News and Events

NEI Statement

Ophthalmic Clinical Trial Design and Endpoints Symposium, November 28-29, 2006

National Eye Institute
National Institutes of Health

More than four million people in the United States have diabetic retinopathy. Nearly two million people over the age of 60 have vision loss from age-related macular degeneration (AMD), and another seven million with AMD are at risk of vision loss. With the development of new drug therapies for wet, or advanced, AMD and possible emergence of new therapies for dry AMD and diabetic retinopathy, possibilities now exist for improving visual acuity in people affected by these diseases. The availability of multiple treatments also offers the opportunity to combine therapies to enhance treatment of retinal diseases that were previously considered untreatable.

This symposium will include a series of invited presentations by representatives of the National Eye Institute (NEI) of the National Institutes of Health, U.S. Food and Drug Administration (FDA), and academia. It also will include a roundtable discussion of outcome variables and clinical trial strategies for evaluating new treatments for AMD, diabetic retinopathy, and other eye diseases. Methods to assess the long-term safety of new therapies after they have been marketed to the public will also be discussed. Presenters and members of the panels will include experts from academia, the NEI, the FDA and the Centers for Medicare and Medicaid Services (CMS).

This symposium will not decide policy, but will provide avenues for discussions of important questions in the topic areas of: visual acuity parameters as outcome measures; endpoints for diabetic retinopathy; study design and endpoints for wet AMD; study design and endpoints for dry AMD; and post-marketing drug surveillance.

Some of the questions to be addressed are:

Are there measures of visual acuity other than the proportion of patients with either a three-line gain or a three-line loss that can be used as primary study outcomes?
How do we assure that statistically significant differences in visual function outcomes are clinically important?
Are there clinical trials of diabetic retinopathy that do not require a three-year follow-up period?
What is the role of anatomic endpoints (e.g. development of proliferative diabetic retinopathy or diabetic macular edema) in diabetes trials?
What primary outcome variables can be used for clinical trials in which multiple therapies are used to treat wet AMD?
How can anatomic endpoints (e.g. development of abnormal blood vessels beneath the retina or of visible retinal lesions) be used in AMD trials?
Does rate or extent of anatomic progression of dry AMD represent a clinically important study outcome?
What is the best mechanism for obtaining and substantiating data on drug-induced complications in patients receiving therapies for retinal diseases after the drugs have been marketed?

Questions that were submitted in advance from those observing the meeting may also be addressed. The deadline for submitting questions for consideration was November 10th.

The discussion at the symposium will be recorded, and a summary report will be published within the next several months. This report will be made available to the public.

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The National Eye Institute (NEI) is part of the National Institutes of Health (NIH) and is the Federal government's lead agency for vision research that leads to sight-saving treatments and plays a key role in reducing visual impairment and blindness. For more information, visit the NEI Website at http://www.nei.nih.gov/.

The National Institutes of Health (NIH) - The Nation's Medical Research Agency - includes 27 Institutes and Centers and is a component of the U. S. Department of Health and Human Services. It is the primary federal agency for conducting and supporting basic, clinical, and translational medical research, and it investigates the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov/.