Target product profile for a test for the early assessment of treatment efficacy in Chagas disease patients: An expert consensus.

Six to 7 million people are estimated to be infected by Trypanosoma cruzi, the parasite causing Chagas disease. However, only a small fraction of patients are properly diagnosed and treated. Benznidazole (BNZ) and nifurtimox (NFX) are the first-line antiparasitic treatments currently available, both with long administration regimens (60 days) that can produce adverse side effects. Despite the fact they are not 100% effective in patients with chronic disease, they are the only drugs currently registered, and the benefits of their administration have been confirmed in several clinical studies. Currently, clinical trials with new compounds, using alternative regimens that aim to maintain efficacy whilst reducing toxicity, are ongoing and could lead to new therapeutic opportunities and/or policy change. In any case, the absence of a test for the early assessment of treatment efficacy, often called a test of cure (ToC), is a major obstacle to Chagas disease control. Accurately monitoring treatment response would undoubtedly improve patient management and support the conduct of clinical trials. Although treatment efficacy and treatment response may be conceptually different, we are using these terms synonymously for the purpose of the current target product profile (TPP).