Abstract

Objective

To discuss issues in the design of a measurement strategy related to the use of patient-reported outcomes (PROs) in support of a labelling claim.

Methods

In association with the release by the US Food and Drug Administration of its draft guidance on the use of PROs to support labeling claims, the Mayo/FDA Patient-Reported Outcomes Consensus Writing Group was formed. This paper, part of a series of manuscripts produced by the Writing Group, focuses on designing a PRO measurement strategy.

Results

Developing a PRO measurement strategy begins with a clear statement about the proposed label claim that will derive from the PRO data. Investigators should identify the relevant domains to measure, develop a conceptual framework, identify alternative approaches for measuring the domains, and synthesize the information to design the measurement strategy.

Conclusion

A position of “reasonable pragmatism” is recommended such that the best available measurement strategy be considered as evidence for labeling.