ENGAGEMENT: A PATH TO GETTING
RESEARCH INTO PRACTICE
PERSPECTIVES FROM A FORMER “PCORIAN”
Kara Odom Walker, MD, MPH, MSHS, FAAFP
Cabinet Secretary, Department of Health and Social Services
May 15, 2017
ABOUT PCORI
 An independent research institute authorized by Congress in 2010 and governed by a 21-member Board of
Governors representing the entire health care community
 Funds comparative clinical effectiveness research (CER) that engages patients and other stakeholders
throughout the research process
 Seeks answers to real-world questions about what works best for patients based on their circumstances
and concerns
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PCORI’S BROAD AND COMPLEX MANDATE
“The purpose of the Institute is to assist patients, clinicians,
purchasers, and policy-makers in making informed health
decisions by advancing the quality and relevance of evidence
concerning the manner in which diseases, disorders, and other health
conditions can effectively and appropriately be prevented, diagnosed,
treated, monitored, and managed through research and evidence
synthesis...
… and the dissemination of research findings with respect to the
relative health outcomes, clinical effectiveness, and appropriateness
of the medical treatments, services...”
--from PCORI’s authorizing legislation
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INCREASING VALUE AND REDUCING WASTE

2009 estimate: As much as 85% of research funding is avoidably wasted
Stages in research production that lead to waste. Moher et al.
Avoidable waste in the production and reporting of research evidence. Chalmers I, Glasziou, P. Lancet 2009; 374: 86-89.
Increasing value and reducing waste in biomedical research: who's listening? Moher D, et al. Lancet 2015; Online:
Sept.28
Engagement as a Path to Useful, High-Quality Research
Topic Selection
and Research
Prioritization
Evaluation
Proposal Review;
Design and Conduct of
Research
Dissemination and
Implementation of
Results
DOES ENGAGEMENT MAKE A DIFFERENCE?
A systematic review* provides the first international evidence of the impact of patient and public involvement on
research on health and social-care research.
 Literature search from 1995-2009 identified
66 studies
 Analysis showed patient and public
involvement enhanced quality and
appropriateness of research
 Impacts were described for all stages of
research
 But authors note the evidence base on impact
of engagement still needs significant
enhancement
*Health Expectations 2014; 17(5): 637–650.
PATIENT-CENTEREDNESS
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WHAT IS COMPARATIVE EFFECTIVENESS?
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WHY WE NEED CER
“ . . . for want of appropriate
studies, innumerable practical
decisions facing patients and
doctors every day do not rest on a
solid foundation of knowledge
about what constitutes the best
choice of care.”
Institute of Medicine Report June 2009
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EVIDENCE OF EVIDENCE GAPS
Among 16 current guidelines reporting levels of
evidence including 2,711 recommendations:
Level of evidence A – 11%
Level of evidence C – 48%
JAMA. 2009;301(8):831-841
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MORE EVIDENCE: VARIABILITY IN PRACTICE
“Variation in surgical rates is high and represents both
gaps in outcomes research and poor patient decision
quality.”
A Dartmouth Atlas of Health Care Series
http://www.dartmouthatlas.org/downloads/reports/Cerebral_aneurysm_report_09_30_14.pdf
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Comparative Effectiveness Research
Research that . . .
• Compares benefits and harms of at least two different
existing methods to prevent, diagnose, treat, or monitor a
clinical condition or to improve care delivery
• Is performed in real-world populations
• Informs a specific clinical or policy decision (“decisional
dilemma”)
Adapted from Initial National Priorities for Comparative Effectiveness
Research, Institute of Medicine of the National Academies
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PATIENT-CENTERED OUTCOMES RESEARCH
 Patient-centered outcomes research, or PCOR, is a relatively new
form of comparative effectiveness research
 Considers patients’ needs and preferences while focusing on the
outcomes most important to them
 Investigates what works, for
whom, under what
circumstances
 Helps patients and other health
care stakeholders make betterinformed decisions about health
and health care options
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COMPARATIVE EFFECTIVENESS - ALLHAT
•
A total of 33,357 participants aged 55 years or older with hypertension and at least 1 other
CHD risk factor
•
Participants were randomly assigned to receive chlorthalidone, 12.5 to 25 mg/d (n = 15,255);
amlodipine, 2.5 to 10 mg/d (n = 9,048); or lisinopril, 10 to 40 mg/d (n = 9,054) for planned
follow-up of approximately 4 to 8 years.
•
Key question: What is the optimal first line therapy for hypertension?
JAMA 2002; 288; 2981-97.
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Key conclusion:
Thiazide-type diuretics are superior in
preventing 1 or more major forms of
CVD and are less expensive.
They should be preferred for first-step
antihypertensive therapy.
Over a patient’s lifetime,
chlorthalidone was always least
expensive (mean $4,802 less than
amlodipine, $3,700 less than lisinopril)
Adding quality of life did not change
these results
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COMPARATIVE EFFECTIVENESS- ALLHAT
• How do the results of ALLHAT help patients and clinicians make decisions?
• Provides information on harms and benefits of three major antihypertensives.
• chlorthalidone
• amlodipine
• lisinopril
• Large enough to allow subgroups to be examined.
• Age <65 vs. >65
• Men vs. women
• Black vs. nonblack
Diabetes vs. no diabetes
JAMA •2002; 288; 2981-97.
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COMPARATIVE EFFECTIVENESS
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RISING NUMBERS OF CITATIONS FOR “PATIENT-CENTERED
OUTCOMES RESEARCH” IN PUBMED (N=290)
100
90
80
70
60
50
40
30
20
10
0
• Extending patient-centered
outcomes to all research
• Extend engagement to
policy and social
determinants of health
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IMPROVING THE FLOW AND RELEVANCE OF RESEARCH
EVIDENCE FOR IMPLEMENTATION
R Lobb, GA Colditz. Implementation science and its application to population health. Annu Rev Public Health. 2013;34:235–
251
FUNDERS ROLE IN A NATIONAL HEALTH RESEARCH PROGRAM
REGULATION/
APPROVAL
DISCOVERY
YOU
ARE
HER
E
NIH
Industry
Academia
FDA
CMS
Patients
Specialties
Payers
PCORI
CLINICAL &
HEALTH CARE
POLICY
COMPARATIVE
CLINICAL
EFFECTIVENESS
RESEARCH
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QUESTIONS?
COMMENTS?