Objective Biotechnology has promoted the discovery and development of new types of therapeutical agents for use in humans: biotech drugs offer innovative, targeted therapies with enormous potential to address unmet medical needs of patients with cancer, AIDS and other serious diseases. However, the therapeutic application of these novel therapies poses serious problems concerning the connection between cost sustainability and innovative value. The aims of the present study are to assess the level of therapeutic innovation of biotech drugs approved by the European Medicines Agency between 2004 and 2011, to make a comparison with the trend of biotech drugs approved between 1995–2003, as previously reported, and to evaluate their economic impact on the Italian healthcare system.

Methods The data source used was the European Public Assessment Report (EPAR) of human medicines available on European Medicines Agency (EMA) website. The scores for therapeutic innovation were assigned according to the algorithm created by Motola et al. Drug expenditure data was obtained from Information Management System–Health Italy database. The list of drugs under analysis was downloaded from European Medicines Agency website and information on approved drugs were retrieved from the European Public Assessment Reports as well as from PubMed databank.

Results From 2004 to 2011, the European Medicines Agency approved 47 biotech drugs: 43 biopharmaceutical innovators and 4 vaccines. Our analysis involved 33 of the 47 biotech drugs approved: 18 products resulted in important therapeutic innovations, 6 in moderate and 5 in modest therapeutic innovations, 2 in pharmacological innovations and finally, 2 involved only technological innovations. We also evaluated the influence of biotech drugs and their different scores for innovation with regard to expenditure as well as consumption. In 2010 and in 2011, the major part of expenditure and consumption concerned biotech drugs classified as important therapeutic innovations, while moderate, modest, pharmacological and technological innovators revealed very reduced contributions in this regard.

Conclusions Our study revealed that 50% of biotech drugs approved between 2004–2011 represented an important or moderate therapeutic innovation. The remaining biotech drugs did not add any extra value in the decision-making process concerning the application of economic strategies in the healthcare field. These considerations suggest decision-makers should optimise application of health technology assessment strategies aimed at evaluating new technologies and sustainable costs in clinical practice.