CDISC/TransCelerate Collaboration and TransCelerate work streams

-Clinical Data Standards -Common Protocol Template-eSource

July 2016

© CDISC 2012

TransCelerate Clinical Data Standards Project

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Opportunity Description

Goals:• Dedicate effort and investments to assist [partner

with] CDISC, C-Path, NCI-EVS and FDA to • complete CFAST Therapeutic Area [TA] data

standards projects more rapidly• Roll-out new TA data standards every few

months as particular disease areas are completed

• Also facilitate adoption to realize benefits and enable interoperability across companies, foundations, health authorities

• Standards are the key foundation for achieving vision for the semantic interoperability described in recent FDA Guidance for providing regulatory submissions in electronic format (standardized study data)

Long Term Vision:Support the creation of common Clinical Data Standards (therapeutic area specific data in addition to existing ‘foundational’ safety domains) and metadatastandards for integrating clinical data from disparate sources as a foundation for end-to-end data flow

•ADaM = Analysis Data Model•CDASH = Clinical Data Acquisition Standards Harmonization•SDTM = Study Data Tabulation Model

For Internal Use Only © 2014 TransCelerate BioPharma, Inc.

Initiative Overview

Develop industry-wide standards that will allow for increased efficiency in research and development.

What Is Clinical Data Standards (CDS)?

Collaborate with CDISC (Clinical Data Interchange Standards Consortium), C-Path (Critical Path Institute), NCI-EVS (National Cancer Institute – Enterprise Vocabulary Service) and FDAas part of the CFAST (Coalition For Accelerating Standards and Therapies) initiative to develop industry-wide Data Standards in priority Therapeutic Areas (TAs) to support the exchange and submission of clinical research and meta-data, improving efficiency and scientific knowledge.

Work in partnership with CDISC, FDA, NCI-EVS and C-Path.

Utilize an open and transparent process with a well-defined standards governance function.

Ensure engagement and input of clinical and medical professional societies, as well as government agencies.

What Is The Vision? What’s the Approach?

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Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 1414

• CFAST - an initiative to accelerate clinical research and medical product development by creating and maintaining data standards, tools and methods for conducting research in therapeutic areas that are important to public health

• Objective - Identify common standards for representing clinical data for studies of drug products in specific, prioritized therapeutic areas [TA]

• To be implemented using CDISC structures (CDASH, SDTM, ADaM)

• Support integration of clinical data from disparate sources as a foundation for end-to-end data flow

Coalition For Accelerating Standards & Therapies

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 15

Therapeutic Area Standards Governance

CFAST SAC Scientific Advisory Committee

• Prioritizes/Approves Proposals• Approves Projects & Charters• Resources & Oversees Projects

• Provides Scientific Advice to TAPSC• Identifies Risks and Opportunities• Identifies/Engages Relevant Partners

OngoingMaintenance

&Enhancement

of Foundational CDISC Standards

CDISC TA Standards Project TeamsProject Leader +

Clinical leads (SMEs), BRIDG Modeler, Concept Creators, Terminologists, Metadata Analysts, Stats

Consultants, Writers, Communications

Research Community

Research Community

CFAST TAPSCTherapeutic Area Program Steering Committee

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 16© CDISC 2012

Project Organization

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Project Manager

TAClinical Experts

Statistical Analysis Experts

Metadata Developers

CDISC TeamLiaisons

Program Manager –

Rhonda Facile

Process Expert

Technical Writer

SDTM Expert

Terminology Expert

BRIDG Expert

CDASH Team

SDS Team

Other CDISCTeams

ADaM Team

Terminology Teams

QuestionnairesTeam

Review Community: CDISC, C-Path, FDA, Transcelerate, Key Opinion Leaders, Medical Associations, U.S. National Institutes of Health, PMDA, EMA, Innovative Medicines Initiative.

CFAST (SAC) Scientific Advisory

Committee

CDISC VP, Standards,

Terminology and Technical Services –

Barrie Nelson

CFAST (TAPSC)Therapeutic Standards

Steering Committee

CDISC Standards

Review Council

Collaborates

CFAST Governance

CDISC Teams

Program Resource

Project ResourceMedical Writer

© CDISC 2012

SHARE

BRIDG, ISO21090

Protocol, CDASH

SDTM, ADaM

Terminologies

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• Single, trusted, authoritative source for CDISC data standards

• Concepts, metadata, collections, relationships, value sets across the full spectrum of CDISC content

• Links research to healthcare concepts to support interoperability

• Aligned with NCI Semantic Systems

• Access to data standards

• Source to target mapping & traceability

• Transformation logic

Facilitates Data

Exchange

c. Impact Analysis & Inheritance

b. Gov’cwork-flows

a. Change control

cb

a

Adapted from Source by Sue Dubman

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Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 18

Vision and 2016 ObjectivesClinical Data Standards

Vision

Develop industry-wide therapeutic area

data standards that will allow for

increased efficiency in research and development

Support CFAST efforts to Publish 8 or more Therapeutic Area (TA) User GuidesLeverage Prostate Cancer project toadvance next level of SHARE capabilitiesand synergies within TA development in oncologyFacilitate member company adoption of CDISC data standards Collaborate with CPT workstream to support addition of 4-6 new TA librariesSupport CDISC to implement SHARE API and RDF Export

• Enables operational efficiencies in data management and analysis, facilitates demonstrating traceability of data and increases efficiency in conducting trials.

• Enables interoperability across companies, foundations, health authoritiesShare information

& Harmonize processes

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 19

Vision and 2016 ObjectivesCommon Protocol Template (CPT)

Vision

Develop a model clinical trial protocol, including format and core content, to ease

interpretation and enable down-stream automation of many

clinical processes.

Enable automated disclosure for trial registration Complete proof of concept on the ability to automate eCRF production; if successful run a simulation to quantify the benefit and develop plan to scale up Issue at least one public template update and add 4-6 new TA libraries

• CPT enables protocol information to be presented in the same place, meaning the same thing, across multiple sponsors and protocols.

• The ability to export key protocol terms from the Technology Enabled Edition of the CPT facilitates sharing of information and harmonizing content across study documents.

Share information

& Harmonize processes

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 20

eSource Vision and 2016 Objectives

Vision

Moving the industry to optimize the use of electronic data sources to improve global clinical science and global clinical trial execution for patients, sites and sponsors.

Develop Landscape Assessment (current state, gaps, obstacles)

Develop Framework to enable uptake of eSource and define eSource alternatives(Use Cases, Core Principles, Common Terminology, Roles/Responsibilities)

Develop Metrics/KPIs

Data Collection: study level implementation for demonstration projects

Develop Stakeholder Strategy (Health Authorities, Vendors & Vendor Associations, Sites/Patients)

Provides unified voice of TransCelerate to Health Authorities to overcome perceived concerns, move faster to steady state, opportunity to influence and shape the environment

Supports efficiencies in reducing data transcription, providing traceable end to end data flow

Facilitates improves data availability (provides real time data /metadata availability)

Provides sponsors with proof of concept to demonstrate viability of using eSource tools for regulated trials (e.g. wearables)

Enhance Sponsor

Efficiencies

Questions

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