Nusinersen for spinal muscular atrophy: 24 month predicted versus actual analysis
Page last updated: 13 July 2021
Drug utilisation sub-committee (DUSC)
February 2021
Abstract
Purpose
To compare predicted and actual utilisation of nusinersen in Type I, II, and IIIa spinal muscular atrophy (SMA), as requested by DUSC at its October 2020 meeting.
Date of listing on the Pharmaceutical Benefits Scheme (PBS)
Nusinersen was PBS listed on 1 June 2018.
Data Source / methodology
Data extracted from the PBS and Authorities data maintained by Department of Health, processed by Services Australia was used for analyses.
Key Findings
- There were 140 and 160 patients treated with nusinersen during the first and second year of listing respectively, which was higher than estimated.
- There were 591 and 514 nusinersen prescriptions dispensed during the first and second year of listing respectively, which was higher than estimated.
- The most common age group in patients initiating nusinersen treatment were those aged between 0-4 years old (39.9% of patients) and overall, there was a similar gender ratio of female and male initiating patients.
- The data was too immature to analyse the time on nusinersen treatment, the median treatment duration was not reached by 30 September 2020.
- After age standardisation (0-18 years), the Australian Capital Territory (ACT) had the highest number of patients initiating nusinersen treatment relative to its population size.
- Type II SMA was the most common type of SMA in patients receiving nusinersen treatment. Similar patient numbers were observed for Type I and Type IIIa SMA.
