Tissue biopsies can be important parts of testing new drugs, and the FDA has now issued a draft guidance with recommendations for when they should be included in clinical trial protocols.
The draft was published Jan. 6 alongside a press release from the FDA. Comments on the proposed guidance must be submitted by March 10 to be considered for the agency’s final ruling on the draft.
Because of the risks biopsies can pose to patients, the FDA recommends only requiring them as part of trial participation if they are needed to determine a patient’s eligibility for the trial or to assess the primary endpoint or a key secondary measure, the agency said in the draft guidance.
If the biopsy is only needed for a non-key secondary endpoint or for exploratory research, the agency recommends making them optional to trial participants.
For example, if a biopsy is needed to tell whether the investigational therapy is working, or is necessary to screen out patients who may be at higher risk for side effects from the treatment, the FDA recommends requiring them in the trial protocol.
Whatever the reasons for the biopsy, the rationale should be clearly spelled out in the trial protocol, according to the draft guidance.
For trials with children, biopsies should only be required if they could offer a direct benefit or pose minimal risk, the guidance says. Single muscle biopsies, for example, are generally safe, while biopsies of large internal organs like the liver or kidney are riskier and should only be done if there is a chance the patient will benefit directly from it.
The guidance was prepared by the FDA's Oncology Center of Excellence, the Office of the Chief Medical Officer, the Center for Drug Evaluation and Research, the Center for Devices and Radiological Health and the Center for Biologics Evaluation and Research in collaboration with the Office for Human Research Protections in the Department of Health and Human Services.