Today, the U.S. Food and Drug Administration issued a draft guidance with the Office for Human Research Protections that, when finalized, will provide recommendations for including tissue biopsies as part of clinical trials.

The draft guidance is intended, when finalized, to assist industry, clinical investigators, institutions, and institutional review boards in understanding considerations for tissue biopsies in adults and children as part of clinical trials that evaluate investigational medical products and/or that are conducted or supported by the U.S. Department of Health and Human Services (HHS).

“Tissue biopsies in clinical trials are often needed to determine eligibility or to understand the effect of the medical product being studied in the trial,” said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “This new draft guidance builds on the agency’s ongoing efforts to enhance clinical trials by providing recommendations to improve participant safety and further clinical research.”

This draft guidance focuses on considerations for when a tissue biopsy, which involves taking tissue from a trial participant, is included in a clinical trial protocol. All biopsy procedures have a certain amount of risk, but biopsies can be necessary for obtaining information important to the trial, such as to determine clinical trial participant eligibility, to ensure enrolled trial participants have the intended target medical condition the trial is evaluating, or to evaluate primary or key secondary endpoints.

In a clinical trial protocol, a tissue biopsy may be described as a required biopsy that is a condition of trial participation, or an optional biopsy that is not a condition of participation. The draft guidance discusses considerations for determining if a tissue biopsy should be required or optional for both adults and children participating in clinical trials. The draft guidance also addresses risk and benefit considerations specific to tissue biopsies conducted in clinical trials involving children. Additionally, the draft guidance reinforces the importance of obtaining informed consent from participants, including parental or guardian permission for children and, when appropriate, assent from children. The draft guidance also has recommendations for information to include about biopsies as part of informed consent.

The draft guidance, “Considerations for Including Tissue Biopsies in Clinical Trials: Guidance for Industry, Investigators, Institutions, and IRBs,” was developed by the FDA’s Oncology Center of Excellence, Office of the Chief Medical Officer, Center for Drug Evaluation and Research, Center for Devices and Radiological Health, and Center for Biologics Evaluation and Research, in collaboration with the HHS Office for Human Research Protections. Comments on the draft guidance must be submitted within 60 days after publication in the Federal Register to Regulations.gov to ensure the agency considers them.