Rockville, Md. – The vision research community is discussing with the Food and Drug Administration (FDA) how to adopt and/or implement safe and effective endpoint measures for assessing glaucoma therapies in U.S. clinical trials. 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 Sept. 24, 2010.
The meeting is summarized in an article, Glaucoma Research Community and FDA Look to the Future, II: NEI/FDA Glaucoma Clinical Trial Design and Endpoints Symposium: Measures of Structural Change and Visual Function, published in the Oct. 2011 issue of Investigative Ophthalmology & Vision Science. This report includes discussion of the most recent meeting, comparing several imaging technologies for assessing structural changes related to glaucoma progression and treatment. According to the authors, comparing imaging technologies for detecting change related to glaucoma is challenging, for several additional reasons:
- There is no gold standard against which a new instrument can be held;
- Instruments vary in the structures that they assess allowing many variances in quality of image, algorithms and methods;
- Change is population dependent influenced by factors such as age, diseases, prior glaucoma-related surgery etc. and
- Instruments and software are constantly improving.
The position of the FDA is that it is the responsibility of the glaucoma research community to establish definitions of glaucoma progression and consensus about structural-functional relationships that characterize early, moderate and late stages of the disease. It has not yet been determined whether patient-reported outcomes (PRO) 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.
However, as reported, the key to using PROs is developing a well-defined and reliable instrument. In using PROs, the FDA wants vision researchers to establish an association with visual endpoint measures that the FDA already considers a valid measure, which in turns predicts a functional change that will be important in a patient’s everyday life.
A next step for the vision research community is to establish these definitions with emphasis on the clinical relevance to the patient. The FDA is also willing to consider drugs or devices for subgroups of patients with glaucoma.
The report reflects the overall position that using PRO-related instruments in “detecting and treating glaucoma progression will reduce the time and cost of clinical trials and the burden of vision loss related to glaucoma.”
Managed by the Association of Research in Vision and Ophthalmology, this meeting is the last in a series of four NEI/FDA symposia to discuss endpoints in ophthalmic clinical trials.
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The ARVO peer-reviewed journal Investigative Ophthalmology & Visual Science (IOVS) publishes results from original hypothesis-based clinical and laboratory research studies, as well as Reviews, Perspectives, and Special Issues. IOVS 2009 Impact Factor ranks No. 4 out of 45 among ophthalmology journals. The journal is online-only and articles are published daily.
The Association for Research in Vision and Ophthalmology (ARVO) is the largest eye and vision research organization in the world. Members include more than 12,500 eye and vision researchers from over 80 countries. ARVO encourages and assists research, training, publication and knowledge-sharing in vision and ophthalmology.