- NHS England and Novartis have come to a “landmark” agreement to make Zolgensma (onasemnogene abeparvovec) available for spinal muscular atrophy (SMA) type 1 patients on the UK’s NHS
- The one-time gene therapy can help babies to reach milestones such as breathe without a ventilator, sit up on their own and move and walk after a single injection, but has a £1.79 million per dose price tag
- NICE has now published its draft guidance, which will be open for comments until 6 April 2021, when a meeting will then be held to discuss the findings on 13 May 2021
LONDON, United Kingdom – NHS England and Novartis have come to a “landmark” agreement to make Zolgensma (onasemnogene abeparvovec) available for spinal muscular atrophy (SMA) type 1 patients on the UK’s NHS.
The one-time gene therapy can help babies to reach milestones such as breathe without a ventilator, sit up on their own and move and walk after a single injection, but has a £1.79 million per dose price tag, making it officially the world’s most expensive drug.
However, the two parties have struck a deal to secure the innovative gene therapy at a “substantial” confidential discount.
The agreement is a “life-changer for youngsters with this cruel disease and for their families,” noted NHS England Chief Executive Sir Simon Stevens.
He continued, “Spinal Muscular Atrophy is the leading genetic cause of death among babies and young children, which is why NHS England has moved mountains to make this treatment available, while successfully negotiating hard behind the scenes to ensure a price that is fair to taxpayers.
“Although the health service is still under real pressure from covid, and NHS England is also focused on leading the national covid vaccination rollout, today’s agreement is an important reminder that the NHS is looking after millions of other patients too, for whom real medical advances are now possible.”
The initial deal paved the way for the National Institute for Health and Care Excellence (NICE) to publish draft guidance recommending treatment with Zolgensma.
NICE has now published its draft guidance, which will be open for comments until 6 April 2021, when a meeting will then be held to discuss the findings on 13 May 2021.
In the document, the organization determined that because of the uncertainty in the clinical data, the cost-effectiveness estimates for Zolgensma treating type 1 SMA are uncertain. However, they are likely to be within a range of what NICE considers an effective use of NHS resources for highly specialized technologies.
Further, the consultation clarified that because of the limited trial data for children aged 7 to 12 months, their treatment should be discussed by a national multidisciplinary team.
Following the decision, NHS England will begin a “rigorous” process to identify centers of excellence to provide the full range of services required to administer the treatment safely, with new specialist services treating patients later this year.
Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE, said: “The committee concluded that Zolgensma represents an important development in treating SMA which could not only allow babies to gain important motor milestones such as independent sitting and walking but for some babies who are diagnosed before they have symptoms, it might come close to being a cure.”
Up until now, NICE has recommended the disease-modifying therapy Spinraza (nusinsersen) for some people with pre-symptomatic SMA and types 1, 2 or 3 SMA as part of a managed access agreement.
NHS Smart Deals
The arrangement is the latest in a series of “smart deals” that NHS England has agreed to secure innovative medicines for patients. Over the last few years, the NHS has begun a process to identify and pull transformative therapies and technologies into the organization more quickly, hoping to change and modernize the way it treats disease.
Potentially one of the most transformative agreements was the securing of a deal with Vertex Pharmaceuticals at the end of last year to make all three of their UK-licensed cystic fibrosis (CF) medicines available, following a tempestuous three-year back and forth between Vertex and the NHS. After the National Institute for Health and Care Excellence (NICE) rejected Orkambi (ivacaftor/lumacaftor) on cost-effectiveness grounds in 2016, the company refused the NHS’ compromise of a £500 million, five-year funding deal. The two reached a seeming impasse, until the implementation of a managed access agreement (MAA).
Other such “smart deals” include the health service’s agreement to bring Gilead’s Yescarta (axicabtagene ciloleucel) to lymphoma patients, the first agreement of its kind in Europe, and a comprehensive settlement to get Biogen’s Spinraza to patients living with SMA.
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