Ravulizumab (Ultomiris®). HTA ID: 19054

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.

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.