In Europe, a disease is defined as rare when it affects fewer than 5 in 10,000 people (Orphan Drug Regulation EC141/2000). It is estimated that between 5,000 and 8,000 distinct rare diseases exist, affecting between 6% and 8% of the population in the course of their lives. In other words, although rare diseases are characterized by low prevalence individually, the total number of people affected by rare diseases in the EU is estimated at between 27 and 36 million. (EUCERD Joint Action – 2014 Report on the State of the Art of Rare Disease Activities)
With a number of European regulations and policies in place in favor of rare disease research and providing incentives for the development and marketing of these innovative drug products, the number of applications has increased steadily each year.
Although the European marketplace presents a significant opportunity for innovative orphan drug companies to meet unmet patient needs, launching your drug product can present a real challenge if you are not familiar with country specific requirements. With its 28 member states, numerous official working languages and complex regulatory framework, this eBook aims to outline the key requirements for a successful EU product launch.