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