Drug repositioning strategies for rare and orphan diseases

Abstract

This chapter describes the current drug discovery processes and then focuses on drug repositioning strategies and finally highlights collaborative efforts between industry, academia and charities towards a cure for alkaptonuria, a rare metabolic disease. It elaborates on the principles of how academic and industry partnerships interact to perform drug repositioning programmes with the aim of finding new treatments for neglected diseases, rare diseases and some Western diseases. Illustrative examples of drugs in development that have been discovered by such strategies will be covered. It typically takes 10–15 years to develop one drug from time it is discovered to when it is available for treating patients. One recent estimate for the average cost of research and development of a successful drug is US$800 million to US$1 billion. The process of clinical drug development is tightly regulated by licensing authorities such as the Food and Drug Administration in the United States and the Medicines Healthcare Products Regulatory Agency in the United Kingdom.