The top 10 research priorities in cystic fibrosis developed by a partnership between people with CF and healthcare providers

Abstract

There remain many treatment uncertainties in cystic fibrosis (CF). With limited resources, research should focus on questions which are most important to the CF community. We conducted a James Lind Alliance Priority Setting Partnership in CF. Research questions were elicited and then prioritised in successive surveys. A workshop agreed the final top 10. Online methods avoided cross infection and widened participation. The elicitation survey had 482 respondents (1080 questions) and prioritisation survey 677 respondents. Participants were drawn equally from the patient and clinical communities globally. We have achieved a consensus on 10 research priorities which will be attractive to funders.

Gaps in the evidence for treatment decisions in cystic fibrosis: a systematic review

“…Therefore, prioritisation is needed. We have recently undertaken a priority-setting exercise with clinicians, patients and families through the James Lind Alliance 35. This has in turn led to a funding call by the UK National Institute for Health Research 36.…”

Cystic fibrosis year in review 2018, part 2

“…Questions were then refined into 12 broad research topic areas for prioritization by the Clinical Research Advisory Board (a group of CF research experts) and Community Voice (a group of people and families affected by CF) . Similarly, in the UK, in coordination with the National Institute for Health Research James Lind Alliance, identification of key treatment uncertainties led to production of the top 10 research questions . Not surprisingly, the two surveys had similar outcomes, with the US top three prioritization including respiratory microorganism detection and treatment, mental health and treatment burden .…”

“…Not surprisingly, the two surveys had similar outcomes, with the US top three prioritization including respiratory microorganism detection and treatment, mental health and treatment burden . In the UK, treatment burden was ranked at the top of the list, and infection treatment, with two specific infection questions, specific to NTM and Pseudomonas aeruginosa , included in their rankings . Addressing some of these concerns, at the North American Cystic Fibrosis Conference in 2018, the CF Foundation announced a $100 million, 5‐year, infection research initiative, to improve detection, diagnosis, treatment, and outcomes of CF infections…”

Cystic fibrosis: novel therapies, remaining challenges

“…Although research into correcting the underlying defect is a priority, there is still a need for new agents and treatments that address the consequences of the underlying defect, such as infection, inflammation and pancreatic disease, and to improve nutrition and mucociliary clearance. A survey of healthcare providers and people with CF asking what the top research priorities are showed an emphasis on treatments to prevent disease progression, reduce symptoms, improve QoL (including reducing treatment burden) and address issues of adherence . Research at a practice level should also be a priority.…”