Rare Diseases Clinical Research Network (RDCRN) A MODEL FOR - PowerPoint PPT Presentation

Rare Diseases Clinical Research Network (RDCRN) • A MODEL FOR COLLABORATIVE RARE DISEASES RESEARCH WITH PATIENT ADVOCACY GROUPS AS PARTNERS

RDCRN: Background Information • Established in 2003 and expanded in 2009 • 3rd cycle renewal in 2014:  a Network of 22 distinct multi-site Clinical Research Consortia (U54 – no more than $1.25M Total Cost/ year)  Each Consortium studies at least three related rare diseases  A central Data Management and Coordinating Center (DMCC)  Involves Patient Advocacy Groups - CP AG  10 NIH ICs as research partners

About RDCRN • Each consortium conducts at least two multi-site clinical studies, has a training program, and pilot proj ect program. • Collectively, the RDCRN is studying 200 rare diseases in natural history and clinical trials at 240 clinical sites located in the US and in 14 countries. • There are more than 90 active protocols. • 29,000 patients have enrolled in clinical studies. • There have been 174 trainees. • There are 2,290 collaborative consortium members. • There are 98 P AGs as research partners. http://rarediseasesnetwork.epi.usf.edu/

NIH Coalition of Patient Porphyria Rare Disease Clinical NCATS , NCI, NHLBI, NIAID, Advocacy Groups Research Consortium NIAMS, NICHD, NIDCR, NIDDK, Dystonia (CPAG for RDCRN) PAG NIMH, NINDS, ODS Coalition North America Mitochondrial Diseases Consortium PAG Developmental Synaptopathies PAG Primary Immune Deficiency Associated with TSC, PTEN Treatment Consortium And SHANK3 Mutations PAG PAG The Frontotemporal Lobar Brittle Bone Disorders Consortium Degeneration Clinical PAG Research Consortium PAG Chronic Graft Versus Inherited Neuropathies Host Disease PAG Consortium PAG The Data Management and Nephrotic Syndrome Coordinating Center Study Network PAG • Collaborative Clinical Research Rare Lung Diseases Urea Cycle Disorders • Centralized Data Consortium Consortium PAG PAG Coordination and Technology Development Lysosomal Brain Vascular • Public Resources and Disease Network PAG Malformation Consortium Education PAG Rare Kidney • Training Genetic Disorders of Stone Consortium PAG Mucociliary Clearance PAG Vasculitis Clinical Consortium of Eosinophilic Research Consortium PAG Gastrointestinal Disease Researchers PAG Clinical Research in ALS & Related PAG Disorders for Therapeutic Development PAG PAG PAG Rett, MECP2 Duplications Autonomic Disorders Sterol and Isoprenoid and Rett-Related Consortium Diseases Consortium Disorders Consortium

Rare Diseases Clinical Research Network (RDCRN) A MODEL FOR - PowerPoint PPT Presentation

Rare Diseases Clinical Research Network (RDCRN) A MODEL FOR COLLABORATIVE RARE DISEASES RESEARCH WITH PATIENT ADVOCACY GROUPS AS PARTNERS RDCRN: Background Information Established in 2003 and expanded in 2009 3rd cycle renewal in

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Rare Diseases Clinical Research Network (RDCRN) A MODEL FOR - PowerPoint PPT Presentation

Rare Diseases Clinical Research Network (RDCRN) A MODEL FOR COLLABORATIVE RARE DISEASES RESEARCH WITH PATIENT ADVOCACY GROUPS AS PARTNERS RDCRN: Background Information Established in 2003 and expanded in 2009 3rd cycle renewal in

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