National Centre for Pharmacoeconomics

National Centre for

NCPE Ireland

Absolute risk

The probability of an event or outcome occurring in a defined population over a specified time period

Absolute risk reduction

A measure of treatment effect that compares the decrease in risk in the control group with that of a treatment group i.e. Pc – Pt

Adverse event

An undesirable effect of a health technology

Atidarsagene autotemcel (Libmeldy®). HTA ID: 21009

Atidarsagene autotemcel (Libmeldy®) is indicated for the treatment of metachromatic leukodystrophy  characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity, in children with late infantile or early juvenile forms without clinical manifestations of the disease, and in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline.


NCPE Assessment Process Complete
Rapid review commissioned 22/03/2021
Rapid review completed 12/04/2021
Rapid Review outcome A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of OTL-200 compared with the current standard of care.
Full pharmacoeconomic assessment commissioned by the HSE 29/04/2021
Full submission received from Applicant 17/12/2021
NCPE/Benaluxa assessment completed 30/09/2022
NCPE/Benaluxa assessment outcome

The NCPE recommends that atidarsagene autotemcel not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatment*.

NCPE/Beneluxa Joint Technical Summary

Plain English Summary

*This recommendation should be considered while also having regard to the criteria specified in the Health (Pricing and Supply of Medical Goods) Act 2013.