Transcript Mullins_UCDenver_2014_09_04x

CRISP Presentation on PCT Study Design:
Case Study for Patient-Centered PCTs
C. Daniel Mullins, PhD
Professor and Chair
Pharmaceutical Health Services Research Department
University of Maryland School of Pharmacy
Acknowledgment: Parts of this presentation are based on work completed
under contract PCORI-SOL-PCWG-002 funded by the Patient-Centered
Outcomes Research Institute (PCORI) and grant 1R24HS022135-01 funded
by the Agency for Healthcare Quality and Research (AHRQ).
Disclaimers: The views expressed in this presentation are solely those of
the speaker and do not necessarily represent the views of the PatientCentered Outcomes Research Institute (PCORI), its Board of Governors, or
Methodology Committee.
The findings and conclusions in this presentation are those of the author,
who is responsible for its content, and do not necessarily represent the
views of AHRQ. No statement in this presentation should be construed as
an official position of AHRQ or of the U.S. Department of Health and
Human Services.
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What does it mean to be patient centered?
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What does it mean to be patient centered?
right drug or treatment + right patient
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Right Drug or Treatment
How do we know what is the right treatment?
• Evidence-based medicine
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Reliable
Meaningful
• Comparative effectiveness
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Relative value
Implies choices
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Right Patient
Patients are unique: no one-size fits all medicine
• Heterogeneous response to treatment
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Genetic factors
Patient behavior and culture
• Patient-centered outcomes research (PCOR)
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Subgroup analysis
Patient preferences
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What does it mean to be patient centered?
Provide evidence to answer patients’ questions such as:
Given my illness and the treatment outcomes that are most important to me, what should I
expect to happen to me?
What are my treatment options? What are the benefits and harms of those options?
What can I do to help improve the treatment outcomes that are most important to me?
How can my health care team help me make the best decisions about my care?
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Patient-Centered PCTs
Design:
How best to answer patients’ Qs
Outcomes:
Meaningful/actionable for patients
Follow-up:
Patient burden/fatigue; recall bias
Analysis:
Heterogeneity of patients
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WHAT IS PATIENTS?
PATIENTS is an AHRQ-funded Patient-Centered
Outcomes Research (PCOR) Research
Infrastructure Development Grant
(R24HS22135-01)
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WHAT IS PATIENTS?
PATIENTS:
 Empowers patients to propose questions about their health care
concerns and actively participate in studies to answer them
 Combines the expertise of:
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•
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Researchers from the University of Maryland
Health care systems
Patient communities
Advocacy groups
 Challenges the status quo by embracing patients and health care
systems in every component of our research studies
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PCOR is a Win for Everyone
Topics and research
questions
Comparators and
outcomes
Translate/disseminate
results
Continuous
PATIENT
RESEARCHER
Interaction
Not a “onesize-fits-all”
result
Meaningful
research
Informed decision making
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Patient Engagement: Why?
• Makes PCOR meaningful
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Framing the question so it is understood
Framing the answer so it is understood
Framing for cultural appropriateness
Does meaningful imply significant or impactful?
• Helps with patient activation
Having the essential knowledge, skills and confidence for managing one’s own
health and health care
• It shows an honest commitment to being patient
centered
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Patient Engagement: How?
Building/Maintaining Trust and Respect
Identifying
Partnering
Explaining
Doing
Updating
Building/Maintaining Trust and Respect
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10-Step Process for Conducting CER *
Public
Announce-
1. Topic Solicitation
ments
2. Prioritization
3. Framing the Question
* Based on Mullins CD, Adbulhalim AM, Lavallee DC.
Continuous Patient Engagement in Comparative
Effectiveness Research. JAMA 2012; 307(15): 1587-8.
Delphi
Process
Patient
Forums
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10-Step Process for Conducting CER *
4. Selection of Comparators
and Outcomes
5. Creation of Conceptual
Framework
6. Analysis Plan
7. Data Collection
In-person
Meetings
Telephone
Calls
* Based on Mullins CD, Adbulhalim AM, Lavallee DC.
Continuous Patient Engagement in Comparative
* Based
on Mullins Research.
CD, Adbulhalim
Lavallee
DC.1587-8.
Effectiveness
JAMAAM,
2012;
307(15):
Continuous Patient Engagement in Comparative
Effectiveness Research. JAMA 2012; 307(15): 1587-8.
Electronic
Social
Media
Focus
Group
Interviews
Teleconferences
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10-Step Process for Conducting CER *
Teach-Back
Method
8. Reviewing & Interpreting
Results
9. Translation
10. Dissemination
* Based on Mullins CD, Adbulhalim AM, Lavallee DC.
Continuous Patient Engagement in Comparative
* Based on Mullins CD, Adbulhalim AM, Lavallee DC.
Effectiveness Research. JAMA 2012; 307(15): 1587-8.
Continuous Patient Engagement in Comparative
Effectiveness Research. JAMA 2012; 307(15): 1587-8.
Media
Critique
Documents
(e.g.
Patient
Guides)
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Summary
• Pragmatic clinical trials (PCTs) should be
– Pragmatic
– Patient centered
• Patient engagement can enhance PCTs
– Design
– Execution
– Translation and dissemination
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