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Randomised controlled trials as evidence in CAM
Edzard Ernst
The randomised controlled trial (RCT) is one of the
more notable achievements in the history of medi-
cine. Yet, in the realm of CAM, it is a methodology that
seems to be more hated than loved. But why is this so?
One reason for the paucity of support for RCTs is,
in my experience, that many CAM enthusiasts are
unclear what an RCT is. Put simply, an RCT is any
study with a control group that allocates trial-
participants not by choice or selection, but at
random. This is the simplest and probably the only
method to make sure that each study group is com-
parable at baseline. We can, of course, match groups
and make sure they are comparable, but this can only
be done for variables that are known. Randomisation
aims to achieve comparability across groups for all
variables, even those that are deemed irrelevant or
are not known.
If RCTs are that simple, why can anyone not be
taken by this design? Opponents cite numerous argu-
ments against the RCT of which I can mention only
those that are most frequent.
1 RCTs are expensive.
Unfortunately, this is often true. The point to remem-
ber, however, it that randomisation costs next to
nothing. It is the trial that is expensive, not the
randomisation.
2 RCTs are artificial and not generalisable.
This is also frequently true. Many RCTs are on highly
selected patients, conducted under laboratory condi-
tions. Thus, the results of trials may not be as mean-
ingful for ‘real-life’ practice as we want them to be.
Such trials provide answers to the question ‘can this
therapy work?’ If we want to find out whether this
therapy works in real life, we might have to conduct
further studies.
3 RCTs are not suited for the complex, holistic treat-
ments of CAM.
This frequently voiced sentiment is simply not
correct. An RCT can quite easily be designed for even
the most complex and holistic therapy. All one needs
Focus on Alternative and
Complementary Therapies
Volume 17(4) December 2012 219–220
© 2012 The Author
FACT © 2012
Royal Pharmaceutical Society
DOI 10.1111/j.2042-7166.2012.01168.x
ISSN 1465-3753
to do is to allocate patients at random to group A or
B, and treat A with the complex and holistic inter-
vention and B with something else. At the end of the
treatment, one can compare the results of both
groups.
4 Randomisation affects the study outcome.
Informed consent demands that all trial participants
know that they may receive either treatment in a
study. This uncertainty might affect the results.
While, theoretically, this may be a possibility, the few
experimental data we currently have on this issue
seem to suggest otherwise.
5 RCTs are unethical.
There might be situations where RCTs are unethical.
The ethical problems of administering a placebo to
severely ill patients are well understood. But, we
should remember that not all RCTs are placebo con-
trolled. Randomisation itself is rarely unethical and,
if it is ethically questionable, we should of course not
do it.
6 RCTs are not free of bias.
This is entirely true. Randomisation only minimises
selection bias and cannot eliminate all sources of
bias. Yet, I do not see how this can be a good reason
against randomisation. Each form of bias has to be
recognised and eliminated or minimised in different
ways.
7 RCTs often produce unreliable results.
This is true as well. If we had 100 RCTs addressing the
same research question, we probably would find a
degree of disagreement amongst their findings. This
may be due to chance, residual bias or other factors.
In such cases, it is best to conduct a systematic review
of all RCTs. The fact that RCTs are not 100% reliable
is not, however, a sound reason for adopting a less
reliable method.
8 RCTs are open to fraud and misconduct.
Again, this is true. All research can be open to fraud
and misconduct. But why should this be a valid argu-
ment specifically against RCTs? Clearly it is not.
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220 Focus on Alternative and Complementary Therapies
9 RCTs tell us little about harm.
We regularly hear that drugs have been withdrawn
because of unacceptable adverse effects. Those who
oppose RCTs might then point out that all the studies
conducted prior to licensing the drug failed to
produce evidence of these adverse effects. This argu-
ment is based on a misunderstanding: RCTs are not
an adequate tool for detecting risk. In fact, the sample
size of many RCTs is usually far too small for identi-
fying rare adverse effects. In other words, RCTs are
just one of several research tools to assess the value of
a treatment.
The conclusions from all this are fairly clear; the
RCT is by no means a flawless methodology. In fact,
December 2012 17(4)
the RCT can only address a relatively narrow range
of research questions. Yet, when RCTs are well
designed and used for the right purpose, they are
better than any other research design we presently
know.
Edzard Ernst, MD, PhD, FMedSci, FSB, FRCP, FRCPEd,
Editor-in-Chief of FACT, Emeritus Professor, Peninsula
Medical School, Veysey Building, Salmon Pool Lane, Exeter
EX2 4SG, UK.
E-mail: eernst@pms.ac.uk