This guidance provides recommendations for the development of antiretroviral drugs regulated within the Center for Drug Evaluation and Research at the Food and Drug Administration (FDA) for the treatment of human immunodeficiency virus-1 (HIV-1 or HIV) infection. Specifically, this guidance addresses the FDA’s current thinking regarding the overall development program and clinical trial designs for antiretroviral drugs to support an indication for the treatment of HIV-1 infection. The organization of the guidance parallels the development plan for a particular drug or biologic.
This guidance does not address the use of antiretroviral drugs for preventing transmission of HIV-1 infection. Also, this guidance does not address the development of therapeutics without antiviral mechanisms, intended to mitigate or reverse clinical or pathophysiological outcomes of immunologic suppression caused by HIV-1 infection.
Additionally, this guidance does not contain discussion of the general issues of statistical analysis or clinical trial design. Those topics are addressed in the ICH guidances for industry E9 Statistical Principles for Clinical Trials and E10 Choice of Control Group and Related Issues in Clinical Trials, respectively. This guidance also does not contain details regarding nonclinical safety and toxicology studies that should be conducted in standard animal models as described in the guidance for industry Nonclinical Safety Evaluation of Drug or Biologic Combinations.