Cystic Fibrosis Research Priorities Refresh (priority setting in association with the JLA)
About this PSP
In January 2017 the JLA PSP in Cystic Fibrosis published its list of research priorities that had been identified and agreed by people with CF, family members and clinical care teams, globally. A considerable amount of research has been done around those priorities. Some examples of the research being done are in a You said, we did:How the Cystic Fibrosis priorities have been addressed since they were agreed in 2017 summary.
Whilst much progress has been made in addressing many of the research priorities there is still work to be done. There have also been considerable changes in cystic fibrosis treatments and care since the original list of priorities was agreed.
This refresh exercise asked people living with Cystic Fibrosis, their carers and family members, researchers and clinicians which of the original priorities were still important to them and what new research priorities there should be.
See news: September 2022, November 2021
This video shows the new Top 10 research priorities being read out:
The Cystic Fibrosis refreshed Top 10 was published in November 2022.
PSP website
Articles and publications
Key documents
Cystic Fibrosis JLA Research Priorities Refresh Protocol
Cystic Fibrosis JLA Research Priorities Refresh news story about Top 10 announcement
Cystic Fibrosis JLA Research Priorities Refresh FAQs about Top 10 announcement
Cystic Fibrosis JLA Research Priorities Refresh Question Verification Form
Cystic Fibrosis JLA Research Priorities Refresh Engagement Summary
Cystic Fibrosis Refresh Top 10 priorities
- What options are available for those not able to take current CFTR modulators (including rarer mutations, not eligible and unable to tolerate)?
- What is the best way to diagnose lung infection when there is no sputum e.g. children and those on modulators?
- How can we relieve gastro-intestinal symptoms, such as stomach pain, bloating and nausea?
- How do we manage an ageing population with CF?
- Is there a way of reducing the negative effects of antibiotics e.g. resistance risk and adverse symptoms in people with CF?
- What are the long-term effects of medications (including CFTR modulators) in CF?
- What are the effects of modulators on systems outside the lungs such as pancreatic function, liver disease, gastrointestinal, bone density etc?
- What are the effective ways of simplifying the treatment burden of people with CF?
- Can genetic therapies (such as gene editing, stem cell and mRNA technology) be used as a treatment for CF?
- Is there a way of preventing CF related diabetes (CFRD) in people with CF?
The following questions were also discussed and put in order of priority at the workshop:
- How can we recognise and manage the side-effects of CFTR modulators (including those at greater risk e.g. liver disease)?
- Are people with CF at higher risk of certain cancers and what is the best way to detect and manage cancers in people with CF?
- Can exercise replace chest physiotherapy in people with CF?
- How best to manage and support people with CF post-transplantation (e.g. mental health, rejection, modulator use)?
- What is the optimum treatment regimen for eradication of Pseudomonas in people with CF?
- What effective ways of motivation, support and technologies help people with CF improve and sustain adherence to treatment?
- How to improve breathing capacity/lung volume?
Document downloads
For full details of all of the questions identified by this PSP, please see the document below.