NICE finds more SMA patients eligible for £75,000 a vial drug

pharmafile | May 4, 2021 | News story | Manufacturing and Production, Sales and Marketing NICE, sma 

NICE has announced that more people with the rare genetic disorder spinal muscular atrophy (SMA) are to benefit from nusinersen, following a review of data collected as part of the Managed Access Agreement (MAA).

Nusinersen, also known as Spinraza, was created by Biogen and is the first known treatment for SMA. Clinical trials for Spinraza showed promising results in type 1, 2 and 3 SMA, with children with SMA type 1 being able crawl and even walk for the first time following treatment.

However, the drug is one of the most expensive in the world costing £75,000 per vial (excluding VAT; British National Formulary, accessed June 2018) meaning the total annual treatment cost is £450,000 for the first year and £225,000 for subsequent years.

Due to the extraordinary cost of the treatment, the MAA was created between NHS England and NHS Improvement and Biogen in 2019. It allows patients to access treatment with Spinraza whilst collecting data on its impact in groups where additional evidence is required to address the uncertainties identified by the independent NICE appraisal committee in its original assessment.

The MAA is also designed to address the financial risk and challenges for implementation in the NHS, it is not routine commissioning of nusinersen in line with its marketing authorisation.

NICE recently launched a review of MAA data, involving Biogen, patient groups, clinicians, SMA REACH UK, and NHS England and NHS Improvement. The review looked at new evidence that would support a change in the MAA treatment eligibility criteria, finding that type 3 SMA patients who are unable to walk would benefit from Spinraza and should therefore be included in the MAA.

Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Assessment at NICE, said: “There are people with SMA who are not able to access treatment with nusinersen under the terms of the MAA which began in July 2019.

“At the time we made a commitment that we would review new evidence on the potential benefits of nusinersen for type III SMA patients who are not currently receiving it. We are therefore pleased that the review has concluded that it is appropriate to extend the clinical eligibility criteria to allow access to nusinersen for type III SMA patients who aren’t able to walk.

“It will also allow the removal of the rule which meant that patients who had lost the ability to walk needed to regain that ability within 12 months of treatment in order to be eligible for further treatment.”

Service capacity to deliver nusinersen to this wider NHS patient cohort is being developed and will expand further as COVID-19 restrictions on social distancing ease. NHS centres will be in touch with patients as this medicine is rolled out further, according to a statement from NICE.

John Stewart, National Director of Specialised Commissioning, said: “It is fantastic news that the NHS is now able to offer many more patients this potentially life changing treatment – the first of its kind to tackle the causes of spinal muscular atrophy.

“This follows another recent breakthrough for babies and children living with type I SMA after the NHS struck a deal for Zolgensma, the ‘world’s most expensive drug’, making it available for patients at a fair price for taxpayers.”

Kat Jenkins

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