Capturing the patient perspective: patient-reported outcomes as clinical trial endpoints.

Just as clinical trial design and rigor have evolved with improvements in methods and processes, so too have methods for capturing patient data in clinical trials. Substantial evidence suggests that standard physician reporting of symptoms for which we lack objective diagnostics (e.g., pain) is often discordant with patient self-report. Current reporting using the National Cancer Institute Common Terminology Criteria for Adverse Events (CTC[AE]) for symptom capture relies on a filtering system, from patient to physician to medical record to medical record abstraction to data entry, with each step requiring interpretation and the possibility of error. In contrast, patient-reported outcomes (PROs) eliminate the filter and rely on direct report. Furthermore, the lack of validation and training in use of the CTC(AE) creates an inadequate data capture system. Inadequacies might be observed as underreporting or overreporting symptom prevalence and severity compared with patient self-report. Inaccuracies in symptom reporting can lead to missing important prognostic information, lack of understanding of patient adherence with therapies, and lack of information for patient decision making. They can also lead to opportunities lost in terms of labeling claims and comparative effectiveness analyses. New developments in patient-reported outcome (PRO) reporting, including the PRO-CTC(AE) and models for incorporation of PROs in clinical trials, might facilitate routine PRO reporting complementary to CTC(AE) in clinical trials. In addition, the cadre of validated PRO instruments already in existence allows for more in-depth, hypothesis-driven evaluations. For standard toxicity reporting, the time has come for mandatory routine PRO symptom reporting complementary to the CTC(AE).