Strategic Research at the Cystic Fibrosis Trust

Elly Aylwin-Foster

5 minute read

Last month Dr Janet Allen, the Director of Strategic Research at the Cystic Fibrosis Trust came into Friday to talk to us about the work the Trust has been doing with a focus on their digital research and initiatives. The Trust is the only nationwide charity that represents people in the UK with CF; a lifelong multi-organ genetic condition which predominately affects the lungs, pancreas and digestive system. Janet was recently named one of the ‘50 Movers and Shakers in BioBusiness 2017’, in BioBeat’s annual publication showcasing women leaders in life sciences.

We met Janet at our roundtable on Patient Utility, where Friday hosted a discussion with senior attendees from all sectors within healthcare including pharmaceutical, charity, the Dept. of Health, pharmacy and start ups.

We discussed some of the Trust’s ongoing research including; remote monitoring for people with CF (PWCF), the CF Trust’s data-rich UK registry, and their research with the Turing Institute for data sciences.

On funding digital

For a charity that represents a relatively small body of 10,000 people with CF in the UK, the Trust has a mature digital presence and has invested in research with lots of different digital facets, including a digitised clinical trials accelerator, which helps close the gap between participants and researchers.

Having inherited a research budget and charged with coming up with a comprehensive five-year research plan, Dr Allen said that while there wasn’t a conscious effort to fund more digital work at first, it became clear it was an area where they could have a “demonstrable and cost effective impact.”

Owing to the unique nature of CF which means those with the condition can’t meet in-person (due to cross-infection), digital projects seemed like a natural choice for better engaging with and representing the CF community. Continued investment in video call facilities and video conferencing has made both working at the Trust more inclusive for those employees with CF, and for the wider CF body who now have greater access to research conferences and events.

On the longterm ambitions of the CF Registry

The CF Registry is the cornerstone of much of the Trust’s research. It’s a secure centralised database holding health data from over 99% of consenting people with cystic fibrosis across the UK stretching back over 10 years, and is managed by the Trust. One intended use is to make it easier for certain individuals with CF to access personalised medicines that only work for their specific genetic mutations. While gathering the data in itself was a sizeable challenge, Dr Allen stressed that making full use of it is another one entirely. “We have only just scratched the surface of what can be done with the registry – we hold detailed data on many different aspects of care – but we are now starting work to better understand it all. One of the most powerful things we can do is link databases with relevant experts and organisations, and make the most of that combined data and knowledge, instead of simply starting from scratch.” One such study is a collaboration with The Turing Institute [link to Turing homepage]. The Trust press release states, “this project aims to use advanced mathematical techniques on UK CF Registry data to create a method of generating personalised risk scores for people with cystic fibrosis. These scores can then be used by people with CF and their clinical teams to tailor their treatments and other activities to help them effectively manage their condition.”

On ownership of patient data

The issue of who owns patient data in the healthcare ecosystem is a convoluted one. In the instance of CF care, the Trust has an established reputation as the broker between PWCF and clinical professionals, trusted because of the centralised level of data the Trust manages for the Registry and because that data is always anonymised to ensure patients feel secure. “Owning this data helps puts us at the centre of conversations around care and research” Janet says. “We can talk about aspects of patient care with authority because we have enormous amounts of data to support these areas.”

On using CF as an exemplar in cross-population research studies.

When it comes to digital projects, the Trust is well placed to serve as an example and share research. Because CF is present from birth PWCF tend to be young and highly digitally savvy (as opposed to a condition which disproportionally affects older people such as Alzheimer’s). Much of their own education about the condition and their socialising with other PWCF is done online, often in the CF Trust forum, which provides an early place for people to socialise with each other.

Says Janet, “CF may be a rare condition but is the ideal exemplar for ongoing areas in healthcare; because of the centralised nature of care in specialist centres [making studies easier to organise than patient care split across primary and secondary healthcare centres] it provides the perfect chance to scale research to larger communities. It is also relevant to many wider areas of research like antibiotic resistance and access to novel drugs.”

On remote monitoring tools for PWCF and skype clinics.

70% of people with CF in the UK are in fulltime employment or education. This means that going to clinic for a check up – where people are at risk of picking up infections– when you’re already walking a tightrope of time management, is not always the best method of care for some. Creating digital methods for PWCF to talk to their doctors and to manage their own care, such as skype clinics for routine management and self-measurement of lung function, is an exciting new area the Trust has been exploring alongside CF centres themselves.

A feasibility study on remote monitoring is being carried out with the Trust by Professor Andres Flotos, to determine whether largescale remote monitoring is a sensible option for PWCF.

FEV1 (the measure of the speed at which someone can breathe out during the first second of exhalation) is the stalwart marker for determining exacerbations or heightened chest infections, but it’s somewhat of a blunt tool for something which can be extremely hard to monitor or detect. Part of this work is finding out from PWCF which biomarkers are most relevant in detecting an exacerbation, whether it’s prevalence of night time cough, recovery time after exertion or something else altogether.

Janet points out that empowerment, “is a central tenet of remote monitoring, and indeed every aspect of the research we do; it’s about providing PWCF the tools they need as individuals to manage their condition. Those needs will vary from person to person, but a variety of tools should be available to help people manage their condition to the best of their ability.”

Janet Allen

Janet Allen, Director of Research at Cystic Fibrosis Trust

To find out more about remote monitoring, please read the Trust’s press release:

A big thank you to Janet for both her time and passion when discussing such fascinating research with us.

Elly Aylwin-Foster

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