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Standardized Study Data: An Update
Presented at theDCDISC Meeting
Ron Fitzmartin, PhD, MBAOffice of Strategic ProgramsCenter for Drug Evaluation and ResearchU.S. Food and Drug Administration
25 April 2014
2
Disclaimer
The views and opinions presented here represent those of the speaker and should not be considered
to represent advice or guidance on behalf of the Food and Drug Administration.
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Steps Toward the Submission of Electronic Standardized Study Data
Data Standards @ the Foundation of Review
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Traceability
Data Quality
Regulatory Review
Data Standards Protocol Data Capture CSR Submission
Valid
ation
Pred
ictability
Co
mm
on
To
ols
Co
mm
un
ication
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Fiscal Year 2013:Number and Percent of NDAs with
Study Data Submissions in CDISC SDTM*
NDAs# of
Submissions % with
CDISC SDTM Data
New Unique NDAs** 98 61%
All NDAs 223 55%
* One or more explicitly stated SDTM studies (or study data structure that resembled SDTM).
**New unique NDAs with data submissions (not previously submitted).Source: Office of Business Informatics
A Glimpse at Progress:Standardized Study Data in FDA
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Data Standards Governance
Automated ReviewEnvironment
Communication
Support GuidesData Standards Catalog
HelpHelp
Web Pages
eDataTeam
* examples
External Collaborations*
CSC
Project Operations
Strategy, Policy & Guidance
PDUFA V Goals – Section
XII
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“…develop
standardized clinical
data terminology
through open
standards
development
organizations (i.e.,
CDISC)”
“… periodically publish
final guidance specifying
the completed data
standards, formats,
and terminologies that
sponsors must use to
submit data in
applications.”
FDASIA Re-authorized
PDUFA*
*PDUFA: Prescription Drug User Fee Act
Draft Binding Guidances &Tech Guide
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Public Comment Period Ends: 7 May 2014
What is Binding Guidance?
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Binding Guidance =
Guidance = • Recommends,• Suggests, • Encourages
• Requires
Do it! Binding
Guidance has a bite!
“Please”
• Electronic submission of standardized study data required for: – Certain INDs– NDAs– ANDAs– Certain BLAs
• Including amendments, supplements, and reports
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eStudy Data Guidance
Study Data Tech Conformance Guide
11http://www.fda.gov/forindustry/datastandards/studydatastandards/default.htm
• Guide supplements the eStudy guidance.
• Provides technical recommendations / specifications for “e” submission of standardized data.
• Consolidates Common Issues and Study Data Specs + much more.
Data Standards Catalog
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eStudy Data Guidance
Dates Requirement
Begins and Ends
Therapeutic Area (TA)Standards Project Plan
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• Adopt or adapt existing TA data elements
• Harmonize with healthcare standards, as possible
• Implementation Guidance
• Standardized Study Data will become Binding
• Collaborate with SDOs & TA Experts
• Ensure Public Comment
• Identify efficacy endpoints
• Manageable increments
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FDA TA Standards Objectives
Express Requirements in Sustainable
Standards
Open and Transparent
Process
Establish FDA’s
requirements
Implement in Guidance to Industry
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Collaboration on TA Standards Development
Academia Government
IndustryClinical / Medical
Societiesjoint
partnership
active participants
Coalition for Accelerating Standards & Therapies
FDA’s Role in the TA Project Stages
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• Scientific & Technical Input• Planning / Prioritization• Initial Expert Review
• FDA Division Expert Review
• FDA Testing / Acceptance
• Guidance
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FDA’s TA Efficacy Requirements Process
Plan & Scope with the Review Division
Efficacy Requirements
Mapping & Report
Acceptance Testing of
CDISC Published TA
Tech Guide &
Data Standards Catalog
Communicate Results to
CFAST
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• Multiple Sclerosis• Diabetes• QT Studies• Traumatic Brain
Injury• Parkinson’s**• Virology**• Major Depressive
Disorder
• Schizophrenia• Influenza• COPD• Pain**• Alzheimer’s**
• Tuberculosis**• Polycystic Kidney
Disease*• Asthma*
• Rheumatoid Arthritis
• Diabetic Nephropathy
• Renal Transplant• Osteoporosis
• Hepatitis C• Oncology-
Breast• Lipid Lowering
• Complicated intra-abdominal infection
• Complicated skin infections
• ADHD• Irritable Bowel Disorder• Anticonvulsants• Comp Urinary Infection
• Anticoagulants for Atrial Fibulation
• Prevention of Pregnancy• Treatment of Erectile
Dysfunction• Acne
Early Look @ Status*of TA Initiative
*Approved-Ongoing & Planned Projects or Published as of April 2014; **Published by CDISC
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10
20
30
40
50
60
70
80
90
100 ~58 TAs%
