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1
Introduction1
Experts believe well below half of medical treatments patients receive
are supported by adequate scientific evidence (IOM, 2007). Discussions of
health care quality over the past two decades increasingly have employed
terms such as “evidence-based medicine” and “learning health care sys -
tem.” While these terms capture important concepts for professionals,
they may come as a surprise to patients and the public, many of whom
might assume medicine already is based on evidence and that the health
system already does constantly learn of and implement new, better meth-
ods of treatment. Patients and the public might also believe that new
therapies are tested in individuals similar to themselves (e.g., age, gender,
race, ethnicity, disease state) when in reality there is a lack of diversity in
clinical trial patient populations.
What the concepts of “evidence-based medicine” and “learning
health care system” have in common is their reliance on the accumula -
tion of medical knowledge based on science, not hope, and, further, that
clinicians would take that knowledge out of the medical textbooks and
laboratory notebooks and apply it in the care of individual patients and
patient populations.
Clinical trials are the linking step that enables basic research findings
to emerge at the patient’s bedside and in physicians’ examining rooms.
The questions clinical trials seek to answer change over time, depend-
1 The planning committee’s role was limited to planning the workshop, and the workshop
summary has been prepared by the workshop rapporteurs as a factual summary of what
occurred at the workshop.
1
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2 PUBLIC ENGAGEMENT AND CLINICAL TRIALS
ing on advances in basic research and the population health problems
they are intended to address. Potential new treatments must be tested
in humans in order to find out whether they “work” and whether they
cause harm (see Appendix B for an overview of the clinical trials process
set forth in the workshop summary from an earlier Institute of Medicine
[IOM] workshop in this workshop series). Clinical trials can comprise a
series of rather elaborate steps, and the rationale for these trials may not
be well understood by the public. An adequate infrastructure to support
the efficient and effective conduct of the nation’s clinical trials enterprise
can help ensure that the system does not become too narrow a bottleneck,
impeding the flow of discovery from science to practice.
Meanwhile, biomedical research is advancing rapidly, and certain
significant problems in clinical trials have hindered their ability to keep
pace with demands to translate discoveries into improved patient care.
In short, as discussed at a 2009 workshop of the IOM’s Forum in Drug
Discovery, Development, and Translation, there is concern that “the cur-
rent clinical trials enterprise in the United States is unable to produce the
high-quality, timely, and actionable evidence needed to support a learning
health care system” (IOM, 2010a). Among the broad categories of prob-
lems identified at that workshop were:
1. the length of time and high financial cost of clinical trials;
2. delays resulting from the many regulatory requirements for studies
that involve human subjects;
3. the generally fragmented way clinical trials are prioritized and
undertaken;
4. obstacles encountered within academic health centers;
5. lack of involvement of community physicians and their needs in
the development and conduct of clinical trials; and
6. dwindling incentives for and attractions of a career as a clinical
trial investigator.
A final significant problem cited at the 2009 workshop became the
focus of this workshop: the increasing difficulty of recruiting and retaining an
appropriate human subject population for specific clinical trials . As discussed
at that meeting and in subsequent discussions of the IOM Forum, many
clinical trials never recruit the number of people needed, and others
accrue patients far too slowly, and the scale of need is substantial. In the
United States in 2009, there were almost 11,000 clinical trials testing vari -
ous medical interventions, mostly (59 percent) new drugs. Collectively, it
was hoped these trials would enroll some 2.8 million people (IOM, 2010a).
In this workshop, titled “Public Engagement and Clinical Trials: New
Models and Disruptive Technologies,” the issue of participation in trials
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3
INTRODUCTION
was put in a large frame, with the more general problem seen as one of a
lack of support for clinical trials by patients and families, by community
leaders, by academic medical centers, and even by practicing physicians,
who may not always seek out trials for their patients or encourage them
to participate, and, in some cases, even discourage participation. Forum
members have identified that successfully engaging these broad and
diverse publics in the clinical trials enterprise is a substantial challenge.
The workshop covered the topics of recruiting and retaining people
to serve as trial participants, increasing practicing clinicians’ participation
in and referrals to trials, and strengthening the public opinion climate in
which clinical trials are carried out and, ultimately, brought into routine
medical practice.
Workshop co-host Dennis Charney, Dean, Mount Sinai School of
Medicine, set the tone for the meeting at its outset by saying that the ulti -
mate success of his school and its new strategic plan will be based not on
“how many papers we publish, or grants we get.” Success, he said, will
depend on the answer to the question, “Did we contribute to changing the
practice of medicine for the better?” He added that a key element of that
contribution will be the school’s contribution to testing new medicines
and therapies.
According to meeting chair Jeffrey Drazen, Editor-in-Chief, New Eng-
land Journal of Medicine, testing medical treatments through clinical trials
requires that people put themselves at risk. However, the evidence gained
through successful clinical trials can ultimately reduce the uncertainty
in medical decision making. Drazen pointed out that, several decades
ago, the medical literature provided information on results of tests in
very small groups of patients—10, 15, perhaps a hundred. Such small
groups will show a therapeutic effect only if it is dramatic. For more
subtle but meaningful effects and for refinements of existing treatments,
a thousand, or even ten thousand, patients are needed for an effect to be
apparent. Meanwhile, sophisticated statistical tools have been developed
that enable much more refined hypothesis testing. Now, Drazen noted,
the stumbling block is recruiting a sufficient number of patients for the
trials themselves.
Medical progress needs more than a sound scientific idea that can be
translated into clinical care in the form of an intervention, whether that is
a drug, device, or behavioral intervention. Progress requires a population
willing to put itself at risk. “The message of this meeting,” Drazen said,
“is that unless we can persuade more people to put themselves at risk,
the rate at which we will be gathering knowledge will become smaller
and smaller.”
This is not easy, because it requires people to admit that the current
medical system does not know the best answer to their problems. Drazen
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4 PUBLIC ENGAGEMENT AND CLINICAL TRIALS
commented that when patients say to him, “I don’t want to be a guinea
pig,” he responds, “If I treat you based on what we know about your situ -
ation, you are a guinea pig anyway, because I don’t know what works. At
least this way, your therapy will teach us something.” And, he adds, many
other people may benefit from that knowledge in the future.
