Last Thursday, an article was published in the New England Journal of Medicine in which former director of the US Centers for Disease Control and Prevention Thomas Frieden called for greater use of alternative data sources, other than randomized controlled trials (RCTs), for health care decision making.1
Frieden says, “For much, and perhaps most, of modern medical practice, RCT-based data are lacking and no RCT is being planned or is likely to be completed to provide evidence for action. This ‘dark matter’ of clinical medicine leaves practitioners with large information gaps for most conditions and increases reliance on past practices and clinical lore.”1
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The article comes as the US Food and Drug Administration (FDA) looks to flesh out a framework for the use of real world evidence. Some examples of “real world evidence” include: observational studies, routine safety surveillance, patient registries and insurance claims, all to evaluate drugs and medical devices.
FDA announced it would hold a public workshop in collaboration with the Duke-Marglis Center for Health Policy in September on the development of real world evidence framework.
RCTs can be considered the “gold standard” for creating evidence to support the approval of new drugs. Frieden argues that observational studies may be just as appropriate at times for determining health outcomes.
Vinay Prasad, a hematologist-oncologist and assistant professor of medicine at the Oregon Health and Sciences University does not completely agree with this assertion. “When are randomized trials desperately needed? To prove that at least in some circumstances a therapy can work … you have to show that something can work under some circumstances to move forward. You cannot assume this. Only an RCT can tell you this unless the effect size is huge,” Prasad said.
Frieden also argues that RCTs often have insufficient study periods and small sample sizes, which lead to unreliable data. On the contrary, Prasad argues that RCTs are still necessary to hash out the effect of any given pair of treatments.
In addition, Frieden mentions the impractically of conducting RCTs, citing their high cost and difficulty to plan and implement. Prasad argues that those issues are not inherent with RCTs, claiming that innovations could be made to bring down their costs and times. Frieden concludes his argument by stating FDA’s standards for evaluating safety and efficacy should be lowered.
Pearl IRBs’ dedicated clinical team includes experts in the fields of medical practice, science, ethics and clinical research. Our vision is to improve the clinical research process. Our clinical research expertise extends beyond RCTs to real world evidence (RWE) based trials as well. Visit our Get Started page for steps on submitting your study to our AAHRPP accredited IRB.