Research ethics and evidence-based medicine

Abstract

It is increasingly becoming a standard requirement to have to document effectiveness before new interventions are adopted by a health care system. The methods of Evidence-based Medicine, such as meta-analyses or other systematic evaluations of evidence, are often used to document effectiveness or the lack of it. There is an understandable reluctance to fund new experimental interventions when the evidence is not yet complete. Some of the most widely known controversies about the application of Evidence-based Medicine, such as mammography screening for breast cancer, have been concerning disputes about what the evidence says about effectiveness. Similarly, in discussions about the use of Evidence-based Medicine for the issue of allocation of scarce resources, the focus has often been on the problematic assumptions made when utilizing methods, such as Quality Adjusted Life Years. In this paper I wifi argue that there is an additional issue that has not been addressed at all in the literature. In my discussion, I wifi assume that there is no disagreement about the established effectiveness of the intervention. I assume further that there is agreement that the established effective intervention is too expensive for general implementation, i.e. it is not cost-effective. I recognize that both of these claims may more often than not be controversial, but for the sake of argument I will assume that there is no such controversy. The problem I want to address is how we should go about establishing the effectiveness of a cheaper intervention that can be widely implemented. According to Evidence-based Medicine we should conduct a randomized clinical trial with the proposed new, cheaper intervention in one of the treatment arms. I wifi demonstrate that this requirement raises an interesting and difficult ethical issue. The perhaps most famous recent example of an intervention that was considered too expensive to implement is treatment of mother and baby to prevent pen-natal transmission of HIV. An effective, but very expensive intervention was established, which was unaffordable for developing countries. A proposed shorter, and cheaper, intervention was subsequently tested in randomized clinical trials in various countries, most of which included placebo in the control group. These trials were widely criticized because they did not use the established, proven treatment as a control. Those who defended the trials argued that the design was necessary to answer questions of relevance to developing countries. What is interesting about this discussion is that it has only been regarded as a problem for developing countries: in the developed world it has simply been taken for granted that one should use the established, effective treatment in the control group. In this paper I will argue that this is not true: the problem of the choice of control groups is a general one, arising in all situations of resource constraints, not just in situations of extreme lack of resources, such as in developing countries. It follows from this, since no country has unlimited resources for health care, that it is a general problem facing all of us. I will defend this claim by examining a recent case from the UK involving treatment of multiple sclerosis. © 2005 Springer-Verlag Berlin Heidelberg.