PhUSE Annual Meeting, London 2014



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PhUSE Annual Meeting, London 2014 Metadata, Study data standards, Master data, terminology, interoperability: Key concepts underlying compliance to FDA guidance on electronic submission Isabelle de Zegher, MD, MSc, PAREXEL Informatics

Table of Content PhUSE CSS Emerging Technology Working group Definitions Metadata and study data standards Master data Controlled terminology Interoperability Pooling, aggregation, integration How can this help toward esubmission Lessons learned Changing landscape: need for concept based MDR from data collection onwards

Table of Content PhUSE CSS Emerging Technology Working group Definitions Metadata and study data standards Master data Controlled terminology Interoperability Pooling, aggregation, integration How can this help toward esubmission Lessons learned Changing landscape: need for concept based MDR from data collection onwards

PhUSE CSS Emerging Technology FDA/PhUSE Computational Science Symposium (CSS) is a collaborative effort between industry and the FDA to work on implementation of data standard In 2013 we launch a new working group related to specific computational science topics, tools, technologies, and approaches. semantic web applications (now in a dedicated working group) analysis metadata, cloud computing (big data)

Table of Content PhUSE CSS Emerging Technology Working group Definitions Metadata and study data standards Master data Controlled terminology Interoperability Pooling, aggregation, integration How can this help toward esubmission Lessons learned Changing landscape: need for concept based MDR from data collection onwards

Metadata definition project: the data standards soup. 6

Participants Mitra Rocca (co-chair) Mitra.rocca@fda.hhs.gov Marcelina Hungria (co-chair) mhungria@dicoregroup.com Yun Oldshue yun.oldshue@takeda.com Kenneth Stoltzfus kenneth.m.stoltzfus@accenture.com Julie James julie_james@bluewaveinformatics.co.uk Tim Church tim.church@torch.uk.net Gregory Steffens Gregory.steffens@novartis.com Praveen Garg Praveen.Garg@iconplc.com John Leveille jleveille@d-wise.com Aimee Basile abasile@celgene.com Sam Hume shume@cdisc.org

Definitions in scope 4.1 METADATA MANAGEMENT 4.1.1 Metadata 4.1.2 Structural metadata 4.1.3 Descriptive metadata 4.1.4 Study Instance Metadata 4.1.5 Metadata repository 4.1.6 Metadata registry 4.1.7 Data element 4.1.8 Attribute 4.1.9 Class 4.1.10 Data type 4.1.11 Value level metadata 4.2 CONTROLLED TERMINOLOGY, CODE SYSTEMS & VALUE SETS 4.2.1 Controlled Terminology/controlled vocabulary 4.2.2 Code system 4.2.3 Dictionary 4.2.4 Concept 4.2.5 Code 4.2.6 Code list 4.2.7 Value set 4.3 MASTER DATA MANAGEMENT 4.3.1 Master Data 4.3.2 (Master) Reference Data 4.3.3 Master Data Management 4.4 INTEROPERABILITY Categorization of Interoperability (by HL7) 4.4.1 Technical interoperability ( machine interoperability ) 4.4.2 Semantic interoperability 4.4.3 Process Interoperability 4.5 DATA AGGREGATION, INTEGRATION, POOLING 4.5.1 Data pooling 4.5.2 Data integration 4.5.3 Data aggregation

Synonym Definition source Description Example Recommended definition & Approach Reference Data Management; MDM [Gartner Magic Quadrant for Master Data Management of Customer Data Solution] http://www.gartner.com/technology/reprints.do?id=1-1ck9udo&ct=121019&st=sb MDM is a technology-enabled discipline in which business and IT work together to ensure the uniformity, accuracy, stewardship, semantic consistency and accountability of the enterprise's official, shared master dataassets. [Source: Master Data Management] Master Data Management (MDM) is the collective application of governance, business processes, policies, standards and tools facilitate consistency in data definition. The idea of Master Data focuses on providing unobstructed access to a consistent representation of shared information [Source: SAS White Paper on Supporting Your Information Strategy with a Phased Approach to Master DataManagement Master Data Management (MDM) comprises of a set of processes and tools that consistently define and manage the master data and master reference data of an enterprise, which are fundamental to the company s business operations. MDM has the objective of providing processes & tools for collecting, aggregating, matching, consolidating, quality-assuring, persisting and distributing such data throughout an organization to ensure consistency and control in the ongoing maintenance and application use of this information. There are different models for master data management the 2 main extremes are Centralized model where all data are managed within a central data store and pushed to the different applications within an organization. Decentralized model (registry) where the master data are managed within each applications but then reconciled through a registry systems to federate. Specific products from vendors such as INFORMATICA, IBM, Software AG, Set of processes and tools needed for the deployment of master data and master reference data within an organization.

Metadata

Master Data

How Controlled Vocabularies are described and used In define.xml(not machine processable) Controlled terminology code : CDISC CT/ NCI EVS CT Value set CUI for SEX: C66731 Female CUI: C16576 Other (machine processable): OID. URI Concept Identifiers Concepts Women Concept Representation C16576 + F Controlled Terminology C16576 Codes Code System Versioning Code Systems Designations female F (primary) Female ISO 21090 Datatypes the CD Concept Descriptor Code list value set with CDISC example Value Set Definition Value Sets Value Set Versioning C66731 (for SEX) inspired from Julie James, BlueWave Informatics

Interoperability

Data Pooling, Integration, Aggregation Dataset 1 Dataset 2 Dataset 3 AGGREGATION Additional grouping or derivation of data POOLING Storing data together without changing the datasets INTEGRATION: Transformation, mapping or harmonization of data (ETL process)

Next steps Comments welcome Consolidation http://www.phusewiki.org/wiki/index.php?title=metadata_man agement

Table of Content PhUSE CSS Emerging Technology Working group Definitions Metadata and study data standards Master data Controlled terminology Interoperability Pooling, aggregation, integration How can this help toward esubmission Lessons learned Changing landscape: need for concept based MDR from data collection onwards

LESSONS LEARNED AT PAREXEL INFORMATICS Efficient Data Integration and compliance to regulatory standards does not start after pooling (retroactive approach) ; it starts with the protocol (proactive approach) A proactive approach is based on 2 components Agreement on study Master Data (study ID, visit ID,..) and related descriptive metadata Definition of study structural metadata aka study specific data standards as a subset of the enterprise wide variables and value sets contained in a Metadata repository (MDR) To be manageable, variables in an MDR need to be grouped in semantically meaningful clinical research concepts (CRC) 2014 PAREXEL INTERNATIONAL CORP. / 17 CONFIDENTIAL

CHANGING LANDSCAPE : Enforcing data standards from protocol onwards Retro-active approach from paper protocol, Pro-active approach with structural metadata Different interpretations of same protocol Limited standards Time to build integrated SDTM data sets 18 2014 PAREXEL INTERNATIONAL CORP. / 18 CONFIDENTIAL One single interpretation of protocol Increased efficiency, consistency & quality through standards Reduced time for integration and secondary data use

CONCEPT BASED MDR : Protocol is not about variables but about concepts Annotated ecrf for Patient Demography? 2014 PAREXEL INTERNATIONAL CORP. / 19 CONFIDENTIAL? SDTM data set (SAS) (different t variables names and different structures than ecrf)

CONCEPT BASED MDR: CDASH/SDTM can be organized by CRC Concept CDASH Question CDASH Variable Subject What is the sex of the subject? What is the subject s date of birth? What is the ethnicity of the subject? What is the race of the subject? What is the subject s age? What are the age ecrfunits content used? description 2014 PAREXEL INTERNATIONAL CORP. / 20 CONFIDENTIAL SEX BRTHDAT or BRTHYR BRTHMO BRTHDY ETHNIC RACE AGE AGEU SDTM Variable SEX BRTHDTC EHTNIC RACE AGE AGEU SDTM mapping

FROM A VARIABLE TO A CONCEPT-BASED MDR Metadata Repository Data Standard Data Collection (CDASH) Standards link SDTM Standards link ADaM Standards Notes Notes Notes Classical MDR We can expect more than 30.000 variables across all Therapeutic Areas How do we ensure? semantic consistency when authoring completeness when selecting variables 2014 PAREXEL INTERNATIONAL CORP. / 21 CONFIDENTIAL

FROM A VARIABLE TO A CONCEPT-BASED MDR Metadata Repository Data Standard Data Collection (CDASH) Standards link Notes Variable Grouping Mapping ISO21090 SDTM Standards link ADaM Standards Notes Notes Relationships Clinical Research Concept (most granular semantically sound concept) 2014 PAREXEL INTERNATIONAL CORP. / 22 CONFIDENTIAL

CONCLUSIONS Let us speak the same language We need to change the way we consider compliance to data standards and data integration: From a retro-active way (building define.xml at submission) To a pro-active approach (study data standards defined at study set-up) We need new tools to manage metadata: Concept based MDR Grouping variables into semantically meaningful concepts (following industry wide patterns) Linking data collection (CDASH) to data submission (SDTM) variables Linked with controlled terminology 2014 PAREXEL INTERNATIONAL CORP. / 23 CONFIDENTIAL

Dr. Isabelle de Zegher Senior Director, Clinical Information Management PAREXEL Informatics Parc des Collines T +32 2 767 16 48 M +32 478 48 28 54 http://www.phusewiki.org/wiki/index.php?title=metadata_m anagement 2014 PAREXEL INTERNATIONAL CORP. / 24 CONFIDENTIAL