Jumping Ahead to Phase III trial

Phase III TrialA new ophthalmic drug was going to enter a phase II dose-ranging trial in order to determine the dose to use in phase III trials. IDDI proposed to jump ahead and test a dose-effect hypothesis in two pivotal phase III trials, using appropriate statistical methodology to adjust for multiple testing (simulations showed a step-up procedure to be slightly preferable to a closed-test procedure). Although the two trials were far larger than if a single dose had been used, this bold approach resulted in a gain of at least one year of clinical development. The drug has received FDA approval (Food and Drug Administration).