NICE Extends Clinical Eligibility Criteria for Spinraza

LONDON, United Kingdom – The National Institute for Health and Care Excellence (NICE) has completed its review of data collected on Spinraza (nursinersen) as part of the therapy’s Managed Access Agreement (MAA).

The Biogen drug, which is used for spinal muscular atrophy (SMA), will now be available to more patients than it previously was, following new evidence that supports a change in the MAA treatment eligibility criteria.

Specifically, the review assessed whether people with type III SMA who are unable to walk can benefit from Spinraza and therefore should be included in the MAA.

Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Assessment at NICE, explained the extension of eligibility: “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.”

The MAA review involved Biogen, patient groups, clinicians, SMA REACH UK, NHS England, and NHS Improvement.

The MAA was initially set up between NICE, NHS England, NHS Improvement, and Biogen to allow patients to access Spinraza while data can be collected to gauge its impact in certain groups for whom additional evidence is required to address uncertainties.

Spinraza MAA “One of Most Comprehensive in the World”

In May 2019, it was announced that NHS England would be funding Spinraza for pediatric patients, whereas treatment for patients with SMA types 2 and 3 would be administered once services were established.

Under the deal, which NHS called “one of the most comprehensive in the world,” patients with SMA types 1, 2, 3a, and 3b, including adults and siblings who have yet shown symptoms, should benefit from Spinraza.

The Spinraza MAA allows funding for Biogen’s drug while further data can be collected. The MAA was part of NHS’ Long Term Plan, which seeks to secure a string of “smart deals” to ensure investment in world-class innovative technologies while making sure patients access new treatments.