Target product profile for the development of pediatric formulations of new drugs for the treatment of children with T. cruzi infection

BACKGROUND: Infection with the protozoan Trypanosoma cruzi leads to Chagas disease, a neglected tropical disease with potentially serious complications. Infection commonly takes place in childhood which can lead, without timely diagnosis and accurate treatment, to severe cardiac events and early mortality. For many years there were no pediatric formulations. Currently, there are two very effective treatments. Nevertheless, new drugs and shorter treatments are desirable. Therefore, the aim of this report is to provide a target product profile (TPP) to guide the development of pediatric formulations of new drugs for the treatment of children with Chagas disease. METHODS: A review of the relevant literature, focusing on the therapeutic options available for pediatric Chagas disease, was undertaken prior to setting up a TPP working group of recognized leaders in the field. After several drafts, the TPP was established through a consensus agreement by the group. For each attribute, essential and ideal requirements were defined, which specified the required TPP performance and use characteristics. RESULTS: The TPP for the development of formulations of new drugs for treatment of children with T. cruzi infection includes 15 different attributes. The target population was defined as newborns to adolescents up to 18 years. The essential indication for the target product would cover acute/congenital and chronic T. cruzi infection; the ideal indication would include chronic indeterminate, congenital and/or acute vector-borne T. cruzi infection; people who are immunosuppressed, and pregnant and breastfeeding women. An age-appropriate drug formulation, acceptable palatability, dose, and ease of administration were considered to be particularly important for children. CONCLUSIONS: The TPP provides guidance for essential and ideal characteristics of formulations of new drugs for the treatment of children with T. cruzi infection.