NHS chief hails SMA drug deal - and was that a swipe at Vertex?


Children in England with the rare muscle wasting disease spinal muscular atrophy (SMA) will get access to Biogen’s super-expensive new drug Spinraza after the manufacturer struck a deal with NHS officials.

NHS England chief executive Simon Stevens endorsed the agreement, adding what looked like a swipe at Vertex, which has been rowing with the NHS for nearly three years over the price of its cystic fibrosis drugs.

Vertex has gone as far as refusing to send files to NICE for cost-effectiveness assessments, saying that the health system in England hugely undervalues its drugs.

Simon Stevens

Stevens said: “The NHS has now reached one of the most comprehensive deals in the world, which allows us to assess real-world evidence of its long-term benefits.

“This latest deal coming on the heels of a number of other recent successful negotiations demonstrates that there is no reason for other companies not to show equivalent flexibility in order to benefit NHS patients, taxpayers and indeed themselves.”

Recent deals have seen NHS patients get access to CAR-T cancer cell therapies ahead of most other countries, and a multi-company agreement covering cheaper biosimilars of AbbVie’s Humira (adalimumab) inflammatory diseases drug.

In the US Spinraza costs $750,000 for the first year, and $375,000 thereafter, although in the UK the list price is slightly lower at around £450,000 ($581,000) for the first year.

Cost-effectiveness body NICE last year rejected the drug, arguing that it would cost the NHS around £400,000 and £600,000 for a year of good quality life for a single patient.

But NICE had called for further talks to make the drug available, which have culminated in the agreement unveiled today after further negotiations over price and other restrictions.

Biogen and NHS England have reached a “managed access agreement” where the health service will fund treatment for a time-limited period, allowing further data to be collected on Spinraza’s effectiveness.

Once NICE has published final guidance sealing the decision Spinraza will be made available immediately to the youngest and most severely affected patients with type 1 disease.

For older babies, children, and young adults with less severe SMA types 2 and 3, the NHS will provide Spinraza “shortly after” guidance is published, once the services to deliver them are established.

NHS England said in a press statement that this process is “not expected to take more than a few weeks.”

According to NHS England this allows all patients including adults and siblings who are yet to show symptoms, to benefit from the treatment.

Terry O’Regan, vice president and managing director of Biogen UK and Ireland, said the announcement “is a momentous occasion for patients and their families who have fought tirelessly for access to this life-changing medicine.

“This positive outcome has been achieved through intensive and collaborative working between the SMA community, NICE, NHS England, and Biogen. We will continue to work with health authorities to ensure this welcome decision translates into access as soon as possible for those awaiting treatment, which includes providing NHS England with access to nusinersen for type I patients immediately.”