Cystic Fibrosis Target Product Profiles: Focus group documents (topic guides and participant information sheets)

Diagnostics are key to the appropriate detection, treatment and management of many conditions and associated health implications. People with cystic fibrosis are susceptible to recurrent pulmonary infections that over time contribute to deterioration and degeneration of the lung. Launched in 2019, the Cystic Fibrosis (CF) Antimicrobial Resistance (AMR) Syndicate was formed to accelerate the translation and adoption of new CF antimicrobials and diagnostics to the clinic through collaboration (https://cfamr.org.uk/), bringing better treatment options to people with CF, faster.

To catalyse the development of new diagnostics, the CF AMR Syndicate, together with the Newcastle NIHR HealthTech Research Centre (formerly Newcastle NIHR MIC), has worked with the wider community to understand the unmet diagnostic needs and develop a suite of Target Product Profiles (TPPs) for CF lung infection diagnostics. The intention of this is to drive diagnostic discovery, focusing efforts where they are needed the most. Although developed in the UK with input from UK based experts and people with CF, we intend for the impact of these TPPs to be global.

This project involved three main phases, sandwiched between planning and management activities, and a dissemination strategy programme, all guided by an expert advisory group.

Phase 1 involved a landscape analysis to identify and prioritise unmet diagnostic needs via multiple focus groups of clinical experts and people with Cystic Fibrosis (pwCF), as well as a scoping review of the diagnostic space and available diagnostic tests (https://doi.org/10.1016/j.resinv.2024.07.005).

Phase 2 involved drafting the TPPs using existing literature and regulatory documentation, focus groups, one-to-one interviews with key opinion leaders (KoLs), and a web-based survey, 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.

The final TPP documents (and a report from the virtual symposium) are available at: https://cfamr.org.uk/therapeutic-tpp/.