The UK’s National Health System is considering cutting off access to life-altering medications for patients who have cystic fibrosis (CF) due to the expensive cost. The greater cost, however, could be the loss of life that occurs when the medications are not accessible. One mother is speaking out about the impending decision.
“We are being forced into a situation where we are going to have to watch our daughter deteriorate year on year until she dies at a very young age,” said Charlotte Perkins, whose 17-month-old daughter Delilah has CF. “I just can’t do that.”
CF is an inherited condition that causes thick mucus to build up throughout the body, affecting multiple organs and leading to difficulty absorbing nutrients and trouble overcoming respiratory illnesses, among other concerns. About one in every 2,500 babies born in the UK has CF. The median life expectancy for a person with CF in the U.S. reached an all-time high of 53 years since the creation of the new life-saving medications that the NHS is looking to cut. Just 15 years ago, the median age of survival was the mid-30s.
In the UK, the NHS states the median age of survival with CF is currently age 40.
Until recently, CF treatments only worked to try to keep symptoms at bay. The new medications, Kaftrio, Symkevi, and Orkambi — which have different names in the U.S. — work to fix the defect that is caused by CF and thereby thin out the thick mucus that would clog the patient’s body systems. Each of the medications is at different stages of approval and has not necessarily been approved for all age groups yet.
The National Institute for Health and Care Excellence (NICE) has issued a draft guidance that states patients currently taking the life-extending medications can remain on them, but newly diagnosed patients or those who have not yet begun to take the medications due to their age or type of CF mutation will not be allowed to take them.
“We were told to ignore the life expectancies that we were reading online, we were told that children weren’t going to die of this disease anymore,” said Perkins. They had been told Charlotte could begin taking Kaftrio when she turned six. “It’s just unthinkable that they can take these children’s lives away from them, when there is a drug that can stop it, give them a full and healthy life.”
Another little girl named Charli was scheduled to begin taking Kaftrio around Christmas time, but her mother Catherine Mayor was told that this may not happen as planned.
“Knowing that this drug is out there, ready for her, but we can’t have it potentially because of the cost. It’s absolutely, it’s just disgusting. It’s mind blowing,” she said. “It’s absolutely devastating, to be told that everything’s going to be okay, your daughter will have a long, healthy life… now to be told, ‘oh actually, these drugs are really, really expensive, and we may not be able to afford them’.”
But CF patients in the UK aren’t the only ones struggling to get the medication or being told access will be cut off.
Canada’s government-run health care programs have approved the medications in some provinces, but not all, and they all appear to have different rules regarding the medications. Some have not yet approved the medications while others have approved the medications for certain age groups or for individuals with a certain level of lung function.
In Colorado, patients and doctors are worried that the CF miracle medication Trikafta may soon no longer be available in the state. Hannah Pfeiffer was on the transplant list and could hardly walk across the room before she began Trikafta. After she began taking the medication, she began going on seven mile hikes. She was even taken off the transplant list.
However, the price for Trikafta is just under $300,000 a year, which caught the attention of Colorado’s new Prescription Drug Affordability Board. Currently, people with CF in Colorado have access to the medications, and Pfeiffer said she pays about $15 a month for Trikafta and that there are co-pay assistance programs to help cover the costs. She worries that if the price is capped, the manufacturer of Trikafta will no longer sell it in Colorado at all.
A spokesperson for the charity Cystic Fibrosis Trust said its important to remember that the draft guidance from NICE is “not the final decision.”
They said, “We are hopeful that this uncertainty will get resolved in the coming weeks and we have written to the secretary of state and are urging Vertex, NICE, and the NHS to swiftly work together to find a solution.
“We want to urge that the best thing people can do right now is to submit evidence to the NICE consultation before 17:00 on 24 November, and encourage others to do so as well.”
Helen Knight, director of medicines evaluation at NICE said, “We are evaluating the cost-effectiveness of these cystic fibrosis medicines to ensure that taxpayers continue to get value for money.”
