Rare Voices Australia (RVA) welcomes the Hon Mark Butler MP’s announcement that, from 1 August 2022, the Pharmaceutical Benefits Scheme (PBS) listed treatment for spinal muscular atrophy (SMA) will be extended to adults whose symptoms appeared before 19 years of age. Read more.
Until now, there have been no treatment options available on the PBS for adults living with SMA. Advocating for access to this treatment for adults living with SMA has required much persistence and a solutions-focused approach. RVA had previously provided consumer comments regarding this treatment that highlighted the inequity of restricting access to subsets of a rare disease community, and the importance of utilising real-world evidence in Health Technology Assessment (HTA) in the absence of traditional clinical trial data.
Congratulations to RVA Partner Spinal Muscular Atrophy Australia and their community for their dedication to advocating for the best outcomes for the SMA community!