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

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

3. What is a CDE?

4. A fixed representation of a variable comprising A fixed representation of a variable comprising

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

6. What is a CDE? A fixed representation of a variable comprising 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 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 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 A: Male, Female, or Other Q: What is the participant’s sex?

9. What is a CDE? A fixed representation of a variable comprising 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 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. Used in common across multiple sites, projects, initiatives, etc.

11. What is a CDE? A fixed representation of a variable comprising 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. Used in common across multiple sites, projects, initiatives, etc. Typically represent core variables – not all variables 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 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 depressionNine questions to measure depression

13. Why use CDEs?

14. Consistent data collection of core set of variables from different sources (sites, projects, initiatives)  valid sharing & comparing of data allowing: 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

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: 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. The need for a CDE is first identified by 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 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 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 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. Broadly applicable & formally evaluated collections 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 More narrowly focused collections  NINDS CDEs for disease-specific studies  NCI Early Detection Research Network  NEI eyeGENE ophthalmic phenotype CDEs  NIDA substance use disorders CDEs for EHRs  NCATS Global Rare Diseases Patient Registry

24. Three Important Facts About CDEs

25. CDE use and efforts will continue to increase 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)

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 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 NIH has an excellent venue for communicating & coordinating: BMIC

28. Biomedical Informatics Coordinating Committee

29. BMIC established 2007 by NIH Director BMIC established 2007 by NIH Director To improve communication & coordination of clinical- informatics & bioinformatics across NIH To improve communication & coordination of clinical- informatics & bioinformatics across NIH All NIH ICs 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 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 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 Established 2012 by BMIC Focus on the many CDE efforts across NIH Focus on the many CDE efforts across NIH 22 NIH ICs 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 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: 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 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.