TREATMENTS - BETTER RESEARCH
FOR BETTER HEALTHCARE
CHAPTER VIII - Excerpt
BLUEPRINT FOR A REVOLUTION
We have no doubt about
the contribution of medical research to better quality of life and
longevity. Nevertheless, in this book we illustrate how the
existing ‘drivers’ for research – commercial and academic – have not
done enough to identify patients’ priorities. Healthcare will always be
full of uncertainties, of greater or lesser importance, and where
better to address them than the NHS, in which all of us have a
stake. So how do we bring about a revolution in which
uncertainties in everyday healthcare are not dodged, but taken up
as challenges; in which research to test treatments becomes a
vital part of good healthcare?
Before outlining our blueprint for this revolution, let us give you a
feeling for what we hope might happen in the routine setting of a
general practice in future.
individually, none of the suggestions that follows is revolutionary, or
even novel. Taken together and promoted jointly by patients and
clinicians, our seven action points constitute a blueprint for a
revolution in the use and testing of treatments.
1. Encourage honesty
when there are uncertainties about the effects of treatments.
Admitting uncertainty is often hard for health professionals, and it is
sometimes not welcomed by patients. As a result, people are sometimes
being given a false sense of security. If clinicians and patients are
to work together successfully for more efficient assessment of
treatment effects, both must be more ready to acknowledge that
inadequately evaluated treatments can do substantial harm; they must
become more familiar with the methods needed to obtain reliable
evidence. We need to find out the best ways of making this happen.
2. Confront double
standards on consent to treatment offered within and outside clinical
Clinicians who are prepared to admit uncertainties about the effects of
treatments and address them in formal treatment comparisons are subject
to more stringent rules for interacting with patients than are their
colleagues. When there are uncertainties about treatment effects,
participation in controlled trials or other methods of unbiased
evaluation should be the norm. We should ensure that participation in
research on treatment effects is not presented as a risky endeavour,
implying that ‘standard’ practice is always effective and safe.
knowledge about how to judge whether claims about treatment effects are
A condition for change is greater public engagement with the ways in
which bias and the play of chance can seriously distort evidence about
the effects of treatments. One of the most important features of
scientific investigation – recognising and minimising bias – can hardly
as ‘general knowledge’. We need more determined efforts to reduce these
important gaps in understanding, and to make these concepts a routine
part of education, from school age onwards.
4. Increase the
capacity for preparing, maintaining, and disseminating systematic
reviews of research evidence about the effects of treatments
Many of the answers to pressing questions about the effects of
treatments can be readily addressed by systematically reviewing
evidence that already exists, by keeping such reviews up to date, and
by disseminating the results efficiently to professionals and patients.
There is a long way to go before the messages from existing evidence
are readily available in systematic reviews. We should urge the NHS to
make this one of its prime goals, so that reliable information about
the effects of treatments is synthesised and made readily accessible
throughout the health service.
5. Tackle scientific
misconduct and conflicts of interest within the clinical research community
Many people are astonished to find that researchers are not required to
assess systematically what is known already when they seek funding and
ethical approval for new research. The consequence is inevitable –
poorly designed and frankly unnecessary research continues on a scale
that is unacceptable on ethical as well as scientific grounds. We
should press research funders and research ethics committees to ensure
that researchers do not embark on new research of any kind without
referring to systematic reviews of other relevant evidence.
Furthermore, biased under-reporting of research is unethical.
Researchers should be required to publish the results of all research
to which patients have contributed, and to make clear what contribution
the new evidence has made to the totality of relevant evidence.
6. Require industry
to provide better, more complete, and more relevant evidence about the
effects of treatment
In 2005, the House of Commons Health Committee published a hardhitting
report on the influence of the pharmaceutical industry.139 Whenever the
power and influence of the drug industry has been challenged in the
past, the pharmaceutical giants have responded with near threats to
withdraw their research and development activities from the UK and have
issued dire warnings about the consequences for the UK economy.
Successive governments have listened, capitulated, and done nothing to
curb the industry’s excesses. The Committee’s recommendations should
not be allowed to fall by the wayside. In particular, we should demand
that all clinical trials are registered publicly at the outset, and
that all the results are published in full on completion.
7. Identify and
prioritise research addressing questions about the effects of
treatments which are deemed important by patients and clinicians
The portfolios of research funders and academic institutions are
dominated by basic research that is unlikely to benefit patients in the
foreseeable future, and by research directed at maximising profits for
industry. Applied research into questions that offer no potential to
make money, yet matter to patients, has to fight for a toe-hold, even
when it is publicly supported. We should see to it that the NHS does
more to identify what questions patients and clinicians are asking
about the effects of treatments, and that research funders take account
of them in prioritising research to reduce these uncertainties.
A revolution in
testing treatments is long overdue. If professionals and patients act
together, the steps that we advocate are eminently practicable. You the
readers should clamour for change – now.
AN ACTION PLAN: THINGS YOU CAN DO
Identify questions about the effects of treatments that are important
Use the National Library for Health (www.library.nhs.uk) to see if
there is reliable information from up-to-date systematic reviews.
If you can’t find the information you need, ask NHS Direct to help you
(www.nhsdirect.nhs.uk; Tel: 0845
46 47 (England and Wales); 08454 24 24 24 (Scotland)). Their experts
may be able to find it for you.
If there is no reliable information to answer your question, ask NHS
Direct whether anyone is currently preparing a systematic review of
existing evidence, or whether there are any potentially relevant
ongoing controlled trials.
If there is a relevant ongoing trial, consult www.controlled-trials.com or
the specific trial website to see whether you can participate in it,
and with whom you can discuss your options.
Agree to participate in a clinical trial only on condition (i) that the
study protocol has been registered publicly on www.controlled-trials.com; (ii)
that the protocol refers to the systematic reviews of existing evidence
showing that the trial is justified; and (iii) that you receive a
written assurance that the full study results will be published, and
sent to all participants who indicate that they wish to receive them.
If no one seems to be taking an interest in your inadequately answered
questions about the effects of treatments, submit them for
consideration to the NHS Health Technology Assessment (HTA) Programme (www.ncchta.org), the National
Institute for Health and Clinical Excellence (NICE) (www.nice.org.uk),
and relevant medical research charities (www.amrc.org.uk).
Learn to recognise uncertainty; speak up; ask questions; seek honest
Let your doctors know if you want to discuss evidence on the effects of
treatments, and what uncertainties there are about them. You may find
the information at www.ohri.ca /DecisionAid helpful.
Encourage wider education about the effects of biases and the play of
chance, and lobby your MP and others about including this in the
curriculum, beginning at primary school.
Be a healthy sceptic about unfounded claims and media reports of
treatment ‘breakthroughs’; ask pertinent questions; discuss with your
Challenge treatments offered to you or your family on the basis of
beliefs and dogmas, but unsubstantiated by reliable evidence.
Encourage and work with doctors, researchers, research funders, and
others who are trying to promote research addressing inadequately
questions about the effects of treatment which you regard as important.