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"You can observe a lot by watching" - The value of a Registry for Cystic Fibrosis in the UK

Wednesday, November 8, 2023

Respiratory Futures recently talked to Dr Jamie Duckers, Dr Gwyneth Davies, Dr Sarah Clarke and Professor Siobhán Carr on behalf of the UK CF Registry Steering Committee. The article below looks into the Cystic Fibrosis Registry in the UK, the value it provides and outlines at a high level what running a Registry looks like.

Can you tell us a little bit about the background of the Cystic Fibrosis (CF) Registry? When did it begin, and what were some of the motivations for its creation?

We know that data is the lifeblood of modern healthcare and as Yogi Berra said, “You can observe a lot by watching.” Patient registries are organised systems that collect uniform clinical data on a population defined by a particular disease that is followed over time. Enthusiasm is growing for the use of observational real-world data as a basis for regulatory, clinical and health policy decision making. The UK CF Registry was first established in 1995 and is supported by the Cystic Fibrosis Trust in collaboration with the CF community with the key objectives of raising standards of care and research. Web-based software enables the collection of a standardised dataset, including patient-reported outcomes, across all paediatric and adult Specialist CF Centres and associated Network CF Clinics throughout the UK.  What value do registries like this provide? What impact has the Cystic Fibrosis registry had on patients and patient care?

A UK CF Registry Data Report is published annually by the Cystic Fibrosis Trust showing national as well as centre-level analyses. It provides longitudinal data on changing demographics, the occurrence of complications (e.g. diabetes, liver disease, cancer), additional areas (such as increasing rates of pregnancy and employment status) and as such it supports service planning for commissioners and NHS Trusts. This multicentre dataset allows the 60 UK CF centres to benchmark, facilitating local and national quality improvement and audit. The ability to collect patient reported outcome measures (PROMS) has recently been added, meaning as a community, we have the ability to look at the impact of new therapies on wellbeing for people with CF. Registry data have also been used to develop shared decision-making tools to facilitate more informed, person-centred care. Epidemiological research has allowed both national and international collaborations, a recent example being a rapid response to studying the impact of COVID-19 on people with CF in the first wave of the pandemic.

The ability to collect patient reported outcome measures (PROMS) has recently been added, meaning as a community, we have the ability to look at the impact of new therapies on wellbeing for people with CF.

As well as being a useful information resource for the CF community and clinical teams, the UK CF Registry is also of growing importance for clinical trials (see below) and post-market authorisation of new medicines for CF. 

What are some of the challenges of setting up and managing a registry?

The UK CF Registry has been critical to advancing care in CF and is the envy of many other diseases. The success of a registry depends on the community and its engagement. We are grateful that over 99% of people with CF in the UK contribute to this powerful data resource. Patient registries need stringent ethical and data protection and data validation oversight as well as significant administrative and clinical support. These requirements require adequate long-term financial investment ensuring that data capture and coverage is of good quality. The registry also needs to be able to respond to changing landscape of care. A key challenge is to preserve opportunities for longitudinal analysis yet ensure data collection remains relevant for the CF community.  Can you provide a high-level example of what running a trial using a registry looks like?

The UK CF Registry has a growing experience in hosting pragmatic randomised controlled trials. Two clinical trials are in progress, CF START and CF STORM, both funded by the NIHR’s Health Technology Assessment programme. These trials have been possible due to the CF Registry’s software infrastructure, building on systems and processes for routine data collection. In addition to data collection from the core registry, registry modules with outcomes specific to a study can be ‘bolted’ to the core system. These also pull through the relevant core variables to prevent duplication of data collection. Registry trials are attractive in aligning outcome collection to standard care, and therefore associated with a lower study burden than one with specific research study visits. Assessment of eligibility, electronic consent/assent, randomisation, collection of patient-reported outcomes is also possible, and all of these functions are embedded within the registry infrastructure hosting the CF STORM study. The population coverage of the UK CF Registry and engagement with CF centres across the UK means that registry trials may open wider opportunities for trial participation, in line with the ethos of the CF Trust’s Clinical Trial Accelerator Platform (CTAP) programme.