Proxy-Reported Quality of Life and Access to Nusinersen Among Patients with Spinal Muscular Atrophy in Saudi Arabia.

In a recently published study that assessed the impact of nusinersen among seven adult SMA patients with types II and III in Germany who have been followed up for at least 10 months and used valid assessment scales, such as, the Hammersmith Functional Motor Scale Expanded (HFMSE) and the Revised Upper Limb Module (RULM), there was a reported improvement in the patients’ motor function; however, these changes did not reach statistical significance. 17 Additionally, the high list price of this drug makes it not cost effective at various traditional cost effectiveness thresholds. 18 , 19 Using the cost effectiveness threshold of €195,600 per Quality Adjusted Life Year (QALY), which is considered a reasonable threshold for rare diseases in Sweden, nusinersen was not found to be cost effective from the perspective of the health technology assessment body in Sweden, also known as the Dental and Pharmaceutical Benefits Agency.

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