Appraisal of risdiplam in Scotland delayed further

03 August 2021

SMA UK, MDUK and TreatSMA have today written to the Scottish Medicines Consortium (SMC) to raise their concerns about the further delays with processing Roche’s submission for risdiplam.

Though we understand this delay is due to the staff and appraisal committees’ workloads, the Scottish SMA community is already facing considerable difficulties because, although nusinersen has been accepted for restricted use within NHS Scotland for both children and adults, there is as yet still no delivery of this treatment to adults. Additionally, due to nusinersen’s intrathecal method of delivery, as it can be challenging to gain access for the majority with severe scoliosis / spinal surgery, it is potentially considered to be clinically unsuitable.

Until 1st July, those who have SMA Type 1 or 2 for whom nusinersen was considered to be clinically unsuitable, were able to enrol in the risdiplam EAMS. This opportunity is now closed. The risdiplam EAMS was never an option for anyone in the UK who has SMA Type 3. This effectively means that for any new child who has SMA Type 3 for whom nusinersen is considered to be clinically unsuitable, there is no treatment option across the UK. However, for adults in Scotland, whatever their SMA diagnosis, the picture is far more bleak.  Not only is there no longer any possibility of risdiplam via the EAMS for those who have Type 1 and 2 for whom nusinersen isconsidered to be clinically unsuitable , but also effectively, there is no adult treatment option available at all because of the delay in rolling out nusinersen to adults in Scotland.  

As SMA is a progressive condition and every day matters, we urge the SMC to consider giving priority to the appraisal process for risdiplam.