International Clinical Trials Day: Why are Clinical Trials Important?

Blog - 20/05/2025

Today is International Clinical Trials Day, and we would like to take a moment to thank everyone that has supported CF clinical research over the years.

The importance of clinical trials and participants

Throughout the years research has helped us gain a better understanding of cystic fibrosis. Clinical trials, along with other types of research continue to help us improve CF treatment and care, as they are the only way we can assess how well a potential new treatment works, along with understanding whether it is safe and how it may interact with any other treatments you take.

Participating in clinical research can mean you are one of the first people to benefit from a new treatment. However, taking part in a study is a decision that needs to be carefully considered, and it is important that you have a good understanding of what this may involve.

The Clinical Trials Accelerator Platform

Eight years ago, Cystic Fibrosis Trust launched the Clinical Trials Accelerator Platform (CTAP) with the ambition of helping speed up the development of new CF treatments.

Before CTAP, the UK could only support a limited number of CF research studies, meaning a lot of people with CF were missing out on opportunities to take part in research. CTAP changed this by providing the infrastructure and resources needed to increase the number and variety of CF study opportunities throughout the UK.

Through CTAP, a network of 25 CF centres (the majority of which are funded with a CF trial coordinator to oversee the running of CF studies), was formed. This enabled more CF centres from around the UK to run clinical trials, opening up research opportunities for more of the CF community as well as creating a sense of collaboration. All of which has meant that studies can be set up quicker than before, and more people with CF can get involved in research.

The creation of CTAP has strengthened the reputation of the UK as a well-recognised and trusted place to deliver CF studies. CTAP aims to continue to increase the availability of clinical research studies to the UK CF community and support the development of new and transformative treatments for CF.

Importance of representation and diversity

As CF studies continue to evolve, it is important that those who do not benefit from CFTR modulators are included in research. Early phase genetic therapies are a recent addition to CTAP supported studies. These studies include the CF population with rare CF variants, including those who do not respond to or tolerate current CFTR modulator therapies. Read more about genetic therapy studies here.

Having a diverse group of people is critical in clinical research studies, to make sure that findings regarding the safety and effectiveness of new treatments are applicable to the CF community as a whole. That is why having a diverse range of people taking part in research is an important part of CF studies. To ensure that everyone with CF, whatever their background, has the chance to access new and potentially life changing treatments.

Unless a clinical trial is being conducted in a wide range of people, the results of that trial are not going to be relevant to everyone with CF. So, you need to have diversity in clinical trials.
Dr. Maya Desai

As part of the diversity in clinical trials project being led by the Trust, we aim to improve accessibility and participation in CF studies by identifying and addressing the current barriers faced by those underrepresented in the UK CF community.

How can you take part

Taking part in research can mean more than participating in CF studies.

Keeping track of clinical trials

If you are interested in taking part in research, our Trials Tracker lists all CF studies currently open across the UK.

If there are no studies near you, try widening your search area or contact us. You may have the option to travel to a different CF centre to take part in a research study. Some may be able to reimburse you for travel and accommodation costs if you have to travel a long way.

To be the first to know when we add new CF study, sign up to our clinical trials and involvement newsletter.

If you are interested in finding out more about clinical research studies, you can also talk to your CF care team, contact a CTAP trial coordinator, or get in touch with CTAP.

Community and public involvement

Join the growing network of over 300 people with lived experience of CF, to help:

make sure our work is relevant to people with CF e.g., reviewing new resources and shaping projects  
helping researchers design studies to meet the needs of people with CF, making it as straightforward as possible for people with CF to take part.

No additional skills or experience is needed, members of the CF community are experts by experience. This includes people with CF themselves, or parents, partners, or family members of someone with CF.

You can take part in as many or as few opportunities as you want to, and you will be fully supported by the team at all times.

Emily’s Entourage – Clinical Trial Connect

Emily’s Entourage (EE) is a US based charity that supports research into CF treatments for those who are unable to benefit from current CFTR modulator therapies.

They launched the CF Clinical Trial Connect (CTC) database that aims to connect people who are currently not taking CFTR modulators with relevant trial opportunities.

EE has partnered with the Trust so that people with CF in the UK can now also sign up to receive notifications on relevant UK studies they may be eligible to take part in.

I do really encourage people to consider getting involved and to talk to your healthcare team about different opportunities because that’s how we are going to reach the finish line.
Emily Kramer-Golinkoff, Emily’s Entourage

You can hear more about Emily’s CF journey and the CTC database on this week's CForYourself podcast episode for International Clinical Trials Day, with host Lucy Baxter and guests Emily Kramer-Golinkoff and Dr Maya Desai.