Including Patient-Reported Outcomes and Patient-Reported Resource-Use Questionnaires in Studies

Rationale: More efficient and better informed healthcare systems are expected to have improved knowledge of the impact of interventions on patient outcomes and resources used by patients and providers in specific health conditions. Objectives: To describe trends related to putting patients at the center of healthcare decision making, regulatory trends and best practice recommendations for developing high-quality patient-reported outcomes (PROs), and strategic issues related to including PROs in studies. Materials and Methods: We summarize PRO concepts, definitions, and broadly-accepted scientific standards for developing, assessing, and interpreting PROs. Three conceptual models are presented as examples for assessing PROs in relation to other outcomes. We discuss different perspectives for stakeholders, including regulatory issues pertaining to formal guidance for PRO development and for use in trials. We provide examples of PROs used in studies for assessing health outcomes in oncology and resource-use outcomes in low back pain patients. Results: Psychometric scientists working closely with multi-disciplinary teams and regulatory authorities have greatly improved the science of collecting, assessing, and understanding patient-reported outcomes in clinical trials. A simplified framework is presented for strategic considerations for including PROs in studies, such as the appropriate timing for PRO endpoints. Asking patients about their health status and/or use of resources improves our understanding of how interventions and care processes may impact their lives and their budgets. We provide examples from a back pain trial of patient-reported resource-use questionnaires for medicines taken and other services or products used by patients. Conclusions: Healthcare stakeholders are placing increased emphasis on resource use and the impact of interventions on patients, including effects associated with diagnostic tests. Patient-reported outcomes are being used in clinical practice and in clinical research, supported by formal best-practice guidelines. Radiology has a role as an engaged stakeholder in the design, conduct, and interpretation of patient-based evidence, and in its relevance to health policy implementation.