According to a story from express.co.uk, Elle Morris was only 11 years old when she died from a severe lung infection. Elle was born with cystic fibrosis, a serious genetic disorder that causes progressive decline in lung function. Unfortunately, the circumstances of her untimely death were partially the result of where she lived: The UK. The country’s National Health Service (NHS) has refused to approve Orkambi, one of the few treatments designed to treat the disease. Now, Elle has become the face of the campaign to get the drug covered.
About Cystic Fibrosis
Cystic fibrosis is a type of genetic disorder which can have impacts throughout the body, but it is most characterized by the build up of abnormally thick, sticky mucus in the lungs. This mucus becomes a fertile breeding ground and habitat for potentially infectious bacteria. Many patients must take antibiotics for much of their lives. This disorder is caused by mutations of the CFTR gene. Symptoms of cystic fibrosis include progressive decline in lung function, lung and sinus infections, coughing up mucus, fatty stool, poor growth, infertility in males, clubbed digits, and digestive problems. Treatment includes antibiotics and medications or procedures intended to maintain lung function. Lung transplant is an option when lung function declines severely. Life expectancy ranges into the 40s and 50s with good care. To learn more about cystic fibrosis, click here.
Struggling for Coverage
The NHS has rejected Orkambi for coverage over concerns about cost effectiveness. The drug is extremely highly priced and there have been some concerns about its efficacy. However, many patients that use it nevertheless see improvements. The NHS has been in negotiation with Vertex, the company that owns Orkambi, but so far there has been little progress.
Elle was granted access to Orkambi via compassionate use, but it was only after a seven month wait. Elle’s cystic fibrosis was severe and she eventually underwent a double-lung transplant. However, she ultimately died from lung infections in January 2018.
Elle’s mother Becky Whitfield said that she felt honored when she was asked about using Elle’s likeness as part of the campaign to get Orkambi approved.
“I do not want any other CF parent going through the pain that we went through with Elle,” Becky said.