Emicizumab (Hemlibra®) for the prophylactic treatment of bleeding in patients with severe haemophilia A without inhibitors

Zorginstituut Nederland has completed its assessment whether emicizumab (Hemlibra®) for the prophylactic treatment of bleeding in patients with severe haemophilia A without inhibitors can be included in the insured package. Due to its expected high costs the Minister of Medical Care and Sports has placed emicizumab in the so-called ‘package lock’ or ‘sluice’ for expensive drugs. These products can only be accepted into the insured package after the Zorginstituut has advised on their inclusion in the insured package and, where applicable, subject to Ministry negotiations to arrive at a financial arrangement with the supplier. The Zorginstituut advises the Minister to include emicizumab only in the basic package if the treatment costs are at most equal to that of factor VIII prophylaxis.

Criteria for including treatments in the insured package

The Zorginstituut assessed the above-mentioned combination treatment based on the four package criteria effectiveness, cost-effectiveness, necessity and feasibility. Assessing from the perspective of the basic package which is paid from collective premiums, the Zorginstituut looks at whether new care is better than what is currently available. In doing this we look not only at the degree of certainty that has been achieved, from a scientific perspective and from the perspective of societal support, but also at efficiency aspects. The Zorginstituut was advised by two independent committees: the Scientific Advisory Board (WAR) which examines data on established medical science and medical practice and determines the cost-effectiveness, and the Insured Package Advisory Committee (ACP) which considers the societal assessment. Stakeholders are also consulted during the process.

Zorginstuut's advice

The advantage of emicizumab is its greater ease of use than the current standard care. As there is already a well-functioning treatment available for this patient population (factor VIII prophylaxis), this does not justify an additional charge, thus increasing the overall budget impact. As an additional argument, we want to state that a new gene therapy (valoctocogene roxaparvovec) will soon be marketed.

The Zorginstituut advises the Minister to include emicizumab only in the basic package if the treatment costs are at most equal to that of factor VIII prophylaxis. It is important to realize that the treatment centres have already agreed to discounts of up to 60% with the various manufacturers.

This report is a summary of recommendations by Zorginstituut Nederland. The original text of this excerpt is in Dutch.