Need for alternative trial designs and evaluation strategies for therapeutic studies of invasive mycoses

Studies of invasive fungal infections have been and remain difficult to imp lement. Randomized clinical trials of fungal infections are especially slow and expensive to perform because it is difficult to identify eligible pati ents in a timely fashion, to prove the presence of the fungal infection in an unequivocal fashion, and to evaluate outcome in a convincing fashion. Be cause of these challenges, licensing decisions for antifungal agents have t o date depended heavily on historical control comparisons and secondary adv antages of the new agent. Although the availability of newer and potentiall y more effective agents makes these approaches less desirable, the fundamen tal difficulties of trials of invasive fungal infections have not changed. Therefore, there is a need for alternative trial designs and evaluation str ategies for therapeutic studies of invasive mycoses, and this article summa rizes the possible strategies in this area.