Aquila's Lunch And Learn CDISC The FDA Data Standard. Disclosure Note 1/17/2014. Host: Josh Boutwell, MBA, RAC CEO Aquila Solutions, LLC

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1 Aquila's Lunch And Learn CDISC The FDA Data Standard Host: Josh Boutwell, MBA, RAC CEO Aquila Solutions, LLC Disclosure Note This free training session will be placed on Aquila s website after the session is complete. The video may include your webex name, your questions and any answers. By participating you agree to allowing Aquila to publish this information. Note: Datasets are publically available from NHANES or CDISC.org. Joshua Boutwell, MBA RAC 15 years of experience in research, Regulatory Affairs, Regulatory Operations and Publishing Founded Aquila Solutions in

2 Today s Topics 1. Clinical Data and Submission Requirements 2. What is XPT? 3. Future FDA Requirements The Future for CDISC 6. Questions 7. The Next Lunch And Learn Clinical Data and Submission Requirements Phase 3 CSR s can be over 20,000 pages long Under 21 CFR sponsors are required to submit statistical data This data can be hundreds to thousands of Megabytes per study Clinical Data and Submission Requirements Current Requirements: All Phase 2 and 3 CSRs must include a clinical database unless specifically exempted by FDA The data must be presented in a method that the FDA can incorporate and analyze easily You are required to use SAS Transport File extension XPT 2

3 Today s Topics 1. Clinical Data and Submission Requirements 2. What is XPT? 3. Future FDA Requirements The Future for CDISC 6. Questions 7. The Next Lunch And Learn What is XPT? XPT is a Human Readable format called SAS Transport What is XPT? XPT is an open, Human Readable format Called SAS Transport 3

4 What is XPT? I recommend using software: SAS Universal Viewer: What is XPT? The XPT format allows you to create: Tables with unlimited size Define unlimited variables Allows descriptive variable names To represent any type of data What goes into a Database? 4

5 What goes into a Legacy Database? 1. XPT files (unlimited number) Contains your data No restriction on field names No guidance on table names What goes into a Legacy Database? 2. Define.pdf (1 per database) Includes a table to describe your XPTs Must identify the data type and source Links to XPT and annotated CRF as appropriate What goes into a Legacy Database? 3. Annotated CRF (1 per tabulation Database) A blank copy of the CRF used in the Clinical Study Text identify the table and variable name of every blank Links to Define.PDF 5

6 What goes into a Legacy Database? 4. Programs (multiple per Analysis Database) The programs that create the Analysis database Typically programmed in SAS language Provide as both txt (.SAS extension) and PDF The Problem Every Study database is different. Most studies have almost identical data types yet 1. Programs can not be reused 2. Navigation must be relearned 3. Data cannot be correlated between studies Today s Topics 1. Clinical Data and Submission Requirements 2. What is XPT? 3. Future FDA Requirements The Future for CDISC 6. Questions 7. The Next Lunch And Learn 6

7 Future FDA Requirements FDA has been working to standardize data packages for years Implemented the JANUS computer system Develop CDISC standards Strongly recommend conversion But on September 13, 2013 it became official Future FDA Requirements Position Statement FDA recognizes the investment made by sponsors over the past decade to develop the expertise and infrastructure to utilize Clinical Data Interchange Standards Consortium (CDISC)[1] standards for study data. The submission of standardized study data enhances a reviewer s ability to more fully understand and characterize the efficacy and safety of a medical product. The Prescription Drug User Fee Act (PDUFA V) Performance Goals state that FDA will develop guidance for industry on the use of CDISC data standards for the electronic submission of study data in applications. In the near future, FDA will publish guidance that will require study data in conformance to CDISC standards. FDA envisions a semantically interoperable and sustainable submission environment that serves both regulated clinical research and health care. To this end, FDA will continue to research and evaluate, with its stakeholders, potential new approaches to current and emerging data standards. FDA does not foresee the replacement of CDISC standards for study data and will not implement new approaches without public input on the cost and utility of those approaches. Future FDA Requirements Aquila Predictions: 1. A draft guidance release in 2014 Q3/Q4 2. Updated and Active by For CDISC requirement on or around PDUFA VI (2017) 4. Probably a 2 year implementation period 5. Existing studies will probably be exempt. These are our predictions. They are not binding on Aquila or anyone else! 7

8 Today s Topics 1. Clinical Data and Submission Requirements 2. What is XPT? 3. Future FDA Requirements The Future for CDISC 6. Questions 7. The Next Lunch And Learn CDISC is: 1. A strict naming convention for XPT file names 2. A strict naming convention for Field names 3. Rules for handling unusual data 4. A DTD and stylesheet for an XML replacement to the Define.pdf (define.xml) CDISC Components: SDTM Study Data Tagging Model ADaM Analysis Data Model 8

9 A define.xml for SDTM Database A define.xml for SDTM Database (as XML code) <?xml version="1.0" encoding="utf 8"?> <?xml stylesheet type="text/xsl" href="../stylesheets/define2 0 0.xsl"?> <ODM xmlns=" xmlns:xlink=" xmlns:def=" ODMVersion="1.3.2" FileOID=" Define XML_2.0.0" FileType="Snapshot" CreationDateTime=" T17:04:44" Originator="CDISC XML Technologies Team" SourceSystem="MH System" SourceSystemVersion="2.0.1" > <! ****************************************** > <! OID conventions used in this example: > <! MetaDataVersion = MDV. > <! def:leaf, leafid LF. > = <! def:valuelistdef = VL. > <! def:whereclausedef = WC. > <! ItemGroupDef = IG. > <! ItemDef IT. = > <! CodeList = CL. > <! MethodDef = MT. > <! def:commentdef = COM. > <! ****************************************** > <! ****************************************** > <! This example does not include Value Level > <! Metadata (def:valuelistdef) for all items > <! that may need separate definitions. > <! The cases included are included for > <! illustration purposes only. > <! ****************************************** > <def:annotatedcrf> <def:documentref leafid="lf.blankcrf"/> </def:annotatedcrf> <def:supplementaldoc> <def:documentref leafid="lf.reviewersguide"/> <def:documentref leafid="lf.complexalgorithms"/> </def:supplementaldoc> <def:valuelistdef OID="VL.DA.DAORRES"> <ItemRef ItemOID="IT.DA.DAORRES.DISPAMT" OrderNumber="1" Mandatory="Yes"> <def:whereclauseref WhereClauseOID="WC.DA.DATESTCD.DISPAMT"/> <ItemRef ItemOID="IT.DA.DAORRES.RETAMT" OrderNumber="2" Mandatory="No"> <def:whereclauseref WhereClauseOID="WC.DA.DATESTCD.RETAMT"/> </def:valuelistdef> <def:valuelistdef OID="VL.EG.EGORRES"> <ItemRef ItemOID="IT.EG.EGORRES.INTP" OrderNumber="1" Mandatory="No"> <def:whereclauseref WhereClauseOID="WC.EG.EGTESTCD.INTP"/> <ItemRef ItemOID="IT.EG.EGORRES.PRMEAN" OrderNumber="2" Mandatory="No"> <def:whereclauseref WhereClauseOID="WC.EG.EGTESTCD.PRMEAN"/> <ItemRef ItemOID="IT.EG.EGORRES.QRSDUR" OrderNumber="3" Mandatory="No"> <def:whereclauseref WhereClauseOID="WC.EG.EGTESTCD.QRSDUR"/> <ItemRef ItemOID="IT.EG.EGORRES.QTMEAN" OrderNumber="4" Mandatory="No"> <def:whereclauseref WhereClauseOID="WC.EG.EGTESTCD.QTMEAN"/> <ItemRef ItemOID="IT.EG.EGORRES.VRMEAN" OrderNumber="5" Mandatory="No"> <def:whereclauseref WhereClauseOID="WC.EG.EGTESTCD.VRMEAN"/> </def:valuelistdef> <def:valuelistdef OID="VL.EG.EGSTRESC"> <ItemRef ItemOID="IT.EG.EGSTRESC.QTCB" OrderNumber="1" Mandatory="Yes" MethodOID="MT.QTCB"> <def:whereclauseref WhereClauseOID="WC.EG.EGTESTCD.QTCB"/> <ItemRef ItemOID="IT.EG.EGSTRESC.QTCF" OrderNumber="2" Mandatory="Yes" MethodOID="MT.QTCF"> <def:whereclauseref WhereClauseOID="WC.EG.EGTESTCD.QTCF"/> </def:valuelistdef> 9

10 A Demographic table for SDTM Database DM.xpt A Demographic table for unusual SDTM Database suppdm.xpt Issues with your Database Conversion It is difficult to convert legacy to CDISC 1. The data structure must be altered 2. Some data may not fit into the standard 3. Existing analysis programs will not work 4. Duplicates work. Plan ahead and design your biostats to use CDISC 10

11 Today s Topics 1. Clinical Data and Submission Requirements 2. What is XPT? 3. Future FDA Requirements The Future for CDISC 6. Questions 7. The Next Lunch And Learn The Future of CDISC CDISC is under continuous development. other models being developed are: SEND Standard for Exchange of Nonclinical Data (FDA Pilot Study Underway) CDASH Clinical Data Acquisition Standards Harmonization LAB Laboratory Data Model ODM Operational Data Model Aquila Can Help Aquila provides Data Package Review We assess your data package to ensure 1. The files are formatted correctly 2. The necessary data is included in the database 3. The define files and annotated CRF are correct 4. Verify your CDISC database is correct Aquila provides Database Conversion If FDA requires your data be submitted in CDISC, Aquila can convert a legacy database to CDISC and place it in your application. 11

12 Today s Topics 1. Clinical Data and Submission Requirements 2. What is XPT? 3. Future FDA Requirements The Future for CDISC 6. Questions 7. The Next Lunch And Learn Next Lunch and Learn : ecopy esubmissions and devices Topics: 1. What is ecopy 2. Where is it required 3. Things to consider February 7 th PM 1:30 PM EST 15 minute training with 15 minute Q/A