Core Patient-Reported Outcomes in Cancer Clinical Trials

On 9 June 2021 the FDA published the draft guidance. Comments should be submitted by 9 August 2021.

This guidance provides recommendations to sponsors for collection of a core set of patient-reported clinical outcomes in cancer clinical trials and related considerations for instrument selection and trial design. Although this guidance focuses on patient-reported outcome (PRO) measures, some of these recommendations may be relevant to other clinical outcome assessments (i.e., clinician-reported outcome, observer-reported outcome, performance outcome) in cancer clinical trials. Recommendations supplement previous guidance on use of PRO measures in clinical trials by providing additional considerations specific to the cancer clinical trial setting. Guidance specific to PRO endpoints and details of analytic methods are not comprehensively covered. FDA does not endorse any specific PRO measure and examples within this document are illustrative and should not be construed as endorsements.

This guidance is specific to registration trials for anti-cancer therapies intended to demonstrate an effect on survival, tumor response, or delay in the progression of a malignancy. Demonstration of a clinically meaningful improvement in patient-reported symptoms or functional impacts alone (i.e., in the absence of evidence of anti-tumor activity) would be more applicable to supportive care drugs and is outside the scope of this guidance. Refer to the guidance for industry Patient-Reported Outcome Measures: Use in Medical Product Development to Support Labeling Claims for situations where the PRO endpoint will be used as the primary evidence of effectiveness. PRO measurement may not be feasible in all cancer trial populations (e.g., in patients with significant cognitive impairment).

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