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

TL;DR

This paper outlines a target product profile to guide the development of new pediatric drug formulations for treating Chagas disease in children.

Contribution

The paper introduces a consensus-based target product profile for new pediatric drugs to treat T. cruzi infection in children.

Findings

A target product profile with 15 attributes was developed for pediatric formulations of new drugs for Chagas disease.

The profile emphasizes age-appropriate formulations, palatability, and ease of administration for children.

The target population includes newborns to adolescents up to 18 years with acute, chronic, and congenital T. cruzi infections.

Abstract

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.

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.

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.

The TPP provides guidance for essential and ideal characteristics of formulations of new drugs for the treatment of children with T. cruzi infection.

Chagas disease is a neglected tropical infection with serious health consequences. Patients often acquire the infection in childhood from a mother that is living with the infection, while still in the womb. Two drugs appeared during the 60s and 70s that proved to be very effective, particularly during childhood. Since that time, new drugs have been tested but none appeared to have the same efficacy. A sixty-day treatment, alongside different barriers to accessing early diagnosis, leads to very low treatment rates, in particular for children. This is a problem because treatment is most effective in the early stage of infection. New drugs need to be developed to prevent Chagas disease causing serious and life-threatening health issues later in life. Developing pharmaceuticals for children can be challenging, with many important issues to consider, including how the drug is administered, length of treatment time, and the taste. We describe a target product profile produced by a panel of experts, which provides guidance on the important attributes of formulations of potential new drugs for treating Chagas disease in infants and children.

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