Patient-Reported Outcome Measure Development and Validation: A Primer for Clinicians.

A comprehensive definition of health includes the assessment of patient experiences of a disease and its treatment. These patient experiences are best captured by standardized patient-reported outcome (PRO) instruments. A PRO is reported directly by the patient (or caregiver) and provides the patient's perspective into how a disease and its treatment impact their lives. PRO instruments are typically standardized, validated questionnaires with items that are scaled and can be combined to represent an underlying health-related construct such as physical, social, and role functioning, psychological well-being, symptoms, pain, and quality of life. Over the past few decades, PROs have become increasingly used in clinical trials as endpoints to better understand treatment benefits from the patient's perspective and in clinical practice to identify unmet needs of patients, health risk surveillance, and monitor outcomes of care. In this paper, we describe the process for developing standardized PRO instruments, from conceptual model development through instrument validation.