Your CF research priorities

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 eg 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 eg 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*

Four of the refreshed top 10 were research priorities included in the top 10 CF research priority list from 2017, as marked by *

The refreshed CF research priorities are being used to shape how the Trust and other research funders can support and fund CF research. We want to find answers to the questions raised in this list.

Our first job is to make sure that researchers and research funders know about the research priorities. As well as sharing the headlines of the refreshed top 10 list, together with other partners from the project team, we’ll make sure that all the detail of the questions asked by the CF community is available. It is important that researchers and funders can understand the context as much as possible.

The list will continue to be promoted on our website, on the project website at questioncf.org, on social media and at research events and conferences.

In 2016 the Trust was a partner in a project known as QuestionCF to identify the research priorities of people with CF, as well as their families, carers and CF teams. Other partners in the project included the CF community and researchers at the University of Nottingham. The priorities were identified in Priority Setting Partnership (PSP), facilitated by the James Lind Alliance (JLA) team at the National Institute for Health and Care Research (NIHR). The research priorities were published in January 2017.

Since then, 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 from December 2021.

In 2022 the Trust was a partner in a follow up QuestionCF project to refresh the CF research priorities, in light of changes due to the pandemic and access to CFTR modulator medicines such as Kaftrio. The priority refresh project management group included representatives from the CF community, researchers at the University of Nottingham and colleagues from the JLA team.

The refreshed CF research priorities were identified by the CF community in several stages, starting with a survey that opened in January 2022 and finishing in an online workshop in November 2022. In the first survey people were asked to review the top 20 questions from the 2017 CF research priorities and submit new research questions that weren’t included in this list. To find out more about how the CF research priorities were refreshed, see the overview article on our website. The process we followed is described in detail on the James Lind Alliance website.

There’s a frequently asked questions page put together in November 2022. 

The QuestionCF website contains more details of the questions submitted and chosen during the selection of the refreshed top 10 research priorities. This includes the long list of 71 questions considered in the second survey and the shortlist of 17 questions considered in the online workshop. A summary of the methods and the results was published as a research paper in the journal Thorax in June 2023.

The protocol for the CF research priority refresh is available on the James Lind Alliance website, alongside the full dataset of the underlying questions. We have also prepared a summary of the topics included in the underlying questions.

Link to full underlying questions data

Summary of topics included in the underlying questions