The Cochrane Lecture the Best and the Enemy of the Good
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6 6Journal of Epidemiology and Community Health 1994;48:6-15 The Cochrane lecture J Epidemiol Community Health: first published as 10.1136/jech.48.1.6 on 1 February 1994. Downloaded from The best and the enemy of the good: randomised controlled trials, uncertainty, and assessing the role of patient choice in medical decision making Klim McPherson Abstract clear today as it did then. Nowadays, both the This lecture aimed to create a bridge to problem and solution are very familiar to us span the conceptual and ideological gap all. The problem was that much of the health between randomised controlled trials care provided was unevaluated, and therefore and systematic observational compari- possibly of no benefit, and the solution - sons and to reduce unwanted and unpro- randomised controlled trials - were the only ductive polarisation. The argument, secure way of knowing the truth of the matter. simply put, is that since randomisation Cochrane's essential idea was that the reli- alone eliminates the selection effect of able assessment of effectiveness was the only therapeutic decision making, anything key to scientific health care, but such methods short of randomisation to attribute cause as he advocated had been disparaged by the to consequent outcome is a waste of time. medical establishment - paradoxically because If observational comparison does have they seemed to be less scientific than the more any significant part in evaluating medi- traditional basic methods of scientific evalu- cal outcomes, there is a grave danger of ation. A problem of methodological imperia- "the best", to paraphrase Voltaire, lism which is with us still, but in different becoming "the enemy of the good". The guise? first section aims to emphasise the ad- He argued forcibly that only when the effec- vantages of randomised controlled trials. tiveness of a treatment or preventive measure Then the nature of an essential precondi- had been established and quantified, could the tion - medical uncertainty - is discussed issues of efficiency and equity be discussed in terms of its extent and effect. Next, the intelligently. But in the absence of reliable http://jech.bmj.com/ role of patient choice in medical decision information about effectiveness, the pursuit of making is considered, both when out- optimal health policies was bound to be per- comes can safely be attributed to treat- manently elusive. ment choice and when they cannot. There Since then Cochrane's arguments have been may be many important situations in largely and impressively accepted by health which choice itself affects outcome and professionals.3 It is probable that without them this could mean that random compari- we would not have all the welcome recent sons give biased estimates of true thera- developments designed specifically to provide on October 3, 2021 by guest. Protected copyright. peutic effects. In the penultimate section, a more effective health care system. I am the implications of this possibility both referring particularly to the NHS Research for randomised controlled trials and for and Development initiative and the systematic outcome research is pursued and lastly concentration on issues of effectiveness and there are some simple recommendations efficiency in the direction of research and for reliable outcome research. health policy. This is why there is a Cochrane lecture at (JT Epidemiol Community Health 1994;48:6-15) the Annual Scientific Meeting of the UK Department of Public Society of Social Medicine and I am honoured Health and Policy, to be giving the lecture this year. I cannot do London School of Hygiene and Tropical Archie Cochrane's great contribution to our justice to my predecessors, who have contrib- Medicine, Keppel discipline in 1972 was to describe a most uted significantly and importantly to the work Street, London important problem in health care and to posit a spearheaded by Archie Cochrane, but I hope WC1E 7HT on K McPherson satisfactory solution. In his biography,' he says to make some methodological points the of his work on Effectiveness and Efficiency2: question of evaluation of health care, many of Correspondence to: I want to Professor Klim McPherson. "I decided to concentrate on one simple idea which I have made before. Largely, - the value of the randomised controlled trials try to divert any polarisation of methodologies Presented at the Scientific Meeting of the UK Society in improving the NHS - and to keep the book which may, I believe, lead to an unnecessary for Social Medcicine, of medical uncertainties. The Cambridge, UK in short and simple." prolongation September 1993. He did so in a manner which rings as loud and issues are too important in my view. The best and the enemy of the good 7 (1) The value of randomised controlled provide hard unbiased evidence about the rela- J Epidemiol Community Health: first published as 10.1136/jech.48.1.6 on 1 February 1994. Downloaded from trials tive effectiveness of common, important, and Since the publication of Effectiveness and Ef- expensive treatments. It is absolutely no part ficiency2 there have been several attempts to of my argument to disparage randomised com- minimise or falsify the importance and rele- parisons at all - least of all because they are too vance of Cochrane's message. Each succeeds expensive or complicated. The end justifies the only in accentuating its importance. It is im- means most of the time. To accept these kind possible nowadays to deny the requirements of arguments is really to suggest that some for serious research on outcomes, but the gap alternative is generally better and that some- between true knowledge of outcomes and the how it does not matter, because it is inevitable, need to know and to understand, and hence to that most treatments will not be evaluated by meet these requirements, remains large. randomised comparisons. What was most important in 1972, and still Feinstein and others (Colin Dollery, for is for us now, is how to bridge that gap. Last example, in his Rock Carling lecture7) assert year Iain Chalmers gave a masterful demon- that most treatments will be assessed by a stration of the practical extrapolation from mixture of insight, theory, acumen, and un- Cochrane himself to the Cochrane Centre, controlled observation. Of course large effects clearly a central part of the "outcomes move- can be reliably detected like that, but from now ment". This evolved from the National Peri- on I will be discussing uncertainties over small natal Epidemiology Unit and took on the work but important effect differences in common of Iain, Richard Peto, Rory Collins, and others medical practices which are poorly understood in summing the results of randomised trials of and are simply not amenable to evaluation in treatment. There is no question in my mind this way. that these efforts in understanding the effec- There is a more extreme current idea - tiveness of treatments, and hence helping which is just plain wrong - and this is that the people choose between options, are of enor- use of databases which record everything mous value. For me, the most exciting has about patients can easily, as a consequence of been the work on breast cancer treatment.4 recording everything, be used to adjust for all The increasing use ofrandomised comparisons confounders and then compare treatments as if in judging effectiveness is wholly appropriate there had been no selection.8 This has to be and must go much further. dismissed as ridiculous. Unknown and unsus- pected confounding is mostly important and always elusive. IMPEDIMENTS The great idea of Fisher and Bradford Hill, I want to leave aside, for the moment, the so ably developed by many, including Archie extent of the importance of uncertainty in Cochrane, is one of the few panaceas of our health care decision making, and examine time. In the context of medical interventions, briefly part of the existing resistance to the use the essence of the argument has been repea- of randomised controlled trials. Cochrane dis- tedly put by David Byer.' He says that "Epi- cussed impediments to the wider acceptance of demiology is an essential discipline . for these in practice. There are three basic reasons assessing the importance of exposures ... there for not carrying out randomised controlled is a disassociation between the reason for expo- http://jech.bmj.com/ trials where uncertainty exists: sure and the outcome. On the other hand, in (1) Ethical objection (?) medicine, the doctor chooses the therapy pre- (2) Lack of objective outcome measures cisely to affect the outcome and for no other (3) The resistance of those "threatened" by reason". Archie's view was most succinctly randomised controlled trials. put, and poignant nonetheless: "Observational evidence is clearly better than opinion but it is thoroughly unsatisfactory". THE ARGUMENTS on October 3, 2021 by guest. Protected copyright. We must be clear about the basic arguments, because in advocating greater, more system- UNCERTAINTY, SUPPLIER-INDUCED DEMAND, atic, and rigorous use of observational AND CONSUMER PREFERENCES methods5 in judging efficacy (as I shall be Let me restate the basic premise in case subse- doing later) it is very easy to associate oneself quent remarks should be misunderstood. Ran- with a camp one does not wish to be associated domised comparisons provide the most rigor- with. There have been many exponents of the ous assessment of the therapeutic effect of view, encapsulated by Feinstein,6 that "Cause- particular interventions. The problems they effect can be evaluated observationally" is are set to help solve are, however, formidable, scientific heresy only if two cogent scientific for decision making in medicine is rarely quite realities are ignored - the first, that some as simple as choosing the best treatment for things are not amenable to experimental inter- each objective diagnosis, even when it is vention, and the second, that randomised con- known.