Material Type: Metachromatic Leukodystrophy

Metachromatic Leukodystrophy

Sep 2023 | Assessment

Interventions of Interest: Independent appraisal committee voted that across all patient subpopulations, arsa-cel demonstrated a net health benefit when compared to usual care. Arsa-cel would achieve common thresholds for cost-effectiveness if priced between $2.3M and $3.9M. Final Documents ICER’s Chief Medical Officer, David Rind, MD stated: “MLD is a devastating disease for children and their […]