New tool found for patient-centred outcomes in drug-resistant TB trials

Tuberculosis remains a disease where clinical trials have traditionally focused on measures of bacteria, cure rates and drug safety. Researchers led by Conor Tweed set out to fill a gap: the lack of person-centred outcome measures (PCOMs) that capture what matters most to people affected by TB during disease and treatment. To do this they first searched the literature to find existing health-related quality-of-life (HR-QoL) tools and to see how these tools had been used in interventional studies of TB. They then turned to people with lived experience: drug-resistant TB (DR-TB) survivors were invited to take part in focus group discussions in three countries — South Africa, Mongolia and Georgia — to describe which parts of illness and therapy mattered most to them. A second set of focus groups helped prioritise and organise these items into domains. In parallel, a panel of researchers and academic clinicians applied a published framework to rate identified PCOMs. Finally, experienced TB Community Advisory Group members judged acceptability and appropriateness and clinical trial site investigators assessed the feasibility of using the shortlisted measures in trials.

The study used a mix of qualitative and evaluation methods. The initial round comprised nine focus groups with 57 participants and produced priority items grouped under the fields "overall quality of life", "disease symptoms", and "treatment and side effects". A second round of four focus groups with 26 participants ranked the priorities. For "overall quality of life", the top items were duration of treatment, experience of swallowing pills, and how long before being able to contribute to the household. The most important disease symptoms were coughing, weight loss and breathlessness. The top-rated treatment side effects were nausea/vomiting, skin colour changes, and nerve problems. The literature and tools identified were then assessed by researchers, community representatives and clinical trial staff. FACIT-TB was the only measurement tool that these groups rated "good" for appropriateness and at least "fair" to "excellent" across the other evaluation fields, making it stand out from other HR-QoL instruments considered.

The clear outcome from this work is that FACIT-TB emerges as the most appropriate disease-specific PCOM currently available for use in clinical trials investigating novel DR-TB therapeutics. By combining what matters to survivors—shorter treatment, manageable pill burden, and recovery of household roles—with commonly experienced symptoms and side effects, FACIT-TB offers a way to measure patient priorities alongside traditional efficacy and safety endpoints. The involvement of DR-TB survivors across three countries, a formal framework assessment by researchers and clinicians, and review by TB Community Advisory Group members and clinical trial site investigators all point toward FACIT-TB being acceptable and feasible to deploy in trial settings. Using a validated, disease-specific PCOM like FACIT-TB in future trials could ensure that new DR-TB treatments are judged not only on microbiological outcomes but also on how they affect people’s daily lives, symptoms and side effects as reported by patients themselves.