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‘Nothing about them, without them’ is the clarion call of those who are committed to genuine user participation in research. The James Lind Alliance brings patients, carers and clinicians together as priority sharing partnerships to ensure that research is targeted at questions that matter to these interested parties. In 2010, we contributed to the Database of Uncertainties of the Effects of Treatments (DUETs). Three hundred ninety-eight questions regarding treatments and their effects were produced from focus groups composed of people with epilepsy, their carers and relatives, or epilepsy clinicians and researchers.1 The questions were subsequently ranked by participants and thematically grouped to identify similarities and comparisons. The top-ranked questions were all deemed ‘unanswered’ at the time of publication following thorough scrutiny of online databases.2 It was our hope by publishing the report and by uploading all questions to the National Health Service Evidence DUETs webpage2 that we would encourage researchers to preferentially focus on these questions. Epilepsy was one of the first partnership exercises to be launched, and we aim to assess the influence of the process since the priorities were published. We sought to identify if the top 20 ranked questions from professionals and patients had been answered over the last 6 years (table 1) (see online supplementary data file 1).
We searched the PubMed and Cochrane databases for published papers, and the National Institute for Health and Care Excellence, Scottish Intercollegiate Guidelines Network (SIGN), International League Against Epilepsy (ILAE), American Epilepsy Society (AES), Neurocritical Care Society (NCS) and European Federation of Neurological Societies (EFNS) guidance for authoritative statements (from 1 January 2010 to 1 April 2016). Papers were rated as ‘answering’ a question if they contained new data that wholly or in part addressed the uncertainty. Review articles, most case reports and hypotheses without data were not included. We recorded the total number of papers yielded by the search strategy and the number that answered the question.
Clinicians and researchers
Three questions were adequately addressed by a similar question, leaving 17 unique questions (Table 2). The status epilepticus questions had the most papers addressing them—46 targeted papers from a total of 108 identified. ‘Prescribing in pregnancy’ and ‘cognitive and psychiatric effects’ were the next best answered, with 39 and 22 targeted papers, respectively. Question 9, ‘Should the lamotrigine dose be pro-actively increased in pregnancy?’, is the focus of an ongoing trial: EMPiRE Study – Anti-Epileptic drug Monitoring in PREgnancy: an evaluation of effectiveness, cost-effectiveness and acceptability of monitoring strategies.3 We identified no relevant papers for two questions (6 and 14).
Patients and carers
The top 20 questions from patients and carers produced 18 unique uncertainties (Table 2). The top priority was addressed. The clusters that involved allied health professionals such as education (26 targeted papers) and cognition and psychiatric effects (30 papers) were better answered than practical questions about how best to take the medication and service configuration (7 and 1 papers, respectively).
There were 158 relevant papers for the clinicians’ and researchers’ questions (mean: 9.3 per question, range: 0–39) compared with 73 for patients' and carers’ questions (4.1 per question, range: 0–24). There was a trend for the questions to get less well answered as the question’s ranking dropped. Fewer patient questions were addressed by national guidelines than professionals’ questions (6 vs 25). All of the professionals’ questions were answered by research or guidelines, compared with 15 of the patients’ questions.
We recognise that our research strategies were not exhaustive and were unblinded. We demonstrate that researchers could be doing more to answer the questions that matter to patients with epilepsy. In contrast researchers are doing a fine job at answering research priorities that are of interest to them and their peers. Research priorities produced by patients and carers produce only 46% as many papers, are addressed in only 24% as many guidelines and often go unanswered entirely. This is despite patient questions often being more tangible and practical. Many of these practical questions remain entirely unanswered. Interestingly, with question 18 (over the diagnosis of depression in epilepsy), there was much new research about the link between epilepsy and depression, but no results specifically answering patients’ question. This indicates that research in this area is happening, but not being focused to areas with keen patient interest.
We suspect that many of the professionals’ questions were created and rated highly because they were aware of the literature and ongoing research, and because they keep up to date with controversies. In contrast, many of the patients’ questions do not have the research momentum and appear de novo. Many of these questions could be considered partially answered, and now it would be useful to create a more specific list of questions relating to each of the broadly ’answered' topics. Examples of true evidence gaps are management when initial epilepsy treatment fails, and how effective second-line treatment is following initial treatment failure.
Other priority setting partnerships using the DUETs model have looked at the dissemination and impact of their priorities. The childhood disability group asked the Health Technology Assessment and Health Services and Delivery Research programmes to identify which of their top questions were the focus of successful funding awards.4 A year after publication, two were being answered: the top priority regarding therapeutic regimens and the 10th focusing on sleep disturbance. A third priority was the subject of NICE guidance in preparation. The Palliative Care group’s priorities gained immediate traction within their community.5 The All Ireland Institute of Hospice and Palliative Care arranged a local prioritisation workshop to reprioritise the long list to their local circumstances. Using the UK Clinical Research Collaboration database they undertook a research mapping exercise to identify the grants that may answer their top questions. They have taken the process further by both reviewing the out-of-scope questions and, with the Motor Neuron Disease Association, directly commissioning research that answers their long list of priorities. We suggest that it is time for a new epilepsy priority sharing partnership to be convened using the James Lind Alliance model, and we hope that the priorities that they produce can capture the imaginations of charities, grant-administering bodies and researchers.
We are grateful to the patients, their relatives and carers, and the clinicians and scientists who gave up their time to participate in the original study.
Competing interests RHT receives/received research support from Epilepsy Research UK, Action Medical Research, Epilepsy Action and the Dravet Society; is an associate editor of Practical Neurology and web editor of Seizure and has received honoraria from Eisai, Sanofi and UCB.
Provenance and peer review Not commissioned; externally peer reviewed.
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