Diversity in CF clinical trials: How we’re breaking down barriers

Empowering everyone with CF to take part in clinical trials

Over the last decade, the introduction of CFTR modulators, like Kaftrio, has dramatically changed the quality of life for many people with CF. However, there is still a significant minority of the CF population who are not benefiting from these treatments.

It’s important that the landscape of CF research and clinical trials continues to evolve to address the new needs and concerns of the community. Diversity in clinical trial participation is crucial to this, because the participants and research outcomes need to be reflective of the whole CF community to make the biggest difference. 

Feedback from CF centres and clinicians suggests that there are certain groups of individuals with CF who are under-represented in clinical trials, including people from Black, Asian and minority ethnic backgrounds, and lower socioeconomic groups.

Our Clinical Trial Accelerator Platform team are determined to address the barriers to trial access and ensure every person with CF can take part in clinical trials when opportunities arise. To do this, we launched the 'diversity in clinical trials project in November 2022.

Our progress so far

This project is being carried out by a working group which includes colleagues from across Cystic Fibrosis Trust, CF clinicians, CTAP trial coordinators, and members of the CF community.

One area of research for treatments for those unable to benefit from modulators will be in the field of genetic therapies. This is a new area of treatments for the CF community and there is a lot that is not understood about how these therapies work. 

Ahead of the first genetic therapy trials opening in the UK, we developed a number of online resources explaining key concepts and frequently asked questions on genetic therapies and clinical trials. This includes information aimed at the CF community and for CF professionals.

To begin identifying barriers to participation in research, we sent a short survey out to the CF community. We received 30 responses, split between people who did and did not have experience with clinical trials. Common themes that came up were:

• Majority of respondents were interested in taking part in research
• Not all people were hearing about research opportunities in CF clinic
• Reasons for not participating in clinical trials included concerns about safety of new treatments and constraints related to the amount of time required to participate or the distance to study centres
• Respondents commented that flexibility with research appointment schedules, a wider remit for financial compensation, and more information from trusted sources (CF team and nurses) would encourage them to participate in clinical trials. 

We also consulted our CTAP trial coordinators to understand common barriers they face when recruiting people to clinical trials. Their insights aligned closely with the responses from the survey. Coordinators commented that participation in trials can be burdensome (time, finances) and that many secondary costs are not considered by study sponsors (childcare, time off work). 

Additionally, they noted that patient information sheets and consent forms can be dense and inaccessible (not available in alternative languages or formats). 

Finally, they felt that there needed to be improved communication and alignment between clinical and research teams.

What happens next?

As this project continues, we want to ensure we can monitor the progress and ongoing clinical trial participation. We also want to develop a robust evidence base identifying under-represented groups in clinical trials, this will allow us to put together a targeted strategy to improving diversity. 

We plan to do this by reviewing specific metrics in study participation, for example, age, gender, ethnicity, compared to the UK CF population.

We also plan to review all Trust resources about clinical trials, keeping in mind underrepresented groups, and ensuring they are accessible for everybody in the CF community. This might look like translating resources, providing different formats (videos, infographics, etc.) and more. We will engage with the community every step of the way when reviewing these resources.

Finally, we want to continue gathering insights from members of the community on attitudes towards clinical trials, and any barriers to taking part. 

How you can get involved

Would you like to get involved by sharing your thoughts on CF clinical trials? We are looking to recruit people with CF or carers of people with CF to participate in small focus groups discussing participation in clinical trials. Anyone in the community can take part. Please reach out to [email protected] to learn more and get involved with this project.

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