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Research centre’s pandemic response drives adoption of ‘lifechanging’ cystic fibrosis treatment

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A cystic fibrosis (CF) research centre at a South West NHS trust continued to recruit to time and target on all studies despite the upheaval caused by COVID-19, its annual report has revealed.

The efforts and flexibility of staff in the centre, at the Royal Devon and Exeter NHS Foundation Trust (RDE), meant it played a key role in gathering the evidence that allowed a new drug, Vertex Pharmaceuticals’ Kaftrio, to be made generally available to CF patients last autumn. 

The centre, supported by the NIHR Clinical Research Network South West Peninsula, was also one of the highest recruiters of adult patients as a percentage of its total available population in the Cystic Fibrosis Trust’s Clinical Trials Accelerator Platform (CTAP) network. The numbers were revealed in the centre’s recently published annual report, covering the period from February 2020 – February 2021.

The success reflects the local team’s creativity and commitment to ensuring patients had access to trial drugs during an incredibly challenging year, as the COVID-19 pandemic stretched the NHS to new extremes and all those with CF, classed as clinically extremely vulnerable, were forced to shield.

Sophie Whiteley, CTAP Cystic Fibrosis Trials Coordinator at the RDE centre, said: “It’s really pleasing to know that, despite the complexity of CF trials, and like everyone else, the complexity of working in the health service over the past year, we have managed to keep our patients on the trials.

“The impact of Kaftrio can vary from patient to patient, but generally, it is lifechanging. Many patients have seen significant improvement in lung function, and others have a lot more energy, their digestive system has improved, they’re able to control their diabetes better. It brings stability for most people, which has been exactly what’s needed this year to avoid having those frequent admissions and lots of contact with care teams.

“It’s been fantastic to watch it spread out and hit the clinic. Kaftrio is able to be given to 90 per cent of the population who have CF. At the moment it’s licensed for 12 years and up, but soon it will be available for ages 6 – 11, so hopefully these kids will never experience the marked dip that many CF patients get as adolescents.”

Dr Nick Withers, Consultant CF Physician at RDE said: “Working with CTAP has enabled our team to involve more of our patients with CF in research and give them earlier access to a new class of drugs. We are mindful that about 10% of our patients are yet to benefit from these new advances, and remain committed to working with other parties to further advance research into new treatments for all patients with CF.”

Through CTAP, the Cystic Fibrosis Trust brings together CF centres around the UK and funds research staff, to support the CF patient community in accessing and participating in clinical trials. As a result, patients with the genetic condition have the best possible access to potentially lifechanging new treatments. The programme also engages with pharmaceutical companies and researchers to showcase the UK as a desirable country to run CF clinical trials.

To find out more about the CTAP programme, please visit: