Risdiplam (Evrysdi™). HTA ID: 21006

Assessment Status NCPE Assessment Process Complete
HTA ID 21006
Drug Risdiplam
Brand Evrysdi™
Indication For the treatment of 5q spinal muscular atrophy (SMA) in patients 2 months of age and older, with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies.
Assessment Process
Rapid review commissioned 15/03/2021
Rapid review completed 29/03/2021
Rapid review outcome A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of risdiplam compared with the current standard of care.
Full pharmacoeconomic assessment commissioned by HSE 04/05/2021
Pre-submission consultation with Applicant 28/06/2021
Full submission received from Applicant 20/08/2021
Preliminary review sent to Applicant 01/10/2021
NCPE assessment re-commenced 29/10/2021
Follow-up to preliminary review sent to Applicant 26/11/2021
NCPE assessment re-commenced 20/12/2021
Factual accuracy sent to Applicant 18/02/2022
NCPE assessment re-commenced 25/02/2022
NCPE assessment completed 31/03/2022
NCPE assessment outcome The NCPE recommends that risdiplam not be considered for reimbursement until cost-effectiveness can be improved relative to existing treatments*.

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.

The HSE has approved reimbursement following confidential price negotiations. September 2023