Standardized Study Data:
An Update
Presented at the
DCDISC Meeting
Ron Fitzmartin, PhD, MBA
Office of Strategic Programs
Center for Drug Evaluation and Research
U.S. Food and Drug Administration
25 April 2014
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.
2
Steps Toward the Submission of Electronic
Standardized Study Data
3
Data Standards @
the Foundation of Review
Regulatory
Review
Validation
Communication
Common Tools
Traceability
Predictability
Data Quality
Protocol
Data Capture
Data Standards
CSR
Submission
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A Glimpse at Progress:
Standardized Study Data in FDA
Fiscal Year 2013:
Number and Percent of NDAs with
Study Data Submissions in CDISC SDTM*
# of
Submissions
% with
CDISC SDTM Data
New Unique NDAs**
98
61%
All NDAs
223
55%
NDAs
* 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
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Data Standards Governance
Strategy, Policy &
Guidance
Communication
Support Guides
Data Standards
Catalog
Help
External Collaborations*
Project Operations
Web
Pages
eData
Team
Automated Review
Environment
CSC
* examples
6
“…develop
standardized clinical
FDASIA
Re-authorized
PDUFA*
final guidance specifying
data terminology
through open
standards
“… periodically publish
the completed data
PDUFA V
Goals – Section
XII
standards, formats,
and terminologies that
development
sponsors must use to
organizations (i.e.,
submit data in
CDISC)”
applications.”
*PDUFA: Prescription Drug User Fee Act
7
Draft Binding Guidances &Tech Guide
Public Comment Period Ends: 7 May 2014
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What is Binding Guidance?
•
Recommends,
Guidance =
• Suggests,
• Encourages
Binding
Binding
Guidance
Guidance =
has a bite!
“Please”
Do it!
• Requires
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eStudy Data Guidance
• Electronic submission of
standardized study data required
for:
–
–
–
–
Certain INDs
NDAs
ANDAs
Certain BLAs
• Including amendments,
supplements, and reports
10
Study Data Tech Conformance Guide
• Guide supplements the eStudy
guidance.
• Provides technical
recommendations / specifications
for “e” submission of standardized
data.
• Consolidates Common Issues and
Study Data Specs + much more.
http://www.fda.gov/forindustry/datastandards/studydatastandards/default.htm
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eStudy Data Guidance
Data Standards Catalog
Dates
Requirement
Begins and Ends
12
Therapeutic Area (TA)
Standards Project Plan
13
FDA TA Standards Objectives
• Identify efficacy
endpoints
• Manageable
increments
• Implementation
Guidance
• Standardized Study
Data will become
Binding
Establish
FDA’s
requirements
Implement
in Guidance
to Industry
Open and
Transparent
Process
Express
Requirements
in Sustainable
Standards
• Collaborate with SDOs &
TA Experts
• Ensure Public Comment
• Adopt or adapt existing
TA data elements
• Harmonize with
healthcare standards, as
possible
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Collaboration on TA Standards
Development
Clinical /
Medical
Societies
Industry
joint
partnership
Academia
Government
active
participants
Coalition for Accelerating
Standards & Therapies
15
FDA’s Role in the TA Project Stages
• Scientific & Technical Input
• Planning / Prioritization
• Initial Expert Review
• FDA Division Expert
Review
• FDA Testing /
Acceptance
• Guidance
16
FDA’s TA Efficacy Requirements Process
Plan & Scope
with the
Review
Division
Efficacy
Requirements
Mapping &
Report
Communicate
Results to
CFAST
Acceptance
Testing of
CDISC
Published TA
Tech Guide
&
Data Standards
Catalog
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Early Look @ Status*of TA Initiative
• Schizophrenia
%
• Influenza 100
• COPD
90
• Pain**
80
•
• Alzheimer’s**
70
• Multiple Sclerosis
•
• Diabetes
60
• QT Studies
50
•
• Traumatic Brain
40
•
Injury
30
• Parkinson’s**
•
20
• Virology**
• Major Depressive 10 •
Disorder
0
•
• Tuberculosis**
• Polycystic Kidney
Disease*
• Asthma*
• Complicated intra~58 TAs
Rheumatoid
Arthritis
Diabetic
Nephropathy
Renal Transplant
Osteoporosis
Hepatitis C
OncologyBreast
Lipid Lowering
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
*Approved-Ongoing & Planned Projects or Published as of April 2014; **Published by CDISC
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Thank You
Ronald.Fitzmartin@fda.hhs.gov
and the Data Standards Team
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