Research News

Top 10 Research Priorities of the CF Community

Clinical research should focus on the questions which are of highest importance to people living with CF and their clinicians. The James Lind Alliance is a non-profit initiative in the UK which brings together patients, carers, and clinicians in Priority Setting Partnerships (https://www.jla.nihr.ac.uk/ ).

These partnerships identify and prioritise unanswered questions that the community agree are the most important. This ensures that health research funders are aware of the issues that matter most to the people who will use the research in their everyday lives.

In 2017, the James Lind Alliance, working with the CF Trust in the UK, brought together people living with CF, their carers, researchers and clinicians in a Priority Setting Partnership to agree on a “Top 10” list of research questions which should be prioritised for future research (https://www.questioncf.org/top-20-2017/).

Five years on the CF landscape has changed significantly and the team decided to go back to the CF community to find out what they thought the priorities for CF research should be now. The project was called Question CF (www.questioncf.org).

Over 1,600 people globally responded to the survey suggesting over 900 different questions. Through a follow-up survey and workshop the final Top 10 priorities for clinical research in CF were agreed as follows:

1. What options are available for those not able to take current CFTR modulators (including rarer mutations, not eligible and unable to tolerate)? 2. What is the best way to diagnose lung infection when there is no sputum e.g. children and those on modulators? 3. How can we relieve gastro-intestinal symptoms, such as stomach pain, bloating and nausea? 4. How do we manage an ageing population with CF? 5. Is there a way of reducing the negative effects of antibiotics e.g. resistance risk and adverse symptoms in people with CF? 6. What are the long-term effects of medications (including CFTR modulators) in CF? 7. What are the effects of modulators on systems outside the lungs such as pancreatic function, liver disease, gastrointestinal, bone density etc? 8. What are the effective ways of simplifying the treatment burden of people with CF? 9. Can genetic therapies (such as gene editing, stem cell and mRNA technology) be used as a treatment for CF? 10. Is there a way of preventing CF related diabetes (CFRD) in people with CF?

*Priorities 3, 5, 8 and 10 were identified in the Top 10 priorities in 2017 and are still priorities today.

The following additional questions were also discussed at the final workshop and ranked in order of priority: How can we recognise and manage the side-effects of CFTR modulators (including those at greater risk e.g. liver disease)?

11. 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? 12. Can exercise replace chest physiotherapy in people with CF? 13. How best to manage and support people with CF post-transplantation (e.g. mental health, rejection, modulator use)? 14. What is the optimum treatment regimen for eradication of Pseudomonas in people with CF? 15. What effective ways of motivation, support and technologies help people with CF improve and sustain adherence to treatment? 16. How to improve breathing capacity/lung volume?

Cystic Fibrosis Ireland will disseminate these priorities among the research community to see how their work might answer these questions. This list will help clinicians and researchers to decide what areas of CF they should focus their research work on. Significant progress has been made in the last 5 years on the questions identified in the original priority setting partnership. We look forward to seeing the progress that will be achieved on the new priorities over the coming years.