title: Orphan drug development and the impact on non-medical support groups.
abstract: The Orphan Drug legislation in the United States and later in the EU has had a significant impact on patients worldwide who suffer from an orphan condition. Apart from providing statutory encouragement and incentives to pharmaceutical companies to develop therapeutic products it has resulted in the encouragement of patients to come together to form patient bodies to advocate on behalf of patients. Starting in a modest way patient groups have gained experience in working with clinicians and scientists and representing their members to companies and to healthcare providers in national European and global environments. This article describes the history and evolution of the patient body and the coming together of national patient groups through umbrella organisations which have proven to be a powerful advocate for pan European and global collaboration and humanitarian aid. It also will review of some unintended consequences of the legislation.
authors.affiliation: European Gaucher Alliance, UK Gauchers Association, 48/52 Silver Street Dursley, Gloucestershire GL11 4ND, UK. Jeremy@eurogaucher.org
authors.forename: Jeremy
authors.initials: J
authors.lastname: Manuel
authors.affiliation: UK Gauchers Association, European Gaucher Alliance, Evesham House Business Centre, 48/52 Silver Street Dursley, Gloucestershire GL11 4ND, UK.
authors.forename: Tanya
authors.initials: T
authors.lastname: Collin-Histed
mesh_terms: D006282:Health Personnel; D006801:Humans; D007880:Legislation, Drug; D009965:Orphan Drug Production / Q000331:legislation & jurisprudence*; D012657:Self-Help Groups / Q000458:organization & administration*