(175) QuestionCF 2022: refreshing the top 10 research priorities for clinical research in Cystic Fibrosis

Background: The top 10 research priorities for clinical research in CF were identified in 2017 through a James Lind Alliance (JLA) Priority Setting Partnership (PSP), engaging the patient and clinical communities [1]. These priorities have been adopted by funders, leading to at least £23 million of funding in the UK alone. Much has changed in CF over the past 5 years so we are now undertaking a global refresh to identify priorities in post-modulator times.

Methods: Led by a multinational steering group representative of the patient and clinical communities, we have developed a new methodology. An initial questionnaire identified which of the 2017 priorities were still relevant and to gather new questions (Jan-Feb 2022).This was available online in several languages and promoted through our “QuestionCF” social media channels, plus professional, charity and patient group partners. The new suggestions were checked, grouped and umbrella questions formed where possible. A second ranking survey will ask individuals to select their personal top 10 (May-June 2022) and an online workshop involving representatives of the patient and clinical communities will agree the refreshed top 10 priorities in summer 2022.

Results: We had 1608 responses to the first survey from 1370 individual respondents. Of these 74% were from lay people (people with CF and their families and friends) and 26% from health care professionals and researchers in CF. The median age of people with CF answering (or with answers on their behalf) was 27 years (range 0-79). Most respondents with CF (65%) were on modulator therapies, with 14% not yet eligible. We had responses from 29 countries around the world, including North America, Europe, New Zealand & Australia, Africa and the Middle East. We had 971 question suggestions. We will present the results of the next stage and final refreshed top 10 priorities for clinical research in CF at NACFC.

Conclusions: Ours is the first JLA PSP in any condition to refresh its priorities. We are working with the global community to keep CF research relevant and focused on the needs of people with CF.

Acknowledgements: This work was supported by the Cystic Fibrosis Trust and a Research England, Participatory Research grant.

References: [1]Rowbotham NJ, Smith S, Leighton PA, Rayner OC, Gathercole K, Elliott ZC, Nash EF, Daniels T, Duff AJA, Collins S, Chandra S, Peaple U, Hurley MN, Brownlee K, Smyth AR. The top 10 research priorities in cystic fibrosis developed by a partnership between people with CF and healthcare providers Thorax 2018;73:388-390. https://dx.doi.org/10.1136/thoraxjnl-2017-210473