phenx-sickle cell disease project at the national heart, lung, and blood institute/nih ellen m....
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PhenX-Sickle Cell Disease Project
at the National Heart, Lung, and Blood Institute/NIH
Ellen M. Werner, Ph.D.Program Director
Blood Epidemiology and Clinical Therapeutics BranchNational Heart, Lung, and Blood Institute
January 7, 2015
2
Genesis of PhenX-SCD Project NHLBI Clinical Practice Recommendations NHLBI and other HHS op divs have had sought to collaborate
HP2020 Registry and Surveillance in Hemoglobinopathies (RuSH) pilot HRSA NBS (with NICHD, CDC), SCD and Thalassemia
Treatment Demonstration Programs
Goal: Establish consensus measures/identify common data elements (CDEs) in SCD
To enable collaborations For data comparison, validation, replication, harmonization
September 2010-Summer 2014 (unfunded)
Hemoglobinopathies Uniform Medical Language Ontology (HUMLO)
Summer 2014-April 2015 (funded)
PhenX-SCD
Division of Blood Diseases and Resources Molecular Cellular and Systems Blood Science Branch Translational Blood Science and Resources Branch Blood Epidemiology and Clinical Therapeutics Branch
Center for Translation Research and Implementation Science Health Inequities and Global Health Branch Implementation Science Branch Translation Research Branch
Re-organization (continued)
T3T2
Biomedical ModelBiomedical and Socioecological
Model
Bench Research• Fundamental
Discovery Science
– Animal Studies
Bedside• 1st Human Studies
• Controlled Observations
• Phase I Clinical Trials
Patients• Phase II and III
Clinical Trials
Practices• Phase IV Clinical Trials
• Comparative Effectiveness Research
Real World Setting• Dissemination and
Implementation Research
T1Discovery Science
Translation to Humans
Translation to Patients
Translation to Clinical Practices
Translation to Real World
SettingsT4
Focusing on Clinical and Translational Research
Mensah. Global Heart September 2013;8(3): 283-284. Based on and informed by the models of Khoury et al. Genet Med 2007;9:665-74, and the
Harvard Catalyst; The Harvard Clinical and Translational Science Center, available at: http://catalyst.harvard.edu/pathfinder/. Accessed August 28, 2013. 5
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