Michael F. Huerta, Ph.DMichael F. Huerta, Ph.D..Associate Director for Program Development Associate Director for Program Development

National Library of Medicine, NIHNational Library of Medicine, NIH

BD2K CDE Webinar – September 8, 2015BD2K CDE Webinar – September 8, 2015

Common Data Elements*: Common Data Elements*: Communication & Coordination Communication & Coordination

Across NIHAcross NIH

Michael F. Huerta, Ph.DMichael F. Huerta, Ph.D..Associate Director for Program Development Associate Director for Program Development

National Library of Medicine, NIHNational Library of Medicine, NIH

BD2K CDE Webinar – September 8, 2015BD2K CDE Webinar – September 8, 2015

*for human subject research*for human subject research

Common Data Elements*: Common Data Elements*: Communication & Coordination Communication & Coordination

Across NIHAcross NIH

What is a CDE?

What is a CDE?

A A fixed representation fixed representation of a variable comprisingof a variable comprising

What is a CDE?

A A fixed representation fixed representation of a variable comprisingof a variable comprising A precisely specified A precisely specified questionquestion

What is a CDE?

A A fixed representation fixed representation of a variable comprisingof a variable comprising A precisely specified A precisely specified questionquestion

Q: What is the participant’s sex?

What is a CDE?

A A fixed representation fixed representation of a variable comprisingof a variable comprising A precisely specified A precisely specified questionquestion A fixed set of A fixed set of permissible answerspermissible answers, which can be , which can be

discrete or continuous over a rangediscrete or continuous over a range

Q: What is the participant’s sex?

What is a CDE?

A A fixed representation fixed representation of a variable comprisingof a variable comprising A precisely specified A precisely specified questionquestion A fixed set of A fixed set of permissible answerspermissible answers, which can be , which can be

discrete or continuous over a rangediscrete or continuous over a range

A: Male, Female, or Other

Q: What is the participant’s sex?

What is a CDE?

A A fixed representation fixed representation of a variable comprisingof a variable comprising A precisely specified A precisely specified questionquestion A fixed set of A fixed set of permissible answerspermissible answers, which can be , which can be

discrete or continuous over a rangediscrete or continuous over a range

What is a CDE?

A A fixed representation fixed representation of a variable comprisingof a variable comprising A precisely specified A precisely specified questionquestion A fixed set of A fixed set of permissible answerspermissible answers, which can be , which can be

discrete or continuous over a rangediscrete or continuous over a range Used in common Used in common across multiple sites, projects, across multiple sites, projects,

initiatives, etc.initiatives, etc.

What is a CDE?

A A fixed representation fixed representation of a variable comprisingof a variable comprising A precisely specified A precisely specified questionquestion A fixed set of A fixed set of permissible answerspermissible answers, which can be , which can be

discrete or continuous over a rangediscrete or continuous over a range Used in common Used in common across multiple sites, projects, across multiple sites, projects,

initiatives, etc.initiatives, etc. Typically represent Typically represent core variablescore variables – not all variables– not all variables

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 Individual CDEs can be combinedcombined to to

Populate case report formsPopulate case report forms Constitute a validated survey instrument, e.g., Constitute a validated survey instrument, e.g.,

Patient Health Questionnaire-9Patient Health Questionnaire-9• Nine questions to measure depressionNine questions to measure depression

Why use CDEs?

Why use CDEs?

Consistent data collection of Consistent data collection of core set of variablescore set of variables from different sources (sites, projects, initiatives) from different sources (sites, projects, initiatives) valid sharing & comparing of datavalid sharing & comparing of data allowing:allowing: Aggregation of data to increase statistical powerAggregation of data to increase statistical power Rigorous comparison of data & resultsRigorous comparison of data & results

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: EfficiencyEfficiency – off-the-shelf data elements – off-the-shelf data elements QualityQuality – validated instruments & measures – validated instruments & measures ClarityClarity – unambiguously defined data elements– unambiguously defined data elements ReproducibilityReproducibility – from rigorous comparison– from rigorous comparison

How are CDEs Typically Developed?

How are CDEs Typically Developed?

The need for a CDE is first identified byThe need for a CDE is first identified by Research funder (e.g., NIH IC)Research funder (e.g., NIH IC) Regulatory agency (e.g., FDA)Regulatory agency (e.g., FDA) Professional society (e.g., ACC)Professional society (e.g., ACC) Research communityResearch community

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 Stakeholders & expert groups convened to develop or select CDE for identified purposeor select CDE for identified purpose

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 communityIterations & updates w input from broader community

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 CDEs are endorsed by the convening organization and

their use is then required, recommended, encouraged or their use is then required, recommended, encouraged or merely acknowledged as a possibilitymerely acknowledged as a possibility

NIH CDE Collections & Efforts

NIH CDE Collections & Efforts

Broadly applicable Broadly applicable & formally evaluated collections& formally evaluated collections PhenX Toolkit PhenX Toolkit > 350 standard measures of > 350 standard measures of

phenotypes & exposuresphenotypes & exposures PROMISPROMIS Validated patient reported outcome Validated patient reported outcome

measures, ~ 100 computerized adaptive testsmeasures, ~ 100 computerized adaptive tests NIH Toolbox NIH Toolbox – Validated measures of cognitive, – Validated measures of cognitive,

emotional, sensory and motor functionsemotional, sensory and motor functions

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 More narrowly focused narrowly focused collectionscollections NINDSNINDS CDEs for disease-specific studies CDEs for disease-specific studies NCINCI Early Detection Research Network Early Detection Research Network NEINEI eyeGENE ophthalmic phenotype CDEs eyeGENE ophthalmic phenotype CDEs NIDANIDA substance use disorders CDEs for EHRs substance use disorders CDEs for EHRs NCATSNCATS Global Rare Diseases Patient Registry Global Rare Diseases Patient Registry

Three Important Facts About CDEs

Three Important Facts About CDEs

CDE use and efforts will continue toCDE use and efforts will continue to increaseincrease > 40 active FOAs // > 300 FOAs in last few years> 40 active FOAs // > 300 FOAs in last few years Data from Electronic Health RecordsData from Electronic Health Records Use by other organizations (eg., FDA, PCORnet)Use by other organizations (eg., FDA, PCORnet)

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 Communication about and coordination of CDE efforts across NIH is a efforts across NIH is a good thinggood thing

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 NIH has an excellent venue excellent venue for communicating & for communicating & coordinating: coordinating: BMICBMIC

Biomedical Informatics Coordinating Committee

Biomedical Informatics Coordinating Committee

BMIC established 2007 by NIH DirectorBMIC established 2007 by NIH Director To improve communication & coordination of clinical-To improve communication & coordination of clinical-

informatics & bioinformatics across NIHinformatics & bioinformatics across NIH All NIH ICsAll NIH ICs

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 includeBMIC Products include

Working Group on Clinical IT StandardsWorking Group on Clinical IT Standards Working Group on Community Based Standards Working Group on Community Based Standards Portal to NIH-supported Data RepositoriesPortal to NIH-supported Data Repositories Portal to NIH Data Sharing Policies Portal to NIH Data Sharing Policies Common Data Elements Working GroupCommon Data Elements Working Group

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 includeBMIC Products include

Working Group on Clinical IT StandardsWorking Group on Clinical IT Standards Working Group on Community Based Standards Working Group on Community Based Standards Portal to NIH-supported Data RepositoriesPortal to NIH-supported Data Repositories Portal to NIH Data Sharing Policies Portal to NIH Data Sharing Policies Common Data Elements Working GroupCommon Data Elements Working Group

BMIC CDE Working Group

Established 2012 by BMICEstablished 2012 by BMIC Focus on the many CDE efforts across NIHFocus on the many CDE efforts across NIH 22 NIH ICs22 NIH ICs

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 ICsOngoing, common engagement of NIH ICs Sharing lessons learned & best practicesSharing lessons learned & best practices Paper on CDEs & CDE efforts at NIHPaper on CDEs & CDE efforts at NIH

Authors from NCATS, NCI, NEI, NHGRI, NIAMS, NICHD, Authors from NCATS, NCI, NEI, NHGRI, NIAMS, NICHD, NIDA, NINDS & NLMNIDA, NINDS & NLM

NIH rep to CDE efforts (FDA, ONC, CFAST, etc.)NIH rep to CDE efforts (FDA, ONC, CFAST, etc.) Several CDE resources facilitating communication & Several CDE resources facilitating communication &

coordination of CDE efforts at NIH coordination of CDE efforts at NIH

BMIC CDE Resource Portal

Information about CDEs from across NIH:Information about CDEs from across NIH: Glossary of termsGlossary of terms Specific CDE use guidance from ICsSpecific CDE use guidance from ICs Organized and sorted information & links toOrganized and sorted information & links to

NIH/IC CDE collectionsNIH/IC CDE collections NIH CDE tools & resourcesNIH CDE tools & resources

NIH CDE Repository

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