Do doctors have a duty to take part in pragmatic randomised trials?
BMJ 2017; 357 doi: https://doi.org/10.1136/bmj.j2817 (Published 14 June 2017) Cite this as: BMJ 2017;357:j2817
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Randomised controlled trials (RCT) in surgery are frequently pragmatic due to unique challenges related to complex interventions. Whilst once described as “comic opera”, surgical research in the United Kingdom has advanced significantly in recent years due to dedicated surgical trials units, integrated academic-clinical career pathways and trainee-led research collaboratives (1-4). Within established and supported networks, we agree that all patients should be afforded the opportunity to engage in pragmatic, randomised trials.
Unfortunately, research waste is increasingly recognised in academic literature. In surgery, one in five RCTs are discontinued early and one in three completed trials remain unpublished (5). Similar problems are encountered in other medical and allied specialties. It is estimated that up to 85% of all health research is avoidably wasted, equivalent to $170 Billion (6). Failure to disseminate research findings leads to hidden trial data, unrealised knowledge and the risk of unnecessary duplication. It also raises ethical concerns related to futile participation for both patients and clinicians. The AllTrials campaign aims to address these issues by calling for mandatory registration and dissemination of research findings.
Investigators can, and must, do more to encourage participation in pragmatic trials. It is their duty to eliminate waste by ensuring timely and complete dissemination of research findings and facilitating open, patient-level meta-analysis. In surgery, this is important to ensure greater research activity is translated into increased outputs. By reducing waste, it will empower clinicians and patients to participate in future trials with confidence and assurance.
Stephen J Chapman, @SJ_Chapman; NIHR Academic Clinical Fellow, University of Leeds, UK
James CD Glasbey, @DrJamesGlasbey; NIHR Academic Clinical Fellow, University of Birmingham, UK
References
1. Horton R. Surgical research or comic opera: questions, but few answers. Lancet 1996;346:984-985.
2. Clough S. Fenton J. Harris-Joseph H. et al. What impact has the NIHR Academic Clinicsl Fellowship (ACF) scheme had on clinical academic careers in England over the last 10 years? A retrospective study. BMJ Open 2017;7:e015722.
3. Royal College of Surgeons of England. Surgical Trials Initiative. Available at : https://www.rcseng.ac.uk/standards-and-research/research/surgical-trials... [Accessed 16th June 2017].
4. Bhangu A. Kolias AG. Pinkney T. et al. Surgical research collaboratives in the UK. Lancet 2013;382:1091-1092.
5. Chapman SJ. Shelton B. Mahmood H. et al. Discontinuation and non-publication of surgical randomised controlled trials: observational study. BMJ 2014;349:g6870.
6. Chalmers I. GLasziou. Avoidable waste in the production and reporting of research evidence. Lancet 2009;376:86-89.
Competing interests: No competing interests
Do 'patients' have a duty to take part in pragmatic randomised trials?
At the very least patients receiving essentially free medical care from universal health coverage medical systems?
For their very own sake and for the others...
Competing interests: No competing interests
Sir
Surely the most interesting finding in this debate is the fact that all the authors agree on the benefits of doctors participating in pragmatic research; even those who oppose the idea that doctors should be forced to participate agree that participation does benefit society. I would contend that the issue of 'duty' or 'obligation' misses the point. What we need is a system that encourages doctors and patients to participate in pragmatic research. In particular, doctors and patients and health administrators should all be encouraged to participate in comparative effectiveness research. When there are multiple treatments that have been shown to be of benefit for a particular condition, there are benefits to everyone if the medical profession can determine whether one treatment is superior to another, or whether one particular treatment is superior to the alternatives in a specific context. Patients will obviously benefit if they receive the optimal treatment, and the health services will benefit if the correct treatments are given to the correct patients; there will be less prescribing of ineffective treatments, and costs will be reduced if it can be shown that a cheaper treatment is at least as effective as a more expensive treatment. (The one group who would perhaps not benefit are commercial enterprises who market relatively ineffective treatments.)
Unfortunately, there are many obstacles at present to performing multicentre pragmatic randomised controlled trials. I am involved in an international study group - EpiNet - whose goal is to promote large investigator-led observational studies and pragmatic randomised controlled trials to determine the optimal treatment for people with epilepsy. We have been repeatedly surprised how difficult it has been to actually get studies underway; these range from difficulties with obtaining regulatory and ethical approvals, the need to pay for various services, apathy and disinterest from doctors, and suspicion of research by patients.1
I would therefore contend that a better question than: "Do doctors have a duty to take part in pragmatic randomised trials?" would be: "How can the various health services encourage pragmatic randomised trials?"
Peter Bergin, Chairman of EpiNet study group. pbergin@adhb.govt.nz
References
1) Bergin, P.S., Beghi, E., Sadleir, L., Brockington, A., Tripathi, M., Richardson, M., et al for the EpiNet Study Group., 2017. EpiNet as a way of involving more physicians and patients in epilepsy research; validation study and accreditation process. Epilepsia Open. 2017:2(1);20–31 http://onlinelibrary.wiley.com/doi/10.1002/epi4.12033/full
Competing interests: No competing interests
Choice of treatments when there is uncertainty about the effects of alternatives
Charles Weijer and his colleagues are correct to assert that people should be able to choose how they help others when there is uncertainty about the effects of treatments (1); but a flawed ethical analysis prompts them to take too narrow a view of how this should be done. I offer here some principles for guiding choices in publicly-funded health services, noting that principles may differ in health services that reject shared risk, shared benefit, and shared cost.
When there are uncertainties about the effects of alternative treatments already in use, patients should be made aware of these uncertainties and declare any preferences they have for (i) choosing any of the existing treatment alternatives; or (ii) choosing to participate in randomized trials to reduce uncertainties about their relative merits.
If the uncertainties relate to the relative merits of new treatments compared with treatments already in use, patients should be offered the choice of existing treatments, or the choice to participate in randomized trials comparing the inadequately evaluated new treatments, so helping to generate the evidence needed to assess whether the new treatment is more likely than existing treatments to do good than harm, at acceptable cost.
None of the above options exposes patients to “risks primarily for the benefit of other people”, as suggested by Weijer et al. Indeed, for the high proportion of patients with chronic health conditions, reducing uncertainties can be expected to improve the evidence base for their future health choices (2).
The continued promotion of flawed and narrow ethical analyses has resulted in the avoidable suffering of millions of people, the vast majority of whom have not been the participants in research to whom Weijer and colleagues have inappropriately restricted their attention (3).
References
1. Weijer C, Goldstein CE, Edwards SJL. Do doctors have a duty to take part in pragmatic randomised trials?: No. BMJ 2017;37:j2817.
2. Chalmers I. What do I want from health research and researchers when I am a patient? BMJ 1995;310:1315-1318.
3. Chalmers I. Regulation of therapeutic research is compromising the interests of patients. International Journal of Pharmaceutical Medicine 2007;21:395-404.
Competing interests: No competing interests