Confronting therapeutic ignorance

BMJ 2008; 337 doi: https://doi.org/10.1136/bmj.39555.392627.80 (Published 16 July 2008) Cite this as: BMJ 2008;337:a841
  1. Iain Chalmers, coordinator
  1. 1James Lind Initiative, Summertown Pavilion, Oxford OX2 7LG
  1. ichalmers{at}jameslindlibrary.org

    Tackling uncertainties about the effects of treatments will help to protect patients

    This week’s BMJ includes the first of a series of articles on areas of practice where clear and robust evidence is lacking, and where uncertainty exists about management.1

    Our failure to confront uncertainty about the effects of treatment has resulted in the suffering and death of patients, sometimes on a massive scale. Hundreds of thousands of people died prematurely because of failure to tackle uncertainty about the effects on mortality of prophylactic use of antiarrhythmic drugs in myocardial infarction.2 3 When the CRASH trial assessed uncertainty about the effects of corticosteroids in acute traumatic brain injury, it revealed that this treatment had been killing people for more than three decades.4 5 When widespread uncertainties about the effects of caffeine used to reduce episodes of apnoea in premature babies were assessed more than 30 years after the treatment had been introduced, it turned out that this simple treatment reduces the incidence of cerebral palsy and developmental delay.6 Had this uncertainty been dealt with when the treatment was introduced, many of the people currently living with cerebral palsy would have been spared their disabilities (see comment on caffeine citrate on the neonatal formulary website (www.neonatalformulary.com)). These are just three examples that illustrate the consequences for patients of our collective failure to recognise and confront uncertainties about the effects of treatments.7

    Over the past two decades, research relevant to treatment decisions has become more readily available to clinicians and patients. Systematic reviews have become much more readily available, and they have been indispensible in preparing evidence summaries for clinicians and patients, such as those published in BMJ Clinical Evidence and BMJ Best Treatments. These and similar resources have informed the preparation of clinical guidelines and clinical care pathways, and many of them are freely available to everyone through the NHS National Library for Health (www.library.nhs.uk) and NHS Choices (www.nhs.uk).

    Despite this progress, however, the challenge of meeting the information needs of clinicians and patients is far from complete, and patients continue to suffer and die unnecessarily as a result. Thousands of existing questions have not yet been investigated in systematic reviews, and thousands of systematic reviews have shown that the existing evidence does not answer important questions about the effects of many treatments. This challenge will not go away—indeed, resolving one uncertainty almost always results in the recognition of additional uncertainties.

    One contribution to making therapeutic uncertainties more visible is the database of uncertainties about the effects of treatments—DUETs (www.duets.nhs.uk), which is being developed by the specialist libraries of the NHS National Library for Health. Another is the new series of articles—called Clinical Uncertainties—which starts in this week’s BMJ. Readers who wish to highlight uncertainties are invited to submit manuscripts that clarify why uncertainty exists, cite evidence of variation in practice, and refer to relevant ongoing studies. Authors should conclude by advising clinicians and patients how they should deal with the uncertainty.

    It is important to be more open with the public about treatment uncertainties and to help patients who recognise the problem and want to help tackle uncertainties in research.8 Although DUETs and the BMJ’s uncertainty articles will help to raise awareness of therapeutic ignorance, more fundamental changes are needed to reduce the damage being done to patients by failure to confront uncertainties.

    The National Institute for Health and Clinical Excellence can help by making greater use of its option to recommend that treatments be used only within the context of research if evidence about their effects is inadequate.9 A recent judgment by the Healthcare Commission is also relevant—it criticised an NHS trust for putting patients at risk by introducing new equipment without evidence that it was beneficial.10

    The General Medical Council of the United Kingdom has made it clear in its booklet Good Medical Practice that doctors must help to resolve uncertainties about the effects of treatments. Sadly, clinicians who acknowledge the logic of the council’s expectations have to take account of the current disincentives facing those who want to confront therapeutic uncertainty. Hyper-regulation of clinical research has made it easier for clinicians simply to acquiesce in therapeutic ignorance.7 Indeed, clinicians who do tackle uncertainties can find themselves the target of highly publicised attacks by people who believe that their health problems can be attributed to their involvement in clinical research.11 12

    The consequences for patients of acquiescing in therapeutic ignorance can be disastrous, yet, perversely, current attitudes to, and restrictions on, therapeutic research are powerful disincentives to people who wish to confront uncertainties about the effects of treatments. It is up to clinicians, patients, and the public in general to decide whether they wish to continue tolerating this bizarre state of affairs.


    Cite this as: BMJ 2008;337:a841



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