Human African trypanosomiasis: update of the methodological framework for clinical trials
Report of the first meeting of the development of new tools subgroup, Geneva, 24 September 2014
Overview
Researchers involved in clinical trials for the evaluation of new treatment modalities for human African trypanosomiasis (HAT), also known as sleeping sickness, face a number of challenges that are rarely, if ever, encountered in this combination in other diseases. Many of these challenges are related to the fact that both the disease and the populations it affects are neglected and that, prior to 2004, there was no background of generally accepted – and ubiquitously feasible – diagnostic and treatment standards for the planning and conduct of clinical evaluation of new treatment modalities for a disease.
In 2004, the World Health Organization (WHO) organized an expert consultation to establish a methodological framework for clinical trials on HAT in order to facilitate collaboration among research actors and comparison of the data obtained by different groups (WHO 2007). The agreed common criteria were applied from that point by the different researchers, which created a new harmony and a collaborative environment.
During the following decade, thanks to renewed research efforts, new diagnostic tools and new knowledge on assessing treatment outcomes became available (WHO 2013), which may allow improvement of the clinical trial methodology. In addition, the number of HAT cases reported annually to WHO has fallen to fewer than 7000, and the disease has been targeted for elimination. These new facts justify an update of some of the criteria adopted in 2004.
This meeting was framed by the WHO Network for HAT Elimination and it convened specifically the sub-group “Development of new tools”, with the following objectives:
- To review and discuss how the new knowledge made available since 2004 could impact the implementation of clinical trials; and
- To update the consensus framework for the planning, conduct and analysis of clinical trials in the future in a way that would promote the acquisition of data that can be readily compared and used in meta-analysis.