title: The partnership of patient advocacy groups and clinical investigators in the rare diseases clinical research network.
abstract: Among the unique features of the Rare Diseases Clinical Research Network (RDCRN) Program is the requirement for each Consortium to include patient advocacy groups (PAGs) as research partners. This development has transformed the work of the RDCRN and is a model for collaborative research. This article outlines the roles patients and PAGs play in the RDCRN and reports on the PAGs' impact on the Network's success.
  
  
  
  Principal Investigators from the 17 RDCRN Consortia and 28 representatives from 76 PAGs affiliated with these Consortia were contacted by email to provide feedback via an online RDCRN survey. Impact was measured in the key areas of 1) Research logistics; 2) Outreach and communication; and 3) Funding and in-kind support. Rating choices were: 1-very negative, 2-somewhat negative, 3-no impact, 4-somewhat positive, and 5-very positive.
  
  
  
  Twenty-seven of the PAGs (96 %) disseminate information about the RDCRN within the patient community. The Consortium Principal Investigators also reported high levels of PAG involvement. Sixteen (94 %) Consortium Principal Investigators and 25 PAGs (89 %) reported PAGs participation in protocol review, study design, Consortium conference calls, attending Consortium meetings, or helping with patient recruitment.
  
  
  
  PAGs are actively involved in shaping Consortia's research agendas, help ensure the feasibility and success of research protocols by assisting with study design and patient recruitment, and support training programs. This extensive PAG-Investigator partnership in the RDCRN has had a strongly positive impact on the success of the Network.
authors.affiliation: Division of Rheumatology and Department of Biostatistics and Epidemiology, University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: Peter A
authors.initials: PA
authors.lastname: Merkel
authors.affiliation: PCD Foundation, Minneapolis, MN, USA.
authors.forename: Michele
authors.initials: M
authors.lastname: Manion
authors.affiliation: Office of Rare Diseases Research (ORDR), National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, MD, USA.
authors.forename: Rashmi
authors.initials: R
authors.lastname: Gopal-Srivastava
authors.affiliation: National Center for Advancing Translational Sciences (NCATS), National Institutes of Health, Bethesda, MD, USA.
authors.forename: Stephen
authors.initials: S
authors.lastname: Groft
authors.affiliation: Departments of Neurology, Human Genetics and Pediatrics, Emory University School of Medicine, Atlanta, GA, USA.
authors.forename: H A
authors.initials: HA
authors.lastname: Jinnah
authors.affiliation: Departments of Medicine, Pharmacology, and Neurology, Clinical Research Center, Vanderbilt University, Nashville, TN, USA.
authors.forename: David
authors.initials: D
authors.lastname: Robertson
authors.affiliation: Health Informatics Institute and Department of Pediatrics, University of South Florida, 3650 Spectrum Boulevard, Tampa, FL, 33612, USA. Jeffrey.Krischer@epi.usf.edu.
authors.forename: Jeffrey P
authors.initials: JP
authors.lastname: Krischer
mesh_terms: D003142:Communication; D003299:Cooperative Behavior; D003625:Data Collection; D006801:Humans; D017063:Outcome Assessment, Health Care; D010344:Patient Advocacy; D035583:Rare Diseases; D012042:Registries; D012107:Research Design; D014481:United States
keywords: Network; Patient engagement; Rare diseases