Australian patients are being forced to wait as decision-makers focus on spending as little as possible on a breakthrough therapy while their counterparts in England are celebrating its "miracle" impact on patients one year after it was funded by the National Health Service (NHS).
England's NHS issued a statement over night marking the one year anniversary of its funding for Vertex's cystic fibrosis therapy TRIKAFTA (elexacaftor, tezacaftor, ivacaftor and ivacaftor). In the UK, it is called KAFTRIO.
"Thousands of cystic fibrosis patients in England have had their health drastically improved by a 'miracle' treatment, allowing one to give birth to a baby, since the NHS made it available exactly one year ago today," said the NHS in a statement.
"In the past 12 months, NHS patients in England benefited from one of the fastest rollouts in the world of the cystic fibrosis triple-therapy, KAFTRIO, alongside treating hundreds of thousands of seriously unwell patients with COVID."
The NHS said nine-in-10 patients with cystic fibrosis in England now have access to therapies that patients describe as a “miracle”.
According to Professor Stephen Powis, NHS national medical director for England, “Despite the pandemic and the incredible efforts to treat more than 420,000 with coronavirus in hospitals, NHS staff have also delivered one of the fastest rollouts of KAFTRIO in the world, transforming the lives of patients with cystic fibrosis.
“Stories of people like Laura, who have gone on to have miracle babies, is yet another example of how the NHS is offering the latest life-changing treatments for patients and their families."
Laura, now 30, started treatment on KAFTRIO one year ago and her health has improved to the extent she has given birth to a baby boy.
“My KAFTRIO journey has been incredible, not just for me and my health but to start a family as well – to be given this one amazing child that I have wanted for so long. Within the space of a few hours of starting the treatment it started to work – it is the miracle drug," she said.
The UK's minister for health, Sajid Javid, said, “I’m delighted to see so many patients benefitting from this life-changing cystic fibrosis drug. Laura’s incredible story shows how these treatments can truly transform people’s lives, giving them a better quality of life for longer.
“I want to thank NHS staff for making this one of the fastest rollouts of Kaftrio in the world – a remarkable accomplishment. I continue to be inspired by their spirit and determination, especially in the midst of the pandemic, to improve as many lives as possible.”
The NHS said the funding of KAFTRIO was "one of a package made possible" by its advanced commercial capabilities that have "delivered an innovative gene-therapy to cure blindness and the world’s most expensive drug, ZOLGENSMA, which can cure paralysis in babies."
Andrew Wilfin, the medical director of Vertex in UK and Ireland, said, “The innovative agreement that was reached between Vertex and NHS England in advance of the marketing authorisation of KAFTRIO meant immediate access for eligible patients as soon as the licence was granted in August 2020.
“Since then, our data shows that the uptake of the medicine in England amongst eligible patients is one of the fastest we have seen globally. We are delighted that so many people with CF have been able to benefit and we continue to collect real world data which further demonstrates the value that the medicine brings to patients.”
