1. 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

2. 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.

3. 3 Steps Toward the Submission of Electronic Standardized Study Data

4. Data Standards @ the Foundation of Review 4 Traceability Data Quality Regulatory Review Data Standards Protocol Data Capture CSRSubmission Validation Predictability Common Tools Communication

5. 5 Fiscal Year 2013: Number and Percent of NDAs with Study Data Submissions in CDISC SDTM* * 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

6. 6 Data Standards Governance Automated Review Environment Communication Support Guides Data Standards Catalog Help Web Pages eData Team * examples External Collaborations * CSC Project Operations Strategy, Policy & Guidance

7. PDUFA V Goals – Section XII 7 “…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

8. Draft Binding Guidances &Tech Guide 8 Public Comment Period Ends: 7 May 2014

9. What is Binding Guidance? 9 Binding Guidance = Recommends, Suggests, Encourages Requires Do it! Binding Guidance has a bite! “Please”

10. Electronic submission of standardized study data required for: –Certain INDs –NDAs –ANDAs –Certain BLAs Including amendments, supplements, and reports 10 eStudy Data Guidance

11. Study Data Tech Conformance Guide 11 http://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.

12. Data Standards Catalog 12 eStudy Data Guidance Dates Requirement Begins and Ends

13. Therapeutic Area (TA) Standards Project Plan 13

14. 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 14 FDA TA Standards Objectives Express Requirements in Sustainable Standards Open and Transparent Process Establish FDA’s requirements Implement in Guidance to Industry

15. Collaboration on TA Standards Development Academia Government Industry Clinical / Medical Societies joint partnership active participants Coalition for Accelerating Standards & Therapies 15

16. FDA’s Role in the TA Project Stages 16 Scientific & Technical Input Planning / Prioritization Initial Expert Review FDA Division Expert Review FDA Testing / Acceptance Guidance

17. 17 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 Catalog Communicate Results to CFAST

18. 18 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 ArthritisRheumatoid Arthritis Diabetic NephropathyDiabetic Nephropathy Renal TransplantRenal Transplant OsteoporosisOsteoporosis Hepatitis CHepatitis C Oncology- BreastOncology- Breast Lipid LoweringLipid Lowering Complicated intra- abdominal infectionComplicated intra- abdominal infection Complicated skin infectionsComplicated skin infections ADHDADHD Irritable Bowel DisorderIrritable Bowel Disorder AnticonvulsantsAnticonvulsants Comp Urinary InfectionComp Urinary Infection Anticoagulants for Atrial FibulationAnticoagulants for Atrial Fibulation Prevention of PregnancyPrevention of Pregnancy Treatment of Erectile DysfunctionTreatment of Erectile Dysfunction AcneAcne Early Look @ Status*of TA Initiative *Approved-Ongoing & Planned Projects or Published as of April 2014; **Published by CDISC ~58 TAs %

19. Thank You Ronald.Fitzmartin@fda.hhs.gov and the Data Standards Team Ronald.Fitzmartin@fda.hhs.gov 19