Question reference: S6W-23235
- Date lodged: 27 November 2023
Current status: Answered by Jenni Minto on 11 December 2023
To ask the Scottish Government what its position is on whether the Scottish Medicines Consortium methodologies remain fit for purpose in the assessment of gene-edited precision medicines where a single treatment may have lifelong benefit.
In Scotland, the Scottish Medicines Consortium (SMC) appraises the clinical and cost-effectiveness of newly licensed medicines. The SMC has a transparent and robust process that is fully described on its website. It is worth noting that any SMC appraisal is undertaken independently of Scottish Ministers and is based on the clinical and cost-effectiveness of a medicine at a population level.
The SMC’s process allows companies to present a cost-effectiveness case which sets out the predicted health benefits of their treatment (including one-off treatments) over a person’s lifetime, and the SMC’s assessments follow standard methodology for such calculations. In addition, in the assessment of innovative medicines for rare conditions (including gene-edited precision medicines where a single treatment may have lifelong benefit), the SMC process involves additional flexibilities as outlined on its website. By way of an example, in 2021, the SMC was the first health technology appraisal body in the UK to accept a one-off gene therapy for spinal muscular atrophy.