Orphan Drug Development—International Program and Study Design Issues

Abstract

The incentives established by the United States Orphan Drug Act in 1983 initiated significant changes within the pharmaceutical industry regarding resources expended to develop products to treat diseases and conditions lacking commercial markets. America’s solution to the orphan drug problem has had worldwide impact and has contributed to an orphan drug law in Japan, proposed legislation in the European Union, and the initiation of a process whereby most Food and Drug Administration-approved orphan drugs and biological products will be similarly approved in Australia.

The international research community faces unique concerns regarding trial design required to obtain approval of products to treat rare diseases. Although safety and efficacy for orphan products cannot be compromised, certain factors impact the research approach and present obstacles to investigators: the life-threatening nature of many orphan diseases; the fact that no other treatment may be available for patients other than the study drug; and the limited number of patients available for clinical trials. Expanding international support for rare disease research is providing stimulus and motivation to overcome these barriers and to encourage development of treatment for very rare diseases. NOTE: The term drug will be used here to specify drug and/or biological product.