The medicine is under review by Britain’s independent, government-funded National Institute for Health and Care Excellence (NICE), which decides whether a newly licensed medicine is added to the National Health Service (NHS) system.
NICE’s appraisal committee is slated to review a revised submission from Spinraza marketer Biogen on March 6, according to the United Kingdom-based publication Express & Star. A favorable NICE recommendation would mean inclusion of the treatment in the public health program of England and Wales.
The treatment, known generically as nusinersen, is the only approved therapy for nearly all types of SMA in several countries, including the United States, across the European Union, Australia, Brazil, Japan, Switzerland, South Korea, Canada, and Chile.
Although part of the United Kingdom, Scotland has its own NHS system. Pending further efficacy data collection, NHS Scotland will cover Spinraza injections at low or no cost for all of its SMA patients starting in April.
The Scottish Medicines Consortium approved Spinraza last year for use by children in Scotland with SMA type 1. Still, regulatory approval for use does not necessarily mean coverage under a respective country’s public health system.
Last August, NICE recommended against Spinraza’s inclusion in its health system, contending that its cost in terms of Quality Adjusted Life Year gained “is too high for it to be considered a cost-effective use of NHS resources.”
Cost is an issue. In the United States, the treatment carries a list price of $750,000 for the first year (when a higher dose is administered), and $350,000 annually thereafter.
In England, Wolverhampton South West Labour MP Eleanor Smith told the House of Commons that one of her constituents, 10-year-old SMA patient Heidi Prescott, was losing the ability to walk, and spends most of her time in a wheelchair.
“There is a treatment that could help Heidi to slow down the deterioration and prolong her life. It’s called Spinraza,” Smith said at Prime Minister’s Questions, a constitutional convention during which the prime minister answers questions from members of Parliament.
“It is not available in England, but will be in Scotland in April 2019. Can I ask the prime minister, why can’t this treatment be accessible to my constituent, Heidi, and other children in England with this disease?”
That’s when Prime Minister Theresa May relayed the news about the March 6 NICE meeting, adding that she was “pleased that Biogen has submitted a revised submission for the NICE appraisal committee to consider.”
NICE was established in 1999 to reduce variation in access to care and treatment across the NHS in England and Wales. Since then, its role has been expanded to include the promotion of good health, ill health prevention, and social care services.
Affecting up to 1 in 10,000 people, SMA is caused by a mutation in the survival motor neuron gene 1 (SMN1). In a healthy person, this gene makes a protein called the survival motor neuron protein, which is critical to the function of nerves that control muscles. Without an adequate level of SMN protein, motor neurons in the spinal cord are lost, preventing muscles from getting brain signals.
Survival motor neuron 2 (SMN2) is a closely related gene not thought to be affected in SMA, but does not produce sufficient functional SMN protein on its own. By targeting the SMN2 gene, Spinraza has been shown to enhance production of full-length SMN protein.