1. CDISC – 17/12/2012 Carine Javierre Nathalie SABIN

2. AGENDA 1- CDISC organizational structure 2- Strategic Alliances 3- Overview of new regulatory procedure 4- Impacts on CDISC standards

3. CDISC organizational structure CDISC – 17/12/2012

4. CDISC organizational structure 1990: The International Conference on Harmonisation (ICH), Problem: need to internationalize the pharmaceutical market between the three areas :  Europe (EMA),  Japan (MHLW),  United States (FDA) ‏  Need to establish a common procedure for the registration of a new drug CDISC – 17/12/2012

5. CDISC organizational structure Founded 1997, incorporated 2000 Members of the organisation :  Pharmaceutical laboratories  Regulatory Agencies  CRO…  CDISC is a global, open, multidisciplinary, non-profit organization that has established standards to support :  “ Clinical Data Acquisition”,  “Clinical Data Exchange”,  “Clinical Data Submission”,  And “Clinical Data Archiving”. CDISC – 17/12/2012

6. CDISC organizational structure CDISC – 17/12/2012

7. CDISC Around the Globe 7 2000 2008 2002 2001 2010 CDISC – 17/12/2012

8. Strategic Alliances CDISC – 17/12/2012

9. BRIDG Model 3.0.3 Day 3 Advanced - 9 The goal of the BRIDG Model is to produce a shared view of the dynamic and static semantics for the domain of protocol-driven research and its associated regulatory artifacts

10. What is the relationship between CDISC and Health Level Seven (HL7)? CDISC has an Associate Charter Agreement with HL7 that was originally signed in 2001, renewed approximately every two years. – The Charter Agreement encourages a relationship that focuses on harmonization of the CDISC clinical research standards and the HL7 healthcare standards. – To this end, BRIDG was initiated in 2004 and is now a collaborative model (www.bridgmodel.org) governed by a Board comprised of CDISC, HL7, NCI (National Cancer Institute), NIH (National Intitutes of Health) and FDA. CDISC – 17/12/2012 The CDISC President and CEO, Rebecca Kush, was elected to the HL7 Board of Directors. The current HL7 CEO served 6 years on the CDISC Board before taking his HL7 CEO position. These relationships can facilitate understanding in terms of the importance and need to link healthcare and research through standards.

11. What is the relationship between CDISC and FDA? FDA's official position is that of "observer" when involved in CDISC activities. FDA representatives participate as liaisons on CDISC teams, present at CDISC conferences, present at other industry conference on the need for (CDISC) standards. FDA presentations have provided public endorsement of CDISC activities, including encouragement to those developing eSubmissions to use SDTM and ADaM. CDISC was awarded a contract from FDA to teaching reviewers and statisticians about CDISC; through this relationship, there are four different CDISC courses, including SDTM and ADaM, which have been taught now to over 300 reviewers. CDISC – 17/12/2012

12. Overview of new regulatory procedure CDISC – 17/12/2012

13. PDUFA REAUTHORIZATION PERFORMANCE GOALS AND PROCEDURES FISCAL YEARS 2013 THROUGH 2017 XII. IMPROVING THE EFFICIENCY OF HUMAN DRUG REVIEW THROUGH REQUIRED ELECTRONIC SUBMISSIONS AND STANDARDIZATION OF ELECTRONIC DRUG APPLICATION DATA E. Clinical Terminology Standards: Using a public process that allows for stakeholder input, FDA shall develop standardized clinical data terminology through open standards development organizations (i.e., the Clinical Data Interchange Standards Consortium (CDISC)) with the goal of completing clinical data terminology and detailed implementation guides by FY 2017. 1. FDA shall develop a project plan for distinct therapeutic indications, prioritizing clinical terminology standards development within and across review divisions. FDA shall publish a proposed project plan for stakeholder review and comment by June 30, 2013. FDA shall update and publish its project plan annually.

14. Impacts on CDISC standards CDISC – 17/12/2012

15. CDER/CBER’s - Background ~ 30% of unique New Drug Applications (NDA) received by CDER in 2011 submitted CDISC/SDTM data ~ 20% of the Biological License Applications (BLA) received by CBER in 2011 submitted CDISC/SDTM data Although standardization has allowed for use of additional data analysis tools, issues with either the implementation of the standard, or the standard itself, have proven to be inhibitive to the regulatory review process The Center for Biologics Evaluation and Research (CBER) is one of six main centers for the U.S. Food and Drug Administration (FDA), which is a part of the U.S. Department of Health and Human Services.U.S. Food and Drug AdministrationU.S. Department of Health and Human Services The Center for Drug Evaluation and Research (CDER, pronounced "see'-der") is a division of the U.S. Food and Drug Administration (FDA) that monitors most drugs as defined in the Food, Drug, and Cosmetic Act.U.S. Food and Drug AdministrationFood, Drug, and Cosmetic Act CDISC – 17/12/2012

16. Top 7 CDISC Standards Issues 1 - Waste of Space 2 - Extras 3 - Validation Errors 4 - Extended Codelists 5 - ISO Dates 6 - Traceability 7 - Inadequate Documentation CDISC – 17/12/2012

17. What is the FDA looking for…? CDISC – 17/12/2012

18. Q&A Thank you CDISC – 17/12/2012