Governments in the industrialised world have revolutionized drug development over the past 25 years with the introduction of orphan legislation encouraging companies to invest in new products to treat rare diseases. Legislation in the US, Europe, Japan and elsewhere gives incentives to companies to start looking after the medical needs of patients with rare, but life-threatening diseases. The concept of ‘orphan’ derives from the fact that these patients had previously been ‘orphaned’ by the pharmaceutical industry.
