BSP Parasites Online 2021
Schedule : Back to Dr Nathalie Strub-Wourgraft

The value of a data platform for drug development and for regulatory agencies: the case of Chagas disease

Mon21  Jun12:45pm(40 mins)
Conference room 2
Keynote Speaker:
Dr Nathalie Strub-Wourgraft


NTDs can also be called diseases of poverty … affecting poor populations often living in remote aeras in LMICs. Typically, NTDs have been neglected by research and even if several initiatives, such as DNDi, have emerged in the past 20 years, they are characterised by many gaps including tremendous knowledge ones. But NTDs affect over a billion of individuals, result in increased poverty, and significant morbi-mortality: most still require affordable and field-adapted tools to control eliminate or eradicate them by 2030. Indeed, twenty of them have been prioritised by WHO and a detailed roadmap, highlighting the gaps & needs per disease do show that many are still missing proper test and treat strategies. Any treatment will need to be developed according to a plan that will secure its approval by endemic regulatory authorities and acceptance by policy makers for recommendation as a public health measure. As of now, in contrast with other diseases, they are only a very limited amount of guidance from regulators to support developers in building solid R&D strategic plans. Chagas disease, identified over 100 years ago, is a complex NTDs, caused by a parasite transmitted by a triatome. It starts by an acute, often poorly symptomatic disease, and continues by a chronic phase, silent that will – or not, evolve in a final cardiac or digestive symptomatic one, the cardiac complication being until now fatal. For now, most therapeutic approaches tackle the chronic indeterminate phase of the disease, hoping that the effect on the circulating parasite observed after one or 2 years is predictive of cure. But still very little is known and today, we do not have tools to either prevent or definitely cure the chronic phase of Chagas preventing disease progression. There has been a wealth of clinical research conducted in the past decade, aiming at identifying treatments for this indeterminate phase of CD. Sometimes in adults, children, for prevention … A recent study (Maguire B et al. Submitted PlosNTD) shows the diversity and heterogeneity of studies, populations, definition of endpoints, outcomes but also the often-insufficient sample size … so that signal detection from each study remains low. Pooling all individual data in a systematic and consistent way would allow to increase the ability to better analyse the data and respond to critical questions, such as “are there predictors of worse outcome, what is the best predictor of response, are there sub-types (lineages of T. cruzi) related progression and/or treatment effects, among others “ … We will discuss the opportunity of setting up this data-sharing platform for Chagas disease.

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