PURPOSE

This article examines the challenges, opportunities, and successes that have occurred in the incorporation of patient-reported outcomes (PROs) in phase III cancer clinical trials.

METHODS

An informal survey of the leadership of US cooperative group PRO investigators identified diverse trials in which PROs had been measured. Exemplary trials were selected for lessons learned and for examination of successful strategies.

RESULTS

We review four challenging trials in depth, illustrating some of the difficulties in integrating PROs within treatment trials, including issues related to missing data, lack of procedures for monitoring patients and ensuring collection of PRO data, and missed opportunities in publication of treatment and PRO outcomes together. Four examples of successful trials are highlighted.

CONCLUSION

As a result of this review, the authors make specific recommendations related to the use of PROs in phase III trials, focusing on issues related to design, choice of PRO instrument and frequency of administration, analysis, and publication strategies.