This presentation will be presented by Behtash Bahador, CISCRP's Senior Manager of Quality and Compliance at the Disclosure & Transparency for Clinical Data Summit on August 13-14, 2018. To learn more at CISCRP's Lay Summaries visit www.ciscrp.org or contact Jay Matthews at jmatthews@ciscrp.org.

1. Patient Perspectives on (“Lay”) Trial Results Summaries
Behtash Bahador, Senior Manager, Quality and Compliance, CISCRP
2nd Disclosure & Transparency for Clinical Data Summit, August 13 -14, 2018

2. Agenda
1. Stakeholder Perspectives on Value of Trial Results Summaries
2. Patient Communication Best Practices
3. Trial Design and Operational Planning
4. Patient Engagement
5. Questions and Answers
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

3. TRANSITION SLIDEStakeholder Perspectives on
the Value of Trial Results Summaries

4. Patients and the Public
Information Most Interested In Receiving
After Participating in a Clinical Trial
2%
25%
35%
46%
49%
59%
72%
73%
Other
Information about scientific publications
Information about upcoming clinical
research studies
The brand name for the study drug
Drug approval status by the regulatory
agency in your country
Whether I received the study drug or
placebo (sugar pill/inactive substance)
A summary of the study results
My individual study results (i.e.
procedures and test results)
Source: 2017 CISCRP P&I Study, n=12,427, Base: All respondents
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

5. Patients and the Public
Importance of Receiving Trial Results Summaries
Source: 2017 CISCRP P&I Study, n=12,427, Base: All respondents
Very or
somewhat
important
91%
Not very or at
all important
9%
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

6. N. America Africa Europe S. America Asia Pacific
Very 66% 68% 57% 68% 55%
Somewhat 27% 28% 32% 25% 30%
Total 93% 96% 89% 93% 85%
Source: 2017 CISCRP P&I Study, n=12,427, Base: All respondents
Importance of Receiving Trial Results Summaries
By Region
Patients and the Public
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

7. Study Participants
Importance of Receiving Trial Results Summaries
Source: 2017 CISCRP P&I Study, n=2,194, Base: Participated in a Study
Very important
62%
Somewhat
important
29%
Not very
important
6%
Not at all
important
3%
84% report
somewhat or very important
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

8. Study Participants
Source: 2017 CISCRP P&I Study, n=2,194, Base: Participated in a Study
Phase I/IIA
(n=229)
Phase I
(n=356)
Phase II
(n=225)
Phase III
(n=269)
Phase IV
(n=118)
Very or
Somewhat
87% 85% 85% 90% 86%
Importance of Receiving Trial Results Summaries
By Phase
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

9. Research Community
2007 FDAAA: possible dissemination of “summary of the clinical trial and its results that
is written in non-technical, understandable language for patients.”
2013 Declaration of Helsinki: “All medical research subjects should be given the option of
being informed about the general outcome and results of the study.”
2013 PhRMA and EFPIA: “Principle of responsible data sharing.”
2014 EU CT Regulation 536/2014: “Sponsor shall submit to the EU database a summary
of the results of the clinical trial…It shall be accompanied by a summary written in a
manner that is understandable to laypersons.”
2015 Institute of Medicine: “A matter of public transparency and respect for
participants.”
2015 US SACHRP: “Agencies should take steps to promote the return of general results to
subjects, due to the value of providing general results to subjects.”
2017 USA: NIH HHS Final Rule: “Does not require the submission of technical or non-
technical summaries”
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

10. TRANSITION SLIDE
Leveraging Lay Summaries
in Patient Engagement

11. Patients and the Public
Information Most Interested In Receiving
After Participating in a Clinical Trial
2%
25%
35%
46%
49%
59%
72%
73%
Other
Information about scientific publications
Information about upcoming clinical
research studies
The brand name for the study drug
Drug approval status by the regulatory
agency in your country
Whether I received the study drug or
placebo (sugar pill/inactive substance)
A summary of the study results
My individual study results (i.e.
procedures and test results)
Source: 2017 CISCRP P&I Study, n=12,427, Base: All respondents
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

12. Study Participants
Did you receive any reports or updates on the results of
the study once you finished the clinical research study?
Source: 2017 CISCRP P&I Study, n= 2,194, Base: Participated in a Study
Yes
30%
No
53%
I don't
remember
17%
Compares to:
2013 53%
2015 51%
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

13. Sponsor Engagement with Patients
Source: 2017 CISCRP P&I Study, n=12,427
72%
want a
summary
91%
think it’s
important
53% no
updates
or results
Missed
Patient
Engagement
Opportunity
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14. Source: CISCRP Perceptions & Insights Study 2015, n=3,152, Base: Participated in a Study
Of participants whose study… % have discussed experience
“Fell well short of my expectations” 49%
“Greatly exceeded my expectations” 89%
Of participants whose study… % received report/update on results
“Fell well short of my expectations” 10%
“Greatly exceeded my expectations” 48%
Improve Experiences
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15. Investigative Site Engagement
Investigative Sites find value
• 49/50 support overall approach and perceive ethical
obligation
• Reinforce site-patient rapport and provide much-
needed closure
• Minimal burden
• Valuable content
• Sponsor – Investigative Site Relationship
Source: 2016 CISCRP Investigative Site Follow-up Evaluation
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

16. Build Trust and Research Literacy
• Increase visibility, impact and transparency
of scientific research
• Building blocks for broad communication of
research
• Facilitate knowledge transfers between science
and the public
• Invitation for public dialogue
• Increase links with media
Lay summaries enhance science communication, Lauren M. Kuehne, Julian D. Olden
Proceedings of the National Academy of Sciences Mar 2015, 112 (12) 3585-3586; DOI: 10.1073/pnas.1500882112
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

17. More Communication is Better
Summaries of Clinical Trial Results for Laypersons (v2 released 22
Feb. 2018)
• “The summary for lay persons in the EU database should not be
regarded as the only way of communicating with trial
participants…”
• “….sponsors may provide trial results to investigators or third
parties to feedback to patients who have taken part in their trials,
along with an acknowledgement of their contribution and an
expression of appreciation”
Patients also want ongoing communication*
• 86% of trial participants not only want to be told the results of their
trial, but also to be updated regularly after last visit and before
receiving results.
• Preferred frequencies ranged from 3-month to annual update
schedules.
*Getz et al. 2012. Expert Rev. Clin. Pharmacol. 5(2): 149-156
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18. Trial Results Summaries
4 Steps of Engagement with Participants
Informed
Consent
First or Last
Visit
Post-Trial
Outreach
Trial
Results
Summary
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19. Going Beyond Regulatory Requirements
Data
Selection
Data
Presentation
Scope of
Reporting
•Interim results not required by EU CTR but best practice in long-
running studies and vulnerable populations
•Sources and scope can vary when ‘voluntary’ TRS
•Ex. peer-reviewed publications or poster
•Ex. multi-study report across a compound / program
Process
•Some elements of the EU CTR that are not critical for
understanding of results by patient or public audiences can be
omitted.
•Best practices in patient communication can be followed to ensure
the most relevant information is reported
Content
•EU CTR requires PDF; CISCRP has provided audio/visual formats
•Innovative approaches to enhance understanding e.g. interactive
designs, teach-back mechanisms, investigator or patient-physician
involvement
Format
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

20. Click to edit Master title
style
Formats for Trial Results Summaries
Print-and-Web-
Ready Document
Video format Audio Format
EU Guidance: “Ethical considerations for clinical trials on medicinal
products conducted with minors” (ch. 19, p28)
“…efforts should be made to make the laypersons’ summary
understandable by children who participated in the trial (those able
to read, from 6 years on) using age-appropriate material, and
preferably involving children and parents in the preparation.
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

21. Data Selection: Endpoint Reporting
The Law: Annex V Clinical Trials Regulation (EU) No 536/2014:
7. Overall results of the clinical trial
The Guidance: Summaries of Clinical Trial Results for Laypersons
(26 Jan. 2017; v2 released 22 Feb. 2018):
“The primary endpoint(s) and results by trial arm which were
pre-specified by the statistical analysis plan as a primary endpoint.
Sponsors should reference the complete list of outcomes based on all
endpoints available in the technical results summary for each clinical trial in
the EU database including patient relevant secondary endpoints.”
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

22. Study Participants & Public
• Transparent and accessible: “Deserve to know”
• Relevant and tangible: “Want to know”
Sponsor Teams and Partners
• Clinical benefit
• Statistical significance
• Support demonstrated treatment effect
Balancing Stakeholder Interests
Data Selection: Endpoint Reporting
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23. TRANSITION SLIDEBest Practices in Patient Communication

24. The full title of your study is: A 24-week treatment,
multicenter, randomized, double blinded, double dummy,
parallel-group, clinical trial evaluating the efficacy and
safety of aclidinium bromide 400 μg/formoterol fumarate
12 μg fixed-dose combination BID compared with each
monotherapy (aclidinium bromide 400 μg BID and
formoterol fumarate 12 μg BID) and tiotropium 18 μg QD
when administered to participants with stable chronic
obstructive pulmonary disease
Short study title: A study to learn how 2 drugs taken
together affect participants with COPD compared to taking
them separately.
Plain Language: Word Choice
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25. “Side effects”?
Summaries of Clinical Trial Results for Laypersons (v2; 22 Feb. 2018)*
Annex 1 – 6. What were the side effects?
“…define ’adverse reactions’ as those adverse events where
the investigator has indicated that there is a possible causal
relationship between the event and the investigational
medicinal product.”
“Consider using a simple term, such as “side effects” to refer to
adverse reactions, explaining how ‘side effects’ are defined for
the purpose of the lay summary.”
Plain Language: Word Choice
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

26. Plain Language: Word Choice
“Side effects”?
What medical problems did participants have during the study?
This section is a summary of the medical problems participants
had during the study that the doctors thought might be related to
the study drugs. These medical problems are called “adverse
reactions”.
An adverse reaction is considered “serious” when it is life
threatening, causes lasting problems, or requires hospital care.
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27. Narrative Content
o Provide high-level contextual statements
 Each outcome presented
 Safety
o Clear statements to address misrepresentation:
 Statistical significance
 Power
o Present data points clearly (BOTH percentage and
absolute value)
o Formatting matters!
Health Literacy and Numeracy
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

28. Narrative Content
Health Literacy and Numeracy
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

29. Narrative Content
Health Literacy and Numeracy
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

30. Narrative Content
Health Literacy and Numeracy
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

31. Click to edit Master title
style
Patient and Public Review
Long standing health literacy best
practice
Helps ensure readability, identify
perceptions of promotion or bias
EU Lay Summary Guidance:
“Sponsors should consider testing the readability of an
initial version of the study results summary with a small
number of people who represent the target population.”
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

32. • Help make informed decisions
• Beyond lay writing – design, health literacy,
numeracy, patient communication
Experience and
capabilities
• No vested interest in trial outcome
• Patient voice included in every summary
• Developing Summaries with the Patient in Mind
Independent
advocates
• Regulatory Compliance
• Translation
• Distribution
Minimize Operational
Burden
Partners and Patient Advocates
How to ensure Trial Results Summaries are non-
promotional, non-bias and easy-to-understand?
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

33. TRANSITION SLIDETrial Design and Operational Planning

34. Patient Advisory Boards
DISCUSSION TOPICS:
 Study design elements
 Informed Consent Form
 Branding and study positioning communication
 Patient recruitment promotional messages; other patient
facing study materials
 Clinical trial medicine kit design and administration
 New technology solutions
FORMAT:
Structured, facilitated in-person meeting
(single meeting or ongoing series)
COMPOSITION: 6 to 8+ Patients / Caregivers
TIMING/LOCATION: Half-day meeting in convenient metropolitan location
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

35. Patient Journey Workshops
MAPPING EXERCISES:
 Learning about a clinical trial
 Informed Consent Process
 Screening visit
 Treatment period
 Study follow-up
FORMAT:
Structured, facilitated in-person meeting
(add-on to Patient Advisory Board or stand-alone)
COMPOSITION: 6 to 8+ Patients / Caregivers
TIMING/LOCATION: Half-day meeting in convenient metropolitan location
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

36. A Phased Approach
• 2-10 studies, EU, US, and multinational, representative TAs
• Management model, Assess Capabilities, Partner or Vendor selection
• Template development: Align with EU requirements and best practices
Pilot projects:
Establishing
Implementation
•Identify cross-functional implementation team
•SOPs; Work instructions, Budgets, site contracts and communications,
ICF
•Training materials on requirements, responsibility and process
internal + external
Portfolio Wide
Implementation
• Governance structure and active management
• Template and Process improvements
• Don’t insulate, COLLABORATE!
Monitoring and
Refinement
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

37. Set Up for Success
Getting buy-in from all pertinent players is essential to setting the
program up for long term success.
The Right
Team
Members
and Decision
Makers
Clinical Trial
Management
Clinical Trial
Transparency
Legal
Regional
Clinical
Operations
RegulatoryMedical
Drug Safety
Medical
Writing
Senior
Management
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

38. Example Pilot Program
Study Description Study Status Pilot Program Goal
Study 1 Phase II,
Oncology
Pre-initiation Evaluate full program with
volunteers, sites, and
internally over length of
studies
Study 2 Phase III,
Immunology
Pre-initiation
Study 3 Phase II,
Immunology
Recently completed Immediately evaluate
materials with volunteers,
sites, and internallyStudy 4 Phase III,
Oncology
Recently completed
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

39. Patients and the Public
Information Most Interested In Receiving
After Participating in a Clinical Trial
2%
25%
35%
46%
49%
59%
72%
73%
Other
Information about scientific publications
Information about upcoming clinical
research studies
The brand name for the study drug
Drug approval status by the regulatory
agency in your country
Whether I received the study drug or
placebo (sugar pill/inactive substance)
A summary of the study results
My individual study results (i.e.
procedures and test results)
Source: 2017 CISCRP P&I Study, n=12,427, Base: All respondents
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

40. Example Scale-Up Implementation
2018
• Pilot processes with range of studies (e.g., phases, TAs, countries)
• Start with printed communications (current patient preference)
• Engage stakeholders and develop SOPs, templates and trainings
2019
• Standardize and streamline processes (especially document review)
• Expand implementation, but keep scope manageable
• Begin integration with study start-up activities (ICF, site contracts, etc.)
2020+
• Implement programs across portfolio
• Continue simple dissemination to patients (print and/or website)
• Explore additional program elements
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.

41. Thank You! Any Questions?
Behtash Bahador
Senior Manager, Quality and Compliance
bbahador@ciscrp.org
Follow us!
C.I.S.C.R.P CISCRP @CISCRP @CISCRP
©2018 CISCRP All Rights Reserved. No part of this document may be reproduced without written consent from CISCRP.