Issue 23.
Can it work? Does it work? Is it worth
it?
Archie Cochrane defined three concepts related to testing healthcare
interventions. Efficacy is the extent to which an intervention does more good than harm
under ideal circumstances ("Can it work?"). Effectiveness assesses whether an
intervention does more good than harm when provided under usual circumstances of
healthcare practice ("Does it work in practice?"). Efficiency measures the
effect of an intervention in relation to the resources it consumes ("Is it worth
it?"). Trials of efficacy and effectiveness have also been described as explanatory
and pragmatic or management trials, respectively, and efficiency trials are more often
called cost effectiveness or cost benefit studies.
Almost all clinical trials assess efficacy. Such trials typically
select patients who are carefully diagnosed; are at highest risk of adverse outcomes from
the disease in question; lack other serious illnesses; and are most likely to follow and
respond to the treatment of interest.
This treatment is prescribed by doctors who follow a careful protocol;
the comparison will be a placebo or existing best practice, and participants will receive
special attention from staff who supplement or replace those employed in usual clinical
settings. The results of such trials are very useful: if the intervention doesnt
work under such ideal conditions it surely wont work under usual conditions. Most
treatments dont survive this stage of testing, and it makes good sense to sequence
the testing of all interventions through this efficacy stage. However the next stages are
not easy.
A recent BMJ paper by Llewellyn-Jones et al showed these problems. In
attempting to help general practitioners to care for depressed elderly people in
residential care, the authors found little evidence that general practitioners improved
their prescribing habits. Many patients refused to participate or dropped out. The result
was a barely detectable benefit, even among those patients who stuck with the programme.
However managers, planners, and politicians will want to know more than
"Does it work?": they will want to know "Is it worth it?" in
comparison with use of the resources for other needs. Trials such as the one by
Llewellyn-Jones et al show that were just now learning to run with community
trials that tackle difficult challenges in research design and implementation that can
undermine the feasibility of a study or prejudice the interpretation of its findings.
Issues of economic analysis also are being resolved, so that questions
of efficiency can be better addressed. This progress will seem slow to researchers caught
up in it and to all of us waiting for the answers, but in the history of the world
were heading for success at a blistering pace. Our progress is fuelled by efficacy
studies and by researchers and governments intent on reaping the benefits they promise.
Fortunately, many governments around the world are aware of the need
for more and better research into health services and are providing funds for training and
research development. One hopes that they will not lose heart or patience: were
going in the right direction, but trial and error are needed, along with investment in
methodological research to get effectiveness and efficiency studies right.
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More National Institute for Clinical
Excellence (NICE)
Drugs to treat dyspepsia and childhood asthma devices are included
alongside therapies for cancer and multiple sclerosis in the first list of treatments to
be assessed by NICE in the autumn. Dyspepsia drugs can be very clinically- and
cost-effective when used in the right situation; however, there is good evidence that they
are often used inappropriately, the DoH says.
There is much variation in the use of inhaler devices for children with
asthma, and one of NICEs tasks will be to assess the effectiveness of the different
systems.
The roles of glatarimer and interferon beta in the treatment of
multiple sclerosis will be considered in the context of services for multiple
sclerosis patients generally. NICE will review the evidence for the use of
paclitaxel as first-line treatment for ovarian cancer and will also issue advice on the
role of docetaxel in breast cancer.
Hip prostheses, zanamivir and oseltamivir, hearing aids, routine
extraction of wisdom teeth, liquid-based cytology for cervical screening, and coronary
artery stents are also on the initial assessment list.
Professor Peter Littlejohns has been appointed as Clinical Director of NICE and Dr Rod
Taylor as Head of Appraisals. Both will take up their appointments on 1 September.
Professor Littlejohns is currently Professor of Public health and Director of the
Healthcare Evaluation Unit at St Georges Hospital Medical School, London, and Dr
Taylor is a lecturer in health services research at the London School of Hygiene
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Handwashing
Bandolier recently had an article on hand washing in hospital acquired
infection.
Clearly it is considered an issue of importance, and a BMJ editorial1
pointed out how infrequent and sporadic hand washing is in health care workers. One
classic paper quoted showed that while doctors estimated that they washed their hands 73%
of the time before patient contact, the observed frequency was just 9%.
Hands can be a repository of nasty germs, and that hand washing can reduce infection
rates has been known for a long time. A cracking little paper2 showed that
Klebsiella species can live quite happily on hands for up to two and a half hours, and
that instituting hand washing regimens can reduce infections in patients by a significant
amount (down from 23 to 16%).
Handwashing with chlorhexidine regularly gave 98-100% reductions in
hand counts.
There is even a systematic review3 looking at compliance
issues with hand washing and barrier precautions, which is a useful source of literature.
It highlighted two other studies which showed that increased compliance with hand washing
before and after patient contact resulted in large (50% or more) decreases in infection
rates. Three more recent papers of interest are examined here.
The bottom line appears to be simple, hand washing with agents
containing chlorhexidine really does lower the rate at which bacteria get onto the hands,
and that hand washing protocols properly enforced reduce hospital acquired infections.
Its more than that, though. There would appear to be clear evidence of
effectiveness, and of benefits of quality and cost-effectiveness.
It may even be a clinical governance issue.
References
1 Editorial. BMJ 1999 318:686.
2 M Casewell, I Phillips. Hands as a route of transmission for
Klebsiella species. BMJ 1977 2:1315-7.
3 E Larson, EK Kretzer. Compliance with handwashing and barrier
precautions. Journal of Hospital Infection 1995 30 (Suppl):88-106.
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