Organizations Comment on the FDA’s Draft Guidance Expanding Clinical Trial Eligibility

Various medical and patient associations, a patient advocacy organization, and a drug manufacturer expressed support for the U.S. Food and Drug Administration’s (FDA’s) draft guidance expanding eligibility criteria for investigational oncology clinical trials. However, some comments from the groups discouraged the FDA from an approach that separates curative and non-curative settings.

“[The expanded guidelines] will allow hematologists to recommend the best possible treatment path for their patients, which could be participating in a clinical trial rather than being required to first use existing treatments that might be suboptimal for them,” the American Society of Hematology wrote in a comment to the FDA. “This is important because in some hematologic malignancy trials exposing patients to available, but not curable or marginally effective treatments may result in patients being ineligible to participate in clinical trials later.”

Among the updated guidelines is a recommendation that sponsors evaluate patients who have received available therapy and those who have not in two separate cohorts, “particularly if interpretation of efficacy results requires a homogenous patient population.” In their comments, Bristol Myers Squibb argued that in some cases, these groups should be studied together for more statistical power, adding that it may be appropriate “to evaluate subjects with heterogenous prior therapy history together” for safety assessments in a phase I dose escalation study.

In a joint comment, the ASCO Association for Clinical Oncology and the Friends of Cancer Research agreed that the dichotomy between curative and non-curative may be overly reductive. “Reducing potential therapeutic options to curative versus non-curative may neglect other important factors patients consider when seeking a therapy that extend beyond the potential for a ‘cure,’ such as delayed progression or improved quality of life, and suggest that adding these factors would add clarity,” the organizations wrote.

Source:, August 27, 2021.