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| The '''Rare Diseases Clinical Research Network (RDCRN)''' is an initiative of the [[Office of Rare Diseases Research]] (ORDR). RDCRN is funded by the ORDR, the [[National Center for Advancing Translational Sciences]] and collaborating institute centers. The RDCRN is designed to advance medical research on [[rare disease]]s by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation.
| | {{Short description|A network to facilitate research on rare diseases}} |
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| As of October 2014, the network is composed of around 2,600 researchers which includes [[National Institutes of Health]] staff, academic investigators and members of 98 patient advocacy groups. There are 91 studies underway.<ref name="NIH2014">{{cite web|url=https://www.nih.gov/news-events/news-releases/nih-funds-research-consortia-study-more-200-rare-diseases |title=NIH funds research consortia to study more than 200 rare diseases|publisher=National Institutes of Health|date=October 8, 2014|accessdate=December 18, 2015}}</ref>
| | The '''Rare Diseases Clinical Research Network''' (RDCRN) is a collaborative network established to advance medical research on [[rare diseases]]. It is funded by the [[National Institutes of Health]] (NIH) and aims to improve the availability of information and treatment options for patients with rare diseases. |
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| == History == | | ==Overview== |
| The following is a timeline of the Rare Diseases Clinical Research Network: | | The RDCRN was created to address the unique challenges posed by rare diseases, which often affect small patient populations and have limited research funding. The network facilitates collaboration among researchers, clinicians, and patients to enhance the understanding of rare diseases and develop new diagnostic and therapeutic approaches. |
| * As a result of the [[Rare Diseases Act of 2002]], on February 27, 2003, the ORDR (in conjunction with the National Center for Research Resources (NCRR), the General Clinical Research Consortium (GCRC) Program, and other NIH Institutes) requested applications for a Rare Diseases Clinical Research Network.<ref>{{cite web|title=RFA-RR-03-008: RARE DISEASES CLINICAL RESEARCH NETWORK|url=http://grants.nih.gov/grants/guide/rfa-files/RFA-RR-03-008.html|accessdate=8 February 2012}}</ref>
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| * On November 3, 2003, the NIH established the Rare Diseases Clinical Research Network with a Data Technology Coordinating Center and the first Rare Disease Clinical Research Consortia (RDCRCs).<ref>{{cite web|url=http://www.nih.gov/news/pr/nov2003/ncrr-03.htm|title=NIH Establishes Rare Diseases Clinical Research Network|date=November 3, 2003|accessdate=February 8, 2012}}</ref> The founding members of the RDCRN were:
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| :* Rare Disease Clinical Research Center for New Therapies and New Diagnostics, Principal Investigator: Dr. Arthur L. Beaudet (Baylor College of Medicine, Houston, TX)
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| :* [[Vasculitis]] Clinical Research Network, Principal Investigator: Dr. Peter A. Merkel (University Pennsylvania, Philadelphia, PA)
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| :* Rare Lung Diseases Consortium, Principal Investigator: Dr. Bruce C. Trapnell (Children's Hospital Medical Center, Cincinnati, OH)
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| :* Rare Diseases Clinical Research Center for [[urea cycle|Urea Cycle]] Disorders, Principal Investigator: [[Mark Batshaw|Dr. Mark L. Batshaw]] (Children's National Medical Center, Washington, DC)
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| :* [[bone marrow|Bone Marrow]] Failure Clinical Research Center, Principal Investigator: Dr. Jaroslaw P. Maciejewski (The [[Cleveland Clinic Foundation]], Cleveland, OH)
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| :* Nervous System Channelopathies Pathogenesis and Treatment, Principal Investigator: Dr. Robert C. Griggs ([[University of Rochester]], Rochester, NY)
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| :* The Natural History of Rare Genetic Steroid Disorders, Principal Investigator: Dr. [[Maria New]] (Weill Medical College of Cornell University, New York, NY)
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| :* The Data and Technology Coordinating Center, Principal Investigator: Dr. Jeffrey P. Krischer (H. Lee Moffitt Cancer Center and Research Institute, University of South Florida, Tampa, FL)
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| * On February 8, 2009, the ORDR partnered with 10 other NIH Institutes to release two requests for resubmissions for the RDCRN.<ref>{{cite web|url=http://grants.nih.gov/grants/guide/rfa-files/rfa-od-08-001.html|title=RFA-OD-08-001: Rare Diseases Clinical Research Consortia (RDCRC) for the Rare Diseases Clinical Research Network|accessdate=February 8, 2012}}</ref>
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| * On October 5, 2009, the NIH announced funding for 19 rare disease clinical research consortia and a Data Management Coordinating Center through the ORDR, along with the [[National Institute of Neurological Disorders and Stroke|National Institute of Neurological Disorders and Stroke (NINDS)]], the [[National Institute of Child Health and Human Development|Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)]], the [[National Heart, Lung, and Blood Institute|National Heart, Lung, and Blood Institute (NHLBI)]], the [[National Institute of Diabetes and Digestive and Kidney Diseases|National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)]], the [[National Institute of Allergy and Infectious Diseases|National Institute of Allergy and Infectious Diseases (NIAID)]], the [[National Institute of Dental and Craniofacial Research|National Institute of Dental and Craniofacial Research (NIDCR)]], and the [[National Institute of Arthritis and Musculoskeletal and Skin Diseases|National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)]].<ref>{{cite web|url=http://www.nih.gov/news/health/oct2009/od-05.htm|title=NIH Announces Expansion of Rare Diseases Clinical Research Network|date=October 5, 2009|accessdate=February 8, 2012}}</ref>
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| *On October 8, 2014, the NIH announced additional funding of $29 million.<ref name="NIH2014"/>
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| == Rare Disease Clinical Research Consortia == | | ==Structure== |
| The Rare Diseases Clinical Research Network consists of 22 Rare Diseases Clinical Research Centers (RDCRCs) and a Data Management and Coordinating Center (DMCC). | | The RDCRN is composed of multiple [[Consortium|consortia]], each focusing on a specific group of rare diseases. These consortia bring together experts from various fields, including [[genetics]], [[epidemiology]], and [[clinical research]], to conduct [[clinical trials]] and other research activities. The network also includes a Data Management and Coordinating Center (DMCC) that supports data collection, management, and analysis across the consortia. |
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| The RDCRCs, the DMCC, and their Principal Investigators are located at the following institutions: | | ==Goals== |
| | The primary goals of the RDCRN are to: |
| | * Facilitate clinical research on rare diseases by providing infrastructure and support. |
| | * Enhance the understanding of the natural history of rare diseases. |
| | * Develop and test new diagnostic and therapeutic interventions. |
| | * Improve the availability of information and resources for patients and healthcare providers. |
| | * Foster collaboration among researchers, clinicians, and patient advocacy groups. |
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| * Urea Cycle Disorders Consortium (UCDC), Mark L. Batshaw, M.D., Children's National Medical Center, Washington, DC
| | ==Research Activities== |
| * Rett Syndrome, MECP2 Duplications, and Rett-related Disorders Consortium (Rett), Alan K. Percy, M.D., Civitan International Research Center, University of Alabama at Birmingham
| | The RDCRN supports a wide range of research activities, including: |
| * Vasculitis Clinical Research Consortium (VCRC), Peter A. Merkel, M.D., M.P.H., University of Pennsylvania, Philadelphia, PA
| | * [[Natural history studies]] to understand the progression of rare diseases. |
| * Genetic Disorders of Mucociliary Clearance Consortium (GDMCC), Michael Knowles, M.D., CF/Pulmonary Research & Training Center, Chapel Hill, NC | | * [[Clinical trials]] to evaluate the safety and efficacy of new treatments. |
| * Rare Lung Disease Consortium (RLD), Bruce Trapnell, M.D., Cincinnati Children’s Hospital, Cincinnati, OH
| | * [[Epidemiological studies]] to assess the prevalence and incidence of rare diseases. |
| * Autonomic Rare Diseases Clinical Research Consortium (ARD), David Herlie Robertson, M.D., Vanderbilt University Medical Center, Nashville, TN | | * [[Genetic studies]] to identify the underlying causes of rare diseases. |
| * Brain Vascular Malformation Consortium (BVMC): Predictors of Clinical Course, Michael T. Lawton, M.D., University of California, San Francisco
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| * Dystonia Coalition (DC), Hyder A. Jinnah, M.D., Emory University, Atlanta, GA
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| * Rare Kidney Stone Consortium (RKSC), Dawn S. Milliner, M.D., Mayo Clinic College of Medicine, Rochester, MN
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| * Chronic Graft Versus Host Diseases Consortium (cGVHD), Stephanie J. Lee, M.D., M.P.H., Fred Hutchinson Cancer Research Center, Seattle, WA
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| * Inherited Neuropathies Consortium (INC), Michael E. Shy, M.D., University of Iowa, Iowa City, IA
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| * Lysosomal Disease Network (LDN), Chester B. Whitley, M.D., University of Minnesota Twin Cities, Minneapolis, MN
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| * Nephrotic Syndrome Study Network (Neptune), Matthias Kretzler, M.D., University of Michigan, Ann Arbor, MI
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| * Primary Immune Deficiency Treatment Consortium (PIDTC), Morton J. Cowan, M.D., University of California, San Francisco | |
| * Sterol and Isoprenoid Diseases Consortium (STAIR), William Rizzo, M.D., University of Nebraska, Omaha, NE
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| * Porphyrias Consortium (PC), Robert J. Desnick, Ph.D., M.D., Mount Sinai School of Medicine of New York University, New York, NY
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| * North America Mitochondrial Diseases Consortium (NAMDC), Michio Hirano, M.D., Columbia University Medical Center, New York, NY | |
| * Brittle Bone Disease Consortium (BBD), Brendan Lee, MD, PhD, Baylor College of Medicine, Houston, TX
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| * Consortium for Eosinophilic Gastrointestinal Disease Researchers (CEGIR), Marc E. Rothenberg, M.D., Ph.D., Cincinnati Children’s Hospital, Cincinnati, OH
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| * Development Synaptopathies Consortium (DSC), Mustafa Sahin, M.D., Ph.D., Boston Children’s Hospital, Boston, MA
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| * Clinical Research in ALS and Related Disorders for Therapeutic Development Consortium (CREATE), Michael Benatar, M.D., Ph.D., University of Miami, Miami, FL
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| * UCSF Memory and Aging Center Consortium (UMAC), Adam Boxer, M.D., Ph.D., University of California, San Francisco, San Francisco, CA
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| * The Data Management and Coordinating Center (DMCC), Jeffrey P. Krischer, Ph.D., University of South Florida, Tampa, FL
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| == Data Management and Coordinating Center (DMCC) == | | ==Patient Involvement== |
| The RDCRN’s Data Management and Coordinating Center (DMCC) is hosted by the Health Informatics Institute at the [[University of South Florida]] in Tampa, FL. The DMCC supports RDCRN consortia by providing "data management and analysis infrastructure; logistical and administrative assistance; and data coordination, management, and sharing."<ref>{{cite web|title=Data Management and Coordinating Center (DMCC)|url=http://rarediseasesnetwork.epi.usf.edu/about/dmcc.htm|accessdate=10 February 2012}}</ref>
| | Patient involvement is a key component of the RDCRN. The network actively engages patients and patient advocacy groups in the research process, ensuring that their perspectives and needs are considered. This collaboration helps to prioritize research efforts and improve the relevance and impact of the studies conducted. |
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| The RDCRN also provides user-friendly web-based recruitment and referral tools, including the [[RDCRN Contact Registry]]. The RDCRN Contact Registry aims to provide up-to-date information for patients and assist in connecting patients with advocacy groups, expert doctors, and clinical research opportunities.
| | ==Impact== |
| | Since its inception, the RDCRN has made significant contributions to the field of rare disease research. It has facilitated numerous clinical trials, leading to the development of new treatments and improved patient outcomes. The network has also increased awareness and understanding of rare diseases among healthcare providers and the general public. |
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| == References == | | ==Related pages== |
| {{Reflist|colwidth=30em}}
| | * [[Rare disease]] |
| | | * [[Clinical trial]] |
| == External links ==
| | * [[National Institutes of Health]] |
| * [http://rarediseasesnetwork.epi.usf.edu/index.htm Rare Diseases Clinical Research Network (RDCRN)] | | * [[Genetic disorder]] |
| * [http://rarediseases.info.nih.gov/Wrapper.aspx?src=asp/resources/extr_res.asp NIH Office of Rare Diseases Research (ORDR): RDCRN information] | |
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| | [[Category:Rare diseases]] |
| | [[Category:Medical research]] |
| [[Category:National Institutes of Health]] | | [[Category:National Institutes of Health]] |
| [[Category:Rare diseases]]
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| {{dictionary-stub1}}
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A network to facilitate research on rare diseases
The Rare Diseases Clinical Research Network (RDCRN) is a collaborative network established to advance medical research on rare diseases. It is funded by the National Institutes of Health (NIH) and aims to improve the availability of information and treatment options for patients with rare diseases.
Overview[edit]
The RDCRN was created to address the unique challenges posed by rare diseases, which often affect small patient populations and have limited research funding. The network facilitates collaboration among researchers, clinicians, and patients to enhance the understanding of rare diseases and develop new diagnostic and therapeutic approaches.
Structure[edit]
The RDCRN is composed of multiple consortia, each focusing on a specific group of rare diseases. These consortia bring together experts from various fields, including genetics, epidemiology, and clinical research, to conduct clinical trials and other research activities. The network also includes a Data Management and Coordinating Center (DMCC) that supports data collection, management, and analysis across the consortia.
The primary goals of the RDCRN are to:
- Facilitate clinical research on rare diseases by providing infrastructure and support.
- Enhance the understanding of the natural history of rare diseases.
- Develop and test new diagnostic and therapeutic interventions.
- Improve the availability of information and resources for patients and healthcare providers.
- Foster collaboration among researchers, clinicians, and patient advocacy groups.
Research Activities[edit]
The RDCRN supports a wide range of research activities, including:
Patient Involvement[edit]
Patient involvement is a key component of the RDCRN. The network actively engages patients and patient advocacy groups in the research process, ensuring that their perspectives and needs are considered. This collaboration helps to prioritize research efforts and improve the relevance and impact of the studies conducted.
Since its inception, the RDCRN has made significant contributions to the field of rare disease research. It has facilitated numerous clinical trials, leading to the development of new treatments and improved patient outcomes. The network has also increased awareness and understanding of rare diseases among healthcare providers and the general public.
Related pages[edit]
