FDA Finalizes Two Guidances on HIV Drug Development
The US Food and Drug Administration (FDA) on Tuesday finalized two guidance documents to support the development of antiretroviral drug products for the treatment of human immunodeficiency virus (HIV) infection in pediatric patients and to aid the development of systemic drugs for the prevention of HIV-1 infection.The guidance from FDA comes as the agency has approved 29 drugs to treat HIV infection. FDA Commissioner Scott Gottlieb said in a statement that the guidance is part of the agency’s work to address vulnerable or at-risk populations.
“Although aimed at different populations affected by HIV, the end goal of these guidances is the same. We must continue the work of ensuring the safety and effectiveness of drugs used in HIV treatment and prevention so that we can attain the healthier future and set as a goal the eradication of this disease," Gottlieb said.
The four-page guidance on pediatric HIV treatments, finalizing the draft from last May, outlines seven major considerations for sponsors (up from five in the draft). FDA advises sponsors developing antiretroviral (ARV) drugs to include adolescents in the initial phase 3 (efficacy) clinical trials along with adults, or to conduct a separate adolescent study in parallel with the adult phase 3 clinical trials.
“FDA encourages sponsors to have early discussions with the WHO, nongovernmental organizations, the FDA, and others regarding pediatric plans to facilitate the development of drug products to meet the needs of pediatric patients (e.g., selection of formulation, strength and dosage of a drug product),” the guidance says.
The other seven-page guidance, finalizing a draft from last June, offers recommendations for investigational drug products for further development as pre-exposure prophylaxis (PrEP) and which can include the following: “(1) an oral drug product approved for the treatment of HIV-1 infection that is subsequently developed as oral PrEP, (2) an oral drug product approved for the treatment of HIV-1 infection that is reformulated as a long-acting drug product or other delivery system (e.g., injectable, implantable device) for PrEP, or (3) a new investigational systemic drug product that is developed for treatment and/or prevention of HIV-1 infection.”
The guidance is divided into sections on nonclinical considerations, clinical pharmacology and clinical considerations for long-acting systemic drugs, dose selection and systemic drug characteristics that affect end-user acceptability. The guidance also discusses key considerations for phase 3 clinical trial design features, such as enrollment criteria, trial design, efficacy endpoint and other trial features and special considerations for pregnant women and adolescents.
Pediatric HIV Infection: Drug Product Development for Treatment: Guidance for Industry
Human Immunodeficiency Virus-1 Infection: Developing Systemic Drug Products for Pre-Exposure Prophylaxis: Guidance for Industry