Clinical and economic assessment of nusinersen: the Bulgarian perspective

ABSTRACT Introduction Spinal muscular atrophy (SMA) is a very serious debilitating rare condition mainly affecting newborns and infants. Areas covered The aim of current chapter is to present the standard of care and treatment available in Bulgaria from both clinical and economic point of view. The authors are presenting the latest clinical studies in the area of this rare neuromuscular disorder as well as describing a very detailed economic evaluation from the perspective of Bulgarian healthcare insurance fund regarding Nusinersen. A systematic literature review of the published clinical studies of nusinersen for the period March 2015 – March 2019 was performed following predefined criteria. Expert opinion Nusinersen is a significant therapeutic advancement, and is the first option to delay the progression of the disease. A number of clinical trials have demonstrated the efficacy and tolerability of nusinersen and achieving better clinical outcomes after its use compared to placebo. Despite the expected significant increase of the budget for SMA, nusinersen provides new possibilities of treatment of children with SMA in Bulgaria and innovative disease-modifying approach to the unmet medical needs of the patients and their families.