The patient perspective or “patient voice” is critical for the optimization of decisions regarding treatment, as well as the accurate assessment of burden of illness and quality of care. For decades, patient-reported outcome (PRO) measures have facilitated comprehensive assessment and description of how patients feel and function.1 Within the context of drug development, PRO scores often supplement clinical indices that are inadequate for assessing the signs, symptoms, and effects important to patients. In some therapeutic areas, PRO measures are of even greater importance, assessing key indices of efficacy known only to the patient, such as changes in the severity of pain or frequency of gastrointestinal symptoms.
Within the pharmaceutical industry, interest in PROs increased greatly after the US Food and Drug Administration (FDA) released their draft and then final guidance documents describing review criteria for PROs used in labeling claims within the United States.2 The PRO Guidance was developed with input from the FDA's Study Endpoints and Label Development (SEALD) team, and SEALD serves as a consultant to the various reviewing divisions to facilitate and help inform decisions related to PROs in product labeling. In addition to the PRO Guidance, the FDA also has described a qualification process for PRO end points as part of the Drug Development Tools Qualification Guidance, which outlines the process for preapproval of measures before inclusion in a specific clinical trial program.3 The latter guidance was issued, in part, to encourage the collaborative development of clinical assessment measures in a precompetitive space.
