1. NINDS CDE Team The Emmes Corporation
A Common Data Language for Clinical Research Studies: Five (5) New Disease Recommendations from the NINDS/NIH Common Data Element Project
Joy Esterlitz, MS
NINDS CDE Team The Emmes Corporation
Rockville, MD USA
Monday, May 8, 2017
Good afternoon. My name is Joy Esterlitz and I will be speaking about Common Data Elements today. Several years ago the National Institute of Neurological Disorders, or NINDS, began the Common Data Element Project. Today I will address the recent additions and chang es to this important project.

2. Presentation Overview
What is the National Institute of Neurological Disorders and Stroke (NINDS) Common Data Element Project?
What are the 5 new disease areas and who are the collaborators?
What are the Common Data Elements (CDEs) and associated Case Report Forms (CRFs)?
What information does the NINDS CDE Website contain?

3. What is the CDE Project?
NINDS initiated the development of CDEs as part of a project to develop data standards for funded clinical research in neuroscience.
The CDEs are content standards that can be applied to various data collection models and are intended to be dynamic and may evolve over time.
CDEs are not a database.

4. What are the goals of the CDE Project?
Develop common definitions and standardize case report forms (CRFs) and other instruments
Help investigators conduct clinical research through the development of these uniform formats by which clinical data can be systematically collected, analyzed and shared across the research community

5. What are the objectives of the CDE Project?
Identify or develop CDEs used in clinical research
(age, gender, race, etc.)
Present data elements in a standard format available to all
Identify common definitions
(including permissible values, range checks, etc.)
Standardize CRFs and other data collection instruments
Provide information to researchers for clinical data collection and sharing

6. Overall Impact of the CDE Project
Reduce time/cost to develop data collection tools
Reduce study start-up time and cost of overall trial
Improve data quality
Facilitate collection of data
Facilitate data sharing/comparisons between studies and meta-analyses
Promote data standards
Provide information to researchers for database development
Promote reuse of research questions/instruments
Promote data collection in a consistent format
Promote sharing and interpretation of research data
Other data standards out there were not specific enough for NINDS-funded clinical research
Lack of data standards – many studies funded, each with own unique forms and data elements
Limited dollars – constrained funding requires efficiency
NIH Data Sharing Policy – CDEs provide the foundation
Multitude of data formats creates barriers to data sharing
Meta-analysis often requires extensive data re-formatting
Motivation
Trials were costing too much: no one believed in re-use of CRFs
Trials were taking too long and costing too much to get up and going
Data quality varied, no standards
Data collection was not consistent
Comparisons or merging of data between studies were not possible

7. NINDS’ Collaborative Effort for CDE Development and Implementation
Expertise from hundreds of disease specialists worldwide
Other NIH institutes and federal agencies such as the National Library of Medicine
International data standards (e.g., ISO)
Clinical Data Interchange Standards Consortium (CDISC)
Non-profits / foundations
Pharmaceuticals

8. NINDS CDE Collaboration and Partnerships
First new disease area – Cerebral Palsy
Cerebral Palsy CDE Working Group
Partnership with American Academy for Cerebral Palsy and Developmental Medicine (AACPDM)
38 subject matter experts
7 subgroups
Of the 5 new diseases, 4 of them had collaborators…

9. NINDS CDE Collaboration and Partnerships
Second new disease area – Chiari I Malformation
Chiari I Malformation CDE Working Group
Partnership with Chiari & Syringomyelia Foundation
54 subject matter experts
6 subgroups
Of the 5 new diseases, 4 of them had collaborators…

10. NINDS CDE Collaboration and Partnerships
Third new disease area - Unruptured Cerebral Aneurysms and Subarachnoid Hemorrhage (SAH)
SAH CDE Working Group
Partnership with National Library of Medicine
83 subject matter experts
8 subgroups
Of the 5 new diseases, 4 of them had collaborators…

11. NINDS CDE Collaboration and Partnerships
Fourth new disease area – Sports-Related Concussion (SRC)
SRC CDE Working Group
Partnership with U.S. Department of Defense
32 subject matter experts
3 subgroups
Of the 5 new diseases, 4 of them had collaborators…

12. NINDS CDE Collaboration
Fifth disease area - Headache Version 2.0
Headache CDE Working Group
41 subject matter experts
5 subgroups
Why Version 2.0?

13. What is a CDE? Standardized question and potential answers
Semantic value with a CDE name, definition and permissible values
Example:
CDE name: “Tobacco current use indicator”
Definition: “Indicator for whether the participant/subject regularly uses tobacco products…”
Data Type: “Alphanumeric”
Input Restriction: “Single Pre-Defined Values Selected”
A data element is not necessarily the smallest unit of data; it can be a unique combination of one or more smaller units. A data element occupies the space provided by field(s) on a paper/electronic case report form (CRF) or field(s) in a database record.
The CDEs are content standards that can be applied to various data collection models and are intended to be dynamic standards that may evolve over time. The goal of the NINDS CDE Project is to develop standard definitions, code lists, and instructions for the CDEs so that investigators collect them consistently across studies. CDEs are the foundation for interoperability among data systems and are a subset of the universe of all data elements.

14. Question on CRF:
CDE Details (subset of attributes of the CDE shown below):

15. NINDS CDE Disease Areas – over 13,000 CDEs & 800 Instruments
Disease Listing
Chiari I Malformation (new)
Cerebral palsy (new)
Epilepsy*
Headache (version 2.0 new)
Mitochondrial disorders*
Movement disorders
Parkinson’s disease
Huntington’s disease
Multiple sclerosis
Spinal cord injury (SCI)*
Stroke*
Unruptured Cerebral Aneurysms and Subarachnoid hemorrhage (new)
* Includes pediatric-specific recommendations
Traumatic brain injury*
Sports-Related Concussion (new)
Neuromuscular disorders*
Amyotrophic lateral sclerosis
Friedreich’s ataxia
Muscular dystrophies
Congenital, Duchenne/Becker, Facioscapulohumeral, Myotonic
Myasthenia gravis
Spinal muscular atrophy
Myalgic Encephalomyelitis/Chronic Fatigue Syndrome (ME/CFS) (in development)
Biomechanical Sensors in Traumatic Brain Injury (in development)
The NINDS CDE Diseases and Disorders that have CDE recommendations are listed here. Those which are under in red are in development.
OTHER POTENTIAL CDE EFFORTS:
Essential tremors
Loss of consciousness
Chronic Fatigue Syndrome
TBI:
Blast sensory
Sport concussion
Charcot-Marie-Tooth disease

16. CDE Development Process
CDEs are identified, developed, and vetted by experts in the scientific community
Development is transparent and inclusive
NINDS and NINDS CDE Team provide continuous support and guidance
Oversight Committee (OC) will be formed to help maintain disease-specific CDEs
Process is iterative – Steering Committee meets to annually review and update CDEs

17. CDE Development Process - Timeline
Development Step
Typical Timeframe
Introductory Working Group (WG) Web conference meeting
1-2 hours
Subgroups meet every 3-5 weeks via conference call to develop CDEs for assigned areas
6-9 months
Internal WG Review of all subgroups’ CDEs
1 -2 months
Subgroups revise CDEs based on feedback from Internal WG Review
1 month
Public Review of WG’s CDEs
6 weeks
Subgroups revise CDEs based on feedback from Public Review
1-2 months
Post Version 1.0 of CDEs on Web site
TOTAL
12-18 months
We have done this entire process in 6 months

18. Sports-Related Concussion Development Process
The National Institute of Neurological Disorders and Stroke, National Institutes of Health and Department of Defense initiated development of Traumatic Brain Injury Sports-Related Concussion CDEs as a joint effort
SRC CDE Working Group met in August for orientation meeting and met regularly in subgroups from October 2016 – April 2017
This is an example of the development process

19. Sports-Related Concussion CDE Subgroups
Timeframe
Acute Subgroup
Time of Injury until 72 hours
Subacute Subgroup
After 72 hours to 3 months
Persistent/Chronic
3 months and greater post concussion
Already mentioned on previous slides
32 subject matter experts
3 subgroups

20. Sports-Related Concussion on the NINDS CDE Website
First set of CDEs and recommendations for SRC was developed and posted for public review in January 2017.
Version 1.0 of the SRC recommendations will be posted to the NINDS CDE WEBSITE ( in June 2017.

21. NINDS CDE Website Homepage

22. NINDS CDE Website Navigation
The project overview states objectives and goals for the NINDS CDE project. It also provides links to the NINDS Office of Clinical Research and provides a downloadable Overview and Brochure. Links for more information are also presented.

23. NINDS CDE Website Navigation
Next, the CDE Catalog presents a way to quickly search for CDEs within the system. CDEs can be search by Disease, Sub-Disease, Domain, Sub-Domain, Case Report Form (CRF), Classification, CDE Name or Keyword. Copyright and population are also choices. Results are displayed below after the Search button is clicked. Results can be downloaded by clicking on the “CDE Detailed Report” link.

24. NINDS CDE Website Navigation
The CRF Library provides a way to search for CRFs by Disease, Sub-Disease, Domain, Sub-Domain, Copyright status or Keyword. Results are displayed below after the Search button is clicked. Clicking on the links in the search results will provide a report of CDEs for that CRF that can be downloaded.

25. Cerebral Palsy CDE Highlight Summary Document

26. Cerebral Palsy: Notice of Copyright – Instrument Recommendation

27. NINDS CDE Website Learn Portal
The Learn Portal shows a variety of associated information to learn more about NINDS CDEs and the process of development.

28. Questions? Joy Esterlitz, MS The Emmes Corporation
At the end of this month, there will be more than 20 disease areas with over 13,000 CDEs and 900 CRFs available for use through the NINDS CDE project website.
Joy Esterlitz, MS
The Emmes Corporation
Rockville, Maryland, USA
Support: This project was funded by HHSN C.
Questions?