The right prescription for biomedical innovation (The Hill)

THE HILL | If you were a patient suffering from a disease, and you read about a treatment option for your illness, you would probably be eager to learn more. Imagine that the drug, device or other intervention had been on the market for several years, and studies showed that it was especially effective in some patients – but in others, it was useless, or even harmful. You’d want to know which group you were in, to make a fully informed decision about whether to use the drug or device, or not.

Welcome to the world of real-world evidence in health care – information derived from the practical or actual experience of patients.  This world can look very different from the idealized settings of randomized, controlled clinical trials (RCTs) that help regulators determine whether a drug or device can be allowed onto the market.

RCTs are considered the “gold standard” of scientific evidence. By assigning patients randomly either to a “study arm” in which they get the new intervention, or a “control arm” in which they get a placebo or the standard treatment, researchers can isolate those results produced by the new drug.  But these trials are typically filled with relatively small groups of carefully preselected patients, and usually are not reflective of the far broader population of people who may eventually use the new drug, or device, or the practices of the far larger circle of physicians who will prescribe it.

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