Guidance on evidence generation on new regimens for tuberculosis preventive treatment

The global burden of tuberculosis infection and the need to prevent progression to active disease highlight the importance of generating robust and policy-relevant evidence for tuberculosis preventive treatment (TPT). This guidance presents a structured framework to support the generation of high-quality evidence on new TPT regimens, addressing persistent limitations in available data, including low certainty of evidence, incomplete reporting and insufficient consideration of key decision-making criteria.

Developed as a complement to WHO target product profiles, the document provides detailed methodological guidance for researchers, funders and other stakeholders involved in clinical trials and related studies, particularly those conducted in phases 2–4. It outlines key elements of the WHO guideline development process and the application of the GRADE approach, emphasizing the need to generate evidence not only on efficacy and safety but also on feasibility, acceptability, equity and cost–effectiveness. The guidance covers study design, outcome harmonization, data analysis and reporting, and highlights the contribution of qualitative and economic research. By clarifying evidence requirements, it aims to strengthen the certainty, comparability and policy relevance of findings, thereby facilitating the development, adoption and implementation of effective TPT regimens in national programmes.