Developing a valid patient-reported outcome measure (original) (raw)

2011, Clinical pharmacology and therapeutics

Many aspects of patients' experiences with illness, medication, and health care are best captured from patient-reported outcomes (PROs). In this article, we describe the process for constructing quality PRO instruments, from conceptual model development through instrument validation. We also discuss PROs as clinical trial end points and the potential of PRO data for aiding clinicians and patients in choosing from among multiple therapeutic options. Finally, we provide an overview of some existing PRO instruments. WHAT ARE PATIENT-REPORTED OUTCOMES? A PRO is "any report of the status of a patient's health condition that comes directly from the patient, without interpretation of the patient's response by a clinician or anyone else." 1 PROs provide patients' perspectives on their well-being, functioning, symptoms, and experiences with treatment. The use of PROs has become increasingly prevalent in clinical research, reflecting the growing recognition that patient quality of life is an important outcome. 2 The growth in the use of PROs has also been fueled by the development of combination and targeted therapies that produce a variety of side effects best captured by direct patient input. 3 For concepts such as pain, fatigue, emotional distress, satisfaction with care, and impact of symptoms on participation in meaningful activities, patients are the best source of information. Among patients with identical clinical presentations (e.g., hemoglobin levels or tumor characteristics) there may be wide variations in symptoms, side effects, and functional status.