CER 2016 Hernandez patient engagement

Team Science:
Partnering with Patients
Adrian F. Hernandez, MD, MHS
Director, Health Services and Outcomes Research
Associate Director, DCRI

All Rights Reserved, Duke Medicine 2007
What problems are we trying
to solve?
1

A Persistent Problem – Major Gaps in Evidence
3
Tricoci P et al. JAMA 2009;301:831-41

Level of Evidence A
Current Guidelines*
*Guidelines expressing*Guidelines expressing
Level of EvidenceLevel of Evidence
11.7%11.7%
26.4%26.4%
15.3%15.3%
13.5%13.5%
12.0%12.0%
22.9%22.9%
6.4%6.4%
6.1%6.1%
23.6%23.6%
0.3%0.3%
9.7%9.7%
11.0%11.0%
19.0%19.0%
4.9%4.9%
4.8%4.8%
0%0% 10%10% 20%20% 30%30%
AFAF
Heart failureHeart failure
PADPAD
STEMISTEMI
PerioperativePerioperative
Secondary preventionSecondary prevention
Stable anginaStable angina
SV arrhythmiasSV arrhythmias
UA/NSTEMIUA/NSTEMI
Valvular diseaseValvular disease
VA/SCDVA/SCD
PCIPCI
CABGCABG
PacemakerPacemaker
Radionuclide imagingRadionuclide imaging

Trial Hyperinflation
Berndt E, Cockburn I. Monthly Labor Review, June 2014

The Driver is Complexity

Surprisingly….. Less Participation

How Can We Engage Patients
(Participants)?
2

Post
Approval
Studies
Create
stud
y
proc
edur
e
Implemen
t the
Stud
y
Monitor
the
stud
y
Analyze data
and
interpret
results
Dissemi-
nate
study
infor
matio
n
FDA Review
and
Approv
al
Develop
the
study
conc
ept
Secure
Funding
Prepare
the
Stud
y
Proto
col
• Provide input on study
design such as barriers to
participation, study
endpoints, and risk/benefit
perception
• Provide input on study
design such as barriers to
participation, study
endpoints, and risk/benefit
perception
• Recruit study participants
• Serve as a peer advocate
during the informed
consent procedure
• Recruit study participants
• Serve as a peer advocate
during the informed
consent procedure
• Provide
feedback on how
the patient
community will
view results
• Provide
feedback on how
the patient
community will
view results
•Work with research team to ensure
study participants get feedback from
study
•Write newsletter articles or blog
about results
• Co-present results with researcher
at a conference or support group
•Work with research team to ensure
study participants get feedback from
study
•Write newsletter articles or blog
about results
• Co-present results with researcher
at a conference or support group
•Serve on a Data Safety
Monitoring Board
• Provide recommendations
for revising study protocol if
changes need to be made
•Serve on a Data Safety
Monitoring Board
• Provide recommendations
for revising study protocol if
changes need to be made
•Work with researcher to
secure NIH, PCORI,
AHRQ funding
• DART & Halo Therapeutics,
e.g. raise seed funding
•Fundraise for own research
•Work with researcher to
secure NIH, PCORI,
AHRQ funding
• DART & Halo Therapeutics,
e.g. raise seed funding
•Fundraise for own research
• Help finalize eligibility
criteria within the study
protocol
• Assist in creating the
informed consent form
• Advise study recruitment
• Help finalize eligibility
criteria within the study
protocol
• Assist in creating the
informed consent form
• Advise study recruitment
Across the continuum?
• Provide information
on unmet need aand
therapeutic burden
• Interest of research
question to patient
community
• Provide information
on unmet need aand
therapeutic burden
• Interest of research
question to patient
community
• Serve on FDA
advisory
committees or post-
market surveillance
initiatves
• Serve on FDA
advisory
committees or post-
market surveillance
initiatves
Courtesy: Bray Patrick-Lake, MFS

Common problems with protocols from patient
perspective
• Exclusion/inclusion criteria so
stringent that no “real” patients meet
criteria
• Study not feasible
– Procedural burden too high
– Unmanageable dosing regimen
– Too many study appointments
– Location not convenient
– Pediatric patients needing to be
seen during school hours

Perspectives on Patient Engagement
Per Investigator (Worries)
• Single-minded focus on
cures
• Disrupt or delegitimize
the peer review process
• Bring an agenda or too
much emotion
• Lack sufficient experience
to speak to essential
issues of scientific merit
Per Patient (Benefits)
• Inspire scientists
• Improve plans for
recruitment, retention,
outreach, follow up,
dissemination
• Provide “real life”
perspectives on
risk/benefits
• Serve as reminder to
ultimate end product of
concrete health benefits
rather than an ‘academic’
only product

Meaningful Patient Engagement is often a
foreign language
Engagement Worst to Best
• Online reports &
anecdotes
• Patient visits
• The sign off
• Survey
• Focus group
• Patient Stakeholders
• Co-investigators
Translation to Researchers
•隨機雙盲對照研究
• 察性研究观
•病例對照研究
•病例對照研究
•案例系列
•病例報告
•想法，意見

Patient Advocate Review of Research
• Why is this study important?
• Who does this study benefit?
• Do patients agree that clinical equipoise exists?
• Will this change the standard of care?
• Who’s eligible for the study? Are they representative of the
patient population?
• Are there other similar trials?
• Does this fill a gap in research?
• Will patients be interested in enrolling?
• Is the study feasible?
• Is the informed consent readable and clear?
• How will study results be communicated?
Earp, J.L., French, E.A, and Gilkey, M.B. (2008) Research Advocacy in Traditional Settings: Questions of Influence
and Legitimacy. In Patient Advocacy for Health Care Quality: Strategies for Achieving Patient-Centered Care (pp. 445-
478). Sudbury, MA: Jones and Bartlett Publishers.

Can Patient (Participant)
Engagement Matter?
3

What is PROSPER?
• Patient-centered Research into Outcomes Stroke
Patients prefer and Effectiveness Research
• Above all, PROSPER is a research collaboration
– Patients
– Doctors
– Caregivers
– Researchers
• Multiple perspectives and
experiences = better understanding!

Patient-Centered Research
into Outcomes Stroke
Patients Prefer and
Effectiveness Research

Real, live patients and what they say…
“The worse doctor was a… cardiologist”
“The doctors didn’t give me the clot-buster because I was on coumadin”
“Doctors didn’t listen to what I needed”
PROSPER Interview with Lesley Maisch
http://www.youtube.com/watch?v=KubuT1_6Lrw

A Few Questions…
Will statins be as
good for me as
someone younger?
And what dose?
Will statins be as
good for me as
someone younger?
And what dose?
What are the tradeoffs
between long-term health
and potential negative
effects of my medications?
What are the tradeoffs
between long-term health
and potential negative
effects of my medications?
I’ve had a stroke and
worry about falls. Will
blood thinners hurt me?
Which one is better?
I’ve had a stroke and
worry about falls. Will
blood thinners hurt me?
Which one is better?
How do the new
anticoagulants affect
my likelihood of
having another
stroke?
How do the new
anticoagulants affect
my likelihood of
having another
stroke?
Can
antidepressants
improve my quality
of life after stroke?
Can
antidepressants
improve my quality
of life after stroke?

What outcomes are we addressing?
– Days alive and at home (“home-time”)
– Any fatigue or depression experienced as a result of
the stroke
– Quality of life
– Mobility
– Whether patient can complete normal activities like
dressing, eating, shopping
– How well patients are tolerating their medications/any
side effects they are experiencing

PROSPER: Personalized Decisions
http://www.prosper-stroke.org/

Can we do Even better?
4

75m+
http://www.cdc.gov/nchs/fastats/hospital.htm
Patients walk through the doors
of hospitals and clinics each year
with questions about their health
and their care.
How do we study their experiences to
find answers and create solutions that
change care and improve outcomes??

A United Network: PCORnet
PCORnet is a network of patients,
clinicians, researchers, health
systems and health plans united
to efficiently generate evidence to
improve patient-centered
outcomes
“Research Infrastructure
Done Differently”
“Research Infrastructure
Done Differently”

Hallmarks of PCORnet
1. Highly engaged patients, clinicians, health systems,
researchers and other partners
2. A collaborative community supported by robust governance
3. Analysis-ready standardized data with strong privacy
protections
4. Oversight that protects patients, supports the timely conduct
of research, and builds trust in the research enterprise
5. Research that is sustainably integrated into care settings
and with communities of patients

Enabling Pragmatic Research:
eScreening, eEnrollment and eFollowup
25
4 12 16 30
DCRI FOLLOW-UP
•Patient Reported Outcomes
•Medication use
•Health outcomes
Baseline Data
ADAPTABLE
Enrollee
8 20 ….
OR
CMS & Payer Virtual Data Warehouse FOLLOW-UP
• Longitudinal health outcomes
Portal FOLLOW-UP
•Patient Reported Outcomes
•Medication use
•Health outcomes
PCORNet Coordinating Center FOLLOW-UP
• Via Common Data Model
• Longitudinal health outcomes

Conclusions
 Patients are a driving force in improving care and will be for
the research enterprise.
 Meaningful engagement of patient stakeholders across the
research continuum will improve quality, efficiency and
translation of research.
 Patient engagement requires an investment of time and
resources just like any component of research
 Engaging patients as partners for research is inspirational
and fun!

Thanks!
Trials
Trial
s
N

CER 2016 Hernandez patient engagement

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