1. TransCelerate BioPharma, Inc A Global Update
Carlo Maccarrone M.Pharm., Ph.D., ACF
Asia Pacific Regional Director – Clinical Operations
GSK, Australia

2. Session Agenda A Global Update History & Mission
How TransCelerate Operates
TransCelerate Portfolio
Initiative Presentations
Introduction to the Shared Investigator Platform
The Clinical Quality Management System Initiative
The eLabels Initiative

3. TransCelerate - a not for profit entity created to drive collaboration

4. TransCelerate’s Organisational Structure
High engagement from member talent is a key attribute in our success.
External Counsel
Board of Directors
Accounting Firm
CEO
SVP, Global Operations
Audit Firm
Oversight Committee
Portfolio Management
Communication & Engagement
Operations
Quality
Finance
Administrative Asst.
Active Portfolio Subcommittees
Leadership
Project Management
Member Company Talent
Key:
Team Members
In terms of orgnaisation structure, Transcelerate has a small permanent office of 9 staff who largely provide the “enabling support”…
But one thing to stress is that:
“High engagement from members talent is a key attribute in the success..”…
as I said having Cross- Company experts working collaboratively on the same problems to develop solutions is key to success.
The “red” shows the important contribution made by Companies …from personnel on the Board of Directors and Oversight committees, portfolio sub-Cmtts, Team members etc.
in addition to funds used to employ contracted resources.
Over 1,000 people from companies working on projects and a rule that they spend 0.25 FTE on TBI projects
Companies can only join because believe in mission /vision…this is not an al la carte joining
- have to with funding support but also people commitment (must be both) – considered that internal staff working on real projects are critical and don’t just want to resort to using consultants …as they need to emebedd in their own companies.
Permanent Position
Enabling Functions
(Regulatory, Technology, Communications, etc.)
Contracted Resources / Member Resources
Leadership
Project Management
Team Members

5. Collaboration with Key Stakeholders
We embody deep and broad collaboration across many stakeholders, all with a common goal of improving clinical trials.
A key tennet of TransCelerate’s success is that membership is made up of a single stakeholder group – which are BioPharmaceutical companies. History has shown that many consortia struggle to deliver results if they have too many diverse stakeholders, with diverse interests trying to come to a solution.
So Membership is limited to Pharmaceutical Companies. However, for a holistic approach to overcome the challenges it is critical to work closely with the other key stakeholders across the whole of Clinical Research ecosystem.
Investigator Sites: Through a partnership with the Society for Clinical Research Sites – TransCelerate initiatives have Site Advocacy Groups (SAGs) which are a group of investigators and site personnel who work with TransCelerate initiatives to provide feedback on solutions. TransCelerate also works with other site organizations around the world included ACRP and AACI.
Research & CRO Community: To tap into the Clinical Research Community – a CRO Forum was created in early 2015 as a way to enable collaboration with the CRO community. This forum is lead by ACRO (The Association of Clinical Research Organizations) and is open to any CRO. Similar to the way we work with SCRS, there are CRO Liaisons that provide input into TransCelerate initiatives.
Industry Initiatives: There is no shortage of industry organizations focused on improving Drug Development. And TransCelerate believes there is too much opportunity for any two groups to be working on the same thing, so TransCelerate consistently meets with many industry groups (some of which you see here in the top right of the slide) to discuss what each organization is working on, to identify any areas of (correlation or) overlap and to promote and complement, when applicable, each others work.
Regulatory Authorities: Finally, TransCelerate provides frequent updates across the worlds regulatory landscape include the Food & Drug Administration (FDA) in the US, EMA in Europe, PMDA in Japan and many others. While TransCelerate doesn’t meet to seek approval or endorsement, the health authorities have provided immeasurably valuable feedback which TransCelerate has used as input into solution design and implementation.

6. TransCelerate Strategic Priorities
TransCelerate assesses industry challenges and selects initiatives aligned with five strategic priorities:

7. TransCelerate’s Portfolio of Initiatives
TransCelerate’s Portfolio of Initiatives focus on Quality, Patient Safety, and Accelerated Development Timelines.
Active
Design, Develop,
& Deploy
Phase
Clinical Research Awareness & Access
Clinical Data Standards
Clinical Data Transparency
Common Protocol Template
Comparator Network
Patient Experience & Technology
Investigator Registry
eConsent
eLabels
eSource
Placebo / Standard of Care Data Sharing
Quality Management System
Site Qualification and Training
Shared Investigator Platform
Risk Based Monitoring
Realization Phase
Clinical Trial Diversification
Pediatric Trial Efficiencies
Realization Phase
Clinical Trial Diversification
Focus to discuss today

8. The Common Protocol Template Initiative

9. The eConsent Initiative

10. TransCelerate Accomplishments to Date… … …
2013
2014
2015
2016
Designed a Risk Based Monitoring Methodology and issued subsequent publications across the industry showing an improvement in quality metrics, and > 15,000+ Unique downloads of TransCelerate Papers.
Operationalized an effective Good Clinical Practice (GCP) certification program to eliminate investigator sites taking duplicative GCP training with >130,000 GCP training certificates issued by 9 Member Companies and 150 external training providers have attested that the training courses offered meet mutual recognition of minimum criteria.
Launched the Investigator Registry and Shared Investigator Platform that will greatly enhance communication between sponsors and sites.
Operationalized a network of TransCelerate Member Companies to secure Comparator Drugs for studies, >60% of the top products sold globally are now covered in the CN product portfolio; Network has achieved > $150M in sales.
Launched a Common Protocol Template containing common structure and model language to improve accuracy in data recording and speed study start up.
Created a platform to share Placebo data from Clinical Studies across participating companies.
Published a Clinical QMS Conceptual Framework taking a proactive quality approach to all stages of clinical research.
Published a model approach for Data De-identification and Anonymization of Individual Patient Data in Clinical Studies.
Launched BioCelerate - A subsidiary of TransCelerate focused on preclinical collaboration.

11. Stay Informed and Engaged!
Send questions, comments at any time
Newsletter & Monthly s
Sign up on our website to hear from us!
Industry articles and updates
LinkedIn: TransCelerate BioPharma Inc.
Industry articles and updates on our initiatives

12. Co-Country Leads – South Korea
Soo-Yeon Park, GSK
Taehui Ahn, BMS

13. Visit us, for more information: www.TransCelerateBioPharmaInc.com
Watch our “About Us” Video:
I encourage you to stay connected to TransCelerate. If you go to the homepage, you can leave your address to be added to monthly communications And a quarterly newsletter.
I look forward to working with you to see how best we can harness the benefits of this collaboration to Australian Clinical Research.
Visit us, for more information:

14. The Shared Investigator Platform
Kelly Kirsch
Consultant-Shared Investigator Platform
Clinical Development Information and Optimization
Eli Lilly & Co., USA

15. Agenda SIP Overview Value Proposition Stakeholder Benefit / Impact
Accomplishments to Date
Who’s Involved
Roadmap – Now and the Future

16. The Shared Investigator Platform
Unmet Need:
Clinical trial sites must use many different websites, each requiring unique login credentials, to perform clinical trial responsibilities and communicate with their Sponsors. Site staff repeatedly prepare and provide the same information to each of their Sponsors. This is time consuming, cumbersome and often difficult.
Objective:
Reduce the burden on investigative sites by providing them with a single point of access, harmonized content and services, and streamlined interaction with participating clinical trial Sponsors.
Benefits:
Ease of use, reduced burden on sites, and harmonized clinical trial content and services for investigative sites and sponsors.

17. Value Proposition / Benefits When Delivered
Through development of the Shared Investigator Platform…
Unmet Need
Value Proposition
Different platforms, processes, and data sources within sponsors.
High costs and redundant effort in developing and maintaining in dividual sponsor portals, and mastering user and facility data.
Burdensome process – Multiple, unique portals, processes, and to ols increase cost and support needed for sites/investigators, and risk of errors.
Investigator Sites: Increased efficiency and reduced burden due to single sign-on, more unified experience, harmonized content and processes, streamlined use by sites across participating sponsors, and increased visibility to study opportunities across participating Sponsors.
Participating Member Companies: Savings through shared system investment & maintenance costs; and harmonized content and processes, which enhances compliance.
Regulatory Bodies: Harmonized information model allows expanded insights to clinical trials.

18. Stakeholder Impact & Benefit
Member Company
Sites
Clinical Study costs reduced by improvements in study start up, conduct & site management activities.
Increase efficiencies and decrease error through reduction in duplicative work and increased automation.
IT cost savings through shared investment in platform development, maintenance & support.
Increase efficiency, reduce burden on investigators & site personnel in planning & conduct of clinical studies.
Streamline interactions, data exchange with participating Study Sponsors through a single application with single sign on.
Align and harmonize data and processes for interacting with participating Study Sponsors.
Regulatory Authorities
Patients
Potential for streamlined expanded insights into clinical trials.
Less administrative time for site staff leads to more time with patients.

19. Accomplishments To Date
Since endorsement as a workstream by the TransCelerate Board of Directors:
Identified partners for platform development; Cognizant & DrugDev
Engaged with ACRO representatives on the Platform
Established and expanded the Site Advocacy Group engagement efforts with the Society for Clinical Research Sites (SCRS)
Release 1.0 Go Live in January 2016; available for Member Company adoption

20. Who’s Involved

21. SIP Roadmap (www.sharedinvestigator.com)

22. SIP Release 1 and 1.1 Functionality2 3
Single Sign On
Secure Access across Sponsors and Studies
Study Workspace
Sponsor specific workspace for each study
Facility / Department Profile
Centralized information; re-used across studies. User associates to facility or Department
Feasibility Survey
Survey creation, distribution, completion & response management
Document Exchange
Share, Post and Retrieve documents
Home Pages
Dashboards ,News, Links, Reports & Metrics
Secure Access across Sponsors and Studies; Sponsors send registration invitation codes
User Profile
Centralized Site staff information; re-used across studies; PI and Clinical Research User CVs
Training
Centralized Training History; GCP training (mutually recognized) 4 6 5 8 7 9
Task, Alerts, and Notifications
Consolidated reminders & information across studies

23. SIP Release 2 Proposed Functionality1 2 3
Safety Notifications
Upload letters, central distribution & notification with tracking acknowledgement
Study Workspace
Add/manage studies; allow bulk load from MC CTMS. Include tasks, workflows & wizard
Profiles/IR Integration
Wizard-based data entry; enhanced IR integration of master data & profiles 4
Reporting/Metrics
Expanded reporting for: DocX, Safety Letters, and other SIP functions 5
Survey
Add survey analysis & new survey types wizard;
Expand functionality; add Site User dashboard 6
Training
Complete GCP and sponsor-specific training; expand tasks and notifications 7
DocX/eTMF
Integrate to MC eTMF; workflow, versioning, e-signature, forms, document submission & distribution 8
General
Platform-level & core functionality: UI, Workflow, analytics, wizards

24. Thank you!
Questions?

25. The Quality Management System Initiative
Hannah Chen
Director, Asia Pacific/China R&D
Janssen Pharma, China

26. Why Is QMS Important and a TransCelerate Initiative?

27. TransCelerate Member Company Structured Interview Results Indicate Opportunities for Improvement

28. Overview of Clinical QMS Conceptual Framework Concept Paper
What it outlines:
A proactive approach and flexible concept ual framework for holistically managing quality across the spectrum of clinical activities (enterprise-focused)
A conceptual framework to enable consist ent and timely delivery of reliable data that may be used by an organization, its partners, regulators, clinicians, and patients to make informed decisions
No harmonized regulatory guidance defining elements of a conceptual Clinical QMS framework, leading to disparate implementation and inefficiencies
Why it is needed:

29. A Structural, Philosophical, Behavioral Approach
Proactive, Risk-based, Flexible
Foundational Aspects
Processes
Resources, Roles & Responsibilities
Risk Management
Issue Management
Knowledge Management
Partnering
Elements of a Clinical QMS Conceptual Framework
Documentation Supporting Achievement of Quality
Assessing the CQMS
Management Review
Understand the Context
Leadership Commitment to Quality
Organizational Commitment to Quality
Continual Improvement of the Framework

30. What’s the Relationship of QMS, QBD and RBM?
Copyright ©2016 TransCelerate BioPharma Inc., All rights reserved.

31. Messages from the Stakeholders
Strong alignment and agreement with the framework, the approach, the elements and the philosophy
The Conceptual Framework ….
Is a valuable development
Is comprehensive and includes all needed elements
Will enable better partnership between sponsors, sites and other stakeholders
Will provide meaningful guidance for managing quality across clinical development activities
Will benefit from a graphic showing relationship between QMS, QbD, and RBM
Acknowledges the importance of establishing the context in which the organization operates

32. What TransCelerate QMS Heard from Health Authorities
FDA
EMA
PMDA
Suggested ICHQ10 (GMP) was not appropriate for a CQMS
Acknowledged benefit to patients and clinical sites
Acknowledged common goals of a CQMS for industry stakeholders
Suggested examples of practical application would benefit organizations seeking to apply a potential guidance
Provided strong support for a CQMS ICH guidance
Encouraged strong focus on patient safety and speed of medicinal products to patients
Expressed strong interest in TransCelerate CQMS. Acknowledged importance of global harmonization separate from ICH E6 R2
CQMS may benefit patients
Supportive provided flexible company implementation
Health Canada
BfArM
COFEPRIS
Provided strong support for a CQMS ICH guidance
Acknowledged importance of global harmonization separate from ICH E R2
Strong interest in the benefit to patients
Concept paper describes a meaningful framework for clinical quality
Q10 (and Q9)only for manufacturing
Examples useful as practical guidance for new companies
Suggested CQMS should be a standard for clinical trial stakeholders
Noted CQMS guidance will improve quality of all industry organizations
Indicated harmonized processes will reduce (agency) review resources and regulatory timelines
CFDA
MFDS
ANVISA
Strong interest in the benefit to patients
Very interested in standardizing model s of quality to help inspectors
Would find specific examples (case studies) helpful
Supportive to QMS concept for clinical trials
Appreciated difference between ICH Q10 and a variable clinical environment
Good interest; some reticence until they see how this works in practice
Indicated QMS should be a focus to the country to develop not only the industries but sites in Brazil.
Suggested initiative may improve the quality and increase number of qualified sites in Brazil.

33. Issue Management: Issues that Matter “Triage” Framework
Examples of Sources of Issues
Study management and monitoring
Data trending and analytics (including internal surveillance / metrics)
Self-identified issues within the clinical development functions
Issues reported by external parties
Noncompliance to regulatory requirements throughout the clinical development enterprise
Audit and inspection results
Conceptual funnel is a mechanism for filtering issues based on the level of risk.
Setting thresholds to allow focus on the “Issues that Matter”
The number of and the definitions of the thresholds will generally be established during risk assessments.
Issues not meeting definition of “Issues that Matter” are retained for trending and analytics
In aggregate, filtered issues may uncover a potential risk requiring “Issues that Matter” triage.
Trending and analytics will feed back into a risk assessment process.
An issue(s) that matters will move quickly through the funnel for appropriate escalation and CAPA process
The preventive action will link back to risk assessment and evaluation of risk mitigation strategies.

34. Knowledge Management For Clinical Development
“Getting the right information to the right people at the right time”
Clinical QMS Conceptual Framework Concept Paper
“Strategies and processes designed to identify, capture, structure, value, leverage, and share an orga intellectual to enhance the clinical development organization’s performance and the performance of QMS elements, including issue management and risk management, based on two critical activities:
Capture, storage and accessibility of institutional explicit and tacit knowledge, and
Its assimilation, interpretation, dissemination, application and maintenance within the organization”
Value
Connects people with information more rapidly
Reduces unnecessary or redundant re-work across teams/functions
Retains and facilitates sharing of critical knowledge
Enables searching, finding and applying information faster
Enhances decision making
Adapted from The Business Dictionary:

35. QMS Strategy

36. QMS Asia Pacific Sub team: Mission and Team
Define next steps for engaging our QMS Asia Pacific team
QMS Asia Pacific Team
Lead Hannah Chen (JNJ China)
Confirmed members:
Tatsuya Murakami (Pfizer-Japan)
Bokjin Hyun(BMS - Korea)
Ellyne Setiawan (BI - China)
Helen Li (Pfizer - China)
Helen Wong(Merck – China HK)
Keiichi Ueda(Shionogi – Japan)
Janet Chen(AZ - China)
Tomoko Ban(AZ - Japan)
Yumi Sugiura (BMS - Japan)
Shuichiro Tatematsu (UCB -Japan)
Saeki Satoshi (Astellas - Japan)
Charmaine Soon(JNJ-Singapore)
Kiyomi Hirayama (Merck-Japan)
Team Mission:
Review and provide comment on TFA output
Test our messaging for the region and provide feedback (is it understood, are there areas that require more attention, etc)
Take our messaging and evolve it for use in the region
Represent QMS at assigned conferences and meeting
Identify areas that are opportunities for additional messaging to support change management implementations.
Pilot: Help to evolve regional workstream sub-team concept

37. Thank you!
Questions?

38. The eLabels Initiative
Jennie Deem
Regulatory Scientist
Eli Lilly & Co., USA

39. Objectives Introduce TransCelerate eLabels initiative
Current clinical trial labeling challenges
What is an eLabel?
Insights from Patients and Sites
Demonstration
Key considerations for implementation
TransCelerate eLabels plans
The purpose of the meeting today is twofold. One we are pleased to provide an overview of the TransCelerate eLabels initiative and its application for clinical trials. Secondly we welcome your feedback for inclusion into member company’s development of their processes and technology.

40. eLabels Initiative Overview
What does this TransCelerate initiative do?
This initiative will help industry progress on the journey to digitally-supported, patient-centric clinical supply chains. The main output is not an eLabeling system, but an implementation toolkit to facilitate external engagement and uptake in the industry.
What is the approach?
Prepare implementation toolkit to facilitate voluntary, modular adoption of eLabeling and to assist in coordinating Health Authority engagement.
Develop and implement an external engagement strategy including sites, patients and Health Authorities.
Develop technology assessment and proof of concept for regulatory and other stakeholder engagement.
What is the vision?
Act as a catalyst for clinical supply chain transformation, enhanced patient utility, reduced clinical labeling timelines and costs, promotion of consistent, up-to-date information and coordinated, effective Health Authority engagement.
Develop technical plan and charter for e-Labels proof of concepts….

41. Wearables are already used in Clinical trials
How is technology already mainstream and a critically important aspect of our lives?
eDiaries are leveraged extensively in Clinical trials to capture the patient experience
Digital technology is leveraged to secure safe passage on air travel and to pay securely from your mobile device
Wearables are already used in Clinical trials

42. Pain Points of Current Labeling
Common stakeholder pain points across the industry (Pain points may vary by sponsor)
Pain Points of Current Labeling
Low usefulness of booklet labels: Few are opened, limited space and small fonts
Booklet labels may impede functional use of a syringe or auto-injector
Information on label is there to meet regulatory requirements, but may not include helpful information for the patient (eg, administration information)
Patients
Limited to no utility for sites except for the trial alias and kit number to aid in dispensing
Limited or no space to write on the label
Sites
New labeling technology has surpassed regulations
Need to advance public health by accelerating innovation
Health Authorities
Paper labels are static, making updates lengthy and costly
Multiple label groupings are needed to support global trials
Long creation and approval times (multiple months) delay getting medicine to patients
Companies

43. eLabels will be an alternative to booklet labels
Current State
Language neutral content on physical label to identify material
Full regulatory compliant label on electronic device
Universal Label
Study-specific eLabel
Potential Future State
* Detailed data may be added: e.g. SubjectID, investigator name #
TX-4457
Booklet Label
I am sure that you are aware that Pharmaceutical companies employ the use of booklet labels in order to maximize their limited quantity of clinical trial material so that a lot can be used in multiple countries. The booklet labels have a long lead time, are expensive, and surveys show that they are infrequently utilized by the patient. The TransCelerate proposal is to have the full label available, in the country language, via an electronic device. There would still be a physical label attached to the product that would be more universal in nature. For example the lot number, storage conditions, package number, trial indicator, sponsor info, and caution statement would still be contained on the label and would leverage pictograms to in order to make the label more country agnostic.

44. Universal Label Example
The universal label attached to the physical packaging will provide language neutral content that allow for traceability, proper storage, warnings and contact information #
TX-4457
Map ID
Description
Trial or molecule Indicator
Kit Number
www.
Company Address, Website, Logo
Batch or Packaging Reference Number
Storage Conditions
Keep out of the Reach of Children
Device-readable code, e.g., barcode
Caution Statement reference
TX-4457
21 CFR 312
§ Labeling of an investigational new drug.
The immediate package of an investigational new drug intended for human use shall bear a label with the statement "Caution: New Drug--Limited by Federal (or United States) law to investigational use.“
Pictograms from the ISPE Good Practice Guide Booklet Labels, USP Pictograms:

45. Insights on eLabels from Patients
5,000+ previous clinical trial patients surveyed indicated1
(3,000+ US, 109 EU and 1,935 China)
Written and verbal instructions were valued most to ensure compliance
The most helpful instructions via demonstration or verbally from site personnel where patients can ask questions
Most preferred means to obtain information:
(EU and US)
Text message and postal mail (China)
Patient Focus Group (n=12)2
…..and asked for the following information
Medication name
Dosing instructions (especially important when taking multiple medications and/or IMP/placebo from multiple containers)
Contact Information for
Sponsor name
Study nurse
Patient Advocate (specific role for all NIH studies)
Adverse event information
Ability to access only once on their device
Patients expressed dissatisfaction with current labels (booklet and single panels)……
“Font is too small”
“Doesn’t contain the information I need”
Would verbally say it comprised physicians, pharmacists and site staff expressed general support for eLabels; speak to the basic label versus future
**********************
It will be important to collect Health Authority, site, and patient input into the development of our deliverablesthis solution. Our eLabels team has begun to collect feedback from across a number of global sites and are utilizing the ISPE patient surveys that were also conducted globally. So far we have discussed the eLabels project with 23 sites, and over 5000 patients have answered questions regarding their use of labels in clinical trials. The results are summarized on the slide. The sites have been very supportive of the concept of elabels. They agreed that the eLabel would enhance readability, and provide greater efficiency at the sites by reducing manual entry (if this functionality is incorporated), and relabeling at the sites. This could reduce the potential of human error and allow the sites to spend more time focusing on the patient, versus logging in material, restickering, and doing manual accountability.
The patient surveys that were conducted were not specifically focused on elabels, but questions were asked about labels in general. The patients responded that they most value instructions verbally from site personal. This feedback was uniform globally. Elabels will not replace this interaction. However, when patients responded in regards to their preferred means to obtain information, they choose an electronic means. The label was not a preferred method, and was it noted that it is not opened or referenced often.
There was an eLabel pilot conducted in Belgium with 24 patients across 3 different arms. The arms were made up of patients , 40 – 65, and patients greater than 65 years old. And all arms had their dosage instructions modified 4 times over the course of 12 days. The feedback was that the eLabel was clear 92% of the time and the patients felt the eLabel was easy to use or not difficult 96% of the time. Keep in mind that the instructions were changed 4 times, which doesn’t typically occur with static labels.
When shown eLabel prototypes, patients positively responded to:
Appearance: “Clean and simple”
Layout: Easy to find information
User Interface: Colors, layout, bolded headers, etc
Search function
Availability of native language
Update functionality and notifications
Contact info and ability to ease of making a call
Caregiver “labelled bottles with a magic marker to know when to take pills from different bottles”
1Patient Perceptions of Investigational Medicinal Products 2015 surveys in EU (and China), Esther Sadler-Williams 8th March 2016, Frankfurt
2TransCelerate eLabels Patient Advisory Board Sept 2016

46. Insights on eLabels from Investigational Sites
45 sites surveyed in EU, US, Latin America and Japan expressed support for eLabels and acknowledged the potential to:
reduce human error and workload
enhance readability of the label
eliminate relabeling
provide detailed dosing instructions
track compliance
provide notification to patient
allow integration with patient diaries
provide access to supportive videos
Sites
Site Focus Group (n=12)2
…..and asked for the following information
Patient dosing reminders
Electronic ways to improve efficiency and accuracy of drug accountability
Dosing videos and pictures which serve as reminders/information when the patient is at home
Machine readable ways to verify the site picked the right container
Sites indicated patients are motivated and most will use an electronic device to obtain information
Sites also expressed dissatisfaction with current labels (booklet and single panels)……
“Difficult to locate and select correct container based on Kit ID”
“We’d never dispense something like this in a retail setting”
Would verbally say it comprised physicians, pharmacists and site staff expressed general support for eLabels; speak to the basic label versus future
**********************
It will be important to collect Health Authority, site, and patient input into the development of our deliverablesthis solution. Our eLabels team has begun to collect feedback from across a number of global sites and are utilizing the ISPE patient surveys that were also conducted globally. So far we have discussed the eLabels project with 23 sites, and over 5000 patients have answered questions regarding their use of labels in clinical trials. The results are summarized on the slide. The sites have been very supportive of the concept of elabels. They agreed that the eLabel would enhance readability, and provide greater efficiency at the sites by reducing manual entry (if this functionality is incorporated), and relabeling at the sites. This could reduce the potential of human error and allow the sites to spend more time focusing on the patient, versus logging in material, restickering, and doing manual accountability.
The patient surveys that were conducted were not specifically focused on elabels, but questions were asked about labels in general. The patients responded that they most value instructions verbally from site personal. This feedback was uniform globally. Elabels will not replace this interaction. However, when patients responded in regards to their preferred means to obtain information, they choose an electronic means. The label was not a preferred method, and was it noted that it is not opened or referenced often.
There was an eLabel pilot conducted in Belgium with 24 patients across 3 different arms. The arms were made up of patients , 40 – 65, and patients greater than 65 years old. And all arms had their dosage instructions modified 4 times over the course of 12 days. The feedback was that the eLabel was clear 92% of the time and the patients felt the eLabel was easy to use or not difficult 96% of the time. Keep in mind that the instructions were changed 4 times, which doesn’t typically occur with static labels.
“A link for patients to access information on-line would be premium”
When shown eLabel prototypes, sites positively responded with:
Unanimously liked the concept
“Simple and clean”
Liked “push” real time updates and notifications
Making it language friendly allows patients to focus on what is useful to them
Extremely helpful to have detailed dosing instructions
“Patients rip it off or don’t even open it”
1TransCelerate Site Advisory Group and Lilly site interviews (SCRS)
2TransCelerate eLabels Site Advisory Group Sept 2016

47. eMeds/eLabel Technical Pilot1
Objectives
Technical evaluation of the different eLabel and eMeds components
Evaluation of user-friendliness and satisfaction of subjects and sites
Patients Reported
eLabel updates to be very clear and acceptable (92%)
84% of subjects scored smartphone use as easy to very easy, despite only 25% having smartphone experience
Overall positive feedback on ease of using eLabel and clarity of eLabel update notifications
71% of subjects considered eLabel as a very accessible and user-friendly format when viewing it independent of an update
Being notified of dosing changes (eLabel updates) was considered the most important advantage of the eMeds technology
1Janssen eMeds/eLabel Technical Pilot
2 SUBJECT SATISFACTION SURVEYS
+ INDIVIDUAL SUBJECT INTERVIEWS
12 DAYS
24 VOLUNTEERS
3 LABEL UPDATES
18-40 yr
40-65 yr
> 65 yr
Dutch
French
English
“1 morning & 1 evening capsule ”
“2 morning, 1 noon & 2 evening capsule”
“0 morning, 2 noon & 1 evening capsules”
“0 morning, 0 noon & 0 evening capsules”
Morning
Noon
Evening
Day 5
Day 12
Final visit
Ease of eLabel use: Satisfied 63% (Neutral 33%) – aligns with 3rd bullet
1Janssen eMed/ eLabel Technical Pilot

48. eLabels provide benefits across the industry (Benefits may vary by sponsor)
eLabel Benefits
Increases efficiency in clinical development allowing for patients to receive medicines faster
Increases patient safety
Enhanced utility of clinical labels and potential for better compliance, e.g.:
Dosing videos
Supplements to communication
Improved usability (e.g. larger font size)
Patients
Rapid access to up-to-date information
Greater efficiencies in Labeling approaches
Lays a future foundation for engaging with the patient about their medication
Sites
Decreases potential for deviations during extension re-stickering: e.g.: sterility, tamper evident seal, product mix-up, time out of environment
Ensures latest information available for patients
Ties into broader digital and innovation strategies
Health Authorities
Increases operational efficiencies in creation of label
Allows for additional pooling strategies which decreases waste
Decreases reaction time to study changes
Increases options for significant value-adds such as adherence programs, patient analytics, patient education
Companies

49. eLabel Technologies – Push versus Pull Devices may either be provided by sponsor or patients utilize their own device
Pull Technology
Push Technology
Databases
There are multiple systems and means to convey text via an electronic method. In the back-up we list 41 ways and 13 categories that we thought of as we were working on this project. For each technology, each member company sponsor must consider 1) What will be applied to the packaging, 2) how will the label be read and understood, 3) what label content will be embedded in the label vs. in the cloud, URL, etc.. 4) How will the content be updated and acknowledged, and 5)how will the patient receive the label information. There are implementation considerations, pros and cons, for each technology that will need to be taken into account by the sponsor company.
We are going to walk through one demo today that anyone can access regardless of your phone or its operating system. The only requirement is that you have a QR reader which is a free app. Please note that this is a demonstration only and was built in 2012, so the label contents may not have all the regulatory requirements for every country. Each member company sponsor can choose the technology or platform that makes the most sense based upon multiple factors, one of the primary factors is integration with other systems such as IRT, distribution, and labeling libraries. Other factors may be how the sponsor company would like to communicate with the patient. The example we are going to show is a pull system where the patient or the site will scan the bar code in order to obtain their information. Another means could be that the sponsor company would push the information to the patient, similar to a text message, and the patient would have to acknowledge the receipt of the information. I have an example of that and can share with you after the discussion if you are interested.
Please scan the bar QR code if you wish. I will also show you an example. Please note that for this specific example, the label will appear in what ever language you have your phone set. This demonstration is simple to execute and the label information is easy to find.
eLabel and eLabel updates are automatically sent to the patient
Action required by patient to retrieve the eLabel
Demonstration was developed by Sanofi in the ordinary course of business and contributed to TransCelerate to facilitate engagement with Health Authorities.

50. Key Consideration – Redundant Systems and Back-up Plans
Business continuity plan developed
URL printed on universal label to allow user to enter a unique identifier (e.g. Kit Number) to retrieve label
Once label is scanned, it is available in the device history off-line
Can be downloaded to local electronic and/ or hard copy storage
Call number on patient card for trouble shooting and support on device / application usage
Retrain user
Potential Solutions
Digital Examples
Human to Human Examples
Full compliance with 21 CFR Part 11 and Annex 11 Computerized Systems
Potential eLabel Failure Points
Hardware
Software
Device inaccessibility
Loss of power
Broken device
Lost device
Server
eLabel application bug
eLabel application is not properly installed on user’s device
Signal Connectivity
Universal Label
Loss of internet
Loss of wireless signal
Lead in with:
All stakeholder’s we’ve spoken with consistently ask about”:
Elderly or individuals without access to technology (As a reminder, our research is showing this is not as big of an issue as we’d like to believe. Patients are motivated, have a caregiver and there are ways to manage the small minority that truly do not have access)
What if wifi is not available
What about privacy
Each sponsor will need to address these based on the study and where their populate is. Here are some of the ways these issues can be mitigated and this is included in our toolkit.
Unreadable Label

51. Critical Considerations for Sponsor Implementation
Items that must be addressed based upon technology chosen
Patient Privacy
Data Security
Systems Validation
Integration with Other Systems:
Interactive Response Technology (IRT)
Label Content Creation and Approval System(s)
Clinical Supply Demand/Planning and Distribution System(s)
Drug Accountability and Tracking System(s)
Sensor Systems (the Internet of Things)
Study/Site Portals
Enterprise Resource Planning (ERP) System(s)
Based upon the technology that will be chosen by the sponsor company a number of considerations must be addressed. Some examples where either a business continuity plan or controls must in place would include:
What to do in case of loss of power or functionality of the device or if the device is lost.
If the internet is critical to the technology, what other back up means will be available to access the information, such as patient cards, emergency contact information, etc.
Maintenance of patient privacy, data security would need to be addressed. Integration with other systems and system validation will need to performed.
Each of these considerations will need to be addressed by each sponsor company and the solution will vary based upon the technology chosen.

52. Parallel approach results in a shortened timeline
Illustrative Cycle Time Improvement: Conventional Model vs. eLabels Model
Conventional Model
12 – 16 weeks
Up to 30 weeks
Country specific multi-language booklet label design and manufacture
Clinical Supplies packaging
QP release final KIT
Ship to site
Clinic Dispenses KIT
Sequential
QP release final KIT
Ship to site
QA release Country Specific KIT label
Upload country/ language specific label to patient/ caregiver’s device
Clinic Dispenses KIT
Design and approve country specific eLabels
Clinical Supplies packaging
eLabels Model
~ weeks
~1 week
Design Universal Label
Parallel
Parallel approach results in a shortened timeline

53. TransCelerate eLabels Engagement Plans
Gathering stakeholder feedback and evolving the eLabels concepts is a key deliverable of this initiative
FDA
EMA (& other EU agencies)
Health Canada
Industry organizations (ie, EFPIA, ISPE)
eLabels
Concepts
AsiaPac Health Authorities
Mention TransCelerate member companies
Sites
Latin America Health Authorities
Patients (CISCRP)

54. Where we’ve been and where we are going…..
2015
2016
2017
2018
Legend:
eLabels Assets
External Engagements
eLabels Design and Delivery Toolkit
Update
Update
TransCelerate approved initiative
Implementation toolkit for voluntary implementation and to assist in HA engagement
Technology Assessment and Prototype
Technology Landscape Assessment completed
Use cases identified
Proof of concept developed for Health Authority and other stakeholder engagement
Proof of concept update with stakeholder feedback
Tech architecture to be developed
Health Authority Engagements
Ongoing Health Authority engagements
Patient/Site/ Industry Organizations
Ongoing Patient and Site Engagements
Industry organization feedback via meetings and conferences

55. eLabeling Lays the Foundation for Future Innovation
Clinical Supply Labeling – Illustrative Maturity Model
Digital Maturity, Patient Focus
Value
Disconnected
Focus only on paper-based clinical labeling
Link to Repository
Phrase library
Regulations database
Connected
Foundation e-Label functionality
Traceability through adherence and destruction
Digitally Enabled, Patient Centric
Integrated e-Label with expiry updating
Smart packaging with automated temperature tracking
Proactive messaging reminders administration
Links to patient education
TransCelerate is focusing on building the foundation of eLabels to enable future innovation

56. Thank you!
Questions?

57. Appendix

58. Shared Investigator Platform Benefits
Sponsors
Regulators
Investigators
Quality, streamline processes, regulatory compliance, capacity.
Streamlined electronic audit process, insight into trial, harmonized information model.
Productivity (via reduced redundant tasks & streamlined processes), access to information.
Costs related to:
Training
Document exchange
Support & maintain
Help desk
Startup time
Study startup time, redundant training.

59. SIP Enables a Site User to:1
Join the Platform
One login; access to multiple participating sponsors.
Enter data once; maintain your credential centrally for use across SIP sponsors & studies. 2
Build a User Profile
Complete GCP Training
Centralized history. Take GCP training once – credit may be recognized by multiple participating sponsors. 3 4
Prioritize & Manage Your Work
Consolidated view of a site tasks across SIP studies and sponsors.
Maintain information centrally for use across SIP sponsors & studies. Facility is associated to users & studies. 5
Manage a Facility Profile
Central visibility to all SIP Feasibility surveys; shorter surveys due to re-use of SIP user & facility profile data. 6
Complete Feasibility Surveys 7
Access Your Study Workspace
Interact & collaborate with SIP study teams; SIP study materials in one place. 8
Exchange Documents
Post, share and retrieve study documents with SIP sponsors.

60. SIP Enables a Sponsor User to:1
Login to the Platform
Sponsor User has platform access from MC system. 2
Search central information to evaluate PI profiles for new studies. User ownership of profile data improves quality.
Access PI information 3
Assign training across sites; site manager access to centralized training documentation for site staff.
Assign & Approve Training 4
Search central information for new studies; user ownership of profile data improves quality.
Access Facility Profile Data 5
Build/Send Feasibility Surveys
Re-use previous surveys & templates to reduce duplication; centralized surveys & automated follow-up reduce effort. 6
Build a Study Workspace
Unique study workspaces provide centralized access, information & collaboration space. 7
Prioritize Work Across Studies
Consolidated view of tasks and deadlines across SIP studies. 8
Exchange Documents
Post and retrieve SIP study documents quickly and easily. 9
Metrics & Reporting
Quickly and easily access key metrics and standard reports.

61. After Release2* SIP Will Enable a Site User to:
One login; access to multiple participating sponsors
Join the Platform 1
Centrally maintain your credentials; generate a detailed WORD formatted CV.
Build a User Profile 2
Centralized history. Take GCP & Study training Request credit for completed training.
Complete Training 3
Consolidated view of a site tasks across SIP studies and sponsors.
Prioritize & Manage Your Work 4
Maintain information centrally for re-use across SIP Sponsors & studies. Broader facility relationships and affiliations
Manage a Facility Profile 5
Manage and track all SIP surveys (feasibility, study closeout, satisfaction); Site User dashboards; survey analysis
Complete Sponsor Surveys 6
Interact & collaborate centrally with study team; study materials in one place. Manage study site startup activities
Access Your Study Workspace 7
Post, share and retrieve study documents with SIP sponsors; auto complete standard forms using SIP data
Exchange Documents 8
Investigators can view new study opportunities with participating SIP Sponsors; view historical enrollment data
New Opportunities/Historical data 9
Central place to view and acknowledge safety letters from participating sponsors
Access Safety Notification 10
* Orange text denotes proposed R2 new features/functions

62. SIP Public Landing Page

63. Site User Landing Page; Consolidated Task List, quick access to Studies
Notifications, Configurations, Searchable Help
Task Dashboard
Consolidated Task List
Quick Access
to Studies

64. Centralized location for User Profile; Accessible by all SIP Sponsors
Site
Abbreviated CV generated off User Profile Data

65. Study Workspace for Collaboration with Sponsors
Site
Sponsor
Access to Study Milestones, Study Site Addresses and Staff, Study Training, and Sponsor Contacts
Study Task List
Quick access to Study Links
The Latest Study News

66. Securely Exchange Study Documents with Sites; Enter metadata for easier searching
Sponsor

67. Manage Feasibility Surveys in a Central Location; Build Survey Templates
Sponsor