New top 10 CF research priorities - your questions answered

The refreshed top 10 CF research priorities have been 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? 

Five years ago, the Trust worked with the CF community, the James Lind Alliance and researchers at the University of Nottingham, to find out the top research priorities of people with CF, as well as their families, carers and CF teams. These priorities have helped us decide what research we fund and they have allowed doctors and scientists to focus the direction of their studies. This research is ongoing, and you can read about some examples of the exciting work underway in a review article. 

Many people with CF now have access to CFTR modulators, and due to the pandemic, many people’s care will have changed, for example with remote clinic appointments and health monitoring at home. The research priorities need to be current and they need to be relevant, we felt it was time to look at them again. 

These priorities were agreed in consultation with people with CF, their loved ones, CF professionals and CF researchers, in the form of two anonymous surveys widely promoted and open to everyone to complete and an online workshop in November. You can read about the process in more detail in this article on our website.  

The project was facilitated by the James Lind Alliance team at the National Institute of Health and Care Research (NIHR). The detailed plan of how it was conducted and who was involved is available on the James Lind Alliance website.  

At the beginning of the project in January, the QuestionCF management group agreed the interests that we would like to see represented at the workshop in the form of a ‘wishlist’. For example, whether people were able to benefit from CFTR modulators or not, or whether they were post-transplant. 

The number of people joining the workshop was limited to around 18-24 people overall, and 22 people took part. Due to the limited numbers, it wasn’t possible to invite everyone who said they’d like to attend. Half of these spaces were allocated to people from the CF community (people with CF, parents or grandparents of people with CF), and the other half from representatives of professionals working in CF (doctors, researchers, representatives from CF teams).  

We asked people to tell us whether they were interested in taking part in the workshop in a form that was widely promoted. It was part of the second survey, in newsletters from the Trust and across several social media platforms on a number of different accounts. We asked them to tell us about themselves, and why they were interested in taking part.  

The responses were anonymously reviewed against the agreed wishlist, and people were invited to take part. The workshop was professionally facilitated by the James Lind Alliance team at the NIHR who have had lots of experience of helping different patient groups identify their top priorities for research. 

All of the comments and questions that people shared with the project team have been taken into account. Similar questions were grouped together to help agree a top 10 list. All of the questions will be published, so nothing will be lost. 

All of the surveys were anonymous, so researchers won’t be able to work out who gave which answers. You can read more about this in the privacy policy on the QuestionCF website. 

You can view the list of all 17 questions that were taken to the workshop, in the final order agreed at the workshop, on the QuestionCF website.  

It is important to say that while the top 10 questions provide a focus, the questions that appear lower in the list will be available. Researchers and research funders will consider these when planning future CF research.  

We know that everyone has a different experience of CF, so these priorities are not going to be a match for every person’s priorities for CF research. However, we hope that these priorities represent the questions from as diverse a group of people as possible, including people who are post-transplant, have rare mutations and those who are not able to tolerate CFTR modulators.  

The aim of this current project was to refresh the CF research priorities, to see if they had changed over the last five years. The top 20 research priorities identified in 2017 were included in the first survey in January, and analysed alongside all of the questions submitted. Four of the refreshed top 10 priorities announced five years ago are still priorities today.  

This is the first time everyone has seen this refreshed list of top 10 CF research priorities. We’ll be sharing them as widely as possible, so as many people as possible know which areas of CF research have been prioritized. Colleagues at the University of Nottingham will be publishing the results of the analysis. The data will also be shared on the James Lind Alliance website. 

At the Trust, we’ll be talking to CF researchers about how their research studies might answer these questions, and what is the best way to help them get answered. We’ll also be sharing the results with other funders of CF research, so we can work together to get as many studies funded as possible. 

In Spring 2023 we’ll update on the Trust’s plans to address these research priorities, our next steps and how the CF community can get involved.