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Life-changing cystic fibrosis drug may no longer be available due to high costs By Michael Will In January 2022, NHS England released a statement announcing that the “miracle cystic fibrosis” treatment Kaftrio would be available on the NHS after reaching a commercial agreement with Vertex Pharmaceuticals. Kaftrio is the latest, and most effective member of a group of ground-breaking cystic fibrosis treatments called CFTR modulators. Clinical trials of Kaftrio have shown remarkable success, with patients showing improved lung function, a reduction in lung infection occurrence, increases in BMI, enhanced pancreatic function, and increased fertility in females. However, in November 2023, the National Institute for Health and Care Excellence (NICE) stated that despite “large and robust evidence” of the drug’s effectiveness they cannot recommend that Kaftrio be prescribed to new patients in the future due to its high costs. Cystic fibrosis is a relatively common genetic disorder affecting over 10,000 people in the UK alone and over 100,000 globally. In 1989 it was identified that the disease is due to mutations in a single gene that encodes the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein. The CFTR protein is found in epithelial cells lining the airway, pancreas, gastrointestinal tract, reproductive tract, salivary glands, and sweat glands. The CFTR protein is a channel which controls the flow of chloride and bicarbonate, these maintain the correct salt and water concentrations in the tissues. Although cystic fibrosis affects multiple organs, damage to the lungs is the primary cause of mortality, responsible for up to 95% of deaths in cases of cystic fibrosis. The decline in lung function is due to recurring lung infections, which occur due to the thick mucus being difficult to clear and adhering to the airway. These mucus plugs become focal points for bacterial infection and growth. In the 1950s, life expectancy for someone with cystic fibrosis was under one year. With an improved understanding of the disease and new methods of managing the symptoms, the current median age of death for someone with cystic fibrosis is 29-35 years of age. Traditional treatments of cystic fibrosis focus solely on managing the symptoms rather than treating the underlying cause. Patients are given antibiotics for recurrent lung infections, physical therapy to aid in the clearance of mucus from the airways, pancreatic enzyme replacement therapy to replace those lost due to pancreatic damage and insulin to treat cystic fibrosis-related diabetes. CFTR modulators offer an exciting, novel treatment for cystic fibrosis. These small molecules bind to defective CFTR proteins and can partially restore their function. CFTR modulators finally offer the opportunity to target the underlying cause of cystic fibrosis rather than managing the symptoms. Currently, two classes of CFTR modulators have received approval for use in the UK; these are Potentiators and Correctors. These modulators target different causes of CFTR dysfunction. Potentiators improve the function of dysfunctional CFTR which is present in the membrane of cells. This helps people with mutations that result in the production of CFTR which is present in the cell membrane but is not functioning properly. Correctors result in more CFTR successfully reaching the membrane of the cell. This class of CFTR modulator aids people with mutations which cause the cells to produce misfolded CFTR. Misfolded CFTR protein is in the wrong shape and is unstable, the cell has quality control mechanisms which will degrade unstable proteins. The result is that less or no CFTR reaches the cell membrane. Kaftrio which was approved for use in the UK in June 2020, is the latest and most effective CFTR modulator drug to be made available. It is estimated that over 80% of people with cystic fibrosis have CFTR mutations which would benefit from treatment with Kaftrio. Kaftrio is a combination therapy made up of two correctors and a potentiator. It was found that using potentiators and correctors in combination had a positive impact on their effectiveness, resulting in the dual benefits of an increase in CFTR deposition on cell membranes and an increase in the functionality of the deposited CFTR. Patients in clinical trials in the UK reported that Kaftrio had improved their lung function and health to levels they never previously thought possible. A UK trial member named Ben stated that after taking Kaftrio “I am fitter and healthier now than I’ve ever felt in my entire life. Like on Saturday I rode 50 miles on a bike. (I) could never comprehend that in my entire life.” In France, use of Kaftrio has resulted in a major decrease in the number of lung transplants, as even those with advanced cystic fibrosis are also showing significant benefits from the drug. The improvement in health provided by Kaftrio and the future potential of next generation CFTR modulators has provided many cystic fibrosis patients with hope for a future that they previously did not think possible. Cynthia, another patient in UK clinical trials stated that the improvement in her condition due to Kaftrio had her thinking ahead to a future which previously did not seem possible, she stated “maybe I could get that retirement plan, maybe I should think about that. I never got that lifetime ISA because I was never going to get to 50 to use it. I thought I had another 15 years tops.”. Unfortunately, CFTR Modulator drugs are currently undeniably very expensive, and it is likely that many health services around the globe will be unable to afford Kaftrio at the current prices which are £100,000 to £160,000 per patient per year. It is due to these extraordinary costs that NICE declared that it was unable to declare that Kaftrio and other CFTR modulators would be an “acceptable use of NHS resources”. This decision has not yet been made final with a further evaluation meeting due to be held on the 14th of December 2023. Those with cystic fibrosis hold out hope that the NHS and Vertex Pharmaceutics can come to a new commercial arrangement. However, with the exclusive patent on Kaftrio not due to expire, in Europe and the US, until 2037 it may be some time until the potential huge benefits of CFTR modulators are available to many with cystic fibrosis due to the high cost.