Patient-Reported Outcomes (PROs) in Dental Clinical Trials and Product Development: Introduction to Scientific and Regulatory Considerations

Although typically measured with objective assessments, treatment efficacy and disease progression may also be examined using reports directly from the patient (patient-reported outcomes or PROs). PROs can enhance clinician and researcher understanding of an illness and its treatment in multiple ways, ranging from an assessment of core signs and symptoms to assessment of impairments and impacts across a variety of domains (eg, physical, social, occupational, emotional). Regulated drug and medical device development programs are increasingly using PROs as endpoints to support label claims. PRO instruments are commonly implemented in dental clinical trials. Concepts such as dental pain, tooth sensitivity, eating discomfort, and speech impairments may all be assessed via PROs. Although PROs are commonly implemented in trials, it is not clear that the development, use, and interpretation of PROs are consistent with scientific and regulatory best practices. The goals of this article were to introduce the concept of PROs and to provide an introduction to PRO scientific and regulatory principles. These principles can be used as a blueprint for using PROs in clinical research and for evaluating trials that have implemented PROs. When used appropriately, PROs may be useful in understanding the patient's perspective on illness and interventions and this perspective may be critical in fully evaluating the efficacy of dental treatments.