title: Challenges and Hurdles to Business as Usual in Drug Development for Treatment of Rare Diseases.
abstract: Only 10-15 first-in-class new medicines are approved each year by the global pharmaceutical industry for all diseases, of which less than a third is for rare (orphan) diseases. The drug discovery processes to identify rare and common diseases are similar, suggesting it will be impossible to discover new drugs for even a small fraction of the rare diseases using the current paradigm. Different approaches are required to address this large unmet medical need.
authors.affiliation: Institute for Rare and Neglected Diseases Drug Diseases, Mountain View, California, USA. david.swinney@irnd3.org.
authors.forename: D C
authors.initials: DC
authors.lastname: Swinney
mesh_terms: D055808:Drug Discovery / Q000379:methods*; D004345:Drug Industry / Q000379:methods*; D006801:Humans; D009965:Orphan Drug Production / Q000379:methods*; D035583:Rare Diseases / Q000188:drug therapy*