The UK’s National Institute for Health and Care Excellence (NICE) has extended the clinical eligibility criteria for Biogen’s spinal muscular atrophy (SMA) drug Spinraza following a review of data collected as part of a managed access agreement (MAA).
Initially, NICE recommended funding for Spinraza (nusinersen) through the MAA scheme to enable patients to access the drug while more long-term data on its efficacy was gathered. At that time, the recommendation covered Spinraza use for certain patients with types I and II SMA.
The MAA, an arrangement between NHS England, NHS Improvement and Biogen, is designed to address the financial risk and challenges for implementation in the NHS and differs from routine commissioning.
The review assessed whether new evidence could be used to support a change in the MAA treatment eligibility criteria, particularly regarding if people with type III SMA who are unable to walk can benefit from the drug and should be included in the MAA.
“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,” said Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Assessment at NICE.
“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,” he added.
“Biogen is delighted by this decision for non-ambulant type III SMA patients who previously had no therapeutic options available. Biogen remains fully committed to supporting NHS England to ensure this positive decision translates into services for all eligible patients, enabling access to nusinersen, without delay,” commented Samuel Cooper, head of rare diseases business unit at Biogen UK and Ireland.