Patient-Reported Outcomes

Patient-reported outcomes (PROs) are defined as any report that comes directly from a patient. Their use as key outcomes in clinical trials has increased significantly, especially during the last decade. PROs encompass a variety of measurements including health-related quality of life (HRQOL), symptoms, functional status, safety, utilities, and satisfaction ratings. Selection of the PRO in a trial will depend on a variety of factors including the trial’s objectives, study population, disease or condition, as well as the type of treatment or intervention. PROs can be used to inform clinical care and to support drug approval and labeling claims. This chapter will provide an overview of the different types of PROs with examples and their role in healthcare within the context of clinical trials. Summaries of important regulatory documents including the FDA PRO guidance and recommendations (SPIRIT and CONSORT PRO extensions) will also be provided. Considerations when designing clinical trials are described in the last section, highlighting important issues and topics that are unique to PROs. Many methodologic and analytic features of PROs are similar to those of any outcomes used in clinical trials; thus they require the same methodological rigor with special attention to missing data. This chapter is written with a focus on the use of PROs in interventional trials in the United States, although most information can be applied to any context. Information presented in this chapter is relevant to clinicians, researchers, policy makers, regulatory and funding agencies, as well as patients. When used appropriately, PROs can generate high-quality data about the effects of a particular intervention on a patient’s physical, psychological, functional, and symptomatic experience.