Clinical trial design for invasive aspergillosis: Time to cast a different mold

Abstract

Despite the availability of active antifungal agents, invasive aspergillosis (IA) continues to be a significant cause of morbidity, mortality, and resource utilization among the various patient populations at risk. New and more effective therapies are required. Like with other drugs, the regulatory approval of new mold-active agents is based on the results of randomized clinical trials (RCT) which are particularly difficult to conduct and usually take several years to complete at very high costs. The limitations of conventional RCTs for aspergillus-active agents are discussed and novel trial methodologies that are likely to expedite approval of therapies active against aspergillosis are proposed.