Patient groups conducted a survey of those who have SMA Type 3 who were ineligible for treatment and the impact of this exclusion on them. The survey results were collated by SMA UK, presented to the Managed Access Agreement Oversight Committee and forwarded to Biogen. We heard directly from individuals / from parents of 37 children / adults who have no access to treatment.

NICE announced their promised MAA review of this eligibility rule. The review, which would involve Biogen, patient groups, clinicians, SMA REACH UK and NHS England and NHS Improvement, would assess whether new evidence has become available to support a change in the MAA treatment eligibility criteria. Biogen had until 7th December 2020 to submit its evidence. SMA UK, other patient groups and clinicians would also submit any national and international evidence they had collected. All evidence would first be reviewed by an independent External Assessment Centre (EAC) who would then make a recommendation to the Managed Access Oversight Committee (MAOC). Representatives of all the above groups would continue to be part of the MAOC which would then review the evidence and recommendation of the EAC.

Until such time as the outcome of the evidence review would be known, NHS England and NHS Improvement (NHSE&I) agreed to suspend the stopping criterion for paediatric patients who had not regained independent ambulation after 12 months of treatment

SMA UK provided the following evidence to NICE via Biogen:

Submission Summary >

Testimonies from Adults Treated in Europe >

The Impact of not Being Eligible to Access Treatment – Survey Results >

SMA UK took part in this review as a member of the MAA Oversight Committee.

4th May: NICE announced a positive decision to change this rule.