The James Lind Alliance Priority Setting Partnership, which used surveys and workshops to identify the top 10 questions for research into CF, is supported by £15,000 from the Cystic Fibrosis Trust’s Venture and Innovation Awards (VIAs), and co-funded by the University of Nottingham and Nottingham University Hospital Charities.
After two surveys, which were open to all members of the CF community, 10 questions were identified from a list of over 1,000 suggestions.
1. What are the effective ways of simplifying the treatment burden of people with CF?
2. How can we relieve gastro-intestinal (GI) symptoms, such as stomach pain, bloating and nausea in people with CF?
3. What is the best treatment for non-tuberculous mycobacterium (NTM) in people with CF (including when to start and what medication)?
4. Which therapies are effective in delaying or preventing progression of lung disease in early life in people with CF?
5. Is there a way of preventing CF-related diabetes (CFRD) in people with CF?
6. What effective ways of motivation, support and technologies help people with CF improve and sustain adherence to treatment?
7. Can exercise replace chest physiotherapy for people with CF?
8. Which antibiotic combinations and dosing plans should be used for CF exacerbations and should antibiotic combinations be rotated?
9. Is there a way of reducing the negative effects of antibiotics e.g. resistance risk and adverse symptoms in people with CF?
10. What is the best way of eradicating Pseudomonas aeruginosa in people with CF?
In Survey 1, the Partnership gathered more than 1,000 suggestions from over 500 people across the world to identify the top 10 questions for research into cystic fibrosis.
Researchers then whittled down questions by removing those that had been repeated, had already been answered or could not be answered through research. The steering committee grouped the remaining questions into 71 questions that the community were then invited to rank in Survey 2.
Survey 2 saw almost 700 people ranking questions, and the chosen top 20 were then debated in a workshop of 25 people, which included five parents, three people with CF and a number of clinicians and support staff.
Of all the people that took part in Survey 1, 24% were people with CF, 27.5% were family and friends of people with CF and 14% of contributors in Survey 1 submitted questions more than once.
"A passionate and engaged community"
Paula Sommer, Head of Research at the Trust said: “This Priority Setting Partnership reflects how passionate and engaged the CF community is when it comes to cutting-edge research. From tackling issues around nontuberculous mycobacterium (NTM) to our Strategic Research Centre (SRC) exploring CF-related diabetes (CFRD), the Trust is breaking new ground all the time in these vital areas of research. We will continue to work closely with our community to tackle the diverse issues posed by CF, and make a life unlimited by the condition a reality.”
Other areas of research being investigated by the Trust that are featured in the top 10 questions chosen by the Partnership include: the introduction of SmartCareCF, a project exploring ways to reduce the burden of care and the risk of cross-infection through the innovative use of new technologies, our 2016 call for an SRC focussing specifically on the GI tract and pancreas and the CF Start clinical trial, which will investigate the usefulness of prescribing certain antibiotics for infants with CF as a preventative measure.
Professor Alan Smyth, who worked with the Trust to obtain funding for the Partnership, said: “The James Lind Alliance Priority Setting Partnership in cystic fibrosis has brought together over 500 people from across the world and includes an even split of medical staff and lay people. This is the first time such a collaborative and representative process has been undertaken in cystic fibrosis. We will now work with the National Institute for Health Research (NIHR), the Cystic Fibrosis Trust, the Cystic Fibrosis Foundation and the UK Research Councils to get these research questions funded and taken forward by experienced and collaborative researchers.”
Zoe, a parent member of the Partnership steering committee, said: “As a parent it was great to see that patients, parents and carers have an equal voice in this project. The coming together of the 'lay' community with healthcare professionals has been a fantastic knowledge-sharing process from the start, which is reflected in the top 10.”
Read our interview with Professor Smyth and Zoe to find out how people living with CF and those who support them are having an impact on research.
What you might have missed
Last year we spoke to people involved across the Partnership to find out more about the work that goes into making it a success.
Tracey Daniels, a specialist CF physiotherapist and member of the Partnership steering committee, spoke about her passion for finding out more about physiotherapy.
Keith Thompson spoke about how important it is for people with CF to be at the forefront of shaping research.
And Rebecca Sterry, who was involved in a Priority Setting Partnership for Autistica UK, a charity funding and campaigning for research into autism, told us how a Partnership can put the community at the heart of shaping research.
Find out more about research we are funding, and donate today to support our vital work for years to come.