Common Data Elements*: Communication & Coordination Across NIH

1. Common Data Elements*: Communication & Coordination Across NIH Michael F. Huerta, Ph.D. Associate Director for Program Development National Library of Medicine, NIH BD2K CDE Webinar – September 8, 2015

2. Common Data Elements*: Communication & Coordination Across NIH *for human subject research Michael F. Huerta, Ph.D. Associate Director for Program Development National Library of Medicine, NIH BD2K CDE Webinar – September 8, 2015

3. What is a CDE?

4. What is a CDE? • A fixed representation of a variable comprising

5. What is a CDE? • A fixed representation of a variable comprising • A precisely specified question

6. What is a CDE? • A fixed representation of a variable comprising • A precisely specified question Q: What is the participant’s sex?

7. What is a CDE? • A fixed representation of a variable comprising • A precisely specified question • A fixed set of permissible answers, which can be discrete or continuous over a range Q: What is the participant’s sex?

8. What is a CDE? • A fixed representation of a variable comprising • A precisely specified question • A fixed set of permissible answers, which can be discrete or continuous over a range Q: What is the participant’s sex? A: Male, Female, or Other

9. What is a CDE? • A fixed representation of a variable comprising • A precisely specified question • A fixed set of permissible answers, which can be discrete or continuous over a range

10. What is a CDE? • A fixed representation of a variable comprising • A precisely specified question • A fixed set of permissible answers, which can be discrete or continuous over a range • Used in common across multiple sites, projects, initiatives, etc.

11. What is a CDE? • A fixed representation of a variable comprising • A precisely specified question • A fixed set of permissible answers, which can be discrete or continuous over a range • Used in common across multiple sites, projects, initiatives, etc. • Typically represent core variables– not all variables

12. What is a CDE? • A fixed representation of a variable comprising • A precisely specified question • A fixed set of permissible answers, which can be discrete or continuous over a range • Used in common across multiple sites, projects, initiatives, etc. • Typically represent core variables – not all variables • Individual CDEs can be combined to • Populate case report forms • Constitute a validated survey instrument, e.g., • Patient Health Questionnaire-9 • Nine questions to measure depression

13. Why use CDEs?

14. Why use CDEs? • Consistent data collection of core set of variablesfrom different sources (sites, projects, initiatives)  valid sharing & comparing of dataallowing: • Aggregation of data to increase statistical power • Rigorous comparison of data & results

15. Why use CDEs? • Consistent data collection of core set of variables from different sources (sites, projects, initiatives)  valid sharing & comparing of data allowing: • Aggregation of data to increase statistical power • Rigorous comparison of data & results • Can be used to promote research: • Efficiency – off-the-shelf data elements • Quality – validated instruments & measures • Clarity– unambiguously defined data elements • Reproducibility– from rigorous comparison

16. How are CDEs Typically Developed?

17. How are CDEs Typically Developed? • The need for a CDE is first identified by • Research funder (e.g., NIH IC) • Regulatory agency (e.g., FDA) • Professional society (e.g., ACC) • Research community

18. How are CDEs Typically Developed? • The need for a CDE is first identified by • Research funder (e.g., NIH IC) • Regulatory agency (e.g., FDA) • Professional society (e.g., ACC) • Research community • Stakeholders & expert groups convened to develop or select CDE for identified purpose

19. How are CDEs Typically Developed? • The need for a CDE is first identified by • Research funder (e.g., NIH IC) • Regulatory agency (e.g., FDA) • Professional society (e.g., ACC) • Research community • Stakeholders & expert groups convened to develop or select CDE for identified purpose • Iterations & updates w input from broader community

20. How are CDEs Typically Developed? • The need for a CDE is first identified by • Research funder (e.g., NIH IC) • Regulatory agency (e.g., FDA) • Professional society (e.g., ACC) • Research community • Stakeholders & expert groups convened to develop or select CDE for identified purpose • Iterations & updates w input from broader community • CDEs are endorsed by the convening organization and their use is then required, recommended, encouraged or merely acknowledged as a possibility

21. NIH CDE Collections & Efforts

22. NIH CDE Collections & Efforts • Broadly applicable & formally evaluated collections • PhenX Toolkit > 350 standard measures of phenotypes & exposures • PROMIS Validated patient reported outcome measures, ~ 100 computerized adaptive tests • NIH Toolbox – Validated measures of cognitive, emotional, sensory and motor functions

23. NIH CDE Collections & Efforts • Broadly applicable & formally evaluated collections • PhenX Toolkit > 350 standard measures of phenotypes & exposures • PROMIS Validated patient reported outcome measures, ~ 100 computerized adaptive tests • NIH Toolbox – Validated measures of cognitive, emotional, sensory and motor functions • More narrowly focused collections • NINDS CDEs for disease-specific studies • NCI Early Detection Research Network • NEIeyeGENE ophthalmic phenotype CDEs • NIDA substance use disorders CDEs for EHRs • NCATS Global Rare Diseases Patient Registry

24. Three Important Facts About CDEs

25. Three Important Facts About CDEs • CDE use and efforts will continue toincrease • > 40 active FOAs // > 300 FOAs in last few years • Data from Electronic Health Records • Use by other organizations (eg., FDA, PCORnet)

26. Three Important Facts About CDEs • CDE use and efforts will continue to increase • > 40 active FOAs // > 300 FOAs in last few years • Data from Electronic Health Records • Use by other organizations (eg., FDA, PCORnet) • Communication about and coordination of CDE efforts across NIH is a good thing

27. Three Important Facts About CDEs • CDE use and efforts will continue to increase • > 40 active FOAs // > 300 FOAs in last few years • Electronic Health Records • Other relevant organizations (eg., FDA, PCORnet) • Communication about and coordination of CDE efforts across NIH is a good thing • NIH has an excellent venue for communicating & coordinating: BMIC

28. Biomedical Informatics Coordinating Committee

29. Biomedical Informatics Coordinating Committee • BMIC established 2007 by NIH Director • To improve communication & coordination of clinical-informatics & bioinformatics across NIH • All NIH ICs

30. Biomedical Informatics Coordinating Committee • BMIC established 2007 by NIH Director • To improve communication & coordination of clinical-informatics & bioinformatics across NIH • All NIH ICs • BMIC Products include • Working Group on Clinical IT Standards • Working Group on Community Based Standards • Portal to NIH-supported Data Repositories • Portal to NIH Data Sharing Policies • Common Data Elements Working Group

31. Biomedical Informatics Coordinating Committee • BMIC established 2007 by NIH Director • To improve communication & coordination of clinical-informatics & bioinformatics across NIH • All NIH ICs • BMIC Products include • Working Group on Clinical IT Standards • Working Group on Community Based Standards • Portal to NIH-supported Data Repositories • Portal to NIH Data Sharing Policies • Common Data Elements Working Group

32. BMIC CDE Working Group • Established 2012 by BMIC • Focus on the many CDE efforts across NIH • 22 NIH ICs

33. BMIC CDE Working Group • Established 2012 by BMIC • Focus on the many CDE efforts across NIH • 22 NIH ICs • BMIC CDE WG Products • Ongoing, common engagement of NIH ICs • Sharing lessons learned & best practices • Paper on CDEs & CDE efforts at NIH • Authors from NCATS, NCI, NEI, NHGRI, NIAMS, NICHD, NIDA, NINDS & NLM • NIH rep to CDE efforts (FDA, ONC, CFAST, etc.) • Several CDE resources facilitating communication & coordination of CDE efforts at NIH

34. BMIC CDE Resource Portal • Information about CDEs from across NIH: • Glossary of terms • Specific CDE use guidance from ICs • Organized and sorted information & links to • NIH/IC CDE collections • NIH CDE tools & resources

35. NIH CDE Repository • Structured human & machine readable definitions of NIH CDEs allowing • Search for individual CDE or sets per FOA, etc. • Compare & harmonize similar but distinct CDEs • Select or create CDEs with minimal duplication • Etc.