Updates on CDISC Activities Sergiy Sirichenko DC CDISC User Group Meeting, DC, 2014-03-20

Outlines Standards Updates Updates from FDA Upcoming events Other SDTM 1.4/IG3.2 Therapeutics: Multiple Sclerosis, Alzheimer, MCI and Asthma Dataset-XML CDASH SAE Updates from FDA New FDA Guidances on Standard Data for Electronic Submissions Define.xml v2 SI units Upcoming events CDISC Interchanges and User Groups PharmaSUG Other

New SDTM version December, 2013 http://www.cdisc.org/sdtm SDTM v1.4 Version of new SDTM IG is 3.2, rather than 3.1.4 New design of SDTM IG document as PDF portfolio  New IG-AP describes how to handle data about Devices “Associated persons” who are not study subjects New variables were added into SDTM model New special purpose domains 11 new domains in IG

New domains in SDTM IG 3.2 Interventions Events Findings Trial Design EC – Exposure as Collected PR – Procedures Events HO – Healthcare Encounters Findings DD – Death Details IS – Immunogenicity Specimen Assessment MI – Microscopic Findings MO – Morphology RP – Reproductive System Findings SS – Subject Status SR – Skin Response Trial Design TD – Trial Disease Assessment

UG for Multiple Sclerosis V1.0 February, 2014 http://www.cdisc.org/therapeutic Draft version 1.0 based on SDTM v1.4 Comments Due April, 1st Examples use the following SDTM domains Examples cover CE – Procedure Agents MH – Medical History MO – Morphology NV – Nervous System Findings * OE – Ophthalmic Exams * FA – Findings About TS – Trial Summary DI – Device Identifiers * * DU – Device In-Use * * Note: * Domain was not published in SDTMIG v3.1.3 and is not final; * SDTMIG for Medical Devices Medical History and Disease Course Relapse Visual Acuity and Contrast Sensitivity Optical Coherence Tomography Imaging Visual Evoked Potential

UG for Alzheimer’s Disease and Mild Cognitive Impairment V2.0 December, 2013 http://www.cdisc.org/therapeutic Provisional version Based on SDTM v1.4 Examples are added for CSF biomarker and imaging biomarkers endpoints QS Terminology Ten additional clinical scales relevant to AD and MCI Examples are adjusted with new SDTM/SDTM IG versions; Family History examples moved from MH to APMH (Associated Persons Medical History) domain ApoE genetics data examples are moved from SC to PF (Pharmacogenomics Findings) domain

Therapeutic Area Data Standards User Guide for Asthma v1.0 November, 2013 http://www.cdisc.org/therapeutic Provisional version based on SDTM v1.4 Examples use the following SDTM domains AG – Procedure Agents** CM – Concomitant Medications AE – Adverse Events HO – Healthcare Encounters* MH – Medical History LB – Laboratory Test Results RE – Respiratory System Findings** FA – Findings About SR – Skin Response* DI – Device Identifier* DU – Device-in-Use* Note: * Domain is new in SDTMIG v3.2; ** Domain was not published in SDTMIG v3.1.3 and is not final.

http://www.cdisc.org/stuff/contentmgr/files/0/2356ae38ac190ab8ca4ae0b222392b37/misc/cfast_ta_program_overview_february_2014.pdf

CDASH Serious Adverse Event Supplement Version 1 November, 2013 http://www.cdisc.org/cdash Final version Extends AE domain to include data elements needed for SAE info Will allow the sponsor to generate E2B message for reporting ICSR Allows to skip data re-entry

Dataset-XML Specification V1.o November, 2013 http://portal.cdisc.org/CT/Review%20Documents/Forms/AllItems.aspx Dataset-XML Was developed as an alternative to SAS Xport Require define.xml to read data FDA is running an evaluation pilot

All “CDISC Standards Latest Updates” http://www.cdisc.org/

New FDA Guidances on Standard Data for Electronic Submissions Study Data Technical Conformance Guide and Data Standards Catalog Guidance on Electronic Submissions: Standardized Study Data Guidance on Submissions in Electronic Format--Submissions under the Federal Food, Drug, and Cosmetic Act February, 2014 Draft Guidance 90 days period for public comments

Study Data Technical Conformance Guide New expected documents Study Data Standardization Plan Study Data Reviewer’s Guide Additional requirements. E.g., “The Subject Elements domain should be included…” “The AE domain should include all adverse events that were recorded in the subjects’ case report forms, regardless of whether the sponsor determined that particular events were or were not treatment-emergent.” New Sections. E.g., #8. Data Fitness Study Data Validation Rules Conformance (data standards) Quality (support meaningful analysis) Study Data Traceability Legacy Data Conversion Plan and Report aCRFs for legacy data elements “Record significant data issues, clarifications, explanations of traceability, and adjudications in the Data Guide”

CDER/CBER Position on Use of SI Units for Lab Tests October, 2013 http://www.fda.gov/ForIndustry/DataStandards/StudyDataStandards/ucm372553.htm “CDER and CBER are evaluating an approach to transition to general acceptance of laboratory data in clinical trials that are measured and reported in Système International (SI) units instead of U.S. Conventional units. The objective is to establish an agency-wide policy on the acceptance of SI units in product submissions. … While this evaluation is underway, sponsors are strongly encouraged to solicit input from review divisions as early in the development cycle as possible to minimize the potential for conversion needs during NDA/BLA review. CDER and CBER encourage sponsors to discuss this issue with FDA before the start of Phase 3 trials.  In some cases the issue may warrant discussion with FDA at the End-of-Phase 2 meeting. …”

FDA is now accepting Define.xml v2.0 http://www.fda.gov/ForIndustry/DataStandards/StudyDataStandards/default.htm

CDISC Meetings 2014 Interchange Europe : April, 7th - 11th, Paris, France USA: November, 10th – 14th, North Bethesda, MD Use Groups Boston: March, 28th, hosted by PAREXEL, Bilirica, MA Oncology Area Standards CDISC End-to-End Atlantic: April, 17th, hosted by Celgene, Summit, NJ 6 presentations Webex available Details including agenda and registration info on http://cdiscportal.digitalinfuzion.com/CDISC%20User%20Networks/default.aspx

PharmaSUG June 1st-4th, San Diego, CA http://www.pharmasug.org/us/index.html The Pharmaceutical Industry SAS® Users Group Annual Conference Online Registration is open Lowest fees until April, 28th Paper abstracts are published Special “Data Standards” section (19 papers)

Other Instem is sharing their sample SEND data Contact Jennifer Feldmann submit@instem.com for details. Inlcude "Send me SEND" in the subject  New OpenCDISC release in March will support SDTM IG3.2 Define.xml v2.0 Dataset-XML