Patients have their say on research priorities
David Holmes
Traditional ways of prioritising research are giving way to a more democratic process, with researchers working in partnership with patients. David Holmes reports.
Chronic fatigue syndrome researchers face death threats from militantsthe depressing headline of a recent investigation by the Observer newspaper in the UK. The campaign of intimidation and threats against scientists by a minority of activists who claim that researchers suppressed evidence that chronic fatigue syndrome has a viral cause is extreme, shocking, and vanishingly rare. But, atypical as such a case clearly is, it serves as a reminder of the sometimes complicated relationship between patients and the researchers who seek to better understand and treat their diseases.
Perhaps one of the most jarring aspects of the chronic fatigue syndrome militancy is that the picture of conflict between patients and researchers that the extremists' actions paint is so at odds with the increasingly close and fruitful relationships being established between many patient groups and research groups. Traditionally, setting priorities for research has been the province of clinicians and academics, but there has been an increasing realisation that the public should be more involved in decisions about what is researched and how that research is designed and undertaken. Chris Polman directs the Multiple Sclerosis Center at the VU Medical Center in Amsterdam and advises several research foundations, including the Dutch MS foundation, Reuma, and the Arthritis Foundation, and has been involved in designing trials for multiple sclerosis treatments since they first became available in the 1980s. He told The Lancet Neurology that it's critically important that patients and patient groups are involved in decisions on trials, especially in indicating what's important to them and what's not so important.
INVOLVE is a UK-based advisory organisation funded by the National Institute for Health Research and founded on the belief that research which reflects the needs and views of the public is more likely to produce results that can be used to improve practice in health and social care. Sarah Buckland has been at the organisation since its inception in 1996. Now Director, Buckland says she has seen a huge change in attitudes towards public involvement in research. Back when INVOLVE first started there was a lot of reticence or a lack of understanding about why involving the public makes a difference, Buckland told The Lancet Neurology. And yes, there are still researchers and others who can't quite see the value or are wary of involving the public for various reasons, but acceptance really is beginning to grow and more people are realising how patients have managed to contribute to research and improved the quality.
Susanne Sorensen, Head of Research at the Alzheimer's Society in the UK, agrees that researchers are much more open to the idea of involving the public than they have been in the past. Crucial to this increased acceptance, Sorensen says, is the growing realisation that contrary to what many people thought, [involving the public] is not a conservative force, it is not a force that directs us only towards care because many of them are very keen on basic science and discovering more about the aetiology of the disease.
UK patient charities have led the way in terms of getting patients involved in research. Launched in 1999, the Alzheimer's Society's research network is probably the oldest managed programme to get patientsor more often carersinvolved in setting research priorities, and was set up to provide a grassroots counterpoint to the old boys' network that prevailed in neurology in the 1990s. The same people sat on panels at the national medical research council, the Alzheimer's Society, the Alzheimer's Society Research Trust etc, and they awarded each other funding, Sorensen recalls. That doesn't mean that it wasn't good research, because they were probably the best people around, but in the Alzheimer's Society we have a big grassroots membership and felt that we really needed to have those voices heard and those voices influencing what we fund.
The research network has expanded steadily in the 12 years since it began, and it has provided a template for similar programmes at the Multiple Sclerosis Society, Parkinson's UK, and countless others. However, listening can be a complicated exercise. Around 200 lay volunteers in the research network each get all the proposals that the society receives for its responsive-mode funding. We get about 50 comments in on each funding application from our members, which is a good spread, Sorensen explains. These comments are then worked into the process to decide on what applications get funded. The volunteers have a real input into what we select for funding, says Sorensen. We also ask them every other year to set the priorities for our research programme; we ask them what is important to them, what would you like to be researched, what would you like to get out of research.
Listening to its research network members has led the Alzheimer's Society to back successful projects that probably would not have been funded elsewhere. Notably, the society supported several groups that were investigating tau rather than amyloid. If you go back to about 2000 there was still a big discussion between tauists and amyloidists, and people interested in tau were seen as second class and often didn't get funding elsewhere, Sorensen explains. Well, we funded researchers looking at tau consistently and that has turned out to be very fruitful and very influential. The logic of the patients and carers who make up the research network was simple: if everybody else is going with amyloid and it is not leading anywhere at the moment, we should look somewhere else. Sometimes they take a simple position like that: we must take a risk on something else, says Sorensen. It was seen as quite risky at the time, which is amusing now.
For Polman, taking the ideas of patients on board is essential for improving the design of trials to ensure that the chosen endpoints are relevant. For example, he says, if you want to measure the severity of disease then a neurological exam exists for reflexes etc, but I've never heard a patient complain about slow reflexes because that's not something that patients experience in daily life, it's something that the doctor tries to evoke that's not relevant. And according to INVOLVE's Buckland, input from patients can also improve recruitment to trials. There are lots of examples of how patients have been able to say that if you do research in this way then people won't want to take part, and that can be as small as changing the times that you're asking people to come in for a treatment.
Improving the design and conduct of clinical studies and building trust between researchers and the public are important products of these interactions. But there is a danger that some patient involvement can amount to little more than a box-ticking exercise. Getting the right patients involved is imperative, argues Sorensen. It's no good to have a universal group of patients sitting in your hospital, because somebody with arthritis might not have very pertinent opinions about dementia research, with all its specific problemsand there are so many issues about taking people in for interventions etc that might be completely different for other patient groups. Getting the right type of lay, lived experience is as important to getting salient patient input as getting the right type of clinical expertise is to the design of clinical research. That's a big problem in public and patient involvement at the momentthat groups are being formed in hospitals who have to provide a lay point of view but they can only really generalise if it's not their lived experience, says Sorensen. I think it's difficult, and I think the charities are probably the better sources for patient involvement because they tend to be about specific types of disease.
But although charities are well placed to provide an interface between patients and researchers, they also have to operate within tight financial constraints. Keeping a small army of volunteers abreast of the latest scientific developments and enabling them to attend site visits and be fully trained and supported does not come cheap, and many balk at the cost. Cost is not the only issue, according to Buckland. It is time consuming and it does take more thinking through when researchers are doing it, she says. Where the thinking upfront hasn't happened and there's not enough time, or the time and energy and thought hasn't been put into how things might work, sometimes things do go wrong. But then what research project doesn't have things go wrong. In Sorensen's opinion, although the financial and logistical burdens are something other charities might attack, the benefits in terms of innovative and high-quality research far outweigh the negatives. I really think that if you're spending public money, you have to ensure that you bring people with you and that you try to address what they want.
Addressing research questions that matter to the people affected by neurological disorders, in a way that satisfies the practical and ethical demands of patients and their families, is central to the success of clinical research. Finding new ways of involving patients and the wider public in clinical studies is likely to become increasingly important in the future, as researchers recognise the enormous value of working in partnership with patient groups.
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