NHS Pays for Babies to Receive Revolutionary £1.8m Gene Therapy Drug
Babies in the UK have started to receive Zolgensma, from Novartis Gene Therapies, on the NHS following a confidential deal. The drug, valued at £1.8m per treatment, is one of the most expensive medicines on the market.
The deal between NHS England and Novartis Gene Therapies will surely spread hope to dozens of families.
Zolgensma provides a single dose to treat spinal muscular atrophy (SMA), a condition suffered by one in 10,000 babies and which sees few of them survive beyond their second birthday. The treatment stops the worsening of the condition, and so the earlier it is given the better.
Zolgensma contains a healthy copy of a missing or faulty gene called SMN1. That is inserted into a harmless virus, which delivers the replacement gene into the nucleus of motor neuron cells, to stop the cells from gradually dying. The healthy motor neuron cells produce the missing SMN protein, which is vital for muscle function.
Four specialist NHS centres have been selected to provide the treatment to the babies.
Before this agreement, the only two ways that babies in the UK could have been treated would either have been if the family had been able to raise sufficient funds to pay for the treatment, or if they were the lucky recipients of a generous programme in which Novartis Gene Therapies provided the drug for free to some patients - although this could only be available to a limited number of babies selected at random around the world.
Sir Simon Stevens, NHS England Chief Executive, hailed this as "fantastic news that this revolutionary treatment is now available for babies and children". He said Zolgensma was among life-changing therapies being routinely used by the NHS.
Sally-Anne Tsangarides, the General Manager at Novartis Gene Therapies in the UK, called it a "hugely important step for babies in England". She added: "We thank all those who have been involved in the landmark agreement that has made it possible."
Comment
The UK has often been criticised for not being able to pay for medicines that cost a lot of money, but this is a good news story which points to something different. This highlights that the NHS will spend money on drugs that can make a huge difference.
The next step should be new born screening for SMA, which happens in some other countries. The earlier the condition can be detected and the babies can be treated, the more impact the treatment can have. Often, by the time SMA is discovered, irreversible damage has already been done.