Unmet diagnostic needs in cystic fibrosis infections and exacerbations: focus groups to inform target product profiles (TPPs)

囊性纤维化感染和急性发作中未满足的诊断需求:焦点小组讨论以指导目标产品概况 (TPP) 的制定

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Abstract

BACKGROUND: In addition to new antimicrobials for people with Cystic Fibrosis (pwCF), new diagnostics are needed to detect and diagnose infections, guide clinical care, and inform clinical decision making. To determine unmet diagnostic needs in pulmonary infection and exacerbation diagnostics in Cystic Fibrosis (CF), the required diagnostic test characteristics and priorities of different stakeholders involved in the care of pwCF were collected and analysed. METHODS: Three focus groups (two clinical and one pwCF) were conducted and used to inform a wider project to deliver a suite of target product profiles (TPPs) for CF lung microbiological infection and/or exacerbation diagnostics. Thematic analysis was performed on the focus group data. RESULTS: Participants described their experience of current practice and existing diagnostics for detection, diagnosis, and management of infection and exacerbations in CF in the UK National Health Service (NHS). Unmet needs included: monitoring modalities and testing for treatment efficacy; acquiring samples with good clinical utility; more acceptable sampling methods; and faster microbiology and culture-based testing.Greater communication between the laboratory and clinical teams, and equity of care across UK CF centres was also highlighted. TPP characteristics of importance to pwCF and clinical representatives included 'accuracy', 'time to results', and 'patient acceptability'. Both participants groups highlighted the need for suitable alternatives to sputum sampling and emphasised the need for novel biomarkers for the early detection and diagnosis of both infection and exacerbations. Amongst clinical representatives, test accuracy was generally valued over the time to results for a clinical test in a non-acute setting. CONCLUSIONS: Focus groups offered rich and detailed insights into the opinions of clinical staff and pwCF alike which informed further stakeholder engagement and shaped the content, scope and characteristics of TPPs. Early and rapid detection would have a positive impact on clinical care and inform earlier clinical decision making.

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