LEEDS, UK – The parents of a five-month-old girl diagnosed with cystic fibrosis are expressing their fear of losing access to a life-changing drug that could improve her quality of life. Layla has been in and out of the hospital since her diagnosis, and her parents had hoped that she would be given access to Kaftrio, which could significantly improve her lung function and overall well-being.
The National Institute for Health and Care Excellence (NICE) published draft guidance deeming the drug too expensive to be offered on the NHS, costing £200,000 per year for each patient. An update is expected later this month on whether the drug would remain available to new patients, while those currently taking the drug will be able to continue regardless of the outcome.
Mum Rula Samara-Sellers expressed her worries, saying that she and her husband had not been able to sleep properly due to fears that Layla would not be able to access the drug when she turns two. She emphasized the ethical and inhumane decision to put a value on a child’s life, stating that it felt like being handed a death sentence.
Another mother, Jemma Davidson, whose daughter Elliette, 12, also has cystic fibrosis, supported calls for the drug to continue to be prescribed. She highlighted the significant positive impact Kaftrio had on her daughter’s life and expressed concerns about other patients not being able to access it.
NICE has stated that they are “liaising with key stakeholders to determine the most appropriate next steps and will provide an update later this month.” They reassured that access to treatments will continue during the ongoing appraisal, supporting both new patients and current patients taking the treatments.
More than 10,000 people in the UK have cystic fibrosis, and Kaftrio has shown to significantly improve lung function, which is crucial for patients’ overall quality of life. The ethical and financial considerations around accessibility to life-changing drugs like Kaftrio continue to raise concerns and reveal the significant impact on patients and their families.