NICE rejects funding for gene modulator drugs for new Cystic Fibrosis patients.

A local young student at Sir John Leman High School – Isabella Ellyatt, unfortunately suffers from Cystic Fibrosis (CF). After receiving her diagnosis, without hesitation she began treatment of the gene modulator drug. Positive results came flooding in after starting this treatment. Results from her FEV test (measures amount of breath a person can exhale in one second) increased from 40% in June to 53% in October. Herself and her mother have noticed a significant improvement within her physical and mental health. This includes a change in height, healthy weight gain and a more energetic personality. Due to the Gene modulator drug, it has improved an immense amount of people’s lives locally and nationally. Isabella Ellyatt stated that after undertaking this treatment: “I feel a lot better than I did before.”

On the 3rd of November, NICE stated that although the gene modulator drug is clinically effective (Kaftrio, Orkambi and Symkeri) it is too expensive for the NHS. Many people across the country are infuriated by this as many parents begin to struggle with their new-borns struggling with CF symptoms. Once Isabella Ellyatt heard this news, she strongly believes that: “they should let everyone have the chance to have the pill.”. This heart-breaking story not only effects our local people, but it interferes nationally as new patients will no longer be able to receive this treatment.

What does the gene modulator drug do?

The gene modulator drugs (also known as Kaftrio, Kalydeco, Orkambi and Symkeri ) can successfully correct misfoldings in the faulty proteins in people with delta 508. This treatment can increase patient's uptake into the cell membrane and helps channels remain open at all times. This enables the body to function effectively and helps tremendously by preventing future respiratory infections. By following this treatment, (triple combination therapy) it can span CF patients' life expectancy from ages 40s to 80s!

Why do new patients desperately need this drug?

Cystic Fibrosis (CF) is an inherited condition that can affect exocrine glands which produces abnormally thick mucus. This leads to the increase of blockage to the pancreas, intestines and bronchi which further results in dangerous respiratory infections. The main symptoms vary for CF which includes frequent chest infections, wheezing with shortness of breath, demanding a vast diet in comparison to others to be able to maintain a healthy weight as well as frequent bowel movements. CF patients are more likely to develop further conditions as they age towards adulthood. This includes CF-related diabetes and osteoporosis (bone disease causing thin, weak bones). Therefore, without the gene modulator drug, CF patients will no longer be able to carry out a normal daily lifestyle in comparison to a healthy person.

In conclusion, 4wk consultation, committee meeting will occur in December to further discuss what should happen to new CF patients who can no longer access the gene modulator drug.