Kate Cardente owns her 3-month-old daughter, Ainsley, while undergoing genetic therapy for spinal muscular atrophy (SMA). SMA is a disease that is the leading genetic cause of death in children. Ainsley receives a unique infusion of Zolgensma.
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LONDON – A drug labeled “the most expensive drug in the world” has been approved by the UK’s National Health Service, a move that could change the lives of babies and children suffering from a rare genetic disorder.
Innovative gene therapy called “Zolgensma” is not cheap, with a reported list price of £ 1.79 million ($ 2.48 million) per dose, NHS England said in a statement on Monday.
The drug will be used for babies and young children suffering from spinal muscular atrophy, a rare and often fatal genetic disease that causes paralysis, muscle weakness and progressive loss of movement.
Babies born with severe type 1 SMA – the most common form of the condition – have a life expectancy of just two years.
The drug will be available on the UK health service, which provides free healthcare at the point of delivery, “at a fair price for taxpayers, following a major confidential deal with the NHS England,” its chief executive Simon Stevens said on Monday. . NHS England did not disclose the price paid.
The NHS is primarily funded by the general tax government, so the medicines and treatments it approves and uses must go through a rigorous cost-effectiveness analysis.
Zolgensma, which is manufactured by Novartis Gene Therapies (part of the US pharmaceutical drug Novartis), has been shown in studies to help babies reach milestones such as ventilator-free breathing, self-sitting and crawling and walking after one. infusion. treatment.
The latest data suggest that Zolgensma may provide a rapid and sustained improvement in the motor function of young children with type 1 SMA and may prolong their life.
80 babies and young children a year could benefit from gene therapy, the NHS said.
Life changer
Stevens said the agreement was a “life changer for young people with this cruel disease and their families.”
“Spinal muscular atrophy is the leading genetic cause of death among infants and young children, which is why NHS England has moved the mountains to provide this treatment, while successfully negotiating behind the scenes to ensure a fair price for taxpayers. ”
He said the agreement showed that while the health service remained under pressure due to the Covid-19 pandemic, the NHS was still “cared for by millions of other patients, for whom real medical progress is possible”.
The agreement with Novartis Gene Therapies provides a substantial reduction in confidentiality for NHS patients in England and paves the way for the National Institute for Excellence in Health and Care (NICE) – the public body that issues guidelines on the cost-effectiveness of medicines and treatments used by the NHS – to publish draft guidelines recommending treatment with Zolgensma.
The terms of the agreement mean that some young children who do not currently meet the NICE recommendation criteria will also be eligible to be considered for treatment by a national clinical team of the country’s leading experts in the treatment of SMA.
The NHS has said it is ready to quickly follow the introduction of highly complex and innovative gene therapy and will not wait until NICE publishes the final guidelines to begin. This approach is supported by NICE, given the importance of administering the single treatment as soon as possible, he added.
The approval of the drug marks the second medical treatment now available for young people with SMA. He added that future treatment options also look promising, as another SMA is currently being reviewed by NICE.