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1. BACKGROUND

The pharmaceutical market is highly regulated with regulations as important factors that impact therapeutic research and innovation on several levels. Innovators must proof safety, clinical efficacy and cost-effectiveness of their products before and after obtaining marketing authorisation. Pharmaceutical firms are among the top investors in research and development (R&D) and their innovative success has greatly expanded the treatment arsenal over the last century. However, despite the biotech revolution and advances in genomics, the sector continues to have attrition rates and lengthy product development, translating into higher R&D costs and drug prices. Indeed, pharmaceutical spending tends to increase at a faster pace than total health spending or GDP in OECD countries while numerous unmet medical needs still exist. A number of important policy measures seek to promote drug innovation. These include those intended to encourage university technology transfer (the Bayh-Dole Act in the United States in 1980 and Allegre’s law in France in 1999), to reward innovation using market incentives (the 1984 Hatch-Waxman Act in the US), and to reduce regulatory costs (the foundation of the European Medicine Agency (EMA) in 1995). One policy common to both the US and the European Union is Orphan Drug legislation (Kesselheim AS,(2011).

Rare disorders is the name of diseases, with varied aetiology and low-prevalence for the majority of which there is no treatment available. They are frequently life-threatening or chronically debilitating with significant impact on quality of life. About 5000 identified diseases are classed …