NHS approved drug costing $ 2.5million

LONDON – A drug that has been called the ‘world’s most expensive drug’ has been approved by the UK’s National Health Service in a move that could change the lives of babies and children with a rare genetic condition.

The innovative gene therapy called “Zolgensma” isn’t cheap, with a list price of £ 1.79million ($ 2.48million) per dose, NHS England said in a statement on Monday.

The medicine will be used for babies and young children with spinal muscular atrophy, a rare and often fatal genetic condition that causes paralysis, muscle weakness and progressive loss of movement.

Babies born with severe type 1 ADS – the most common form of the disease – have a life expectancy of just two years.

The drug will be available on the UK health service, which provides free medical care at the point of delivery, “at a price that’s fair to taxpayers after a historic confidential agreement reached by the NHS England,” chief executive Simon Stevens said Monday. . NHS England has not disclosed the price paid.

The NHS is primarily funded by the government from general taxation, so the drugs and treatments it approves and uses must go through rigorous cost-benefit analysis.

Zolgensma, which is manufactured by Novartis Gene Therapies (which is part of the US pharmaceutical company Novartis), has been shown in studies to help babies achieve milestones such as breathing without a ventilator, sitting alone, and crawling and walking past. only one infusion. treatment.

The latest data suggests that Zolgensma can quickly and sustainably improve motor function in young children with type 1 SMA and prolong their life.

Up to 80 babies and young children a year could potentially benefit from gene therapy, the NHS said.

Stevens said the deal had “changed the lives of young people with this cruel disease and their families.”

“Spinal muscular atrophy is the leading genetic cause of death in babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating behind the scenes to secure a price fair to taxpayers. “

He said the deal showed that while the health service remained under pressure due to the Covid-19 pandemic, the NHS was also “caring for millions of other patients, for whom real medical progress is now possible “.

Deal with Novartis Gene Therapies secures drug for NHS patients in England at a substantial confidential price and paves the way for the National Institute for Health and Care Excellence (NICE) – the public body that provides advice on cost-effectiveness 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 referral criteria will also be eligible for treatment by a national clinical team made up of the country’s leading experts in the treatment of SMA.

The NHS has said it is ready to speed up the introduction of the highly complex and innovative gene therapy and will not wait for NICE to release final guidelines to begin. This approach is supported by NICE given the importance of administering the spot treatment as early as possible, he added.

The drug’s approval marks the second medical treatment now available for young people with ADS. He added that future treatment options also look promising, with another SMA drug currently being reviewed by NICE.