England’s drug cost regulator has stopped short of endorsing Evrysdi, Roche’s treatment for the rare genetic disorder spinal muscular atrophy, but will make it available to patients as it gathers more data.
The National Institute of Health and Care Excellence (NICE) has published guidance recommending Evrysdi for its managed access agreement (MAA) program. The move allows the National Health Service to examine a therapy further with real-world, long-term evidence while offering it at a discounted price.
It also allows NICE to further assess the treatment from a pricing perspective. The list price of the Evrysdi is £7,900 ($10,663) per 60 mg (80 ml) vial.
The MAA status for Evrysdi will extend to March 2024, when Roche is expected to resubmit new evidence. NICE explained that it could not recommend Evrysdi for full commissioning at this time because “cost-effectiveness estimates were substantially higher than the range normally considered a cost-effective use of NHS resources.”
NICE took the action despite agreeing that Evrysdi can be considered a life-extending treatment. In draft guidance earlier this year, NICE turned down the medicine.
The move makes the treatment available to the roughly 1,500 people in England and Wales with SMA. The disease is a progressive neuromuscular condition that leads to muscle weakness, loss of movement and difficulties with breathing and swallowing. Those with the Type 1 form of the disease usually die before reaching the age of 2.
In making its recommendation, NICE acknowledged the severity of the disorder and the attractiveness of the option that Evrysdi provides as an oral treatment. Biogen’s Spinraza, the first among a group of new SMA treatment options, is injected directly into the spinal cord. NICE expanded access to that drug earlier this year.
The new oral option "should lead to greater adherence to treatment, along with giving access to a treatment to those who aren’t able to have other currently recommended options,” Meindert Boysen, the director of NICE, said in a statement.
Novartis’ Zolgensma, a one-time gene therapy, has been approved for children aged 2 and younger. The company is working to expand the drug’s reach to older patients with a new formulation. Despite its cost of £1.79 million ($2.03 million), it won a NICE recommendation earlier this year, making it the most expensive drug ever sanctioned by the organization.