There is growing consensus among patients, clinicians, payers, regulators, and product developers that incorporating the patient voice is necessary in product development and testing to support product-labeling claims. PROs provide the most direct method for gauging individuals’ symptoms, functional status, participation, health status, and quality of life. PROs play a particularly important role in the approval process for chronic conditions where the goal of treatment is not curative but rather to alleviate symptoms, and improve functional ability and quality of life. While the application of PRO instruments in the regulatory process has developed over the past 6 years with the creation of the Food & Drug Administration (FDA) PRO Guidance and the European Medicines Agency (EMA) paper on use of PROs in oncology studies, there remain methodological and technical challenges associated with the development of a PRO measurement strategy that is based on scientifically rigorous and patient-centered approaches.
This workshop will review advances in the application of PROs in the regulatory approval process, and best practices for engaging patient and clinician partners in the PRO development and selection process. The workshop will also include an interactive component that will prioritize recommendations for implementing PRO measurement strategies that triangulates the patient, clinician, and industry perspectives, and set a research agenda moving forward.