Page last updated:  7th April 2025

The National Institute for Health and Care Excellence (NICE) recommends which medicines NHS England should fund. To do this it reviews clinical and cost effectiveness evidence about the medicine. If it shows promising potential but there is significant uncertainty in the clinical evidence, it may be recommended for time-limited NHS funding. This is via a Managed Access Agreement (MAA).

This is a way for people to receive the new medicine, while further evidence is collected to assess its long-term benefits.

When NICE made its decision to recommend nusinersen (Spinraza), the long-term benefits for patients were still very uncertain.

In July 2019, the Nusinersen MAA was finalised.

In May 2021, Variation 1 was agreed. This followed a review of the eligibility criteria which had excluded the majority of those people who had a clinical diagnosis of SMA Type 3 and lost the ability to walk. You can read the background to this change and the part SMA UK played in contributing to this.

In February 2022, Variation 2 was agreed. This was to bring the eligibility, monitoring and stopping criteria in line as far as possible with the new MAA for risdiplam.

In November 2022, Variation 3 was agreed. This was in large part in recognition of the difficulties there had been with data collection during the pandemic, especially PROMs data.

In March 2025, Variation 4 was agreed. This extended the MAA to give NICE time to review the data via the Multi Technology Assessment (MTA – see link below) and issue new guidance.

The MAA was to last for five years, from 24th July 2019 to 23rd July 2025. The MAA will now expire automatically after six years on 30 September 2026 if it has not expired earlier as a result of the publication of the NICE reappraisal of nusinersen.

During this time, NICE recommended that the NHS should fund risdiplam. This was again via an MAA. Risdiplam started to be available via NHS funding in January 2022.

As there were now two drugs, NICE decide to review the clinical and cost effectiveness of both drugs at the same time. They set up a Multi Technology Assessment to do this.

Read more about NICE’s  Multi Technology Assessment and how SMA UK is involved >

See NICE’s Website – Nusinersen