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Arch Ophthalmol. 2004;122:1043-1044.

	Since this article does not have an abstract, we have provided the first 150 words of the full text and any section headings.

Prospective, randomized, and controlled clinical trials have been in use for nearly three quarters of a century¹ and are the cornerstone of rational drug development.^2-3 In fact, the Food and Drug Administration and other health authorities generally require that at least 2 prospective, randomized clinical trials demonstrate therapeutic efficacy prior to granting approval. Such pivotal trials are time consuming and expensive. Recent estimates suggest that pivotal, or phase 3, trials routinely recruit hundreds to thousands of patients, typically take 2 to 4 years to complete, and commonly cost $30 to $50 million. Phase 3 trials also expose large numbers of patients to the risk of drug-related adverse effects. For these reasons, pharmaceutical companies, the Food and Drug Administration, and international health authorities have adopted a staged approach to new drug testing in humans.

Phase 1 trials are the first studies to be performed in humans and are designed to investigate . . . [Full Text of this Article]

Emmett T. Cunningham, Jr, MD, PhD, MPH; Loni da Silva, MS, RAC; Evelyn M. Harrison, MBA; Marlene W. Modi, PhD; Denis O'Shaughnessy, PhD; David R. Guyer, MD