Clinical trials of drugs and devices for treating glaucoma, a group of diseases
that damage the optic nerve and can lead to blindness, may move forward more quickly as a result of the recent Glaucoma Clinical Drug Trial Design and Endpoints Symposium. Held recently at NIH, the symposium was jointly hosted by the National Eye Institute and the Food and Drug Administration.
The symposium brought together glaucoma investigators, officials from FDA and NEI and representatives from the pharmaceutical industry to discuss ways to improve clinical trial design and to speed up approval of new therapies
for glaucoma. NEI director Dr. Paul Sieving welcomed the more than 170 attendees and lauded the increasing collaboration between NIH and FDA. “We at NEI are proud to be part of this wider research community that is making progress in helping move therapeutic compounds toward clinical trials,”
A major topic of discussion was how to determine appropriate endpoints and outcome variables for glaucoma clinical trials. An endpoint is a measurable change in the structure or function of ocular tissue as an outcome of the trial.
However, structural changes in the optic nerve often do not coincide with changes in vision, making it difficult to predict when or if an observed change will result in a clinically significant loss of vision.
Advances in visual imaging technologies allow researchers to better detect structural changes in the nerve fiber layer of the retina and the optic nerve head, whereas measurements of a patient’s functional vision may vary from visit to visit. “Structural measurements are more likely than visual function outcomes to be used in the future as primary endpoints,” explained Dr. Wiley Chambers II, acting director of the Division of Anti-Infective and Ophthalmic Products of FDA’s Center for Drug Evaluation and Research. “But they must be highly correlated with the ability to predict functional changes in vision to assure the clinical utility of a new therapy.”
“The open dialogue between clinical researchers and FDA staff provided a unique opportunity for researchers to get a common understanding of the necessary
components of research projects from the FDA’s perspective,” said Dr. Frederick Ferris III, NEI clinical director. “It also provided a collaborative forum for the development of acceptable new outcome variables, including validated quality of life measures, for use in future glaucoma research protocols.”
The symposium, moderated by NEI grantees Dr. Robert N. Weinreb of the University of California, San Diego and Dr. Paul L. Kaufman of the University
of Wisconsin, builds on the success of the Ophthalmic Clinical Trial Design and Endpoints Symposium, the first collaborative meeting between NEI and FDA held in November 2006. That symposium focused on therapies for age-related macular degeneration and diabetic retinopathy.