1 / 41

The Rare Diseases Clinical Research Network (RDCRN)

The Rare Diseases Clinical Research Network (RDCRN). Data current as of August 31, 2014. Rare Diseases Clinical Research Network (RDCRN) is coordinated by Office of Rare Diseases Research, NCATS.

gannon
Download Presentation

The Rare Diseases Clinical Research Network (RDCRN)

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. The Rare Diseases Clinical Research Network (RDCRN) Data current as of August 31, 2014 Rare Diseases Clinical Research Network (RDCRN) is coordinated by Office of Rare Diseases Research, NCATS. Funding and programmatic support is provided by ORDR in collaboration with participating NIH Institutes.

  2. Rare/Orphan Diseases • The FDA defines an orphan disease as: • A condition affecting <200,000 Americans Or • A disease with greater prevalence but no reasonable expectation that drug development costs are recoverable by U.S. sales • There are an estimated 6,000 rare diseases • Approximately 25 million Americans have a rare disease

  3. Challenges for Rare Diseases Research • Disease often not well characterized or defined • Rarity means: • Recruitment for trials is usually quite difficult • Study populations become widely dispersed • Few expert centers for diagnosis, management, and research • Often little high-quality evidence available to guide treatment

  4. Goals of the RDCRN • Facilitate clinical research by: • Creation of Consortia focused on related diseases • Cost-sharing research infrastructures • Establishing uniform protocols for data collection • Making meaningful large-scale studies possible • Longitudinal cohorts, pilot projects, and randomized trials • Directly engage patients and their advocates • Train new investigators in rare diseases research

  5. RDCRN - Established by the Office of Rare Diseases Research- Started in 2003--currently in 2nd 5 year cycle - 1st cycle (10 Consortia) supported by ORDR, NCRR, NINDS, NIAMS, NICHD, NHLBI, NIDDK - 2nd cycle (19 Consortia) supported by ORDR, NINDS, NIAMS, NICHD, NHLBI, NIDDK, NIAID, NIDCR, NCI- Currently involves 241 institutions world-wide- Collectively studying >200 diseases- 30937 patients enrolled in studies

  6. Coalition of Patient Advocacy Groups (CPAG) DHHS-NIH ORDR, NINDS, NIAMS, NICHD, NHLBI, NIDDK, NIDCR, NIAID, NCI Chronic Graft Versus Host Disease Consortium Dystonia Coalition North America Mitochondrial Diseases Consortium Genetic Disorders of Mucociliary Clearance Consortium Primary Immune Deficiency Treatment Consortium Porphyria Rare Disease Clinical Research Consortium The Data Management and Coordinating Center Vasculitis Clinical Research Consortium Rare Kidney Stone Consortium Lysosomal Disease Network • Collaborative Clinical Research • Centralized Data Coordination and Technology Development • Public Resources and Education • Training Nephrotic Syndrome Rare Disease Clinical Research Network Inherited Neuropathies Consortium Urea Cycle Disorders Consortium Angelman, Rett and Prader-Willi Syndromes Consortium Molecular and Epidemiologic Characterization of Salivary Gland Carcinomas Consortium Brain Vascular Malformation Consortium Autonomic Rare Diseases Clinical Research Consortium Sterol and Isoprenoid Diseases Consortium

  7. DHHS-NIH ORDR, NINDS, NIAMS, NICHD, NHLBI, NIDDK, NIDCR, NIAID, NCI Coalition of Patient Advocacy Groups (CPAG) The Data Management and Coordinating Center • Collaborative Clinical Research • Public Resources and Education • Centralized Data Coordination and Technology Development • Training

  8. Rare Diseases Clinical Research Network (RDCRN) NIH NIAID ORDR / NCATS Program Coordination NIAMS NIDCR NHLBI NCI NICHD NINDS NIDDK Steering Committee RDCRN Consortia, DMCC and CPAG OSMBs/DSMBs Angelman, Rett and Prader-Willi Syndromes Consortium Genetic Disorders of Mucociliary Clearance Consortium Porphyrias Consortium Urea Cycle Disorders Consortium Autonomic Rare Diseases Clinical Research Consortium Inherited Neuropathies Consortium Primary Immune Deficiency Treatment Consortium Vasculitis Clinical Research Consortium Brain Vascular Malformation Consortium Lysosomal Disease Network Rare Kidney Stone Consortium Data Management and Coordinating Center (DMCC) Chronic Graft Versus Host Disease Consortium Nephrotic Syndrome Study Network Salivary Gland Carcinomas Consortium Coalition of Patient Advocacy Groups (CPAG) (> 95 PAGs) Dystonia Coalition North American Mitochondrial Disease Consortium Sterol and Isoprenoid Diseases Consortium

  9. RDCRN Data Management and Coordinating Center (DMCC) • Supports RDCRN by providing technologies, tools, and support of study design and data analysis • On-line protocol management system • Patient enrollment/randomization • Data entry and collection with data standards • Adverse event reporting • Protocol training for research staff • Members’ website: documentation, databases • Hosts RDCRN public website (>2 million hits/year) • Oversees the RDCRN Contact Registry

  10. RDCRN Websitehttp://rarediseasesnetwork.org • Portal to websites for each Consortium • Portal to members’ website • Portal for patient advocacy groups • RDCRN Contact Registry • RDCRN Media Center

  11. RDCRN Web Stats for Last 12 Month Period

  12. RDCRN Protocol Activity - 17 Consortia with NIH approved protocols- 88 Accruing protocols- 7 Accrual goal met- 241 Clinical Sites

  13. Consortia Protocol Accrual

  14. Protocol by NIH Institute

  15. RDCRN Patient Contact Registry - Enrollment open to patients with diseases under study by Consortia- Provides international on-line system for communication, recruitment, research- 182 Diseases represented- 13708 Registrations

  16. Contact Registry Accrual

  17. RDCRN U.S. Sites

  18. RDCRN North America Sites

  19. RDCRN European Sites

  20. RDCRN World Sites

  21. U.S. Geographic Distribution of Contact Registrants

  22. North America Geographic Distribution of Contact Registrants

  23. European Geographic Distribution of Contact Registrants

  24. World Geographic Distribution of Contact Registrants

  25. Start of Consortium-Specific Slides

  26. BVMC U.S. Sites

  27. BVMC North America Sites

  28. BVMC European Sites

  29. BVMC World Sites

  30. BVMC U.S. Geographic Distribution of Contact Registrants

  31. BVMC North America Geographic Distribution of Contact Registrants

  32. BVMC European Geographic Distribution of Contact Registrants

  33. BVMC World Geographic Distribution of Contact Registrants

  34. BVMC Contact Registrants by Disease

  35. BVMC Contact Registry Accrual Over Time

  36. BVMC Accrual by Protocol

  37. BVMC Accrual (cumulative) by Protocol

  38. BVMC Accrual (cumulative) Over Time

  39. BVMC Trainees

  40. BVMC Recent Publications

  41. www.rarediseasesnetwork.org/bvmc www.rarediseasesnetwork.org

More Related