ABSTRACT
The Cures Act specifically calls on FDA to assist sponsors in incorporating complex adaptive and other novel trial designs into proposed clinical protocols and applications for new drugs and biological products to facilitate more efficient product development. One of the key innovations in drug development is to seek consolidation of the phases and to rapidly expand accrual with “seamless” trial designs. Consider a two-stage winner design with two dose groups of an experimental treatment and one control group. However, the larger the correlation, the more the multiplicity, leading to more correction for the critical value. When designing the study protocol, one can plan for the interim analysis time and assume a correlation value based on information from other studies. A trial to treat age-related macular degeneration is conducted using the seamless Phase II/III select-the-winner design. At the first phase of the trial, patients are randomized to the standard of care active control, doses 1.0 or 1.5 mg of the test treatment.
