A new drug is introduced to the market. It has been approved (after stringent scrutiny) by regulators, who require ever more convincing evidence for safety and efficacy. Aside from the increased costs of the new treatment compared with the old, what could the problems be?Even on the cost front, many would argue that there is little cause for concern. We have entered an era where placebo-controlled clinical trials demonstrate that new treatments work, in contrast to the demonstrations of efficacy of the sort available for earlier treatments. There is general agreement that, if we were to operate only in accordance with the demonstrations of efficacy from clinical trials of the type now done, the health services would be more effective and efficient and, ultimately, costs would fall. Furthermore, the use of many new drugs in recent years has appeared to be justified by economic models based on figures from clinical trials and a range of assumptions, such that a new antidepressant or antipsychotic costing several thousand pounds a year can be transformed (by costs offsets) into a treatment that is less expensive than that using an older agent costing £50 per year or less.
Treatment effects and treatment effectivenessThere are many problems with this scenario, however. When they were introduced, randomised controlled trials (RCTs) were a significant step forward in terms of evaluative technologies for new treatments. The assumption of a null hypothesis means that their primary purpose was to show that treatments did not work -to stop therapeutic bandwagons in their tracks. Within psychiatry, for instance, the first RCTs demonstrated that cortisone did not work for schizophrenia (Rees, 1997). A recent illustration of this function of RCTs lies in demonstrations that debriefing, which had all but become a social movement (Raphael et al, 1995), does not work -at least when given indiscriminately (Bisson et al, 1997).What RCTs did historically was to demonstrate to the opponents of treatments such as chlorpromazine that the first antipsychotics did have some treatment effect, whatever these critics might still think about the overall benefits. Now, in complete contrast to the original intentions behind their use, RCT evidence is used to fuel therapeutic bandwagons. It is sold as evidence that the treatment works (actually does good) rather than evidence that treatments have an effect (which may be put to good use in judicious hands). There is no philosophical or methodological basis for this development.Randomised controlled trials originated within epidemiology. Some epidemiologists had and continue to have considerable misgivings about the capacity of randomisation to overcome the problems of external validity that result from the sampling methods adopted by this approach. The alternative is to use large simple trials with 'hard' end-points such as mortality (Healy, 1997). The problems inherent in RCTs are compounded in companysponsored RCTs, which explicitly recruit samples of convenience. This approach offers intern...