FDA encourages including incurable cancer patients in trials, regardless of prior therapies
The FDA on Thursday called to include those with incurable cancers (when there is no potential for cure or for prolonged/near normal survival) in appropriate clinical trials, regardless of whether they have received existing alternative treatments.
Historically, many cancer clinical trials have required that participating patients previously received multiple therapies, according to Richard Pazdur, director of the FDA’s Oncology Center of Excellence.
But the new, 2-page draft guidance recommends expanding the eligibility criteria “such that, with appropriate informed consent, patients may be eligible for inclusion in trials of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 non-curative setting.”
FDA also said the informed consent documents that patients sign before participating in the trial should clearly state whether there are other treatment options known to confer clinical benefit, and should include a discussion of possible benefits, risks, and uncertainties.
Vincent Rajkumar, cancer doctor and professor at the Mayo Clinic, told Endpoints News that what the guidance is describing already occurs in practice, and that he fully supports it.
“We risk losing a lot of potentially active drugs because we are testing them too late after exhausting all other treatments by which time it’s pretty hard for any drug to work,” he said. “So trying new drugs early in disease course with informed consent is something I fully support.”
The guidance also calls on sponsors to evaluate patients who have received available therapy(ies) and those who haven’t in separate cohorts, “particularly if interpretation of efficacy results requires a homogenous patient population.”
Generally, FDA said, eligibility criteria should require that patients have received available therapy(ies) that could cure a substantial proportion of patients, such as with available treatment options for pediatric acute lymphoblastic leukemia, classic Hodgkin lymphoma, or testicular cancer, in clinical trials evaluating investigational cancer drugs.
“Alternatively, such available therapy should be administered to all patients in the trial, where the investigational drug is added to such therapy (i.e., add-on trial),” FDA said. “However, eligibility criteria in which patients receive an investigational drug(s) in lieu of available therapy is reasonable in the non-curative setting (i.e., when 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.”