Onasemnogene abeparvovec (Zolgensma®). HTA ID: 20021

This HTA is being undertaken as part of the BENELUXA collaboration. It is a joint assessment between Ireland, Netherlands and Belgium with Austria acting as reviewer. Please see www.beneluxa.org for further information.

Assessment Status Assessment process complete
HTA ID 20021
Drug Onasemnogene abeparvovec
Brand Zolgensma®
Indication For the treatment of patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1, or patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to 3 copies of the SMN2 gene.
Assessment Process
Rapid review commissioned 14/04/2020
Rapid review completed 13/05/2020
Rapid review outcome A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of onasemnogene abeparvovec (Zolgensma®) compared with the current standard of care.
Full pharmacoeconomic assessment commissioned by HSE 27/05/2020
Concept dossier received 08/06/2020
Response to Applicant 29/06/2020
Final Complete Dossier accepted 02/11/2020
First draft report complete** 07/12/2020
Final Report** 02/04/2021
NCPE assessment outcome The NCPE recommends that Onasemnogene abeparvovec (Zolgensma®) not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatments.*


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

Executive summary Zolgensma Beneluxa (Ireland)Final Version

Plain English Summary

**The Beneluxa process differs slightly from the process in Ireland where the Drug Committee received only the final report.  In the Beneluxa process the respective committees in the Netherlands (WAR) and Belgium (CTG) receive draft versions of the report (2 drafts).  Edits, if appropriate are made in this time and the report is finalised after the second round of reviewing.

The HSE has approved reimbursement following confidential price negotiations October 2021.