Skip to Content

Clinical Trials for Macular Degeneration

In order to receive approval from the U.S. Food and Drug Administration (FDA), a new drug or treatment must be proven to be both safe and effective by undergoing a rigorous series of controlled unbiased studies. To prevent bias, neither the patient nor the examiners can know which patients received the actual treatment and which were the untreated (or “control”) subjects.

These are called “double blind” or “double masked” studies and usually yield the most reliable results. The medication is coded and patients are placed at random into either the treatment or control group. When the study is concluded, the code is revealed and it is then possible to determine who received the actual drug and who received the inactive substance, or placebo.

As defined by the U.S. National Institutes of Health, most clinical trials are designated as Phase I, II, or III, based on the questions the study is seeking to answer:

  • In Phase I clinical trials, researchers test a new drug or treatment in a small group of people (20-80) for the first time to evaluate its safety, determine a safe and effective dosage range, and identify possible side effects.
  • In Phase II clinical trials, the study drug or treatment is given to a larger group of people (100-300) to determine if it is effective and to further evaluate its safety.
  • In Phase III studies, the study drug or treatment is given to even larger groups of people (1,000-3,000) to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
  • In Phase IV studies, after the Food and Drug Administration has approved the drug, continuing studies will determine additional information, such as the drug’s risks, side effects, benefits, and optimal use.

To learn more about clinical research on treatments for macular degeneration, you can visit the following resources: