with a prevalence of <5 per 10000 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.


OBJECTIVE
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).


METHODS
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.


RESULTS
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 10000 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.


CONCLUSIONS
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.
Orofino
Javier
J
Cellerix, Madrid, Spain.
Soto
Javier
J
Casado
Miguel A
MA
Oyagüez
Itziar
I
D005602:France; D005858:Germany; D006801:Humans; D007558:Italy; D009965:Orphan Drug Production / Q000191:economics*; D011306:Prescription Fees; D035583:Rare Diseases / Q000188:drug therapy*; D013030:Spain; D006113:United Kingdom