USA – FDA Recommends Use of Placebo Only in Certain Oncology Trials

Given the ethical challenges of using a placebo in randomized controlled clinical trials for therapies to treat hematologic malignancy and oncologic disease, the US Food and Drug Administration (FDA) recommends that a sponsor use a placebo-controlled design only in select circumstances, according to new draft guidance released Thursday.

Such circumstances include “where surveillance is standard of care,” or with specific trial “design features (e.g. if the trial uses an add-on design, when the endpoint intended to support a labeling claim has a high degree of subjectivity, such as patient reported outcomes).”

And when considering the use of a placebo control, FDA says sponsors should provide a rationale for the trial design and a detailed description in the protocol and statistical analysis plan of the proposal for blinding and unblinding…