ABSTRACT
This chapter focuses on the US and Europe, since these are main markets currently where orphan drug legislation is relevant because of the size of the markets and the development of the healthcare systems. It outlines the history of the incentives for the development of products for rare diseases, the process of obtaining designation as an orphan drug and some of the key factors to consider when developing a drug to treat a rare disease. The chapter shows that the development of an orphan drug requires a different approach, both in the thinking of the company doing the development and in the way the regulators make their assessment. The development of medicines to treat diseases is a commercial business, driven by market forces as it has been for more than a century, spurred on by the high gains to be made from medicines for common diseases such as high cholesterol, asthma, cardiovascular disease, hypertension, diabetes and gastrointestinal disorders.
