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December 2002 • Volume 141 • Number 6
Editorials
Randomized
trials and recruitment tribulations: Rethinking the research enterprise
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See related article,
p 798.
In their article exploring Australian pediatricians' attitudes toward randomized
controlled trials (RCTs),1 Caldwell
et al identify reasons why clinicians are disinclined to enroll their patients
in clinical experiments. Many factors appear to influence pediatricians'
enrollment of patients in RCTs, including pediatricians' assumptions about
parental social class and attitudes about research, pediatricians' own opinions
about the treatments under study, and concerns for the doctor-patient
relationship. A variety of misgivings dissuade some pediatricians from enrolling
patients in any trials and lead others to enroll patients erratically and
selectively.
Unwillingness on the part of many pediatricians to enroll their patients in RCTs
represents a challenge to the pediatric scientific community. Researchers'
mandates to provide the best evidence on which to base improvements in health
care for children compels us to conduct more pediatric RCTs, and therefore to
involve more children. The ethical issues presented by children's participation
in trials are substantial, but far from insuperable. Many successful US models
for broad participation in pediatric RCTs exist. In oncology the merger of
several groups has formed the highly successful Children's Oncology Group (http://www.childrensoncologygroup.org/).
Rheumatology has the Pediatric Rheumatology Collaborative Study Group and the
Pediatric Rheumatology Research Network; neonatology has the National Institute
of Child Health and Human Development Neonatal Research Network and the Vermont
Oxford Network, and, among pediatric primary care practitioners, the American
Academy of Pediatrics Center for Child Health Research's Pediatric Research in
Office Settings is currently conducting its first RCT. These groups all have
identified strategies for involving large groups of pediatricians in enrolling
their patients in RCTs, and we have much to learn from their experience. In
addition, site management organizations working with the pharmaceutical industry
and specializing in pediatrics have emerged, and these groups have developed
similar expertise in involving pediatricians in RCTs.
The inconsistency described by Caldwell et al in participant recruitment among
the pediatricians who do enroll their patients in trials represents a
scientific challenge of its own. RCTs are unquestionably the best study design
for evaluating treatment effectiveness, as they maximize internal validity—the
degree to which a study's results can be attributed to the hypothesized effect
under investigation.2 Yet, when
patient recruitment into an RCT has been systematically non-random because
participants have been enrolled according to pediatricians' preconceptions or
personal standards, the evidence gained from the trial may be of limited
external validity, or generalizability. The paradoxic consequence of
nonrandom recruitment is that particular RCTs might offer internally valid
answers on the effectiveness of new treatments, but that the answers could not
be generalized to patients outside the study group.
What can be done to get more pediatricians to enroll their patients in clinical
trials? Given that networks and study groups have had the most success involving
pediatricians in enrolling their patients in RCTs, maintaining or establishing
core support for existing research networks (the National Institutes of Health,
Agency for Healthcare Research and Quality, and Maternal and Child Health Bureau
all currently support networks) and creating new networks must be a priority for
federal agencies. In addition, investigators conducting RCTs on their own should
learn from the experiences of the networks, which foster a truly collaborative
ethos toward research. Under this ethic, research is no longer under the
exclusive control of researchers. Practicing clinicians also are deemed to have
an important role to play in research. Investigators working in research
networks learn to take note of and incorporate clinicians' input on research
questions, protocols, and procedures, making them all partners in the research
process. This approach results in better and more easily implemented protocols
that encourage pediatrician participation. In addition, participating
pediatricians gain a sense of ownership of the research, and those who feel
themselves to be part owners of research done in RCTs are likely to be more
enthusiastic about enrolling their patients as participants. Pediatricians who
participate in research have reported additional benefits for themselves,
including learning about research design and procedures, gaining new insights
into the human subjects process, and perhaps most importantly, feeling a sense
of affiliation with a larger enterprise for the good of children.
The good to be gleaned in transferring a sense of ownership of the research
enterprise applies not only to pediatricians, but also to the participants
in trials (or more likely, their parents). This should be an objective for all
those enrolling patients into trials. The word subjects connotes a
passivity that is inconsistent with the actual role of patients in trials, who
are often taking medicines daily, coming in for blood tests, and are anything
but inactive. We must be able to convince parents (and when they are old enough,
our patients) that they also are vital partners in the research.
Finally, what can be done to minimize the threat to the external validity of
RCTs represented by inconsistent recruitment into RCTs? First, pediatricians
need education about the importance of uniform and consistent enrollment
procedures to the integrity of the research. Second, enrollment needs to be made
as streamlined as possible, so that pediatricians are not tempted to wait for
slack times to enroll participants. Last, there is no substitute for strictly
tracking the enrollment process, so that those reviewing the research can
understand who was eligible for an RCT, who was enrolled, and why eligible
participants were not enrolled.
Enhancing the recruitment of patients in pediatric RCTs represents not only a
challenge to the pediatric research community, but also an opportunity to
rethink participation in the research enterprise. When we learn to structure
RCTs so that we treat all—scientists, clinicians, and patients—as partners in
the research endeavor, then recruitment of patients will be less of a problem.
| References | TOP |
1. Caldwell PHY, Butow PN, Craig JC. Pediatricians' attitudes towards randomized controlled trials involving children. J Pediatr 2002;141:798-803.
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2. Last JM. A dictionary of epidemiology. New York: Oxford University
Press; 1983.
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