Package advice ravulizumab (Ultomiris®) for the treatment of PNH and aHUS
The National Health Care Institute has completed its assessment whether ravulizumab (Ultomiris®) can be included in the insured package. Ravulizumab is indicated for the treatment of certain groups of patients with paroxysmal nocturnal haemoglobinuria (PNH) and atypical haemolytic uraemic syndrome (aHUS). The National Health Care Institute has concluded that ravulizumab for the aHUS indication does not meet the statutory criterion of ‘established medical science and medical practice’. Therefore, ravulizumab is not eligible for reimbursement from the basic insurance scheme for the treatment of patients with aHUS. For the PNH indication, ravulizumab does comply with the ‘established medical science and medical practice’. The National Health Care Institute advises the Minister to include ravulizumab only for the indication PNH in the health insurance package, provided the price negotiations with the marketing authorisation holder successfully deliver a net price that does not exceed that of the existing treatment.
Registered indication
Ravulizumab (Ultomiris®) is indicated for the following indications:
- treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH):
- in patients with haemolysis with clinical symptom(s) indicative of high disease activity;
- in patients who are clinically stable after having been treated with eculizumab for at least the past 6 months.
- treatment of patients with a body weight of 10 kg or above with atypical haemolytic uraemic syndrome (aHUS), who are complement inhibitor-naive or have received eculizumab for at least 3 months and have evidence of response to eculizumab.
Package advice
The National Health Care Institute has concluded that ravulizumab for the aHUS indication does not meet the statutory criterion of ‘established medical science and medical practice’. Due to the limitations in the design of the studies, there is a very low quality of evidence. This results in very little confidence in the effect of ravulizumab on the crucial outcome measures. Therefore, ravulizumab is not eligible for reimbursement from the basic insurance scheme for the treatment of patients with aHUS.
The National Health Care Institute advises the Minister to include ravulizumab only for the indication PNH in the health insurance package, provided the price negotiations with the marketing authorisation holder successfully deliver a net price that does not exceed that of the existing treatment with eculizumab. Since there is an equal value compared to eculizumab, which is already being reimbursed, and there are no indications that one product is preferable to another, we advise the Minister to take into account during the price negotiations the existing discount on eculizumab. The price reduction recommended at the time for eculizumab, the arrival of biosimilars and other treatment options in the (near) future, and the doubts about the relationship between the asking price and the investments made by the marketing authorisation holder should also play a role in the negotiations.