The U.S. Food and Drug Administration (FDA) has issued a new piece of guidance with important implications for cancer patients considering whether to take part in a clinical trial. The FDA is advising drug developers and clinical trial sponsors not to give placebosthat is, the inactive "nothing drugs" used for randomizationto patients with serious medical conditions, including some types of cancer.
It is important to note that in many clinical trials for cancer, patients will indeed receive the most up-to-date treatment, or the so-called “standard of care.”
Read MoreThe NCI technically defines a placebo as "an inactive substance or other intervention that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or other intervention are compared to the effects of the placebo."
In some cancer clinical trials that are "randomized," patients who enroll are randomly assigned to receive either a placebo or the new experimental drug being studied. If the clinical trial is "double-blinded," that means that neither the patients or the doctors running the clinical trials know who's in which group. This is an important part of a clinical trial design, because it creates a safeguard against bias, and the possibility that results could be skewed.
"Whenever we do experimentswhether they're in the lab or in a clinical trialwe have to try to make sure that the results we achieve are unbiased," Dr. Alana Welm, an associate professor in the Department of Oncological Sciences at the University of Utah and an Investigator at the Huntsman Cancer Institute, told SurvivorNet in a previous conversation about clinical trial design. "And sometimes, we have natural biases that are not with any intent… but to ensure that the results are trustworthy, we do a process called randomization."
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But when you've been diagnosed with a serious illness such as cancer, the possibility of receiving a "nothing pill" instead of treatment can, in and of itself, become life-threatening. That's why Dr. Sharpless and the FDA are urging the drug development industry to avoid placebos in certain clinical trials, especially when it comes to cancers for which there is another available treatment option.
Instead of Placebos, Doctors Often Use "Standard-of-Care" Treatment in Clinical Trials
The thing about cancer clinical trials is that getting zero treatment, isn't usually the reality of the go-to "placebo arm" anyway. Instead, when patients with life-threatening cancers enroll in randomized clinical trials, the two groups are often broken into the new, experimental drug and a "control" group that receives the "standard-of-care" treatment. Treatments considered standard of care are those that are accepted by experts as the go-to treatment for a specific cancer. Standard-of-care, in other words, is the most commonly used treatment. In many cases, it might involve a combination of chemotherapy, surgery, or radiationbut not always.
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Sometimes, in what the FDA document calls an "add-on" trial, both groups of participants receive a standard-of-care treatment, and then the placebo and/or the experimental drug are given as an "add-on" to that treatment. This is acceptable as well, the FDA saysas long as the patients in the "other" arm continue to receive treatment.
Dr. Welmalong with many other expertstold SurvivorNet that using standard-of-care as the "other" random arm of the clinical trial is already quite common for cancer trials.
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"The most common [placebo] in cancer would be a randomization to a standard of care, which is the approved therapy for your stage of the disease, versus that same therapy plus the experimental therapy."
And while this practice is already commonand it is not likely that a patient with a very serious cancer would receive nothing at all in a clinical trialthe FDA guidance document is an important recognition, and one that will likely guide cancer clinical trial design going forward (though the document is not legally binding).
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