The status of clinical trials: Cause for concern

<p>Abstract</p> <p>Background</p> <p>Americans see clinical research as important, with over 15 million American residents participating in NIH-sponsored studies in 2008 and growing yearly.</p> <p>Methods</p> <p>Documents reporting NIH supported Clinical Research projects were reviewed.</p> <p>Results</p> <p>When compared with other studies, the number of interventional Phase III and Phase IV trials have decreased from 20% to 4.4% from 1994-2008.</p> <p>Conclusions</p> <p>This finding most likely has occurred for several reasons. One reason is that the physician lacks an infrastructure for designing and carrying out trials. This lack is because of an absence of a coordinated effort to train clinical trialists. It is clear that the Nation needs a more purposeful approach to developing and maintaining the infrastructure for designing and conducting clinical trials. Building it de novo trial by trial is profoundly inefficient, to say nothing about time consuming and error prone.</p

Evaluating treatments in health care: The instability of a one-legged stool

<p>Abstract</p> <p>Background</p> <p>Both scientists and the public routinely refer to randomized controlled trials (RCTs) as being the 'gold standard' of scientific evidence. Although there is no question that placebo-controlled RCTs play a significant role in the evaluation of new pharmaceutical treatments, especially when it is important to rule out placebo effects, they have many inherent limitations which constrain their ability to inform medical decision making. The purpose of this paper is to raise questions about <it>over-reliance </it>on RCTs and to point out an additional perspective for evaluating healthcare evidence, as embodied in the Hill criteria. The arguments presented here are generally relevant to all areas of health care, though mental health applications provide the primary context for this essay.</p> <p>Discussion</p> <p>This article first traces the history of RCTs, and then evaluates five of their major limitations: they often lack external validity, they have the potential for increasing health risk in the general population, they are no less likely to overestimate treatment effects than many other methods, they make a relatively weak contribution to clinical practice, and they are excessively expensive (leading to several additional vulnerabilities in the quality of evidence produced). Next, the nine Hill criteria are presented and discussed as a richer approach to the evaluation of health care treatments. Reliance on these multi-faceted criteria requires more analytical thinking than simply examining RCT data, but will also enhance confidence in the evaluation of novel treatments.</p> <p>Summary</p> <p>Excessive reliance on RCTs tends to stifle funding of other types of research, and publication of other forms of evidence. We call upon our research and clinical colleagues to consider additional methods of evaluating data, such as the Hill criteria. Over-reliance on RCTs is similar to resting all of health care evidence on a one-legged stool.</p

Use of Pharmacoeconomics Information—Report of the ISPOR Task Force on Use of Pharmacoeconomic/Health Economic Information in Health-Care Decision Making

Objectives: Despite the growing number of pharmacoeconomic (PE)/health economic (HE) studies, very little is known about their use by decision makers. The objectives of the Task Force were to ensure that the good research practices of PE/HE studies pay attention to the needs of health-care decision makers and to develop a “toolbox” for the health-care decision maker wanting to interpret and use PE/HE studies. Methods: The membership of the Task Force consisted of individuals involved in making decisions about the availability or use of medicines and researchers into the use of economic evaluations. The group communicated by E-mail and face-to-face meetings. A literature review of decision makers’ attitudes toward PE/HE studies and published economic evaluation guidelines was undertaken. In addition, a focus group discussion was held with opinion leaders in managed care pharmacy. Results: The literature review identified 16 surveys of decision makers’ attitudes toward PE/HE studies and 15 published guidelines that outlined reporting requirements for economic evaluations. These were reviewed and classified. Based on the published literature and comments from decision makers, seven additional reporting requirements for studies were specified. Conclusions: While the Task Force's additional reporting requirements may be helpful to decision makers, they raise a number of issues. These include the feasibility of meeting the additional requirements, whether decision makers should receive more education in economic evaluation, and whether there should be more study of health-care decision-making procedures themselves.Peer Reviewedhttp://deepblue.lib.umich.edu/bitstream/2027.42/73067/1/j.1524-4733.2003.64245.x.pd