Overcoming Organizational Challenges of Integrating Patient-Reported Outcomes in Oncology Clinical Trials
Patients with cancer frequently experience multiple symptoms that may cause significant distress and may impair physical, emotional, and social functioning and health-related quality of life. Drug development in oncology is characterized by a high attrition rate of new compounds, faster development times encouraged by the regulatory process, studies that are often open and single-arm, and emphasis on survival-related endpoints, creating unique challenges for the inclusion of patient reported outcomes (PROs). These challenges to include PRO-related endpoints in oncology research are further exacerbated by downward pressure on budget and resources and also an overly rigorous application of the US Food and Drug Administration's PRO guidance, which can in turn prevent study teams from optimally including PROs in oncology clinical trials. With increasing calls for demonstration of value of new cancer drugs from payers, patients, and their caregivers, study teams should consider the utility of PROs beyond regulatory needs. Optimal implementation of a PRO strategy in oncology research can be achieved by applying the PRO guidance to the greatest extent possible, making use of off-the-shelf PRO measures to capture concepts of interest, discussing plans with the regulatory bodies early in the process, and treating PRO-related endpoints with the same level of rigor as other endpoints.
Gnanasakthy, A., & DeMuro, C. (2015). Overcoming Organizational Challenges of Integrating Patient-Reported Outcomes in Oncology Clinical Trials. Therapeutic Innovation & Regulatory Science, 49(6), 822-830. DOI: 10.1177/2168479015608413