Elosulfase alfa in the treatment of mucopolysaccharidosis type IVA: insights from the first managed access agreement.

ABSTRACT: Managed access agreements provide a crucial mechanism whereby real-world data can be collected systematically to reduce uncertainty around available clinical and economic data, whilst providing the opportunity to identify patient sub-populations who are most likely to benefit from a new treatment. This manuscript aims to share learnings from the first managed access agreement, which was initiated following positive conditional approval in 2015 from the National Institute for Health and Care Excellence (NICE) for elosulfase alfa, an enzyme replacement therapy for the treatment of mucopolysaccharidosis type IVA (MPS IVA). This managed access agreement enabled the collection of comprehensive real-world data for patients with MPS IVA, with results demonstrating that patients starting elosulfas

SUBMITTER: Stevens B 

PROVIDER: S-EPMC8467187 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature