By the 1990’s the European Union had fallen behind other developed economies in the encouragement and support its pharmaceutical regulatory framework offered to the development of new treatments for rare diseases. The aim of the Orphan Regulation was to remedy this. We examine critically the interplay of the legal instruments underpinning the scheme of orphan incentives with the judgments of the Court of Justice and conclude that the definitions and tests embodied by the Orphan Regulation, which decide which products should be rewarded, have served patients with rare diseases well.