Robin Feldman, MBA & Joy Esterlitz, MS NINDS CDE Team The Emmes Corporation Rockville, MD USA. Thursday, September 14, 2017

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1 The National Institute of Neurological Disorders and Stroke (NINDS) and American Academy of Cerebral Palsy and Developmental Medicine (AACPDM) Cerebral Palsy Common Data Elements (CDE) Recommendations Robin Feldman, MBA & Joy Esterlitz, MS NINDS CDE Team The Emmes Corporation Rockville, MD USA Disclosure Information AACPDM 71 st Annual Meeting September 11-16, 2017 Speaker Name: Joy Esterlitz, MS Disclosure of Relevant Financial Relationships: I have no financial relationships to disclose. Disclosure of Off-Label and/or investigative uses: I will not discuss off label use and/or investigational use in my presentation Thursday, September 14, Disclosure Information AACPDM 71 st Annual Meeting September 11-16, 2017 Speaker Name: Robin Feldman, MBA Disclosure of Relevant Financial Relationships I have no financial relationships to disclose. Disclosure of Off-Label and/or investigative uses: I will not discuss off label use and/or investigational use in my presentation Presentation Overview What is the National Institute of Neurological Disorders and Stroke (NINDS) Common Data Element Project? What are the Common Data Elements (CDEs) and associated Case Report Forms (CRFs)? What information does the NINDS CDE Website contain? What was the process to develop the Cerebral Palsy CDEs? 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. 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 CDEs are not a database

) Standardize CRFs and other data collection instruments Provide information to researchers for clinical data collection and sharing Overall Impact of the CDE Project Reduce the time and cost needed

2 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 Overall Impact of the CDE Project Reduce the time and cost needed to develop data collection tools CDE Goals Promote standardized, consistent and universal data collection Improve data quality and facilitate data sharing Improve opportunities for meta-analysis analysis and comparison of results from various studies 7 8 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 NINDS CDE Current Diseases Amyotrophic Lateral Sclerosis Cerebral Palsy Chiari Malformation Congenital Muscular Dystrophy Duchenne Muscular Dystrophy/Becker Muscular Dystrophy Epilepsy Facioscapulohumeral Muscular Dystrophy Friedreich s Ataxia Headache Version 2.0 Huntington s Disease Mitochondrial Disease Multiple Sclerosis Myasthenia Gravis Myotonic Dystrophy Neuromuscular Diseases Parkinson s Disease Spinal Cord Injury and Pediatric SCI Spinal Muscular Atrophy Sports Related Concussion Stroke Traumatic Brain Injury Unruptured Cerebral Aneurysms and Subarachnoid Hemorrhage 9 NINDS CDE Collaboration and Partnerships Cerebral Palsy American Academy for Cerebral Palsy and Developmental Medicine (AACPDM) Chiari I Malformation Chiari & Syringomyelia Foundation Unruptured Cerebral Aneurysms and Subarachnoid Hemorrhage (SAH) National Library of Medicine Sports-Related Concussion (SRC) U.S. Department of Defense What is a CDE? Standardized question and potential answers Semantic value with a CDE name, definition and permissible values Example: CDE name: Clinical event or milestone type Question Text: Milestone Definition: Type of clinical event or milestone pertinent to the disease or disorder Data Type: Alphanumeric Input Restriction: Multiple Pre-Defined Values Selected

Question on CRF: CDE Development Process CDE Details (subset of attributes of the CDE shown below): CDEs are identified, developed, and vetted by experts in the scientific community Development is

Committee meets to annually review and update CDEs 13 14 CDE DEVELOPMENT EVELOPMENT: : PROCESS (CONT CONT.

national conference or via web conference Subgroups meet every 3-5 weeks via conference call to develop CDEs for assigned areas Internal WG Review of all subgroups CDEs Subgroups revise CDEs based on

3 Question on CRF: CDE Development Process CDE Details (subset of attributes of the CDE shown below): 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 CDE DEVELOPMENT EVELOPMENT: : PROCESS (CONT CONT.) DEVELOPMENT STEP NINDS invites Working Group (WG) members and WG Chair(s) NINDS works with Chair(s) to divide WG into subgroups and to nominate subgroup Chair(s) Introductory meeting of WG at national conference or via web conference Subgroups meet every 3-5 weeks via conference call to develop CDEs for assigned areas Internal WG Review of all subgroups CDEs Subgroups revise CDEs based on feedback from internal WG review Public Review of WG s CDEs Subgroups review CDEs based on feedback from Public Review Post version 1.0 of CDEs on the website Total TYPICAL TIMEFRAME 2-4 weeks 2-4 weeks 1-2 hours 6-9 months 1 month 1-2 months 6-8 weeks 1 month 2-4 weeks months CDE DEVELOPMENT EVELOPMENT:PROCESS (CONT CONT.) Version 1.0 of the CDEs is not the end CDEs are dynamic and will evolve over time NINDS has created oversight committees to continually review the general and disease specific CDEs CDE Terminology Classifications Exploratory Supplemental Supplemental - Highly Recommended* Disease Core General Core * Classification term of Basic used for Traumatic Brain Injury CDEs NINDS CDE Disease Areas over 13,000 CDEs & 900 Instruments 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 Disease Listing 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) 17 3

CP CDE DEVELOPMENT EVELOPMENT:PROCESS CP CDE TIMELINE Initial CP CDE Project meeting held in October 2015 How were the CP CDEs developed?

review from September 2016 to October 2016 Version 1.0 CP CDEs were posted on NINDS CDE website (www.commondataelements.ninds.nih.

Palsy and Developmental Medicine (AACPDM) Seven Working Groups and a Steering Committee Participant Characteristics and Disease/Injury Related Events Health, Growth, Genetics, Comorbidities, and Labs

WORKFLOW AND CONCEPTUAL FRAMEWORK* * Schiarti et al.

4 CP CDE DEVELOPMENT EVELOPMENT:PROCESS CP CDE TIMELINE Initial CP CDE Project meeting held in October 2015 How were the CP CDEs developed? Individual working groups met via teleconference monthly for 6 9 months CP CDE working groups internally reviewed all the working groups in June 2016 CP CDEs posted to NINDS CDE Website for public review from September 2016 to October 2016 Version 1.0 CP CDEs were posted on NINDS CDE website ( on December 15, CEREBRAL PALSY CDE PROJECT ALSY CDE P Collaboration between the National Institutes of Neurological Disorders and Stroke (NINDS) and the American Academy of Cerebral Palsy and Developmental Medicine (AACPDM) Seven Working Groups and a Steering Committee Participant Characteristics and Disease/Injury Related Events Health, Growth, Genetics, Comorbidities, and Labs Neuroimaging Diagnostics Neuromotor Skill and Functional Assessments Neurocognitive, Social, and Emotional Assessments Engagement and Quality of Life Assessments Integrated Across WGs CP CDE PROJECT WORKFLOW AND CONCEPTUAL FRAMEWORK* * Schiarti et al. (Submitted for publication) CP CDE PROJECT ROJECT OVERALL PURPOSE Standardize data collection and assessment in studies of children and youth by: Indentify valid and reliable measures including assessment and evaluative tools to be recommended for routine use in research studies and clinical practice in all types of studies Recommend a set of CDEs and measures that comprehensively represent functional profiles across all domains of the International Classification of Functioning, Disability and Health (ICF) Identify CDEs/ measurement gaps that could be addressed in future research initiatives CEREBRAL PALSY SELECTION CRITERIA Selection criteria applied by all the working groups: Applicable to children and youth aged 0 to 18 years and Represents a relevant areas of study in CP and Has documented validity and reliability testing CDEs and instruments had to meet all selection criteria in order to be included in the CP CDEs set

5 NINDS CP CDE CLASSIFICATION CRITERIA CDEs Classification Core Supplemental Highly Recommended Supplemental Exploratory Definitions A data element for recording essential information applicable to any CP study including therapeutic areas and study designs. Consistent with all NINDS diseasespecific CDE sets A data element which is recommended for use whenever applicable Adataelementthathassomeevidenceof validity and is commonly collected in clinical studies in CP. Use depends upon the study design A data element that is emerging or that requires further validation in CP 25 CEREBRAL PALSY DOMAIN WORKING GROUPS DEVELOPMENT PROCESS All WGs reviewed existing CDEs and instruments defined from other diagnostic groups in the NINDS CDE project including: Friedrich s Ataxia Stroke Epilepsy Spinal Muscular Atrophy Duchenne Muscular Dystrophy/ Becker Muscular Dystrophy Traumatic Brain Injury 26 INTEGRATED ACROSS WORKING GROUPS Reviewed all the instruments recommended by the six domain Working Groups recommended: 226 measures were identified by the Working Groups 122 (54%) were proposed for inclusion in Version 1.0 CP CDEs. 34 classified as Supplemental Highly Recommended 61 classified as Supplemental 31 classified as Exploratory NUMBER OF INSTRUMENTS AND CRFS REVIEWED AND RECOMMENDED BY WORKING GROUPS Working Group Standardized Instruments (Number) Number of CRFs (Number) Participant Characteristics and 8 2 Disease/Injury Related Events Health, Growth, Genetics, Comorbidities, and Labs Neuromotor Skill and Functional 92 6 Assessments Neurocognitive, Social, and Emotional 66 N/A Assessments Engagement and Quality of Life 32 N/A Assessments Neuroimaging N/A 1 Total NINDS CDE Website Homepage NINDS CDE Website Navigation

Cerebral Palsy Data Standards Page Cerebral Palsy Case Report Form Case Report Form 31

Dictionary CDE 33 34 NeuroImaging Cerebral Palsy MRI CDE Details NINDS CDE CP Outcome

6 Cerebral Palsy Data Standards Page Cerebral Palsy Case Report Form Case Report Form Cerebral Palsy Case Report Form Cerebral Palsy Case Report Form CDE Details = Data Dictionary CDE NeuroImaging Cerebral Palsy MRI CDE Details NINDS CDE CP Outcome Measures Recommendations Notice of Copyright Question Text CDE Name Permissible Value

Cerebral Palsy: Notice of Copyright Instrument

Palsy Overview Page 39 40 Cerebral Palsy History

7 Cerebral Palsy: Notice of Copyright Instrument Recommendation NINDS CDE CP Outcome Measures Recommendations CDE Details = Data Dictionary Classification CDE Detailed Report Cerebral Palsy Overview Page Cerebral Palsy History and Acknowledgements Page Feedback and Suggestions Form

Resource Listing Document CP CDE Highlight Summary 45

8 Start-UP Resource Listing Cerebral Palsy CDE Start-Up Resource Listing Document 44 Cerebral Palsy CDE Start-Up Resource Listing Document CP CDE Highlight Summary 45 Cerebral Palsy CDE Highlight Summary Document NINDS CDE Website Navigation

Cerebral Palsy : CRF NINDS CDE Website Navigation 49 50 Cerebral Palsy:

CDE Name 51 52 NINDS CDE Website Learn Portal At the end of this month,

CRFs available for use through the NINDS CDE project website. Questions?

9 Cerebral Palsy : CRF NINDS CDE Website Navigation Cerebral Palsy: CDE Details Cerebral Palsy: Notice of Copyright Instrument Recommendation CDE Name NINDS CDE Website Learn Portal 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. Questions? NINDSCDE@emmes.com Support: This project was funded by HHSN C

TBI Research at NIH. Ramona Hicks, PhD NIH/NINDS

TBI Research at NIH. Ramona Hicks, PhD NIH/NINDS TBI Research at NIH Ramona Hicks, PhD NIH/NINDS Objectives 1. Overview of TBI research opportunities at NIH 2. Highlight a few projects Common Data Element Project Federal Interagency TBI Research Informatics