The Nusinersen Managed Access Agreement (MAA)
The Nusinersen Managed Access Agreement (MAA)
Page last checked: 16th December 2022
Last updated: 16th December 2022
NICE makes recommendations on new medicines by reviewing clinical and cost effectiveness evidence. When a medicine shows promising potential but there is significant uncertainty in the clinical evidence, it may be recommended for time limited NHS funded access in England as part of a Managed Access Agreement. This is a way for patients to receive promising new treatments, while further evidence is collected to assess the long-term benefits of a new medicine. Based on the evidence available when NICE made its decision about recommending nusinersen (Spinraza) the long-term benefits for patients were still very uncertain.
The initial Nusinersen Managed Access Agreement was finalised in July 2019. You can read this here.
In May 2021, Variation 1 was agreed following 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 ambulation. You can read the background to this change and the part SMA UK played in contributing to this change, here.
In February 2022, Variation 2 was agreed. This was to bring the eligibility, monitoring and stopping criteria in line as far as possible, given their different methods of administration (intrathecal injection versus oral), 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.
This MAA has been agreed between:
- NICE
- NHS England and NHS Improvement
- Biogen (the drug company that makes Spinraza™)
The following groups / individuals have signed up to the agreement:
- SMA UK (Patient Group)
- Muscular Dystrophy UK (Patient Group)
- TreatSMA (Patient Group)
- Professor Francesco Muntoni, FRCPCH, FMedSci: Dubowitz Neuromuscular Centre and MRC Centre for Neuromuscular Diseases (a lead clinician who represents the healthcare professionals at treatment centres and the SMA REACH UK registry)
This MAA will last for five years, from 24th July 2019 to 23rd July 2025.
The MAA data collection period will last for a minimum of three years and automatically cease at the end of the sixth year (July 2025), unless NICE guidance is published sooner.
The MAA has been designed to allow enough time for additional evidence to be generated for NICE. At the end of the MAA period, NICE will review the new evidence and review its guidance to indicate whether the medicine should be recommended to use in the NHS – this may result in a difference to what the NHS will pay for the drug for example. While most topics recommended for managed access go on to be recommended for routine use on the NHS, there is no guarantee that it will be recommended when it is reviewed by NICE.
When Biogen resubmits the new evidence available to NICE it will include the data that’s been collected from patients throughout the MAA (see What data is being collected and why?). Patient groups and clinicians can also contribute to this process and make submissions.
The NICE appraisal committee will then evaluate the evidence submissions. They will consider both the clinical and cost effectiveness of the drug and make a final decision on whether nusinersen should continue to be funded on the NHS after the MAA has expired.