Onasemnogene abeparvovec (Zolgensma®) is indicated 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.
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.
|NCPE Assessment Process||Complete|
|Rapid review received||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|
|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.*|
**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.
*This recommendation should be considered while also having regard to the criteria specified in the Health (Pricing and Supply of Medical Goods Act) 2013.
The HSE has approved reimbursement following confidential price negotiations October 2021.