Health authorities in England and Wales have approved the use of the drug Spinraza, leaving Ireland, with Estonia, as the only EU states barring access to the medicine.
The National Health Service (NHS) said it had reached what it called "one of the most comprehensive deals in the world, which allows us to assess real-world evidence of its long-term benefits".
The "managed access agreement" with pharma group Biogen will fund treatment for a limited time and collect data on the drug's effectiveness.
NHS England did not disclose the price it will pay for the drug. Biogen said it "applauded" the decision by the National Institute for Health and Care Excellence (Nice) to recommend funding for Spinraza in the United Kingdom.
Spinraza, which is administered by spinal injection every four months, is currently the only approved treatment available for patients, mostly children, with the rare muscle-wasting disease spinal muscular atrophy (SMA). The often fatal condition affects fewer than 100 people in Ireland, among them 26 children.
Nice, Britain’s healthcare cost agency, said last August that it could not recommend Spinraza as a cost-effective treatment. Ireland’s National Centre for Pharmacoeconomics (NCPE) reached the same conclusion in March.
The HSE is now looking again at the possibility of approving the drug after Biogen dropped its asking price. It is due to make its decision in the coming days.
At the time of the initial rejection back in March, the HSE said the drug would cost about €600,000 in the first year to treat each of the Irish children suffering with the disease and €380,000 a year thereafter “with an estimated budget impact in excess of €20 million over a five-year period”.
At the time, NCPE director Prof Michael Barry noted that the additional funds allocated for new medicines in 2019 – around €10 million – had already been almost exhausted by the end of February.
Biogen claimed it had submitted pricing for its portfolio of biosimilars – like-for-like therapies for expensive biologic drugs that have come off patent – that would make “significant savings to the health budget, and substantially offset the cost of nusinersen [Spinraza]”.
SMA is the leading genetic cause of infant mortality, affecting one in every 10,000 live births. About 60 per cent of patients have the most severe Type 1 form of the disease, which often leads to paralysis, impaired breathing and death by the second birthday. – Additional reporting Reuters.