Stephen C. Groft, Pharm.D. Office of Rare Diseases Research (ORDR) National Center for Advancing Translational Science (NCATS) National Institutes of
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1 Stephen C. Groft, Pharm.D. Office of Rare Diseases Research (ORDR) National Center for Advancing Translational Science (NCATS) National Institutes of Health (NIH) Department of Health and Human Services Best Practices in Clinical Study Design for Rare Diseases The George Washington University Washington, DC April 30, 2013
2 International Rare Diseases Research Consortium Co-operation At International Level To Stimulate, Better Coordinate & Maximise Output Of Rare Disease Research Efforts Around The World Establish and Link Global Rare Diseases Community: Research Investigators, Protocols and Clinical Trials, Academia, Industry, Government, and PAG, and Regulatory Partnerships
3 IRDiRC Governance Document Defines the aims of the consortium; mandate and composition of committees and working groups; rules for procedures, nomination of members, conflicts of interest etc. IRDiRC Policies and Guidelines Will establish a common policy framework addressing various aspects of research, such as, sharing of data and samples, common quality standards, interoperability and harmonisation of ontologies and bio-banks, rapid release of research results, etc.
4 nyul/fotolia.com Yuri Arcurs/Fotolia.com IRDiRC Vision And 2020 Goals In Rare Diseases Research 200 New Therapies Means to Diagnose Most Rare Diseases
5 32 committed members Europe E-RARE Consortium (EU) North America Canadian Institutes for Health Research (CA) FDA Office of Orphan Products Development (US) Genome Canada (CA) rtguest/fotolia.com European Commission (EU) EURORDIS (EU) French Association against Myopathies (FR) French National Research Agency (FR) German Federal Ministry of Education and research (DE) Italian Higher Institute of Health Research (IT) Italian Telethon Foundation (IT) Lysogene (FR) Netherlands Organisation for Health Research and Development Prosensa (NL) Spanish Carlos III Health Institute (ES) UK National Institute for Health Research (UK) Asia BGI (CN) Australia Western Australian Department of Health (AU) Genetic Alliance (US) NHGRI Mendelian Disorders Genome Centres (US) National Center for Advancing Translational Sciences (US) National Cancer Institute (US) National Institute of Neurological Disorders and Stroke (US) National Institute of Arthritis and Musculoskeletal and Skin Diseases (US) National Institute of Child Health and Human Development (US) National Eye Institute (US) NKT Therapeutics (US) NORD (US) Office of Rare Diseases Research NCATS (US) PTC Therapeutics (US) Sanford Research (US) Shire (US)
6 IRDiRC Governance Structure Executive Committee Chair Paul Lasko Scientific Committees Diagnostics Chair Kym Boycott Interdisciplinary Chair Hanns Lochmueller Therapies Chair Josep Torrent-Farnell Working Groups EU-funded SUPPORT-IRDiRC provides organisational support for Scientific Committees and Working Groups.
7 How Does It Work? Identify And Define Shared Strategic Goals Agree To Approach Jointly And Pool Resources Let Each Organisation Use Its Own Funding Mechanisms/Timing Agree To Share Data / Standards Alignment Flexibility - Commitment
8 Working Groups To Advise The Scientific Committees Ontologies Sequencing Model Systems Genomics Bioinformatics Bio-banks Registries Biomarkers Ethics Repurposing Drugs Therapies Regulatory Issues
9 Exploratory Workshop Reykjavik Launch Washington Working Groups Scientific Committees IRDiRC Timeline IRDiRC 2013 Conference 3000 diagnostics 6000 diagnostics Support-irdirc Scientific Secretariat 50 new applications for market authorisation 200 New Applications For Market Authorisation
10
11 Contact Information Office of Rare Diseases Research, NIH Phone: Web Site : Genetic and Rare Diseases Information Center Toll-free: TTY: GARDinfo@nih.gov Web site:
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