It's all about hemophilia. - With regard to the periodically recurring information in the public space regarding limitations in access to hemophilia treatment, we assure that every patient with hemophilia has access to modern preventive and stabilizing treatment for his disease. The hemophilia treatment program in Poland is one of the best in Europe - it ensures continuity of therapy, safety and a high level of treatment. No other group of patients has such an organized, special system of drug delivery and collection. All logistics and patient services are handled by the National Blood Center," reads the Health Ministry's announcement.
Ministry of Health: Honest discussion needed
Published Sept. 5, 2025 07:57
The Health Ministry recalls that hemophilia treatment includes four key clinical components, which are considered each time reimbursement decisions are made:
- The effectiveness of the treatment, that is, the level of activity of the agent that ensures proper hemostasis of the patient,
- Safety of use dependent on patient activity,
- Treatment administration time interval,
- Convenient method of administration.
Reimbursement applications
An application to expand the indications for emicizumab treatment in both children and adults is currently being processed. In addition, an application is under consideration for reimbursement of a blood clotting factor with higher efficacy, administered once a week. This solution could provide an alternative to the previously used blood clotting factors in the hemophilia treatment program and to emicizumab.
The ministry stresses that emicizumab and clotting factor concentrates are two different therapies used to prevent bleeding in patients with hemophilia. The concentrates have an immediate effect, which makes it possible to quickly stop bleeding. For this reason, they are also administered to patients treated with emicizumab, as rapid control of bleeding is crucial to saving health and life.
New therapies
The drug Hemlibra (emicizumab) is no longer the only subcutaneous drug used to prevent bleeding in patients with hemophilia. At the end of 2024. The European Medicines Agency has approved two new formulations, marstacymab and concizumab, for use in patients with hemophilia A and B. Discussions are currently underway with the responsible parties to make them available as part of guaranteed services.
The process analyzes two therapies with different mechanisms of action for the same disease entity. In order to develop the most favorable eligibility criteria for patients for the drug program, a meeting was held with experts. They indicated that patients with difficult intravenous access, including children under the age of 4, would benefit most from emicizumab prophylaxis. Other patients should receive prophylaxis with long-acting clotting factors, or even with ultra-long-acting clotting factors. Administration of the drug once every 7-10 days significantly improves quality of life, while effectively protecting against bleeding.
Experts stress that despite the less invasive nature of subcutaneous administration, intravenous clotting factors may be more beneficial for some patients. The mechanism of action of emicizumab corresponds to maintaining factor activity at a level of about 9-15%, which is comparable to mild hemophilia. This protects against spontaneous bleeding, but the risk of post-traumatic or surgery-related bleeding is higher than with clotting factor concentrates. Patients taking emicizumab are not advised to participate in contact sports - in this case it is necessary to use intravenous clotting factors, which are available free of charge.
Costs of treatment
The Ministry of Health indicates that the annual cost of prophylaxis for one patient ranges from 400 to 900 thousand zlotys and is publicly financed. Prophylaxis does not completely eliminate the risk of bleeding, so emergency treatment is also necessary, which can generate additional expenses of several hundred thousand zlotys a year. The total cost of therapy for a patient with hemophilia can be as much as PLN 2 million a year.
Reimbursement of a drug is not a one-time investment, but a long-term commitment by the state. The most costly treatment of a single case over the past five years has consumed PLN 43 million in public funds, with the daily cost of treating inhibitor-related complications reaching up to PLN 200,000.
Need for dialogue
The ministry stresses that negotiations with pharmaceutical companies and the search for compromise solutions are necessary to guarantee effective therapy for patients. There is criticism of the actions of some entities, including lobbying organizations and foundations, which promote only one product, leaving out important facts. The Ministry points out that such practices can lead to unfavorable financial deals for the state.
Patient priority
The Ministry of Health is unequivocal about its priority to provide patients with free access to effective and safe treatment. Therefore, the ministry is determined to make available long-acting therapies with high efficacy for prophylactic treatment of patients with hemophilia under the age of 18 as part of the drug program, while securing public funds to finance the continuation of therapies reimbursed to date.
Source: MZ
