Integration of Patient Reported Outcomes in Drug Development in Genitourinary Cancers

Patient reported outcomes (PROs) are increasingly utilized in cancer drug development, and are of particular importance in genitourinary cancers due to symptom burden, multiple treatment options with similar efficacy, and often prolonged duration of disease. Here we review current data and perspectives related to use of PROs in drug development for genitourinary cancers, including insights on the regulatory process for drug approval.

The FDA is committed to incorporating PRO data into the regulatory process for development and approval of new cancer drugs, but challenges exist due to lack of standardization of PRO instrument choice and analytic approach, missing data, and difficulty isolating treatment effect from disease-related effects. We review guidance for standardization of PRO methodology that is nonetheless tailored to disease state and anticipated effects of treatment. PRO and efficacy data should be simultaneously analyzed and reported for best clinical practice. Multiple disease-specific PRO instruments exist for genitourinary cancers. While clinicians, researchers, and regulatory bodies alike recognize the importance of PROs in cancer drug development, challenges remain regarding implementation of best practices.