PPT-Rare Diseases Clinical Research Network

Data Management and Coordinating Center RDCRN DMCC Rosalie Holland LDN Investigator Meeting at WORLDSymposium 2016 March 4 2016 The Rare Diseases Clinical Research Network RDCRN is coordinated by the Office of Rare Diseases Research ORDR NCATS Funding and programmatic support is provided by ORDR in collaboration with participating NIH Institutes. CampaignTracker. A brief overview of its design and function and what we have learned so far.. Background & Purpose. The . CampaignTracker. is a tool built to house Rare’s reporting tools and processes that provide a consistent global language with which to measure and assess the progress and quality of Rare’s work around the world.. Batten Disease. June 29-30, 2010 . FDA Public Meeting. “Considerations regarding the review and . regulation of articles for treatment of rare diseases”. Tracy VanHoutan, . Board Member . of the Batten Disease Support and Research Association (BDSRA). DISEASES. “ARE WE READY FOR THE CHANGE THAT WE NEED TO FIND EFFECTIVE TREATMENTS FOR ORPHAN DISEASE?”. 2. YES!. Rare disease is very ‘popular’!. Rare disease <200,000 Americans have disease. Latvian . Alliance for Rare . Diseases. Baiba . Ziemele. . 22.08.2015.. Rare diseases. Rare diseases are life-threatening, chronically debilitating, rare and with a high degree of . complexity. < 5 in 10 000 people. in the United States. Paul Melmeyer. Associate. . Director. of Public Policy. National . Organization. for Rare . Disorders. (NORD). Where. . W. e. . W. ere. What. . We. . H. ave . Accomplished. 65. th. Annual . Bohan. Lecture. Scott J. Weir, . PharmD. , PhD. 08 October 2011. Drug Focused Translational Research. Translational Research Defined by the Clinical Problem. Breast Cancer. Clinical Problem. inclusion 1 6.19 Rare diseases See Background Paper 6.19 (BP6_19Rare.pdf) Background In the EU, a disease is considered to be rare when the number of people affected is less than 5 per 10 000. There About NCATS and Translational ScienceNCATS is one of 27 Institutes and Centers at the National Institutes of Health. The Center was established to transform the translational process so Translation is ARTICLEAdvancements in science and technology have helped researchers develop new treatments for some of the most common diseases known to man. Diseases that were once considered death sentences are n Rare Disease DayFrequently Asked QuestionsWhat is a rare disease The most complete listing rarediseasesinfonihgov/diseasesWho is affected by rare diseasesManybut not allrare diseases are genetic Some | Version 02 | March 2020 : Orphanet nomenclature and classification of rare diseases www.orpha.net www.orphadata.org Procedural document on the Orphanet nomenclature and classification of rare begs the question: how long will it take other countries to similarly oer access to genomic medicine? What key barriers exist to the implementation of genomic medicine?Given the current disparities i Page 53 of 60 Ralf‐Dieter Hilgers*, Franz KönigDepartment of Medical Statistics, RWTH Universit Aachen, Pauwelstr 30, D‐ 52074 Aachen, Germany Dr. RalfDieter Hilgers, Department of Medical Statis . to. . care. and . on. European . cooperation. EESC . public. . hearing. :. Ensuring. . strong. European . solidarity. . for. . rare. . disease. . patients. Prof. Dr. . Ildiko. . Horvath.