| Assessment Status | Assessment Process Complete |
| HTA ID | 19054 |
| Drug | Ravulizumab |
| Brand | Ultomiris® |
| Indication | For the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH): in patients with haemolysis with clinical symptom(s) indicative of high disease activity and in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months. |
| Assessment Process | |
| Rapid review commissioned | 04/12/2019 |
| Rapid review completed | 02/03/2020 |
| Rapid review outcome | A full HTA is recommended to assess the clinical effectiveness and cost effectiveness of ravulizumab compared with the current standard of care, on the basis of the proposed price relative to currently available therapies. |
| Full pharmacoeconomic assessment commissioned by HSE | 04/03/2020 |
| Pre-submission consultation with Applicant | 28/06/2021 |
| Full submission received from Applicant | 05/11/2021 |
| Preliminary review sent to Applicant | 28/02/2022 |
| NCPE assessment re-commenced | 25/03/2022 |
| Factual accuracy sent to Applicant | 29/04/2022 |
| NCPE assessment re-commenced | 10/05/2022 |
| NCPE assessment completed | 30/05/2022 |
| NCPE assessment outcome | The NCPE recommends that ravulizumab (Ultomiris®), for the treatment of adult patients with PNH, be considered for reimbursement provided certain conditions are met*. These are that the cost of ravulizumab should not exceed any eculizumab products currently available or anticipated to be available in the near future. A price premium over eculizumab is not justified given that both treatments appear to have similar efficacy. In addition, ravulizumab did not demonstrate an improvement in treatment-related burden compared with eculizumab in clinical trials. |
*This recommendation should be considered while also having regard to the criteria specified in the Health (Pricing and Supply of Medical Goods) Act 2013.
