Evaluation of person-centred outcome measures for use in clinical trials of tuberculosis therapeutics

BackgroundThere is a need for person-centred outcome measures (PCOMs) in tuberculosis (TB) clinical trials to capture the priorities of affected individuals relating to both disease and treatment, alongside the traditional efficacy and safety analysis. MethodsWe conducted a literature search to identify existing health-related quality-of-life (HR-QoL) measurement tools and instances where they have been used to evaluate TB disease in interventional studies. DR-TB survivors were recruited for focus group discussions in South Africa, Mongolia and Georgia to determine the most important aspects of disease and treatment experience from their perspective. A second round of focus groups then worked to prioritise these items and group them within different domains. A panel of researchers and academic clinicians evaluated existing PCOMs identified in the literature search using a published framework. Finally, an assessment was carried out by a experienced TB Community Advisory Group members for appropriateness and acceptability, and clinical trial site investigators for feasibility of the PCOMs. ResultsThe initial round of 9 focus groups involving 57 participants identified priority items grouped under the fields "overall quality of life", "disease symptoms", and "treatment and side effects". In the second round of four focus groups involving 26 participants, the highest ranked items under "overall quality of life" were duration of treatment, experience of swallowing pills, and how long before being able to contribute to the household. The most important symptoms were coughing, weight loss and breathlessness. The highest ranked side effects were nausea/vomiting, skin colour changes, and nerve problems. FACIT-TB was the only measurement tool assessed that was rated "good" for appropriateness, and "excellent", "good" or "fair" in all other fields by community representatives, researchers and clinical trial site staff. ConclusionsThe FACIT-TB measurement tool was identified as the most appropriate disease-specific PCOM currently available for use in clinical trials investigating novel DR-TB therapeutics.