RCSI to lead €3m study to test cystic fibrosis drug outside clinical trials

30 Jul 2020

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RCSI will lead a study into how a new cystic fibrosis drug called Kaftrio performs among the general population after promising clinical trial results.

The Royal College of Surgeons in Ireland (RCSI) will lead a €3m international project, dubbed ‘Recover’, to see whether a new cystic fibrosis drug can be released globally. The triple-combination drug called Kaftrio (TriKafta in the US) demonstrated significant improvements in clinical trials of people with the most common form of cystic fibrosis.

Pending final approval from the European Medicines Agency, expected in the next few weeks, the RCSI-led project will examine how the drug affects people in their day-to-day lives. Starting in August, the study will recruit patients and examine how they react to the drug over a two-year period across cystic fibrosis centres in Ireland and the UK.

In addition to collecting routine health measurements, researchers will study detailed imaging, functional, biological and quality of life measurements that could not be collected in clinical trials.

‘Essential to confirm the trial findings’

Prof Paul McNally, lead researcher on the Recover study, said this will help the team understand why different people might respond differently to the drug and how it might affect the “treatment burden” that people with cystic fibrosis live with.

“Clinical trials occur under very controlled conditions and exclude many sick people with cystic fibrosis,” he said. “‘Real-world’ studies, such as Recover, are essential to confirm the trial findings and examine in-depth effects on the everyday lives all people with cystic fibrosis.”

In terms of funding for the study, €2.85m was awarded by the US Cystic Fibrosis Foundation, €112,000 from the Cystic Fibrosis Trust in the UK and €110,000 from Cystic Fibrosis Ireland.

Dr Lucy Allen, director of research at the Cystic Fibrosis Trust, said this study was crucial to “understand the full impact of this drug”.

“In the short term, the results of the Recover study may speed up access to Kaftrio around the world. In the longer term, it will provide information on tailoring care for each individual with cystic fibrosis,” she said.

Colm Gorey was a senior journalist with Silicon Republic