| 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*. |
*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