The document discusses CDISC standards and their implementation. It begins by asking what the CDISC standards are, when is the best time to implement them, and the timeline for requiring them in FDA submissions. It then explains that CDISC develops data standards for medical research through an open consensus process. Implementing standards early in a clinical program saves time and money compared to converting legacy data later. The FDA will require CDISC standards like SDTM and ADaM for submissions through binding guidance issued in 2012 as part of critical path initiatives and legislative acts. Resources are available to stay up to date on standards development.

1. What we need to know about CDISC
Shannon Labout, CCDM
Director, Data Management

2. WHAT WE NEED TO KNOW ABOUT CDISC
 What are the CDISC standards?
 What is the best timing to implement CDISC
standards in a clinical program?
 What is the timeline for requiring CDISC
Standards in FDA Submissions?

3. WHAT ARE THE CDISC STANDARDS
 CDISC organized as a non-profit standards
developing organization (SDO) in 2000
 CDISC works closely with other SDOs (e.g.,
HL7, ISO) and with the FDA
 CDISC develops standards for medical
research (started with volunteers in 1990s)
 CDISC standards development is an open,
consensus-based process, powered by
volunteers
 www.CDISC.org

4. http://www.cdisc.org/foundation-standards

5. TIMING OF IMPLEMENTATION MATTERS!
1. If you choose to convert your legacy data
to SDTM at the end of the process
Allows you to hold on to your old processes
and standards
But…
Adds significant time, effort, and expense at
the end of the process
Legacy data conversion can introduce other
significant problems

6. LEGACY DATA CONVERSION PROBLEMS
 No 1:1 relationship between CRF and SDTM
domains/variables means you will have to
spend a lot of time mapping data to the
right domains/variables
 Collected values may not map to controlled
terminologies that are required
 No standards definitions/assumptions make
the data less “combinable” – may not be
able to map with the same meaning

7. TIMING OF IMPLEMENTATION MATTERS!
2. Streamline your processes by building (industry)
standards into your process up-front:
 Saves time, money, effort
 Automatically generate deliverables throughout
 Build a standard library of CRFs, database structures,
SAS programming, etc.
 Reduce (or eliminates) mapping and re-programming
throughout the process
 Makes data more combinable, re-usable
 Allows data sharing with vendors and other research
partners more easily
 Supports FDA objectives
 Standard format for data submissions and data
repository
 Builds in transparency and traceability

8. IMPLEMENT STANDARDS FROM THE START
 ODM – designed to hold CDISC content plus
audit trail, based on XML
 PRM – structured protocol information makes it
reusable downstream (registries, EDC, CSR,
labeling), based on XML
 CDASH – standard metadata for CRFs is
harmonized with other CDISC standards –
makes it easier to get to SDTM and ADaM
 Controlled terminologies with standard
definitions
 Everyone collecting the same information the
same way makes that data more usable and
meaningful in a data repository

9. WILL CDISC STANDARDS BE REQUIRED?
Two Perspectives

10. YES! CDISC STANDARDS WILL BE REQUIRED
Supporting the Vision, Innovation and Opportunity
of the Critical Path Initiative

11. YES!! CDISC STANDARDS WILL BE REQUIRED

12. Whichever way we choose to look at it…
CDISC STANDARDS ARE NO
LONGER OPTIONAL FOR FDA
SUBMISSIONS

13. 2004
Critical Path
Initiative

14. http://www.fda.gov/ScienceResearch/SpecialT
opics/CriticalPathInitiative/CriticalPathOpport
unitiesReports/ucm077262.htm

15. CRITICAL PATH INITIATIVE
 The Critical Path Initiative (CPI) is FDA's
national strategy to drive innovation in the
scientific processes through which
medical products are developed,
evaluated, and manufactured.
 FDA was hoping that industry would
voluntarily adopt standards…

16. 2004 Critical 2006 Federal
Path Register
Initiative Notice

17. FEDERAL REGISTER NOTICE
 In 2006, FDA told us the requirement was
coming
all submissions to be electronic
all submissions to follow standard formats
(e.g., SDTM)
 They were still hoping we would voluntarily
adopt standards…

18. 2004 2006 2008 2010
Critical Federal Federal Federal
Path Register Register Register
Initiative Notice Notice Notice

19. WHAT’S HAPPENED SINCE THEN?
 ARRA $$ invested in FDA’s standards
implementation
 CDER Computational Science Center
 Cross-Center FDA Data Standards Council
 New Software and Tools
 New Review Environment
 Janus V1, V2, V3
 CDISC training developed for FDA
reviewers
 CDISC Standards are being built in at FDA

20. FDASIA INTRODUCES THE REQUIREMENT
2012
FDASIA and
PDUFA V

21. FDASIA
 Food and Drug Administration Safety and
Innovation Act of 2012
Signed into law July 2012
Became effective October 2012
a few key points
Reauthorizes PDUFA and MDUFA
Creates User Fees for Biosimilars and
Generics
Section 1136 requires electronic
submissions in a standard format to be
referenced in binding Guidance

22. PDUFA V IT PLAN
Objective
“FDA is committed to achieve the long-term
goal of improving the exchange, review,
and management of human drug and
biologic applications throughout the product
life cycle through strategic investments in
automated, standards-based information
technology (IT).”

23. BINDING GUIDANCE
FDA will issue final guidance no later than 12
months from the close of the public comment
period on the draft guidance. Such final
guidance and any subsequent revisions to
the final guidance shall be binding on
sponsors, applicants, and manufacturers no
earlier than twenty-four months after
issuance of the final guidance.

24. DRAFT GUIDANCE ISSUED 2012
 Harmonized cross-center
 CDER – Drug applications
 CBER – Biologics applications
 CDRH – Device / Radiological applications
 Specifies the requirement for electronic submission in
a standard format
 FDA publishes the standard formats they will accept
on their data standards web page
 References these CDISC standards
 SDTM - tabulations of collected data
 Controlled Terminology – allowed values
 ADaM – analysis datasets (supports CSR)
 SEND – pre-clinical data (based on SDTM)
 Define.xml – standard metadata format
http://www.fda.gov/ForIndustry/DataStandards/StudyDataStandards/default.htm

25. FREE WEBINAR “CDISC WILL BE REQUIRED!”
AVAILABLE ONLINE AT
HTTP://KESTRELCONSULTING.TRAININGCAMPUS.NET

26. DEVELOPING TA STANDARDS
 FDA has published intent to help industry create
therapeutic area (TA) specific standards by
2017 (PDUFA V commitment letter)
 High priority therapeutic areas published on
FDA website
(http://www.fda.gov/Drugs/DevelopmentApprovalProcess/Form
sSubmissionRequirements/ElectronicSubmissions/ucm287408.h
tm#_ftnref10)
 Working with CDISC and others to develop TA
 Standards development open to all who can
participate (http://www.cdisc.org/therapeutic)

27. FDA Timeline
This assumes standardization projects for therapeutic areas would be scoped narrowly enough
to be accomplishable within a 12-month period, and that subsequent projects in those areas
would build on the results.

28. CDISC Timeline

29. HOW WE CAN STAY UP TO DATE
 Monthly CDISC webinars
Updates on what’s being released for public
review – new domains, new terminology, etc
Free to attend live webinar
Archives are available on demand to CDISC
members
 CDISC Newsletter (monthly, free)
 CDISC website (www.cdisc.org)
 CDISC Education & Events
Private and public courses
Conferences (Interchange) in US, EU and AP

30. RESOURCES
 www.cdisc.org/foundational-standards - Starting
point for all the standards
 http://www.cdisc.org/content6283 - Technical
development plan (Gantt chart)
 http://www.fda.gov/ForIndustry/DataStandards/
StudyDataStandards/default.htm
 Email accounts for questions
(cber.cdisc@fda.hhs.gov, cder-edata@fda.hhs.gov,
cdrh.cdisc@fda.hhs.gov)
 http://www.opencdisc.org/
 Validation of your submission
 Help with Define.xml

31. TAKE AWAY MESSAGES
 It’s time to implement CDISC standards - no
longer optional for FDA submissions
Right now – FDA wants SDTM/ADaM
In the near future – FDA will require
SDTM/ADaM
 Update / improve your processes to take
full advantage of these industry standards
 Get involved with standards development
Contribute your TA-specific knowledge
Help develop the standards we all will have
to use

32. THANK YOU!
Shannon Labout, CCDM
Director, Data Management
slabout@sdcclinical.com