ninds cde team - aacpdmcp cde working groups internally reviewed all the working groups in june 2016...
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Robin Feldman, MBA & Joy Esterlitz, MS NINDS CDE Team
The Emmes CorporationRockville, MD USA
Thursday, September 14, 2017
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
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Disclosure InformationAACPDM 71st 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
Disclosure InformationAACPDM 71st Annual Meeting | September 11-16, 2017
Speaker Name: Robin Feldman, MBA
Disclosure of Relevant Financial RelationshipsI 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 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?
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What is the CDE Project?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.
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What are the goals of the CDE Project?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
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What are the objectives of the CDE Project?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
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Reduce the time and Reduce the time and cost needed to develop cost needed to develop
data collection toolsdata collection tools
Promote standardized, Promote standardized, consistent and consistent and universal data universal data
collectioncollection
Improve data quality Improve data quality and facilitate data and facilitate data
sharingsharing
Improve opportunities Improve opportunities for metafor meta--analysis and analysis and comparison of results comparison of results from various studiesfrom various studies
CDE GoalsCDE Goals
Overall Impact of the CDE ProjectOverall Impact of the CDE ProjectOverall Impact of the CDE ProjectOverall Impact of the CDE Project
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NINDS’ Collaborative Effort for CDE NINDS’ Collaborative Effort for CDE Development and ImplementationDevelopment 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 / FoundationsPharmaceuticals
NINDS CDE Current DiseasesNINDS CDE Current Diseases
Amyotrophic Lateral SclerosisCerebral Palsy
Chiari MalformationCongenital Muscular Dystrophy
Duchenne Muscular Dystrophy/Becker Muscular
DystrophyEpilepsy
Facioscapulohumeral Muscular Dystrophy
Friedreich’s AtaxiaHeadache Version 2.0Huntington’s DiseaseMitochondrial Disease
Multiple SclerosisMyasthenia Gravis
Myotonic DystrophyNeuromuscular Diseases
Parkinson’s DiseaseSpinal Cord Injury and Pediatric
SCISpinal Muscular Atrophy
Sports Related ConcussionStroke
Traumatic Brain InjuryUnruptured Cerebral Aneurysms and Subarachnoid Hemorrhage
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NINDS CDE Collaboration and PartnershipsNINDS CDE Collaboration and Partnerships
Cerebral Palsy–American Academy for Cerebral Palsy and Developmental Medicine (AACPDM)
Chiari I Malformation–Chiari & Syringomyelia FoundationUnruptured Cerebral Aneurysms and Subarachnoid Hemorrhage (SAH)–National Library of Medicine
Sports-Related Concussion (SRC)–U.S. Department of Defense
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What is a CDE?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”
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CDE Details (subset of attributes of the CDE shown below):CDE Details (subset of attributes of the CDE shown below):
Question on CRF:Question on CRF:
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CDE Development ProcessCDE 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
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CDE DCDE DEVELOPMENTEVELOPMENT: P: PROCESSROCESS ((CONTCONT.).)CDE DCDE DEVELOPMENTEVELOPMENT: P: PROCESSROCESS ((CONTCONT.).)
DEVELOPMENT STEPTYPICAL
TIMEFRAME
NINDS invites Working Group (WG) members and WG Chair(s) 2 - 4 weeks
NINDS works with Chair(s) to divide WG into subgroups and to nominate subgroup Chair(s) 2 - 4 weeks
Introductory meeting of WG at national conference or via web conference 1 - 2 hours
Subgroups meet every 3 - 5 weeks via conference call to develop CDEs for assigned areas 6 - 9 months
Internal WG ReviewInternal WG Review of all subgroups’ CDEs 1 month
Subgroups revise CDEs based on feedback from internal WG review 1 - 2 months
Public ReviewPublic Review of WG’s CDEs 6 - 8 weeks
Subgroups review CDEs based on feedback from Public Review 1 month
Post version 1.0 of CDEs on the website 2 - 4 weeks
Total 12 - 18 months
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Version 1.0 of the CDEs is not the end Version 1.0 of the CDEs is not the end –– CDEs are CDEs are dynamic and will evolve over timedynamic and will evolve over time
NINDS has created oversight committees to NINDS has created oversight committees to continually review the general and disease specific continually review the general and disease specific
CDEsCDEs
CDE DCDE DEVELOPMENTEVELOPMENT:: PPROCESSROCESS ((CONTCONT.).)CDE DCDE DEVELOPMENTEVELOPMENT:: PPROCESSROCESS ((CONTCONT.).)
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CDE Terminology CDE Terminology –– ClassificationsClassifications
Exploratory
Supplemental
Supplemental -Highly
Recommended*
Disease Core
General
Core* Classification term of “Basic” used for Traumatic Brain Injury CDEs
NINDS CDE Disease Areas NINDS CDE Disease Areas –– over 13,000 CDEs & 900 Instrumentsover 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 sclerosisSpinal 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)
Disease Listing
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How were How were the CP CDEs the CP CDEs developed?developed?
CP CDE DCP CDE DEVELOPMENTEVELOPMENT:: PPROCESSROCESSCP CDE DCP CDE DEVELOPMENTEVELOPMENT:: PPROCESSROCESS CP CDE TCP CDE TIMELINEIMELINEInitial CP CDE Project meeting held in October 2015
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 (www.commondataelements.ninds.nih.gov) on December 15, 2016
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
CCEREBRALEREBRAL PPALSYALSY CDE PCDE PROJECTROJECT
CP CDE PCP CDE PROJECTROJECT –– WWORKFLOWORKFLOW ANDAND CONCEPTUALCONCEPTUAL FRAMEWORKFRAMEWORK**
* Schiarti et al. (Submitted for publication)
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CP CDE PCP CDE PROJECTROJECT OOVERALLVERALL PURPOSEPURPOSEStandardize 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
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CCEREBRALEREBRAL PPALSYALSY SSELECTIONELECTION CCRITERIARITERIA
Selection criteria applied by all the working groups:
Applicable to children and youth aged 0 to 18 yearsand
Represents a relevant areas of study in CPand
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
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NINDS CP CDE CNINDS CP CDE CLASSIFICATIONLASSIFICATION CCRITERIARITERIA
CDEs Classification Definitions
Core
A data element for recording essentialinformation applicable to any CP studyincluding therapeutic areas and studydesigns. Consistent with all NINDS disease-specific CDE sets
Supplemental – Highly RecommendedA data element which is recommended foruse whenever applicable
Supplemental
A data element that has some evidence ofvalidity and is commonly collected inclinical studies in CP. Use depends uponthe study design
ExploratoryA data element that is emerging or thatrequires further validation in CP
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CCEREBRALEREBRAL PPALSYALSY DDOMAINOMAIN WWORKINGORKING GGROUPSROUPSDDEVELOPMENTEVELOPMENT PPROCESSROCESS
All WGs reviewed existing CDEs and instruments defined from other diagnostic groups in the NINDS CDE project including:Friedrich’s AtaxiaStroke EpilepsySpinal Muscular Atrophy Duchenne Muscular Dystrophy/
Becker Muscular Dystrophy Traumatic Brain Injury
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IINTEGRATEDNTEGRATED AACROSSCROSS WWORKINGORKING GGROUPSROUPSReviewed 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
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NNUMBERUMBER OFOF IINSTRUMENTSNSTRUMENTS ANDAND CRFCRFSS RREVIEWEDEVIEWEDANDAND RRECOMMENDEDECOMMENDED BYBY WWORKINGORKING GGROUPSROUPS
Working Group Standardized Instruments
(Number)
Number of CRFs
(Number)Participant Characteristics and Disease/Injury Related Events
8 2
Health, Growth, Genetics, Comorbidities, and Labs
28 11
Neuromotor Skill and Functional Assessments
92 6
Neurocognitive, Social, and Emotional Assessments
66 N/A
Engagement and Quality of Life Assessments
32 N/A
Neuroimaging N/A 1
Total 226 20
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NINDS CDE Website HomepageNINDS CDE Website Homepage
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NINDS CDE Website Navigation NINDS CDE Website Navigation
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Cerebral Palsy Data Standards PageCerebral Palsy Data Standards Page
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Cerebral Palsy Case Report FormCerebral Palsy Case Report Form
Case Report Form
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Cerebral Palsy Case Report FormCerebral Palsy Case Report Form
CDE
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Cerebral Palsy Case Report Form
CDE Details = Data Dictionary
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NeuroImagingNeuroImaging –– Cerebral Palsy Cerebral Palsy MRI CDE Details MRI CDE Details
Question Text
Permissible Value
CDE Name
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NINDS CDE CP Outcome Measures NINDS CDE CP Outcome Measures RecommendationsRecommendations
Notice of Copyright
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Cerebral Palsy: Notice of Copyright Cerebral Palsy: Notice of Copyright Instrument RecommendationInstrument Recommendation
Classification
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NINDS CDE CP Outcome Measures NINDS CDE CP Outcome Measures RecommendationsRecommendations
CDE Details = Data Dictionary
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CDE Detailed ReportCDE Detailed Report
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Cerebral Palsy Overview PageCerebral Palsy Overview Page
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Cerebral Palsy History and Acknowledgements PageCerebral Palsy History and Acknowledgements Page
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Feedback and Suggestions FormFeedback and Suggestions Form
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StartStart--UP Resource ListingUP Resource Listing
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Cerebral Palsy CDE StartCerebral Palsy CDE Start--Up Resource Listing Up Resource Listing DocumentDocument
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Cerebral Palsy CDE StartCerebral Palsy CDE Start--Up Resource Listing Up Resource Listing DocumentDocument
CP CDE Highlight SummaryCP CDE Highlight Summary
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Cerebral Palsy CDE Highlight Summary DocumentCerebral Palsy CDE Highlight Summary Document
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NINDS CDE Website NavigationNINDS CDE Website Navigation
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Cerebral Palsy : CRFCerebral Palsy : CRF
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NINDS CDE Website NavigationNINDS CDE Website Navigation
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Cerebral Palsy: CDE DetailsCerebral Palsy: CDE Details
CDE Name
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Cerebral Palsy: Notice of Copyright Cerebral Palsy: Notice of Copyright –– Instrument RecommendationInstrument Recommendation
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NINDS CDE Website Learn PortalNINDS CDE Website Learn Portal
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Support: This project was funded by HHSN271201200034C.
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.