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The James Lind Alliance (JLA), launched just over a year ago, is named after the Scottish naval surgeon who conducted a randomised trial in scurvy, showing the superiority of citrus fruit over other treatments. The core aim of the JLA, which is a UK based organisation, is the proper involvement of patients, working together with clinicians (and not just doctors but nurses, therapists, and others), to identify important treatment uncertainties and to plan shared priorities in research (http://www.lindalliance.org).1 It was formed to address a number of shortcomings and problems in current therapeutic research.
There are mismatches between what researchers are researching and unanswered questions asked by patients and clinicians
Firstly, there are mismatches between what researchers are researching and unanswered questions asked by patients and clinicians; linked with this, there are mismatches between treatment outcomes studied by researchers and those regarded as important by patients. Doctors’ and patients’ priorities are not necessarily the same, as several studies have demonstrated. For example, in a survey of research publications on osteoarthritis of the knee and the preferences of patients and clinicians for the type of research needed, it was found that commercially funded drug trials dominated research. But what patients wanted was more research on knee replacement, education and advice, and physiotherapy, all of which were rated as more important than further drug research.2 In another example, methods were developed to involve patients with rheumatoid arthritis in identifying research outcomes. It emerged that fatigue—previously not within the core set of outcome measures, as prioritised by rheumatologists—proved to be the outcome of greatest concern to patients. As a result, it was included in the core set of outcome measures for future trials.3 In Alzheimer’s disease, a survey of the views of patients and carers highlighted the importance of improvements in mood, motivation, concentration, confidence, and ability to manage daily living.4 Attention has also been drawn to mismatches in the research priorities of different stakeholders in mental health research.5
Patients and clinicians need to set priorities for research, rather than industry
Secondly, patients and clinicians need to set priorities for research, rather than industry. Chalmers and colleagues drew attention to the decline in non-commercially funded clinical trials in the UK between 1980 and 2002.6 Research that has been prioritised by patients and clinicians together, with the greatest relevance to the needs of patients, ought to be well placed in the competitive market for non-commercial funding, and should create pressure for the provision of more funding of this type. But why should research funding bodies be influenced? It has been demonstrated that patients and the public can make pertinent comments on health technology assessment and influence commissioned research,7 and the argument that addressing therapeutic questions rated as important by patients will lead to greater relevance of the resulting research seems intuitively obvious. Of course, the extent to which patient involvement will improve outcomes of research remains to be seen; this will require careful assessment. However, the overwhelming argument is that therapeutic research planned jointly by patients and clinicians will address the issues of greatest importance to patients.
Thirdly, although pharmaceutical and medical technology companies are important partners in therapeutic research, they should not be permitted to dictate the research agenda. The commercial imperatives for industry are obvious and will almost inevitably distort research priorities. In addition, investigating new indications for drugs already licensed is an important area of little interest to the pharmaceutical industry, despite the good track record of this type of research. Examples of such “indication switches” include aspirin for cardiovascular and cerebrovascular protection, amoxicillin for peptic ulceration, amitriptyline for neuropathic pain, thalidomide for leprosy, and metformin for polycystic ovary syndrome.8 The hard commercial fact is that unless costs can be covered by patent, pharmaceutical companies are, on the whole, reluctant to find new uses for old drugs. Furthermore, there is a tendency in commercially funded trials not to test a new drug head-to-head against the best existing treatment, and this can lead to difficulties for clinicians in advising patients about the most appropriate treatment. And finally, there are problems of ownership of trial data and publication bias, with a tendency not to publish negative trials and worse still, deliberate non-reporting of adverse data.9
Therapeutic research planned jointly by patients and clinicians will address the issues of greatest importance to patients
The JLA proposes a process in which organisations representing patients and clinicians form working partnerships, to consider what research about treatments on a particular topic or disease they would like to see organised. Their work is supported and informed by the Database of Uncertainties about the Effect of Treatments (DUETs), which is populated by questions found through extensive searches for treatment uncertainties voiced by both patients and clinicians. Priority setting meetings, at which there are approximately equal numbers of patients and clinicians, supported by DUETs and the appropriate Cochrane review editor, will consider treatment uncertainties, examine existing evidence, identify the need for systematic reviews where there are none, and set priorities for future therapeutic research—which hopefully will then stand a better chance of being funded. Meetings will be independently chaired and all participants will be required to complete a declaration of competing interests.
As might be expected, translating the core philosophy of the JLA into practice has not proved entirely straightforward. However, a working partnership of thoracic physicians (the British Thoracic Society) and patients with asthma (Asthma UK) will shortly be considering priorities in asthma research. In preparation for this meeting, some important but unexpected treatment uncertainties have been collected in DUETS (see http://www.duets.nhs.uk). In addition, discussions are now taking place towards a possible working partnership in epilepsy.
Neurology is a specialty embracing a number of headline common conditions and a multitude of less common, often disabling diseases. The methods proposed by the JLA for identifying and agreeing research needed to address the many treatment uncertainties we face with our patients on a daily basis seem particularly well suited to the conditions we have to treat. Without capturing the insights and experiences of our patients in a systematic way, we risk underestimating or overlooking treatment uncertainties and outcome measures of greatest importance to them.
In the UK, neurologists already work closely with their patients and the many patient charities, both individually and through the links between the Association of British Neurologists and the Neurological Alliance. These links could be strengthened further by in JLA working partnerships, focusing on the therapeutic research that matters most to patients.
Competing interests: I am a member of the James Lind Alliance Steering Group (as is the Editor).
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