Health State Utility Values in Fabry Disease: Insights from the Pegunigalsidase Alfa Clinical Trials.

ABSTRACT:

Introduction

Fabry disease (FD) is a rare lysosomal storage disorder that is associated with pain and progressive damage to the renal, cardiac, and cerebrovascular systems. Enzyme replacement therapy (ERT) is one of the treatment options for FD and the most recently approved ERT agent, pegunigalsidase alfa, has shown clinical efficacy in three phase 3 clinical trials of adults with FD: BALANCE, BRIDGE, and BRIGHT. Recent published guidelines support the mapping of health utility state data to the EuroQol-5 Dimension-3 Level (EQ-5D-3L) index to align with the preferred methodology used by the National Institute for Health and Care Excellence (NICE). Therefore, the primary objective of this study was to estimate EQ-5D-3L values in clinical trials of pegunigalsidase alfa for FD for fu

SUBMITTER: Azimpour K 

PROVIDER: S-EPMC11868215 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature