Big pharma moves from 'blockbusters' to 'niche busters'

Abstract

Since its passage in 1983, the US Orphan Drug Act has led to the approval of more than 350 drugs for around 200 rare diseases, mostly thanks to small biotech startups looking for a unique niche in the marketplace. Yet with the demise of big pharma's traditional business model, some of the worlds largest drug makers are aggressively entering the rare disease sector.