Use of patient-reported outcomes in medical product development: a report from the 2009 NEI/FDA Clinical Trial Endpoints Symposium.

Abstract

On October 13, 2009, the third in a series of meetings to discuss endpoints in ophthalmic clinical trials was held at the National Institutes of Health (NIH). This event, about patient-reported outcomes (PROs), was an opportunity for the vision community and the U.S. Food and Drug Administration (FDA) to meet and discuss the FDA requirements for adding new endpoints to the evaluation of ophthalmic treatments and products. The meeting was attended by researchers, clinicians, policymakers, and representatives from industry and vision associations. The first two meetings, in 2006 and 2008, were held to discuss endpoints and clinical trial strategies for evaluating new treatments for age-related macular degeneration (AMD) and diabetic retinopathy and glaucoma treatments, respectively.1,2 The objectives of this conference were to provide definitions of PROs, to describe the importance of PROs, to assess what is known and what should be known about PROs in ophthalmology, to provide insights on how the FDA evaluates development and validation of PRO instruments, and to report on clinical trial design issues relevant to PROs. An ancillary issue was the use of PROs in labeling claims and patient-information materials. The meeting was organized by the Association for Research in Vision and Ophthalmology (ARVO) and co-chaired by Rohit Varma, MD, PhD, Frederick Ferris, MD, and Neil Bressler, MD. Representing the FDA were Laurie Burke, RPh, PhD, Wiley Chambers, MD, Malvina Eydelman, MD, Danica Marinac-Dabic, MD, PhD, and Paivi Miskala. All participants and their affiliations are shown in the box on page 6096). Growing evidence indicates the importance of vision-related PROs in clinical trials for evaluating medical drugs and devices, not only for medical product labeling, but also to expand the understanding of clinical trial outcomes.3–7 Although the FDA has incorporated labeling for PROs in areas outside of ophthalmology, the issues and challenges relevant to ophthalmology are just beginning to be understood. Most clinical trials in ophthalmology use visual acuity measured by an eye chart to assess changes in vision related to experimental conditions. However, as many clinicians and researchers are aware, objective measurement of visual acuity (and/or visual field) may not adequately describe the total impact of a treatment on a patient's visual world.