Orphan drug designation – Europe, the USA and Japan

Abstract

Patients with rare diseases deserve the same quality, safety and efficacy in medicines as other patients with more common conditions. However, for rare diseases, the cost of developing and bringing a drug to market would not normally be recovered by expected sales. In order to encourage development and research, a supportive legislative and regulatory framework known as orphan drug designation has been adopted for medicines for rare diseases in a number of countries and regions. Although similarities exist, the criteria and processes for designation are not internationally harmonised and this editorial summarises orphan drug designation in Europe, the USA and Japan.

Keywords::

• Europe
• Japan
• orphan drug designation
• rare disease
• USA

Acknowledgments

The author would like to thank Maria Mavris (EURORDIS – European Organisation for Rare Diseases) for comments on the manuscript during preparation. The views expressed are those of D J O'Connor and do not necessarily reflect the official positions of the MHRA or the EMA or their respective committees.