The current status of orphan drug development in Europe and the US

Intractable Rare Dis Res. 2014 Feb;3(1):1-7. doi: 10.5582/irdr.3.1.


Orphan drug legislation has been introduced in a number of countries in order to stimulate the development of treatments for rare diseases by introducing commercial incentives for companies wishing to undertake that development. In order to navigate the maze of regulatory regulations and procedures so that companies can make proper use of the orphan drug incentives, specialist knowledge is required. This article will review the current status of orphan drug development in the EU and the US, explain the incentives and procedures, and touch on the role of patient organisations in the process.

Keywords: Rare diseases; orphan designation; orphan drugs; patient organisations.

Publication types

  • Review