Cystic Fibrosis Courage: A Mother of Two Patients Tells All

According to a story from The Sunday Post, it is an interesting time to be a cystic fibrosis patient in the UK these days. Just weeks after The Scottish Medicines Consortium (SMC) announced its rejection of two critical drugs for the disease, the drugs were suddenly approved in a reversal of the prior decision after an agreement with the drugs’ manufacturer, the US pharma company Vertex, was reached. Mother Angela Glassford, whose son and daughter both have cystic fibrosis, describes the challenges they have faced.

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.

No Easy Day

Angela describes an overwhelming situation in which her children are forced to reckon daily with their diagnosis while still trying to have some semblance of a regular existence. The children must endure a dizzying array of therapies, surgeries, and procedures in an uphill struggle to slow the progression of their disorder. Nevertheless, the approval of Orkambi and Symkevi for coverage on the NHS is cause for celebration and could make a major difference in the lives of her kids.

However, Angela says that regulators like the SMC need to have a more efficient and faster-moving decision making process in place. The process also needs to take into account the perspectives of patients, not just a detached cost-benefit analysis. At the end of the day, cystic fibrosis is a progressive disease that worsens over time, so any treatment delays translate directly into lost survival time for that patient.

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