PHP39 The “Weight” of Orphan Drugs in the European Pharmaceutical Policy. A Focus on the Expenditure and the Utilization of Orphan Drugs in Five European Union Countries

One of the most challenging problems for pharmaceutical policy is how to pay for very expensive new drugs for rare diseases, known as “Orphan drugs” (ODs). Aims of this work were to compare the expenditure and utilization of ODs among five European Union Countries (France, Germany, Italy, Spain, UK) and identify the ATC group with the major impact on Italian ODs expenditure and utilization. European database from IMS and the AIFA internal database were consulted for the period 2009-2010 according to the ODs approved by European Medicines Agency. In all 5EU both utilization and expenditure increased in the year 2010 compared to 2009 ranging respectively around 13%-28% for the expenditure and around 7%-17% for the utilization. Italy is the third after Germany (917 Ml €) and France (828 Ml €) for the expenditure and the second after Germany (22 Ml standard units) for utilization. 80% of the ODs authorised by EMA is available in Italy, the remaining 20% is not accessible (marketing reasons). During 2010 Italian expenditure for ODs amounted to 5,5 Ml €, with an increase by 19% compared to 2009. According to the utilization, the value of ODs amounted to 20 Ml standard units, with an increase of 15% compared to 2009. “L” ATC code (antineoplastic) represents the highest expenditure (63% of total ODs) and utilization (60% of total ODs). Within “L” ATC group, Imatinib is the most used molecule (146 Ml € and 8,5 Ml standard units) with an average cost/patient/year of 38,500€ (assuming weight 70kg) ranging from 5,700€ (Anagrelide) and 44,500€ (Nilotinib). Results suggest that ODs expenditure and utilization is rapidly growing up, particularly for some ATC groups. The increase of ODs' utilization is a common challenge among EU countries in order to guarantee a balance between patient access and budget sustainability.