LEEDS, UK – The parents of a baby girl with cystic fibrosis are facing a difficult situation as the National Institute for Health and Care Excellence (NICE) has published draft guidance deeming the life-changing drug Kaftrio too expensive to be offered on the National Health Service (NHS). Rula Samara-Sellers and husband Connor expressed deep concern about potentially losing access to the drug that could significantly improve the life of their daughter, Layla.
Layla, who is five months old, has been in and out of hospital since being diagnosed with cystic fibrosis not long after she was born. Her parents had hoped that Layla would be given access to Kaftrio, which has the potential to help her live a more normal life. The fear of losing access to this drug has been described by the parents as being like living with a “death sentence.”
More than 10,000 people in the UK have cystic fibrosis, an inherited condition that causes sticky mucus to build up in the lungs and digestive system. Kaftrio has been reported to significantly improve lung function, helping patients breathe more easily and enhancing their overall quality of life.
The decision by NICE has left Layla’s parents and many others in the UK concerned about the potential impact on their loved ones’ lives. Jemma Davidson, whose daughter Elliette, 12, also has cystic fibrosis, has supported the calls for Kaftrio to continue to be prescribed, stating that her daughter’s life had changed completely since taking the drug.
NICE has stated that it will provide an update later in the month on whether the drug will remain available to new patients. In the meantime, access to treatments will continue while the appraisal is ongoing. This will be the case for people starting the treatments as well as those already taking the treatments.