Clinician-rated Outcome Assessments in Orphan Disease Research
Within the world of clinical research, clinical outcome assessments (COAs) can measure patients’ health status and define end points that can be interpreted to indicate the benefits of a medical intervention on how patients feel, function, or survive. COAs can take many forms—clinician-rated, patient-rated, interview-based, functional performance, observer-rated, and still others—and individual COAs can be used in different ways in different settings. This article focuses on clinician-rated outcome assessments (ClinRos), some of which may be novel; others may be well-established and familiar to individuals practicing in clinics and research settings alike. When the right ClinRo is selected and patient data is collected in a consistent manner over time, it can enable key insights into a patient’s state of mind, behavior, cognitions, visual acuity, limb movement, gait, and much more, providing clinical context for measures reflecting pharmacological attributes of a novel therapy.