Nobody in a clinical trial wants to be given a placebo, whatever that is…

“Placebo” is often used in a negative way. But that’s not necessarily true. Placebos are sometimes used in clinical trials. A placebo is a biologically neutral substance packaged to look like the real drug. Although often referred to as a sugar pill, many placebos are not even pills. They are simply inactive compounds packaged to look, feel, and appear like an active drug. They may be an injection, IV infusion, capsule, a cream, an inhaler, a skin patch, or take any other form that is used by an active drug.

Placebos are used when the purpose of the research is to compare different approaches to treatment. The placebo helps ensure that the study is double blinded which means neither the doctor nor the patient knows which treatment is being administered. Many cancer patients are understandably concerned that they might get a sugar pill instead of a real treatment. This is often the first question the physician researcher hears when a clinical trial is brought up as an option, “Will I get the real thing or just a sugar pill?”

When placebos are used

The fundamental principle that defines when placebos are appropriate is rather simple: if you have cancer, and a treatment that works is available, a placebo cannot be used instead. Period‑‑end of story. Research is not and cannot be about withholding effective treatments from patients with cancer. A placebo can be used in two research situations:

1          There is no effective treatment and the standard treatment would be observation or perhaps supportive treatment (treatments designed to lessen symptoms but not treat the disease).

2          There is an effective treatment and this treatment is being given to all patients in the study. The placebo is being added to the known effective treatment to allow a comparison between the standard treatment and a combination of the standard treatment plus a new drug of unknown effectiveness.

The Placebo Effect (this is kind of weird)

The most mysterious benefit of having a placebo in a study is that placebos actually do work! Now you may think we are completely losing our minds (always a possibility), but it is true! The degree to which placebos work varies considerably depending on what disease is being studied, but it stands to reason that if we are going to devise a fancy, expensive, patented new drug with an unpronounceable name, it should produce results better than a mere sugar pill. According to the American Cancer Society, placebos have an effect on about one in three patients. Often the result is a reported improvement in a symptom or in quality of life. Sometimes it could actually be a new side effect. The mechanisms through which placebos work may represent, to some extent, the importance of the mind-body connection.

A placebo can reinforce patients’ belief that they will get better and so they actually do. In general, placebo effects are seen as symptoms or things people feel. The actual shrinkage of cancer tumors would be extraordinary. Nevertheless, many studies of new cancer drugs look beyond tumor shrinkage and length of life following a cancer diagnosis. They examine quality of life, symptoms of cancer, and side effects of the drug. Placebos allow us to see the true picture of what a new drug can offer beyond what you could get from a sugar pill.

There’s more to learn about the placebo. Watch for the next chapter!

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(c) 2012 Tom Beer and Larry Axmaker