The FDA has published draft guidance for trial investigators and sponsors, recommending that patients with incurable cancers be considered for oncology trials regardless of whether they have received existing alternative treatment options.

Many trials have historically required that participants previously received multiple therapies. But according to the guidance, the FDA believes that incurable cancer patients, if given sufficient information to make an informed decision, should be eligible for participation in cancer trials even if they haven’t tried existing alternative treatment options for their cancer.

“If there is no scientific rationale for excluding these patients, then clinical trial eligibility criteria should be broadened to include these patients, with appropriate informed consent,” the guidance says. “With appropriate informed consent, patients may be eligible for inclusion in trials of investigational drugs, including first-in-human trials, regardless of whether they have received available therapy in the noncurative setting.”

In addition to disclosing appropriate alternative procedures or courses of treatment, if any, that could help the patient, the informed consent should clearly state that other treatment options known to provide benefit exist and should include a discussion of the possible benefits, risks and uncertainties associated with the investigational drug.

The agency also advised sponsors and investigators to consider evaluating patients who have been given available therapies and patients who haven’t in separate cohorts, particularly if a homogenous patient population is needed to evaluate efficacy.

Trials evaluating investigational cancer drugs should require that patients, in general, have received available therapies that offer a potential cure in a substantial proportion of patients, the guidance says.

“However, eligibility criteria in which patients receive an investigational drug(s) in lieu of available therapy is reasonable in the noncurative setting (i.e., there is no potential for cure or for prolonged/near normal survival) when patients have been provided adequate information to make an informed decision on trial participation,” the guidance states.

Comments are due on the draft guideline by Aug. 24.

Read the full guidance here: https://bit.ly/3A5y6ZQ.