The vision community is discussing with the Food and Drug Administration (FDA) how vision-related patient-reported outcomes (PROs) might be used in improving the labeling of ophthalmic treatments and products. The group — composed of researchers, clinicians, policymakers and representatives from industry and vision associations — attended a one-day symposium sponsored by the National Institutes of Health's National Eye Institute (NEI) and the FDA on October 13, 2009.
The meeting is summarized in an article, Use of Patient-Reported Outcomes in Medical Product Development: A Report from the 2009 NEI/FDA Clinical Trial Endpoints Symposium, published in the December issue of Investigative Ophthalmology & Vision Science (IOVS). The report includes discussion on defining PROs, insights on how the FDA evaluates development and validation of PRO instruments, and information on clinical trial design issues relevant to PROs.
According to the authors, while the FDA has incorporated labeling for PROs in other areas, the issues and challenges pertinent to ophthalmology are just beginning to be understood. Research suggests there is growing evidence that vision-related PROs in clinical trials is important for evaluating medical drugs and devices for product labeling, as well as to expand the understanding of clinical trial outcomes.
It is well known that clinical trials in ophthalmology assess changes in vision using an eye chart. However, in the report, clinicians and researchers point out that an objective measurement of how well a person sees does not adequately describe the total impact a particular treatment may have on an individual's "visual world."
It has not yet been determined whether PROs are suitable as primary endpoints in clinical trials. FDA representatives recognize that PROs can be useful in evaluation of drugs and devices but also acknowledge the challenge in developing appropriate PRO instruments.
A next step for the vision community is to provide data that will verify an instrument's content validity and other measurement properties for the intended clinical trial population. FDA representatives encouraged ophthalmic drug and device manufacturers to schedule meetings with the agency in advance of a phase 3 trial to determine whether instruments are appropriate.
The report reflects the overall position that "PROs in labeling will help clinicians and surgeons make better decisions for treatment of patients based on the outcomes the patients value most for themselves."
Managed by the Association of Research in Vision and Ophthalmology, this meeting is the third in a series of four NEI/FDA symposia to discuss endpoints in ophthalmic clinical trials.
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