USA – FDA Finalizes Guidance on Placebos and Blinding for Cancer Trials

The US Food and Drug Administration (FDA) on Wednesday finalized guidance recommending that drugmakers only use placebos in cancer clinical trials in select circumstances due to ethical concerns that would arise when alternative therapies are available.

According to guidance, placebos should only be considered in randomized controlled trials to treat oncologic disease or hematologic malignancy “when surveillance is standard of care” or in studies with “certain trial design features (e.g., when the trial uses an add-on design).”

“A placebo-controlled study design may be useful or preferred in maintenance therapy, in add-on trial designs, in trials of adjuvant therapies (for which standard of care is surveillance), and for indications where no treatment is available,” FDA writes.

The guidance provides a set of considerations for sponsors weighing a placebo-controlled trial design, noting that sponsors “should provide the rationale for the trial design” and a description of their plan for blinding and unblinding patients during the trial…