FDA Approval of Antiretroviral Agents: An Evolving Paradigm

Abstract

The approach to traditional approval of antiretroviral agents in the United States has recently evolved. As in the past, companies can provide confirmatory clinical endpoint data following accelerated approval; alternatively, they can base their claim of efficacy primarily on data demonstrating a sustained reduction in plasma HIV-1 RNA. This change was fueled by issues associated with conducting long-term clinical endpoint trials given the rapidly changing treatment strategies for HIV and the availability of potent combination regimens. The Food and Drug Administration (FDA) hopes that this new approach will help direct more resources to ambitious, collaborative clinical trials. The rapid evolution of traditional approval also illustrates the importance of close collaboration between industry, academia, and the FDA. This field would not have moved forward so rapidly without consensus initiatives such as the Surrogate Marker Collaborative Group, which provided evidence suggesting that a virologic response, in terms of changes in HIV-1 RNA, is a strong predictor of clinical benefit.