Patient Engagement in Health Economic and Outcomes Research: Current and Future ISPOR Initiatives, presentation from the ISPOR 20th International meeting Philadelphia, May 2015, by the Patient Centered Special Interest Group

1. FORUM
PATIENT ENGAGEMENT IN HEALTH ECONOMIC
AND OUTCOMES RESEARCH:
CURRENT AND FUTURE ISPOR INITIATIVES

2. Moderator
FORUM
Todd Berner, MD
Co-Chair Patient Engagement In Research Working Group
ISPOR PATIENT CENTERED SPECIAL INTEREST GROUP

3. Speakers
FORUM
Rob Camp,
Communications Manager, EUPATI,
Barcelona, Spain
Amie Scott, MPH,
Business Health Research Analyst, SEAS Capital Partners, LLC,
Plymouth, MI, USA
Don Husereau, MSc,
Senior Associate, Institute of Health Economics
Adjunct Professor, Epidemiology and Community Medicine,
University of Ottawa, Ottawa, Canada
Emil Chiauzzi, PhD,
Research Director, PatientsLikeMe,
Cambridge, MA, USA

4. FORUM ISPOR Involvement with Patients
PATIENT CENTERED SPECIAL INTEREST GROUP
Patient Engagement In Research Working Group
PATIENT REPRESENTATIVE ROUNDTABLE
European
North American

5. FORUM
PATIENT ENGAGEMENT IN RESEARCH WORKING GROUP
Goal:
To determine how best to involve patients and their
representatives in the research process by identifying:
 The stages at which patients should be involved
 The level of their involvement in each stage
 The challenges that will face the researchers
 Recommendations

6. FORUM
Goal:
 provide an opportunity for patient representatives to learn
and discuss how they can participate in the assessment of
a new health technology (drug, medical device, diagnostics,
biopharmaceutical, vaccines)
 increase their involvement in health policy decision making.

7. Patient involvement in health
technologies research
Are they involved enough?
ISPOR 20th International Meeting
Philadelphia PA 18 May 2015
The project is receiving support from the Innovative Medicines Initiative Joint Undertaking under grant agreement n°
115334, resources of which are composed of financial contribution from the European Union's Seventh Framework
Programme (FP7/2007-2013) and EFPIA companies.

8. Patient involvement is patient engagement is not
recruitment! (recruitment is recruitment)
It is a process of utilizing the expertise we have to
improve the research process and shape it together
What do I mean by patient involvement?

9. Why patient involvement/engagement?
We make the research better
A wide range of stakeholders can give research teams
advice about research questions, procedures and conduct
Stakeholders, especially community stakeholders
(patients), have critical knowledge about local cultures and
dynamics of the disease that trial sponsors/designers may
lack. Their feedback can help ensure that the research and
procedures are culturally sensitive and appropriate
This advice can lead to better (and faster) recruitment,
better retention, better adherence, better data, and better
likelihood of uptake of interventions should they be safe
and effective (ie, better research)
It is their right

10. Are there Guidelines?
Standard practices for stakeholder engagement.
They provide trial funders, sponsors, and
implementers with systematic guidance on how
to effectively engage with stakeholders in the
design and conduct of clinical trials.
YES, they exist and you can adapt any of them!

11. Community Stakeholders are:
Patients and individuals and groups who are
ultimately representing the interests of people who
would participate in a trial.
patient advocates, representatives
ex-patients (cured, etc. with experience!)
family, caregivers
Who is a Community Stakeholder?

12. Examples of Research Stakeholders

13. • Respect
• Mutual Understanding
• Integrity
• Transparency
• Accountability
• Community Stakeholder Autonomy
Guiding principles

14. Where can it be applied?
Formative Research Activities
Stakeholder Advisory Mechanisms
Stakeholder Engagement Plan
Stakeholder Education Plan
Communications Plan
Issues Management Plan
Site Selection
Protocol Development
Informed Consent Process
Current standards
Access
Co-morbidities and Care
Trial-related harm
Accrual, follow-up and exit
Trial closure and dissemination
Post-trial access

15. Stakeholder Advisory Mechanisms
Community Advisory Boards (CABs) are one of many
ways (formal or informal) that research teams can
engage with stakeholders

16. Ex. about Protocol Development

17. • involvement in defining the priorities in
research activities
• involvement in better protocol design, e.g.
defining trial endpoints
• involvement in benefit-risk assessment,
• involvement in data monitoring committees
• A reminder of multidisciplinarity
Concrete involvement

18. Implementation, monitoring, and
evaluation
• Impact
– Did the engagement improve the research? How?
– Did stakeholders provide useful feedback on the design of
the study and the protocol?
– How do various stakeholders feel about the quality of
the engagement process and the relationships?
– Did community stakeholders feel their inputs and feedback
were listened to and addressed?
– All stakeholders can be involved in the evaluation
process and give their perspectives

19. Application/implementation
• It is a learning process!
• It will be different in every setting. There
isn’t one answer. And there won’t be one
answer.
• It will depend on country, institution,
sponsor, etc.

20. EUPATI Training and Information
• By 2017, there will be a searchable education website with
teachable and downloadable information in 7 languages
– providing user-friendly information and education resources on the
research development process that will further community
understanding of these issues
– create the leading public library of patient-friendly information on
research development and research in the seven most common
languages in Europe
– establish a sustainable website and information service to provide
up-to-date information
– to show you how to get involved in research and development, to
support ongoing and future research and clinical trials
– Supported by 4 patient umbrella org’s, 3 universities, 7
int’l NGOs and some 30 pharmaceutical companies; non-
disease-specific, non-treatment specific

21. Our future
There are very competent and willing science-
oriented partners in the patient community
• EUPATI is working on increasing the number of
well-educated advocates
• Education, education, education
• Implementation, implementation,
implementation

22. Thank you
• To all of you
• www.patientsacademy.eu
• rob.camp@patientsacademy.eu

23. PATIENT ENGAGEMENT IN HEALTH ECONOMIC AND OUTCOMES
RESEARCH: CURRENT AND FUTURE ISPOR INITIATIVES
Emil Chiauzzi, Ph.D.
Research Director
PatientsLikeMe
May 18, 2015

24. A patient network that improves lives & a real-time research platform that
advances medicine
About PLM
patients data insights
• 25+ million structured data
points
• 3+ million free-text posts
• 10+ PROs
• Increasing third-party data
integration
• 300,000+ patients
• 2,000+ conditions
• Ongoing
engagement
• Deep trust
• 60+ peer-reviewed papers
• Patient-generated
taxonomy
• Safety monitoring platform
• Endpoints in clinical trials
• Dozens of biotech/pharma
relationships

25. What Members Do
• Document changes to
symptoms and new
treatments, triggers and
side effects
• Generate a real-world
outcomes research data
Learn
about living with and
treating life-changing
conditions
• Answer the question
“Is this normal?”
• Learn from aggregated
patient treatment and
symptom data
Connect
with others sharing those
experiences
• Search patients based on age,
gender, disease, treatment,
symptoms, interests, etc.
• Day-to-day support
• Live better, together
Track
their history and progress
over time

26. Structured Data Overview
Patient Experience & QoL
PatientsLikeMe Data FieldsPatient Reporters Stakeholders Data Utilization
Care
Management
Access &
Reimbursement
Research
Registries
CER, PCOR,
HEOR
• Industry
• Regulators
• Payers/Plans
• Researchers
• Providers
• Patients
Conditions
Age
Gender
Race
Ethnicity
Location
Payer type
Active Safety
Surveillance
Diagnostic Journey
Symptoms
Primary / Secondary
Hospitalizations
Comorbidities
Treatments
Effectiveness
Dates of Therapy
Indication for Use
Adherence History
Side Effects
Stop Reasons
Advice / Tips
Related Data
Labs, Tests, BMI
Free Text Narrative

27. Engagement Over Time
Member Communications
• 89% of logins are triggered
by an engagement
message
• These communications
include monthly
newsletters, blog posts,
social media, etc.
• Dedicated team of
Community moderators
Tracking and Social
• Patients can signup for
reminders to update their
profile
• Patients can view other
member profiles and
connect
• “Data for Good” campaign
Research Opportunities
• Patients can participate in
longitudinal health studies with
PLM partners
• PLM supplements traditional study
recruitment by sending targeted
outreach to potential subjects

28. Givebacks

29. • Funded by RWJF
• World’s first open-participation
research platform for creating
patient-reported health outcome
measurements
• A hub where researchers, clinicians
and patients can work together to
increase the understanding of
disease – faster than ever before
• Year 1 (2013): researcher-led
measure development
• Year 2 (2014): patient-led measure
development www.openresearchexchange.com
Open Research Exchange

30. Wearables
“Out of box” experience
Tailored communications
Informed consent
IRB review
Research protocol design
Device procurement
Data architecture design
Product enhancements
Study participant selection
Study execution
Insight generation

31. Trial Access: Leveraging the PLM Platform
 Attitudes towards
clinical trial aspects
(randomization,
open access)
 Opinions on pt
involvement in CT
 Triggers for interest
in clinical trials
 Exposure to media
and offline
marketing channels
 Past experiences in
clinical trials
 Ability to
participate in future
trials
 Concerns / factors
in deciding to
participate
Key domains
relevant to trial
design
X
 Age
 Sex
 Geography
 Primary condition
 Quality of life
 Disease
progression
=
 Ability to quickly
identify trial
issues before
beginning trial
 Design a more
specific / targeted
survey around a
specific clinical
program
 Understand
patient
perspectives on
clinical trials
across conditions
Trial design
effects
Stratification

32. PatientsLikeMe Publications

33. Contact Information
Dr. Emil Chiauzzi, PhD
PatientsLikeMe Research Director
echiauzzi@patientslikeme.com
617-674-1846

34. As a newly formed SIG, to guide our direction and
provide materials that are in line with the needs of the
ISPOR community, we conducted an informal survey.
The objective of conducting this informal survey
was to better understand how aspects of patient
engagement in outcomes research was perceived
and implemented among the ISPOR community.

35.  Setting
ISPOR Annual Meeting (Montreal, June 2-4, 2014)
ISPOR-EU Meeting (Amsterdam, November 8-12, 2014)
 Structured Interview conducted by SIG members
Interviews guided by an survey tool consisting of
29 questions (closed and open-ended).
 Purposive sample
Requested conversation regarding patient engagement
with ISPOR members attending outcome sessions

36.  Analysis of informal survey
 Patient Engagement along Outcomes Research Continuum2
 Preparation: Agenda setting
 Execution: Study design, recruitment; Data collection and
analysis
 Translation: Dissemination, implementation and evaluation
1Carman, 2013; Exhibit 1: A multidimensional Framework for Patient and Family Engagement in Health and Health Care
2Domecq, 2014; Figure 3 Methods and Phases of Engagement

37. Framework: Preparation, Execution, Translation
Domecq, 2014
n = 142
studies

38. Primary Work Areas N=39 N=7443
Industry 22 (56.4%) 3385 (45.5%)
Health Research 7 (17.9%) 1871 (25.1%)
HealthCare Communications 0 (0.0%) 334 (4.5%)
Academia 7 (17.9%) 1101 (14.8%)
Managed Care 2 (5.1%) 116 (1.6%)
Government 1 (2.6%) 502 (6.7%)
Clinical Practice 1 (2.6%) 134 (1.8%)
Montreal meeting
(n=21, 53.8%)
Amsterdam meeting
(n=18,46.2%)
* Primary work areas reported by ISPOR membership
between September 15, 2013 –September 15, 2014
Respondent Characteristics

39. N=39 Yes No
Are you aware of any patient engagement activities,
initiatives in your organization/industry/academia?
32 (84.1%) 4 (10.3%)
Awareness of Patient Engagement at current
organization

40. N=39 Yes No
Don’t
Know
Brought patients/advocacy groups in to your
organization to meet with researchers?
18 (46.2%) 7 (17.9%) ---
Involve patients in discussion with approval or
review agencies?
11 (28.2%) 16 (41.0%) 2 (5.1%)
Personal Experience: Patient Engagement in
Preparation Phase

41. N=39 Yes No
Don’t
Know
Involve patients or patient community in study
design?
16 (41.0%) 10 (25.6%) ---
In research methods or clinical trials? 2 (5.1%) 25 (64.1%) ---
In write up of patient information leaflets? 4 (10.3%) 23 (59.0%) ---
In informed consent document creation? 4 (10.3%) 22 (56.4%) 1 (2.6%)
Collecting Data from patients? 29 (74.4%) 3 (7.7%) ---
Qualitative Data Collection ? 24 (61.5%) 8 (20.5%) ---
Quantitative Data Collection? 15 (38.5%) 17 (43.6%) ---
Involve patients in recruitment plans for studies? 3 (7.7%) 24 (61.5%) ---
Personal Experience: Patient Engagement in
Execution Phase

42. N=39 Yes No
Involve patients in discussion of results to facilitate
interpretation?
12 (30.8%) 16 (41.0%)
Personal Experience: Patient Engagement in
Translation Phase

43. Challenges: Patient Engagement in Research
‘
It’s a bad idea’
‘Not helpful if not complete information from patients’
‘Concerns about social media, challenges about regulation, approach may be seen
as commercial’
‘Access to participants without all kinds of approvals, how to make interactive and a
win-win situation’
‘Costly, so should be need based’
‘Patient advocacy groups feeling alienated due to budget constraints’
‘Keeping the underserved community engaged’
‘How prepared are patients to engage?’
‘Motivating clinicians to involve patients in clinical routine, patient follow-up, missing
data after the intervention/engagement’

44. Conclusions
 No shared notion of consistent structure of
methodology for patient engagement in OR
among the ISPOR community; No one
mentioned any frameworks used.
 Currently no standard checklist or structure in
methodology that meets the needs of the ISPOR
outcomes research community
 Although our sample may be skewed, affirmed
the need for a toolkit of practical methodology for
use in health outcomes research

45.  Update on Patient Representatives
Roundtable Europe & CASE REPORT on
patient involvement in payer healthcare
decisions to access new therapies
FORUM

46.  First European Roundtable - 5 November
2013, ISPOR 16th Annual European
Congress in Dublin, Ireland
 Second European Roundtable – 1-
November 2014 - ISPOR 17th Annual
European Congress, in Amsterdam, The
Netherlands

47.  Promote opportunities for collaboration
 How to improve Ensure representation and
understanding of patient perspectives in
research? HTA?
 How to help researchers understand patients
and patients understand researchers and
create linkages

48.  1 page template developed by the European
Roundtable similar to EUPATI Case Report
for clinical research
 Gather descriptions of HTA interactions with
patients to
 Understand good practices/successes
 Understand continued challenges/barriers

50. Sign up as Review Group Member
• Business card to Kelly
• Sign-up sheet
• Website: Interest Groups
• Join ISPOR Special Interest Groups
Select ISPOR Patient Centered Special Interest Group

51. FORUM SLIDES are AVAILABLE
Go to the ISPOR PHL MEETING
home page and click on the orange
Released Presentations menu
OR
via this link to ISPOR’s
Released Presentations webpage:
http://www.ispor.org/Event/ReleasedPresentations/2015Phila
delphia