Neglected Diseases: Drug Development for Chagas Disease as an Example

Abstract

Neglected diseases (NDs), also known as neglected tropical diseases, are a group of chronic diseases that disproportionately affect the poorest populations in the world. Largely ignored, children comprise a significant proportion of the voiceless victims of NDs. Unfortunately, the limited efforts to identify new, safe, and effective treatments for NDs, and the even larger void in drug development efforts for children, make young patients with such disorders a highly neglected population. Lack of specific pediatric drug studies makes the actual rate of adverse drug reactions and the dose–response relationships largely unknown, leading to significant guesswork when pediatric doses and monitoring schedules are planned. In this chapter, the authors have discussed the principal challenges, and some potential solutions, in the pharmacotherapy of pediatric NDs. Chagas disease has been used as an example of a predominantly pediatric disease with chronic complications that may only surface later in adulthood.