Interventions of Interest:
- atidarsagene autotemcel (“arsa-cel”, Orchard Therapeutics)
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.
ICER’s Chief Medical Officer, David Rind, MD stated:
“MLD is a devastating disease for children and their caregivers. It is extremely rare, and most doctors have never seen a case. We again see a gene therapy potentially filling the promise of treating a disease that was previously untreatable, delivering remarkable clinical benefits and extending life. If the manufacturer prices arsa-cel in line with its price in Europe, despite being very expensive, arsa-cel would be cost-effective.”