1. The NINDS Common Data Elements Project February 20, 2014 Wendy R. Galpern, MD, PhD NINDS / NIH American Society for Experimental NeuroTherapeutics | 16 th Annual Meeting

2. Disclosure None American Society for Experimental NeuroTherapeutics | 16 th Annual Meeting

3. Acknowledgement Disease experts NINDS – Joanne Odenkirchen (jo21x@nih.gov)jo21x@nih.gov Contractors ( KAI [2007 – 2012] ; EMMES [2012 - ] )

4. Learning Objectives Recognize how the use of common data elements (CDEs) can facilitate clinical research efforts Understand the development processes for the NINDS CDEs and the available products American Society for Experimental NeuroTherapeutics | 16 th Annual Meeting

5. What is the NINDS Common Data Elements Project? Effort to facilitate clinical research across a variety of neurological disorders Disease experts developed: – Standardized format for CDEs – Common documentation and CRFs All materials are publically available

6. Why is the development of CDEs important? Clinical trials and research studies – Study-specific Case Report Forms – Various definitions for same items – Comparison between studies difficult

7. Why is the development of CDEs important? Clinical trials and research studies – Study-specific Case Report Forms – Various definitions for same items – Comparison between studies difficult Systematically collect, analyze, share data Decrease study start-up time and cost Facilitate data sharing and comparisons across studies

8. What is a Common Data Element (CDE)? A logical unit of data, pertaining to one kind of information Has a name, precise definition, and clear enumerated values (codes) if applicable Example – Element name: Year of initial diagnosis – Definition: Year physician first diagnosed disorder – Code value: YYYY (4-digit numeric)

9. Objectives of CDE Project Identify CDEs – Age, sex, race, handedness, etc. Standardize CDE format Develop common documentation (definitions, permissible values, etc.) Create standardized case report forms

10. NINDS CDE Disease Areas Epilepsy Headache Mitochondrial disorders Movement disorders Parkinson’s disease Huntington’s disease Multiple sclerosis Spinal cord injury Stroke Neuromuscular disorders ALS Friedreich’s ataxia Muscular dystrophies – Becker, congenital, Duchenne, FSH Myasthenia Gravis Myotonic dystrophy Spinal muscular atrophy Traumatic brain injury Completed Under development Planned General CDEs

11. General Core CDEs (used by most, if not all, studies) Disease-specific Core CDEs (used by all studies in a specific disease) Disease-specific Supplemental CDEs (extended set; “commonly” collected, but not core) Disease-specific Exploratory elements (not yet validated, under development) Links to other NINDS repositories Classification of CDEs

12. Impact of the CDE Project Reduce time/cost to develop data collection tools Reduce study start-up time Promote data collection in a consistent format Improve data quality Facilitate comparisons between studies and meta-analyses

13. Who is involved in the CDE Project? Working groups of international experts in disease area Identify instruments most likely to be used in clinical research studies Develop CDEs: common methods for recording information collected

14. Example List subgroups Parkinson’s disease subgroups General and MotorEpidemiology / Environment PsychiatryFunctional Neurosurgery CognitiveOther Non-Motor ImagingQuality of Life NeuropathologyOperations GeneticsStatistics and Scale Metrics

15. What is the CDE Process? Internal Working Group review period (revisions as needed) Develop recommendations Core Supplemental Exploratory Recommendations posted for public comment (revisions as needed) CDEs posted for public use ~ 8 mo ~2 mo

16. What is the CDE Process? Internal Working Group review period (revisions as needed) Develop recommendations Core Supplemental Exploratory Recommendations posted for public comment (revisions as needed) CDEs posted for public use ~ 8 mo ~2 mo Iterative process Review and update recommendations

17. NINDS CDE Products Recommendations – CDEs Template CRFs Data dictionaries – Existing instruments (copyright) Additional documentation – Summary tables – Guidelines for use (e.g., screening, rating, diagnostic)

18. How to find the CDEs Search internet for “NINDS CDE” www.commondataelements.ninds.nih.gov

20. Click “Tools” to access CDE catalog and CRF library

21. CDE Catalog

22. CRF Library

23. Collaborative effort: Development and implementation Hundreds of disease experts worldwide Other NIH institutes National Library of Medicine Harmonization with international data standards CDISC (PD CDEs will be CDISC standard) NINDS funded studies Non-profits / foundations

24. Impact on Clinical Care and Practice A series of recommendations for data collection across clinical research in neurological disorders has been developed and are publically available Use of the CDEs will increase efficiency by harmonizing data collection across the research community The NINDS encourages use of the CDEs by investigators world-wide who are conducting clinical research studies

25. THANK YOU