|Assessment Status||Assessment Process Complete|
|Indication||Is indicated for long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) deficiency.|
|Rapid review commissioned||08/02/2017|
|Rapid review completed||13/03/2017|
|Rapid review outcome||Full Pharmacoeconomic Assessment Recommended|
|Full pharmacoeconomic assessment commissioned by HSE||30/03/2017|
|Pre-submission consultation with Applicant||22/08/2017|
|Full submission received from Applicant||13/11/2017|
|Preliminary review sent to Applicant||16/03/2018|
|NCPE assessment re-commenced||11/04/2018|
|Factual accuracy sent to applicant||17/05/2018|
|NCPE assessment re-commenced||05/06/2018|
|NCPE assessment completed||08/06/2018|
|NCPE assessment outcome||The NCPE recommends for the infantile presentation, that sebelipase alfa (Kanuma®) not be considered for reimbursement unless cost effectiveness can be improved relative to existing treatments. For the paediatric adult presentation, robust evidence of additional clinical benefit of sebelipase alfa has not been demonstrated. Therefore the NCPE recommends that sebelipase alfa not be considered for reimbursement.|
These recommendations should be considered while also having regard to the criteria specified in the Health (Pricing and Supply of Medical Goods) Act 2013.
The HSE approved reimbursement following confidential price negotiations July 2019.