​ Executive Summary Common Protocol Template (CPT)

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 2 What is TransCelerate? Our vision ​ To improve the health of people around the world by accelerating and simplifying the research and development of innovative new therapies. Our mission ​ To collaborate across the global research and development community to identify, prioritize, design and facilitate implementation of solutions designed to drive the efficient, effective and high-quality delivery of new medicines. TransCelerate is a not-for-profit entity created to drive collaboration across the pharmaceutical industry as a means to developing solutions for overcoming inefficiencies Our core values  Quality  Transparency & Openness to new ideas  Trust & Integrity  Collaboration  Courage

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 3 Why a Common Protocol Template? ​ Heard at a June 2015 DIA meeting… “Patient recruitment is challenged by the complexity of protocols. Sponsors do the same things too many different ways. Just make it the same!” “If you have standards without traceability, then you aren't really CDISC compliant.” Investigator FDA “Just because ‘subject’ is in the regs, doesn’t mean you have to use it. Patients think of ‘subject’ as a verb, and who wants to be subjected to something? Please use ‘trial participant’ instead.” Patient Advocate

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 4 ​ Problem Statement – Format and core content of study protocols vary from sponsor to sponsor making interpretation difficult for study sites, IRBs, and regulators. – Study protocols have become increasingly complex and lengthy driving up cost and time. – Manual set-up of clinical systems based on non-standard “manual” protocols is time consuming, costly, and prone to error. ​ Solutions – Develop a streamlined model clinical trial protocol, including format and core content, to ease interpretation and enable down-stream automation of many clinical processes. – Develop model protocol endpoint definitions, incorporate into piloting of the template. Background: Why a Common Protocol Template? ​ Guiding Principles – “Common” in a CPT: information always in the same place, means the same thing. – Is it better? Move beyond incremental improvements and current limitations. – It has to be better for everyone, but Investigators/Sites are the first priority. – A human readable template that paves the way for an automated solution and facilitates the use of data standards.

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 5 Near Term Benefits to Sites Protocols streamlined and organized with an investigator focus Reduced burden on sites working on multiple studies The Common Protocol Template Potentially Benefits Many Stakeholders Sponsor Sites IRB Patient Regulator Near Term Benefits to Sponsors Reduction in redundant protocol content Enabling of therapeutic area standards Improved conduct of the study and quality of data collected Potential Future Benefits to Sponsors Automation of downstream processes and reuse of content Enabling of therapeutic area standards in additional TAs Enabling collaborative clinical trials Potential Future Benefits to Sites Opportunity to harmonize additional documentation Enable consistency of some case report forms Near Term Benefits to IRBs Increased consistency between sponsor protocols: easier review and faster approval Potential Future Benefits to IRBs Enabler for automation of IRBs submissions Near Term Benefits to Patients Increased efficiency / quality in clinical development for participants & future patients Potential Future Benefits to Patients Improved access to protocol information Getting medicines faster, for participants & future patients Near Term Benefits to Regulators Protocols streamlined, increased consistency between sponsor protocols to ease review Potential Future Benefits to Regulators Increased ease of data interpretation and ability to compare protocols (improves input on protocol design) Increased use of data standards enabling end-to-end use of metadata and traceability Near Term Potential Future State

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 6 A common protocol template structure with harmonized language Streamlined content enables identification of critical information for end users Begin working towards common endpoint definitions to align with Clinical Data Interchange Standards Consortium (CDISC) Therapeutic Area (TA) data standards. Asthma and Diabetes available in the first release. Reconnect processes (protocol, eCRF, development) Transformation of the design process o Analytics-driven trial design, modelling, scenario planning Role-based access to protocol information (Principal Investigator [PI], Ethics, Participants) Connection to other systems Common Protocol Template is Intended to Prepare for the Future State Human- Readable Protocol IRB/IECs Sites Regulators Foundation Machine- Readable Protocol Metadata driven processes Content Reuse Disclosure SAP CSR eCRF Statistical Output Future

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 7 Current State/Future State: Establishing Our Direction Where we started… – Paper-based, manual processing Where we are… –Electronic, document based Where we should be… –Digital, Cloud-based, computable, re-usable Today Manual processes and interventions Duplication of company efforts Disconnected interfaces Lot of customization Long cycle times Variable quality High costs Rework Patient-centric clinical trial design Automated/seamless interfaces / parallel processing Quality by design Shorter cycle times Cost efficient “Integration of Data Analytics” Incremental Transformational

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 8 Advisory Committee eProtocol Advisory Committee S TAKEHOLDER F EEDBACK “…to inform the development of a common human readable and electronic protocol template and provide a forum for key stakeholders to inform future direction and provide know-how (Committee Charter)” “EMA has not agreed to take up a position on the Advisory Committee…..Happy to provide comments on any draft templates…” FDA 18-Mar-2016: “Today we’re announcing a draft clinical trial protocol template developed by the Food and Drug Administration (FDA) and National Institutes of Health (NIH) that should help with that. …..We are aware of other efforts in this area, including one undertaken by TransCelerate Biopharma Inc. (TransCelerate), which has issued a common protocol template intended to be the basis for a forthcoming electronic protocol. Although our initial target audiences differ, we plan to collaborate with groups like TransCelerate to help ensure consistency for the medical product development community.” Other Advisors or Influencers

Copyright ©2015 TransCelerate BioPharma Inc., All rights reserved. 9 Value Proposition – Why Should Sponsors Adopt? Quality The expertise of 18+ member companies has been tapped to develop the template – the work has been done for you. The template includes TA –specific libraries based on sponsor best practices. Sponsors will have the opportunity to suggest revisions, and the template will be maintained and updated over time. Broad adoption will help drive greater efficiency for investigators, sites, sponsors,, and regulators. Efficiency Sponsors can spend less time on low-value customization, and reduce time managing template maintenance. The template is easy for authors to use. Sponsors can adopt in a phased approach, and can choose to use either the Word version or Tech-Enabled version. You don’t have to go “all in” at the outset. Compliance The template is supported by FDA, and Health Authority feedback to date has been unanimously positive. Quality + Efficiency + Compliance = Value Harmonization Value

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