title: The role of patient advocacy organisations in neuromuscular disease R&D--The case of the Dutch neuromuscular disease association VSN.
abstract: This article investigates to what extent patient advocacy organisations play a role in influencing R&D and policymaking for rare neuromuscular diseases. The Dutch neuromuscular disease organisation VSN is studied in depth. A brief history of the VSN is sketched along with the international embedding of the organisation. Then, a more general perspective is provided on the reasons and extent of the involvement of patient organisations (and especially the VSN) in innovation processes. Lastly, internal mechanisms are presented that can best be applied by these organisations. The VSN adheres to a rare, long-term vision on drug innovation that requires long-term planning and policy and vision creation and steering the direction of science and technology. At the same time, other actors like scientific organisations and science policymakers and managers can benefit from these lessons to learn how to deal with patients and patient organisations in the future.
authors.affiliation: Department of Innovation and Environmental Studies, Utrecht University, Van Unnik 1013A, P.O. Box 80115, NL-3508 TC Utrecht, The Netherlands. w.boon@geo.uu.nl
authors.forename: Wouter
authors.initials: W
authors.lastname: Boon
authors.forename: Ria
authors.initials: R
authors.lastname: Broekgaarden
mesh_terms: D003256:Community Participation; D003254:Consumer Advocacy; D003255:Consumer Organizations / Q000592:standards / Q000639:trends*; D003659:Decision Making, Organizational; D015195:Drug Design; D004353:Drug Evaluation, Preclinical / Q000639:trends; D004345:Drug Industry / Q000331:legislation & jurisprudence / Q000639:trends; D004335:Drug and Narcotic Control / Q000331:legislation & jurisprudence / Q000639:trends; D005653:Fund Raising; D006291:Health Policy / Q000331:legislation & jurisprudence / Q000639:trends; D006801:Humans; D017146:Models, Organizational; D009426:Netherlands; D009468:Neuromuscular Diseases / Q000188:drug therapy* / Q000503:physiopathology / Q000628:therapy*; D009936:Organizational Innovation; D010344:Patient Advocacy / Q000592:standards / Q000639:trends*; D010358:Patient Participation; D018802:Patient-Centered Care; D010817:Physician-Patient Relations; D010818:Practice Patterns, Physicians'; D011369:Professional-Patient Relations; D011787:Quality of Health Care; D035583:Rare Diseases; D012106:Research; D035844:Researcher-Subject Relations; D057170:Translational Research, Biomedical / Q000592:standards / Q000639:trends*