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Should placebo-controlled trials be eliminated in favor of comparative-effectiveness research?
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The two types of research go hand in hand.
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It is critical to understand the distinction between clinical trials and comparative-effectiveness research. In oncology, this distinction is extremely important. Clinical trials are designed to determine efficacy in a defined population of patients, whereas the goal of comparative-effectiveness research is to look at a broader population of patients to see, for example, if the benefit demonstrated in a clinical trial showing efficacy can be translated to observed effectiveness in a more general population. The problem with this concept of comparative-effectiveness research in oncology is that we are moving more and more to subsets of patients in terms of measuring benefits of outcomes. That does not mean comparative-effectiveness research has no place in oncology. It certainly can be useful, but it will not replace what we do in clinical trials and that includes, in select circumstances, the use of placebo-controlled trials.
There are many examples where the placebo was essential in measuring the efficacy of the experimental treatment. It is important to understand that in oncology comparative-effectiveness research should not replace clinical research. In fact, one of the concerns that has been expressed is that if there is an overemphasis on comparative-effectiveness research will stifle innovation and potentially detract from clinical trial efforts. I personally don’t believe that will happen. I think these two types of research should integrate hand in hand.
Al B. Benson III, MD, is a Professor of Medicine at the Feinberg School of Medicine at Northwestern University and Associate Director for Clinical Investigations at Robert H. Lurie Comprehensive Cancer Center at Northwestern University.
Placebo-controlled trials are sometimes necessary.
Placebo-controlled trials are sometimes necessary. Comparative-effectiveness research and placebo-controlled trials are not mutually exclusive.
The type of control for a trial depends entirely on the circumstances. There are some clinical situations in which no treatment, ie, placebo, is appropriate.
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For instance, when investigating adjuvant therapy in kidney cancer, there are three large, ongoing trials with placebo controls. Placebo is appropriate in these trials because observation is the current standard of care in this setting. In contrast, in metastatic kidney cancer, there are now many active agents.
Therefore, a placebo-controlled trial would be inappropriate, as there are data to support that there is at least some clinical activity to many therapies in the treatment-refractory setting.
In the metastatic setting, trials that compare efficacy of established agents would thus have a greater impact on clinical practice by informing the risks and benefits of one active approach vs. another.
Brian Rini, MD, is Staff at the Cleveland Clinic Taussig Cancer Institute, Ohio.