Many patients have concerns about enrolling in clinical trials that involve placebo dosing, assuming that they may not receive treatment. What should be explained to them regarding placebo use in controlled trials? — Name withheld on request
Patients often have fears about enrolling in a clinical trial and potentially not receiving treatment. They need to be reassured that they will receive standard-of-care treatment, at the least. In addition, explaining the following points can help patients understand how placebos are used in clinical trials.
A placebo is an agent that has no effect on the disease or patient (sometimes called a sugar pill), and are used in certain phase 3 trials only. Patients must be offered at least the current standard-of-care treatment available for their condition. More importantly, their health care provider would never advocate for a patient to receive a placebo if a treatment is available.
Phase 3 trials often compare standard therapy with or without an investigational agent vs the standard therapy alone. These trials are either open label — both clinicians and patients know the treatment being administered — or double blind — neither clinicians nor patients know which treatment is being administered. Placebos are not used in open-label trials.
Patients are sometimes disappointed if they are not enrolled into the investigational arm. Often they assume that the investigational arm is going to demonstrate superior response rates than standard of care. This is not necessarily true. The intent of the phase 3 clinical trial is to determine which treatment is more effective in the most patients. Therefore, investigational arm is not always better, as some patients respond as well or better when treated with the standard therapy. — Kara Yannotti, MMH, BSN, CCRP