ABSTRACT
This chapter starts off with a concise overview of orphan drug development. It is generally acknowledged that the introduction of specific orphan drug legislation in various jurisdictions across the globe has made, and is still making, an enormous difference in the lives of millions of rare disease patients. The chapter is written with a small start-up company with an interest in orphan drug development in mind as the main target readership, providing a sound mix of the necessary theoretical and helpful practical information. The chapter is also of interest to representatives of venture capital firms, patient organisations and even more mature SMEs stepping into the orphan drug arena. To benefit from the incentives, a sponsor has to apply and obtain an orphan designation for its product from the Food and Drug Administration (FDA) or European Medicines Agency (EMA)/European Commission in the US and the EU, respectively.
