The development of patient-focused diagnostic target product profiles for detection of infection and exacerbation in people with cystic fibrosis

<ns3:p> Background Infections and exacerbations are common for people living with Cystic Fibrosis (pwCF). This project aimed to develop patient-focused diagnostic target product profiles (TPP) to address unmet diagnostic needs for pwCF with international relevance. Methods This project involved three phases, guided by an expert advisory group of key opinion leaders (KoLs) including methodologists, pwCF, and clinical staff, and including lived experience perspectives at every stage. Phase 1 involved a landscape analysis to identify and prioritise unmet diagnostic needs via focus groups with clinical experts and pwCF, as well as a scoping review of the diagnostic space and available diagnostic tests. Phase 2 involved drafting the TPPs using evidence from existing literature and regulatory documentation, focus groups, one-to-one interviews with KoLs, and web-based surveys, to define ‘minimal’ and ‘optimal’ characteristics for each TPP. Phase 3 refined and validated the TPP content through additional interviews, a two-round modified Delphi exercise, and a virtual symposium. Results A comprehensive document detailing four TPPs was drafted and released freely for use. The document includes aspirational TPPs covering diagnostics for managing acute pulmonary exacerbations, rapid pathogen identification, and antimicrobial susceptibility tests, as well as a detailed TPP defining <ns3:italic>in-vitro</ns3:italic> diagnostic tests for rapid detection of non-tuberculous mycobacteria (NTM) pulmonary infections. The document contains additional guidance for diagnostic development and research needs identified for pwCF. Conclusions The project consulted with over 150 individuals and experts in CF infection management, and diagnostic development. The TPP guidance document supports research and development of patient-focused diagnostics for the benefit of pwCF. </ns3:p>