Guidance on evidence generation on new regimens for tuberculosis treatment

This guidance document provides recommendations on generating evidence to support the development and evaluation of new tuberculosis (TB) treatment regimens and to inform World Health Organization (WHO) guideline development. It explains the relationship between evidence generation, regulatory approval and WHO policy recommendations, and outlines the methodological approaches used by WHO to assess evidence through the Grading of Recommendations Assessment, Development and Evaluation (GRADE) framework. The document addresses challenges commonly encountered in TB research, including low-certainty evidence, inconsistent reporting and limited data on feasibility, acceptability, equity and cost–effectiveness.

Focused primarily on late-stage clinical trials for drug-susceptible and drug-resistant TB, the guidance describes key considerations for study design, participant selection, efficacy and safety outcomes, treatment duration, follow-up, data analysis and data sharing. It also provides guidance on generating evidence beyond clinical benefits and harms, including evidence related to values, resource use, equity, acceptability and feasibility. In addition, the document summarizes relevant WHO processes linked to the development of new TB medicines and regimens, including guideline development, prequalification and coordinated scientific advice procedures.