AQW 7480/22-27

Mr Danny Donnelly
Alliance Party
East Antrim

Tabled Date: 26/02/2024
Answered On Date: 28/02/2024
Priority Written: Yes

Question:
To ask the Minister of Health what action he will take to ensure the continuity of the provision of Risdiplam for people with neuromuscular conditions.

Answer:

NICE TA755, when originally issued in December 2021, initially recommended the use of Risdiplam as an option for treating 5q spinal muscular atrophy (SMA) in people 2 months and older with a clinical diagnosis of SMA types 1, 2 or 3 or with pre-symptomatic SMA and 1 to 4 SMN2 copies. In accordance with DoH Circular HSC (SQSD) 12/22 the Department of Health made provision for patients on this basis.

In December 2023, NICE updated its recommendation to include people of all ages with a clinical diagnosis of SMA types 1, 2 or 3 or with pre-symptomatic SMA and 1 to 4 SMN2 copies to take account for the license extension to the marketing authorisation.

In accordance with DoH Circular HSC (SQSD) 12/22 which sets out the process for endorsement, implementation, monitoring and assurance in Northern Ireland, the Department endorsed the updated NICE TA as applicable in Northern Ireland on 26 January 2023.

My Department have issued a Service Notification to HSC Trusts and other relevant providers and stakeholders on 19 February 2024 setting out the expectations for implementation.

Subsequently, hospital clinicians, on behalf of their patients are currently able to make application to the SPPG for the use of Risdiplam for all ages who meet the necessary criteria.

The Department is aware that the lead adult neuromuscular consultant is due to leave post at the end of March 2024 and my Department is currently engaging with Trusts to put arrangements in place for the continuation of this treatment for adults in 2024/25 and beyond.