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Core Patient-Reported Outcomes in Cancer Clinical Trials Draft Guidance for Industry June 2021


Not for implementation. Contains non-binding recommendations.

This guidance is being distributed for comment purposes only.

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Although you can comment on any guidance at any time (see 21 CFR 10.115(g)(5)), to ensure that the FDA considers your comment on a draft guidance before it begins work on the final version of the guidance, submit either online or written comments on the draft guidance before the close date.

If unable to submit comments online, please mail written comments to:

Dockets Management
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852

All written comments should be identified with this document's docket number: FDA-2020-D-2303

Docket Number:
Issued by:
Guidance Issuing Office
Oncology Center of Excellence
Center for Biologics Evaluation and Research
Center for Drug Evaluation and Research

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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