PERS PE C T IV E
Choosing Wisely
as stewards of limited health
care resources will be revealed
by physician-led efforts such as
Choos­ing Wisely. General acceptance of this effort to date by
physicians and the public is encouraging and probably reflects
our enduring trust in physicians
as healers and credible leaders of
health care reform. This trust
must not be squandered; rather,
it should be leveraged to restore
balance in our nation’s health
care investment.
Disclosure forms provided by the authors
are available with the full text of this article
at NEJM.org.
From the Dartmouth Institute for Health
Policy and Clinical Practice (N.E.M., C.H.C.),
the Department of Community and Family
Medicine, Geisel School of Medicine at
Dartmouth (N.E.M), and the Norris Cotton
Cancer Center, Dartmouth–Hitchcock Medical Center (N.E.M., C.H.C.) — all in Lebanon, NH; and the Department of Health
Care Policy, Harvard Medical School (T.D.S.),
the Division of General Internal Medicine,
Brigham and Women’s Hospital (T.D.S.),
Partners Healthcare System (T.D.S.), and
the Department of Health Policy and Management, Harvard School of Public Health
(M.B.R.) — all in Boston.
This article was published on January 22,
2014, at NEJM.org.
1. Choosing Wisely: an initiative of the ABIM
Foundation. 2013 (http://www.choosingwisely
.org).
2. Good Stewardship Working Group. The
“top 5” lists in primary care: meeting the re-
sponsibility of professionalism. Arch Intern
Med 2011;171:1385-90.
3. Stein R. In wake of mammography guidelines, U.S. health task force faces new scrutiny, Washington Post. December 20, 2009
(http://articles.washingtonpost.com/
2009-12-20/politics/36795296_1_task-force
-mammography-guidelines-health-care
-services).
4. Understanding of the efficiency and effectiveness of the health care system. In: The
Dartmouth Atlas of Health Care. Lebanon, NH:
Dartmouth Institute for Health Policy and
Clinical Practice, Center for Health Policy Research, 2013 (http://www.dartmouthatlas.org).
5. Levinson W, Roter DL, Mullooly JP, Dull
VT, Frankel RM. Physician-patient communication: the relationship with malpractice
claims among primary care physicians and
surgeons. JAMA 1997;277:553-9.
DOI: 10.1056/NEJMp1314965
Copyright © 2014 Massachusetts Medical Society.
PCORI at 3 Years — Progress, Lessons, and Plans
Joseph V. Selby, M.D., M.P.H., and Steven H. Lipstein, M.H.A.
T
he Patient-Centered Outcomes
Research Institute (PCORI),
which recently marked its third
anniversary, has established distinctive pathways for funding
and conducting practical research
and a solid foundation of funded studies. The PCORI board of
governors has adopted three
strategic goals to meet its mandate under the Affordable Care
Act. These goals are to increase
the quantity, quality, and timeliness of usable, trustworthy comparative research information; to
accelerate the implementation
and use of research evidence; and
to exert influence on research
funded by others to make it more
patient-centered and useful. To
address the first goal, PCORI
funds comparative clinical effectiveness research (CER)1,2 related to five national priorities:
evaluating prevention, diagnosis,
and treatment options; improving health systems; enhancing
592
communication and dissemination of evidence; addressing disparities in health and health
care; and improving CER methods and data infrastructure.
To ensure that funded research is useful and therefore
more likely to be implemented,
PCORI engages individuals and
organizations representing patients, caregivers, clinicians, delivery systems, payers and purchasers, researchers, policymakers,
and industry in generating research questions. Multistakeholder advisory panels help PCORI
prioritize and refine suggested
questions for targeted proposal
solicitations.3 We require funding applicants to involve patients
and relevant stakeholders on
their research teams throughout
the study — helping to identify
and refine research questions,
choose comparators and outcomes, identify and recruit study
populations, develop recruitment
materials and survey instruments,
and interpret and disseminate
findings. Patients and other
stakeholders, trained by PCORI
in research review, make up 50%
of the merit-review panels that
evaluate applications — the idea
being to keep the focus on relevant questions and lay a foundation for disseminating important
findings.
Applications are scored on
technical merit, including adherence to standards developed by
PCORI’s Methodology Committee; the relevant condition’s burden on individuals and society;
the relevance to patients of the
proposed comparisons, study populations, and outcomes (patientcenteredness); the quality of patient and stakeholder engagement;
and the likelihood that the results could change clinical or
personal practice, improving outcomes. Patient-centeredness, engagement, and likelihood of
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PE R S PE C T IV E
PCORI at 3 Years
PCORI Funding Commitments to Date (through September 2013), According to Priority Area.*
No. of Projects
Funded
National Priorities for Research and Examples of Studies
Total Funds
Committed
millions of U.S. $
Assessment of prevention, diagnosis, and treatment options
65
116.1
41
77.1
25
42.3
31
52.4
30
28.2
Selection of peritoneal dialysis or hemodialysis for kidney failure: gaining meaningful information
for patients and caregivers
Comparing effectiveness of pharmacotherapy, psychotherapy, or combination therapy for treating
depression during pregnancy
Comparative effectiveness of intravenous vs. oral antibiotic therapy for serious bacterial infections
Improving health care systems
Do patient-centered medical homes within an ACO improve patient outcomes during the transition from hospital to home care?
Using telehealth to deliver developmental, behavioral, and mental health services in primary care
settings for children in underserved areas
Comparing the effectiveness of a multidisciplinary care team model with usual serial care for the
treatment of lung cancer in a regional hospital
Communication and dissemination research
Shared medical decision making in pediatric diabetes
Improving communication for chemotherapy: addressing concerns of older cancer patients and
caregivers
Enhancing genomic laboratory reports to enhance communication and empower patients
Addressing disparities
Long-term outcomes of community engagement to address depression outcomes disparities
Reducing disparities with literacy-adapted psychosocial treatments for chronic pain: a comparative trial
Using technology to deliver multidisciplinary care to individuals with Parkinson disease in their homes
Accelerating patient-centered outcomes research and methodologic research
Building PCOR value and integrity with data quality and transparency standards
Facilitating patient-reported outcome measurement for key conditions
Development of a causal-inference toolkit for patient-centered outcomes research
*ACO denotes accountable care organization, PCMH patient-centered medical home, and PCOR patient-centered outcomes research.
changing practice are not explicit
criteria of either the National Institutes of Health or the Agency
for Healthcare Research and Quality and are meant to produce
a different portfolio of funded
projects.
Recognizing the pervasive lack
of the type of information needed for decision making, the
board has solicited CER pro­
posals spanning the spectrum of
clinical conditions. After issuing
broad funding announcements
for each national priority, PCORI
has awarded more than $316
million for 192 studies covering
a wide range of clinical conditions, geographic locations, and
socioeconomic characteristics (see
table for examples; a complete list
of funded projects is available at
www.pcori.org/pfaawards/?viewby
=priority).
We have also begun to issue
funding announcements targeting
specific research questions. The
first such announcement, sponsored with the National Institute
on Aging, requests proposals for
a large clinical trial comparing a
multicomponent intervention with
usual care for preventing injurious falls in the elderly. The sec-
ond calls for proposals to assess
treatment options for black and
Hispanic patients with uncontrolled asthma. The third, jointly
sponsored with the Agency for
Healthcare Research and Quality,
seeks proposals for a registrybased cohort study comparing the
effectiveness of various uterinesparing treatment options in
women with symptomatic uterine
fibroids. Targeted announcements
allow PCORI to direct resources
to areas deemed high-priority by
stakeholders and advisory panels.
To increase the efficiency of
conducting patient-centered CER
n engl j med 370;7 nejm.org february 13, 2014
593
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PERS PE C T IV E
PCORI at 3 Years
and accelerate the implementation
of findings, PCORI recently awarded approximately $100 million
to launch a National PatientCentered Clinical Research Network, PCORnet. This infrastructure, composed of 11 large
health-system–based networks and
18 patient-group–based networks,
will generate interoperable data
sets to support multinetwork
observational and randomized
studies addressing clinical and
service-delivery questions. Key requirements for awardees include
the active involvement of host
health systems and patients in
governing and using the infrastructure, a willingness to host
system-based randomized studies, and a willingness to collaborate with outside researchers on
PCORI-funded research.
More recently, PCORI announced its first 30 “Pipeline to
Proposals” Engagement Awards,4
intended to seed new partnerships between researchers and
stakeholder communities and
build teams that can compete
successfully for patient-centeredresearch funding from PCORI
and other sources.
PCORI’s broad funding announcements have yielded a diverse research portfolio reflecting the major causes of illness
and death in the United States.
Of 162 CER studies, 37 (23%) focus on cancer detection, treatment, or surveillance; 30 (19%)
on mental health; 26 (16%) on
cardiovascular diseases; and 18
(11%) on endocrine disorders, including diabetes mellitus. Crosscutting issues, including self-care
(15 studies) and pain management (7 studies), are also represented. PCORI-funded studies
include many direct comparisons
of two or more options and many
594
others that synthesize existing evidence, then develop and evaluate
decision support tools for improving patient–clinician decision
making. Nearly 45% of studies
feature prospective, randomized
comparisons. Studies typically include patient-reported outcomes
or other patient-relevant outcomes,
often in addition to more traditional outcomes such as rehospitalization, disease progression, or
the occurrence of complications
or death.
PCORI has found stakeholder
communities ready and able to engage in research. Patient-centered
questions come from multiple
sources, and innovative partnerships are emerging. Participation
in merit review has been received
well by both scientific and stakeholder reviewers; because the
unfamiliar processes of collaborating with stakeholders have
presented challenges to some researchers, PCORI conducted a
webinar entitled “Promising Practices in Engagement”5 and made
examples of successful engagement plans available online. Some
successful applicants build on
long-standing relationships with
patients, clinicians, or organizations representing them; others
develop new relationships as proposals are prepared. Patient and
family advisory councils of delivery systems or health plans are
frequent partners, as are professional, provider, and payer associations.
Over the next 3 years, PCORI
will commit as much as $1.5 billion to research projects. Broad
funding announcements will continue, but we’ll increasingly focus
on high-priority questions identified by PCORI advisory panels
through targeted PFAs and calls
for larger pragmatic studies. We
continue to refine our merit-review processes and will seek
ways to support new investigators
in conducting patient-centered
outcomes research. The interests
of patients with rare diseases
will be pursued with an advisory
panel. PCORI will also seek additional opportunities to cofund
and comanage clinical research
with others.
PCORI will disseminate its
methodology standards, work
with the research community in
developing additional needed
standards, and work with a clinical trials advisory panel to enhance PCORI-funded clinical
trials. Finally, we will actively
evaluate the effectiveness of and
best methods for engaging patients and other stakeholders in
research — a fundamental requirement of PCORI research that,
though reasonable and appealing,
needs a firmer evidence base.
PCORI has laid an innovative
foundation for producing and
disseminating clinical research,
engaging multiple stakeholders
in the process, and embedding
clinical research within health
care systems and activated patient communities. Ultimately, our
aim is to make research more
useful and more likely to be included in health care decision
making.
Disclosure forms provided by the authors
are available with the full text of this article
at NEJM.org.
From the Patient-Centered Outcomes Research Institute (PCORI), Washington, DC.
Other members of the PCORI board of governors are Grayson Norquist (chair), Debra
Barksdale, Kerry Barnett, Lawrence Becker,
Francis Collins, Allen Douma, Arnold Epstein,
Christine Goertz, Leah Hole-Marshall, Gail
Hunt, Robert Jesse, Richard Kronick, Harlan
Krumholz, Richard Kuntz, Sharon Levine,
Freda Lewis-Hall, Ellen Sigal, Harlan Weisman, and Robert Zwolak. Methodology
committee members include Robin Newhouse (chair), Steven Goodman (vice-chair),
n engl j med 370;7 nejm.org february 13, 2014
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PE R S PE C T IV E
PCORI at 3 Years
Naomi Aronson, Ethan Basch, Alfred Berg,
David Flum, Mark Helfand, John Ioannidis,
Michael Lauer, David Meltzer, Brian Mittman, Sally Morton, Sebastian Schneeweiss,
Jean Slutsky, Mary Tinetti, and Clyde Yancy.
1. Washington AE, Lipstein SH. The PatientCentered Outcomes Research Institute —
promoting better information, decisions,
and health. N Engl J Med 2011;365(15):e31.
2. Selby JV, Beal AC, Frank L. The PatientCentered Outcomes Research Institute
(PCORI) national priorities for research and
initial research agenda. JAMA 2012;307:
1583-4.
3. Beal AC. PCORI’s first advisory panels:
celebrating a talented and diverse group.
Washington, DC: Patient-Centered Outcomes
Research Institute, April 8, 2013 (http://www
.pcori.org/blog/pcoris-first-advisory-panels
-celebrating-a-talented-and-diverse-group/).
4. Beal AC, Sheridan S, Schrandt S. PCORI’s
engagement awards: a new opportunity to
build new research partnerships. Washington, DC: Patient-Centered Outcomes Research Institute, June 17 2013 (http://www
.pcori.org/blog/pcoris-engagement-awards
-a-new-opportunity-to-build-new-research
-partnerships/).
5. Promising practices of meaningful engagement in the conduct of research. Washington, DC: Patient-Centered Outcomes Research Institute, September 19, 2013 (http://
www.pcori.org/events/promising-practices
-of-meaningful-engagement-in-the-conduct
-of-research/).
DOI: 10.1056/NEJMp1313061
Copyright © 2014 Massachusetts Medical Society.
“Misfearing” — Culture, Identity, and Our Perceptions
of Health Risks
Lisa Rosenbaum, M.D.
D
uring my cardiology fellowship, I worked at a women’s
cardiovascular clinic where we
asked every new patient the
same question: “What do you
think is the number-one killer
of women?” Most women said
either breast cancer or heart disease. But Ms. S., a middle-aged
woman with high blood pressure and hyperlipidemia, answered
in a way that sticks with me: “I
know the right answer is heart
disease,” she said, eyeing me as
if facing an irresistible temptation, “but I’m still going to say
‘breast cancer.’”
If helping women understand
their cardiovascular risk were
about right answers, I would
simply have reiterated the facts
about heart disease — that it
takes more women’s lives each
year than all types of cancer
combined (see graphs), that it is
in many ways preventable, and
that, despite what many women
believe, multivitamins and antioxidants do not reduce the risk.
But Ms. S.’s response short-circuited my statistical litany. Her
sense of risk was clearly less
about fact than about feeling.
Would more facts really address
those feelings?
Data on campaigns to educate
women about heart disease reinforced my sense that our efforts
to provide women with the facts
about heart disease were missing
something critical. Although the
first decade of educational campaigns led to a near doubling of
women’s knowledge about heart
disease, in the past few years
such efforts have failed to reap
further gains. Moreover, persistent gaps in perceptions remain
among minority women, who are
often at greatest risk.1
If the next frontier in preventing cardiovascular disease among
women is less about disseminating evidence than about understanding why the evidence may
be hitting a wall, the critical
question is why women might
feel more fearful of other diseases, particularly breast cancer, despite ample evidence suggesting
that heart disease poses a far
greater threat.
“Misfearing,” the term Cass
Sunstein uses to describe the human tendency to fear instinctively
rather than factually, is not unique
to women’s perceived health
threats.2 Decades of research on
risk perception have revealed the
many factors feeding our misfears and associated perceptions.
Tornadoes. Terrorist attacks. Hom­
i­cides. The big, the dramatic, and
the memorable occupy far more
of our worry budget than the
things that kill with far greater
frequency: strokes, diabetes, heart
disease. But interacting with
many of these fear factors is another force we rarely associate
with our individual health perceptions: our commitment to our
cultural groups.
Our desire to belong to something bigger than ourselves is as
fundamental to our nature as our
desire for individual success. Human nature is both selfish and
“groupish,” according to the moral psychologist Jonathan Haidt:
“Our minds contain a variety of
mental mechanisms that make us
adept at promoting our group’s
interests. . . . We are not saints,
but we are sometimes good team
players.”3
If you survived middle school,
you know how powerful the desire to belong to a group can be.
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