CDISC Electronic Submission to FDA

According to FDA Draft Guidance for Industry in Electronic Submission and Study Data Technical Conformance Guide, the pharmaceutical companies will need to provide CDISC Electronic submission to FDA. The paper will explain Data Standard Catalog which will dictate FDA Standards. The paper will discuss how to prepare CDISC electronic submission and what to prepare in CDISC electronic submission.

1. Accenture Accelerated R&D CDISC Electronic Submission to FDA Kevin Lee Clinical Data Strategies, Senior Consultant PharmaSUG 2014 Annual Conference

2. Disclaimer Any views or opinions presented in this presentation are solely those of the author and do not necessarily represent those of the company. 2 © 2014 Accenture All Rights Reserved.

3. Why? 3 © 2014 Accenture All Rights Reserved.

4. Agenda 4 © 2014 Accenture All Rights Reserved. Why do we care CDISC electronic submission? How can we prepare CDISC electronic submission? What do we prepare CDISC electronic submission? Conclusion Questions & Answers

5. Current Status in eSubmission in CDER FDA 5 © 2014 Accenture All Rights Reserved.

6. Current Status in CDISC Submission in CDER FDA 6 © 2014 Accenture All Rights Reserved.

7. Section 745A(a) of the Federal Food, Drug, and Cosmetic Act (FD&C Act) 7 © 2014 Accenture All Rights Reserved. Enhanced by Food and Drug Administration Safety and Innovation Act (FDASIA) on July 9, 2012. Requires that submissions be submitted in electronic format.

8. New FDA Draft Guidance on CDISC eSubmission 8 © 2014 Accenture All Rights Reserved. Guidance for Industry: Providing Regulatory Submissions in Electronic Format – Standardized Study Data Guidance for Industry: Providing Regulatory Submissions in Electronic Format – Submissions Under Section 745A (a) of the Federal Food Drug, and Cosmetic Act

9. How to prepare CDISC eSubmission 9 © 2014 Accenture All Rights Reserved. eCTD (Electronic Common Technical Document) Data Standard Catalog CDISC Standards Study Data Technical Conformance Guide

10. Electronic Common Technical Document 10 © 2014 Accenture All Rights Reserved.

11. Data Standards Catalog 11 © 2014 Accenture All Rights Reserved. Data Standards Catalog Standards Type and Version (i.e., eCTD , xml, SDTM 1.3, ADaM 2.1, Define.xml 2) FDA Center (i.e., CBER, CDER, CDRH) FDA Support start and end date FDA Requirement start and end date Standard Exchange Format Supported Version Implementation Guide Reference FDA Center Date Support Begins (yyyy -mm-dd) Date Support Ends ( yyyy-mm-dd) Date Requirement Begins Date Requirements Ends SDTM XPT 1.3 3.1.3 CBER, CDER 2012-12-01       SDTM XPT 1.2 3.1.2 Amendment 1 CBER, CDER 2013-08-07       SDTM XPT 1.2 3.1.2 CBER, CDER 2009-10-30       SDTM XPT 1.1 3.1.1 CBER, CDER Ongoing 2015-01-28     ADaM XPT 2.1 1.0 CBER, CDER Ongoing       SEND XPT 1.2 3.1.2 CDER 2011-06-13       Define XML 2.0 N/A CBER, CDER, CDRH 2013-08-07       SDTM 1.3 and SDTM IG 3.1.3 is accepted on CBER and CDER from 2012-12-01 on xpt format. SDTM 1.1 and SDTM IG 3.1.1 will be no longer accepted on CBER and CDER from 2015-01-28.

12. Timetables for Standards 12 © 2014 Accenture All Rights Reserved.

13. Standard Exchange Format Supported Version Implementation Guide Reference FDA Center Date Support Begins ( yyyy-mm-dd) Date Support Ends ( yyyy-mm-dd) Date Requirement Begins Date Requirements Ends SDTM XPT 4.1 CBER, CDER 2016-05-06   2018-03-15     Timetable Example (Update – 12 months Implementation) 13 © 2014 Accenture All Rights Reserved. Data Standards Catalog

14. Study Data Technical Conformance Guide 14 © 2014 Accenture All Rights Reserved.

15. What to p repare for CDISC eSubmission 15 © 2014 Accenture All Rights Reserved. CDISC components according to Data Standard Catalog Its electronic formats according to eCTD

16. CDISC Clinical Trial Process 16 © 2014 Accenture All Rights Reserved. PRN (Protocol) eCRF ODM.xml, LAB SDTM TFL ADaM SAP CDASH CSR CT Define.xml FDA Submission Components

17. Study Data Standardization Plan 17 © 2014 Accenture All Rights Reserved.

18. Study Data Reviewer’s Guide (SDRG) 18 © 2014 Accenture All Rights Reserved.

19. Format of Electronic Files according to eCTD 19 © 2014 Accenture All Rights Reserved.

20. Naming Conventions of Electronic Files According to eCTD 20 © 2014 Accenture All Rights Reserved.

21. pdf File Guidance according to eCTD 21 © 2014 Accenture All Rights Reserved.

22. xpt File Formats Guidelines 22 © 2014 Accenture All Rights Reserved.

23. Reports in module 5.3.1.1 of eCTD 23 © 2014 Accenture All Rights Reserved. CSR, SAP, Protocol, CRF

24. CDISC Datasets in module 5.datasets 24 © 2014 Accenture All Rights Reserved. ADaM xpt datasets, Define.xml, ADRG ADaM SAS programs, TFL SAS programs SDTM xpt datasets, Define.xml, SDTM annotated blank CRF, SDRG Any xpt datasets Patient profile

25. Waiver Criteria for FDA Standards 25 © 2014 Accenture All Rights Reserved. Discuss the waiver request with FDA prior to or at the pre-IND meeting and submit the request in writing prior to submission. A written waiver request The specific requirements that sponsors seek for waivers A necessary reason A description of the alternatives No Waivers, but sponsors may apply for it

26. FDA Support 26 © 2014 Accenture All Rights Reserved. – Study Data Technical Conformance Guide: non -binding – Sponsor can arrange pre -submission technical review on sample data Implementation Support – Study Data Standardization Plan – Data Standardization Issues Meeting with FDA on pre-IND or prior to end-of-phase 2

27. Conclusion 27 © 2014 Accenture All Rights Reserved. Before : “should” in FDA documents means that something is suggested or recommended, but not required. After : “should” in Guidance for Industry in Electronic Submission means required. It is better to start with the end in minds.

28. Contact Information 28 © 2014 Accenture All Rights Reserved. Email address : [email protected] Linkedin Profile : www.linkedin.com/in/HelloKevinLee/ Tweet : @ HelloKevinLee Slide share : http://www.slideshare.net/KevinLee56 Blogs : HiKevinLee.tumbrl.com

29. Questions and Discussion 29 © 2014 Accenture All Rights Reserved.