ABSTRACT

One of the most common study designs currently used to evaluate new treatments for patients infected with the human immunodeficiency virus (HIV) is the non-inferiority (NI) clinical trial. This chapter provides an overview of the basic principles for designing, conducting, and analyzing HIV NI trials. Despite the availability of highly active antiretroviral therapy (HAART), there remains a need to develop safe antiretroviral (ARV) treatments that can maintain virological suppression in a broad range of HIV-infected patients from diverse clinical settings. In children infected with HIV, administering co-trimoxazole prophylactically before ARV therapy can reduce morbidity. The general strategy for establishing non-inferiority of an experimental therapy to a standard therapy for HIV is to demonstrate that the difference in efficacy between the two treatments is less than some prespecified margin of non-inferiority. A common inferential mistake is to assume that failure to observe a statistically significant difference in a conventional superiority trial is evidence that the two treatments being compared are similar.