What is the value of research

Those who support the need for research argue that no new treatment should be offered outside the context of a controlled trial, so that the treatment's effectiveness and efficacy can be measured ab initio, not only for the sake of the patient receiving it but also for future patients. This view entails that patients should by custom and practice also be experimental subjects. Few would rather be a guinea pig than the recipient of tried and tested treatment, but the proponents of clinical trials point out that even an established treatment which is given outside the context of a trial is more often than not untested and unproven. Hence patients receiving it are, de facto if not dejure, guinea pigs in a uncontrolled trial whose outcomes are not being measured consistently.

Baum (1986) explained that the surgeon who carries out mastectomy for early breast cancer for 10 years and then switches to lumpectomy for the next 10 years, because custom and practice have changed, is in fact conducting a research project involving 'haphazard allocation'. The surgeon's patients are not receiving the best known treatment, they are receiving the treatment that she thinks is best on the basis of unreliable data. Because the surgeon is acting solely in what she believes to be the best interests of her patients, and her intentions towards them are unmixed with the desire to gain knowledge which will not be of direct benefit to them, the ethics of her behaviour have not, in the past, been openly questioned.

On the other hand, the surgeon might undertake a properly designed controlled trial, in which half her patients were chosen randomly (a technical word which means that patients' treatment is determined by the equivalent of tossing a coin rather than anyone's deliberate choice) to receive mastectomy and half to receive lumpectomy. If she then compared the treatment outcomes for each group, she would produce objectively convincing evidence for which of the two treatments is the better, instead of continuing in uncertainty or, worse still, thinking she knew which was better when she did not.

Whilst few would deny the need to demonstrate greater certainty than subjective observation allows, the attitude of the surgeon to the individual patients in the trial might nevertheless then be open to rebuke, because arguably she is not doing her best for each one, but treating each as a means to her own end: that of answering the question of whether mastectomy or lumpectomy is the better treatment for early breast cancer. However, Baum would argue that in fact the surgeon is doing the best for each of her patients because she is offering a 50% chance of receiving the best treatment, whichever it is. Since she does not know for certain which is better, it would be wrong for her to offer treatment in any other way than randomly (understood in its technical sense). If she switched to lumpectomy, without finding out for certain whether it was the better treatment, she might be exposing her patients to unknown risks and uncertain benefits. Hence, for the surgeon, putting her patients into such a trial means they are better off than if she offered just one of the two treatments.

The treatment for childhood leukaemia and the side effects of diethylstyl-boesterol are examples of why research is so important. For some decades in the UK, research into treatments for childhood leukaemia has been organized nationally, so that most children presenting with leukaemia will (with their parents' consent) be randomly allocated either the latest proven treatment or the latest novel and experimental treatment. As a result of this collaborative and carefully orchestrated activity, treatment for leukaemia has moved from being mostly unsuccessful to being 50% successful, in that mortality from the condition has dropped from 100% to 50%. The story of treatment development for leukaemia is an astounding success, from the point of view of its consequences. It has not been the result of some single, radical discovery like that of penicillin, which brought about a complete shift in the paradigm of treatment of those diseases which antibiotics can cure. Rather, it took, and continues to take, painstakingly small steps that have inched forward over years. Had such careful research not been conducted, successful treatments for leukaemia may have been developed, but, as Baum would argue, the discoveries would have been haphazard, subject to chance, and unlikely to have been received into the generality of practice so readily, since their efficacy would not be proven in the eyes of others.

Diethylstyboesterol was first synthesized in 1938 and administered to several million pregnant women to prevent spontaneous abortion and premature delivery (Dodds et al., 1938). Much later, in the 1970s, a group of doctors noticed a connection between the mother's exposure to the drug and the likelihood of her child, if female, contracting vaginal cancer (Noller and Fish, 1974). Now there are indications that the cancer risk is not so great as the 1970s observations indicated (Hatch et al., 1998). If the drug had been introduced by means of a properly conducted randomized controlled trial, with follow up of the patients in the trial, the questions about cancer would

The limitations of research

have been discovered by the 1940s or 1950s at the latest. Because the drug was administered in an uncontrolled fashion, risks were only noticed much later on by chance observation and linking of factors in the women concerned, and even then they were not properly quantified.

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