title: Multifaceted roles of ultra-rare and rare disease patients/parents in drug discovery.
abstract: Individual parents and patients are increasingly doing more to fund, discover and develop treatments for rare and ultra-rare diseases that afflict their children, themselves or their friends. They are performing roles in business development that would be classed as entrepreneurial; and their organizational roles in driving the science in some cases are equivalent to those of principal investigators. These roles are in addition to their usual positioning as advocates. Through their efforts and those of the collaborative networks that they have developed, they could be positioned to disrupt the usual course of drug discovery. This can be illustrated using three different ultra-rare disease parent/patient advocate groups and the diseases for which they are developing treatments. This represents an alternative model for pharmaceutical research.
authors: Jonah's Just Begun, P.O. Box 150057, Brooklyn, NY 11215, USA; Phoenix Nest, P.O. Box 150057, Brooklyn, NY 11215, USA.
authors: Jill
authors: J
authors: Wood
authors: Lori
authors: L
authors: Sames
authors: Allison
authors: A
authors: Moore
authors: Sean
authors: S
authors: Ekins
mesh_terms: D002648:Child; D003299:Cooperative Behavior; D055808:Drug Discovery / Q000191:economics / Q000379:methods*; D004345:Drug Industry / Q000191:economics / Q000458:organization & administration; D006801:Humans; D017146:Models, Organizational; D009965:Orphan Drug Production / Q000191:economics / Q000379:methods; D010290:Parents; D010344:Patient Advocacy; D035583:Rare Diseases / Q000188:drug therapy*; D012106:Research / Q000191:economics / Q000458:organization & administration