Researchers at RCSI University of Medicine and Health Sciences are leading a new €3 million study to investigate the real-world effects of the newest cystic fibrosis (CF) medications as they become available for people with CF.
The research will look at how a new triple combination CF drug, Kaftrio, will affect people in their day to day lives, rather than in controlled clinical trial conditions. Kaftrio (known as TriKafta in the US) is a successor to Orkambi.
The project, called RECOVER, has been awarded €2.85m from the Cystic Fibrosis Foundation (US), €112,000 from the Cystic Fibrosis Trust (UK) and €100,000 from Cystic Fibrosis Ireland.
In recent years, a number of promising new medications have been developed that treat the underlying genetic defect in CF.
Pending final European Medicines Agency approval, expected in the next number of weeks, these new medications will be provided for use in the majority of people with CF in Ireland and the UK as part of an existing agreement between the manufacturer and the respective states.
Starting in August 2020, the study will recruit patients and begin to examine clinical outcomes over a two-year period across CF centres in Ireland and the UK.
“Our study will allow us to discover in detail how this powerful new treatment affects the health and everyday lives of people with cystic fibrosis, to understand why different people might respond differently to the drug and to gain insight into how this treatment might affect the very significant treatment burden that people with cystic fibrosis currently endure,” said Professor Paul McNally, Associate Professor of Paediatrics at RCSI, Respiratory Consultant in Children’s Health Ireland and lead researcher on the RECOVER study.