Table 1.

FDA reviewer approach to submitted patient‐reported outcomes data

FDA review questions	Considerations
Which instruments are being used?	Are the instruments “fit for purpose” and well defined and reliable within the context of the clinical trial? Is there evidence of adequate measurement characteristics for the context of use?
Were PRO endpoints in the statistical hierarchy?	Were PRO objectives and endpoints stated clearly within the protocol and statistical analysis plan? Were endpoints clearly constructed based on the research objective?
How much data is missing?	Is data missingness due to technical limitations? Trial conduct? High attrition due to toxicity or disease progression?
Is the assessment timing reasonable given the drug(s) being tested?	Are PROs being assessed at times when symptomatic adverse event or physical function deterioration are most likely to occur in the treatment or disease course?
Can conclusions be made on the strength of results?	Are the results robust and clinically interpretable in order to inform the FDA benefit–risk determination?
What is the best way to share PRO results with the public?	Is the product labeling the most appropriate place for communication of complex PRO results?

Abbreviations: FDA, Food and Drug Administration; PRO, patient‐reported outcome.