Advocacy for Access to Treatments
Advocacy for Access to Treatments
We have been advocating for access to treatments in the UK since autumn 2016 – it continues to be a high priority. We are also members of the UK SMA Newborn Screening Alliance working for the inclusion of SMA in the newborn heel prick blood test.
Advocacy for access to Nusinersen and Risdiplam in 2024
Both treatments have been funded via time limited Managed Access Agreements (MAA) which come to an end this year (2024).
When two or more treatments are used for the same condition, NICE reviews them at the same time. They set up a Multiple Technology Appraisal (MTA) and have done this for these two treatments.
NICE will look at:
- all the evidence that has been collected while the MAAs have been in place
- any new evidence from clinical trials
- international studies
SMA UK, Muscular Dystrophy UK (MDUK) and TreatSMA are consultees to the appraisal committee; this means we have the opportunity to contribute to the decision-making process in a number of different ways. Find out more here
