Current and Future Challenges in Trial Design for Pre-Exposure Prophylaxis in HIV Prevention

Abstract

Abstract Success in establishing efficacy of antiretroviral drugs to prevent acquisition of HIV infection has fundamentally changed the trial design considerations for future experimental drugs. Current trials of potential new antiretroviral agents for pre-exposure prophylaxis are using active control designs – where all trial participants receive an active antiretroviral drug. Current trials of other experimental approaches, such as vaccines and monoclonal antibodies, permit use of the proven prevention agent FTC/TDF for all trial participants. In the future, if even more effective prevention methods are approved, active control designs would anticipate very few infection events and not provide statistically robust evidence. A potential alternative is to conduct placebo randomized trials limited to participants for whom current prevention tools are not acceptable.