Treatment system for hemophilia patients in Poland: duality of pathways

What does the treatment system for hemophilia patients look like in Poland, and in what direction should this system develop?

Currently, patients with hemophilia are primarily managed through the National Hemophilia Treatment Program, which has been a bright spot in the Polish health care system for many years. It is a program that takes care of patients in a very comprehensive way and guarantees them access to treatment that is effective. Today, it can significantly expand by including precisely in this plan modern therapies that are already available throughout Europe, but which are nevertheless still very limited for the Polish patient. This modern treatment is guaranteed only for a handful of pediatric patients in the drug program, i.e. this additional treatment pathway that appears in this group of patients is the drug program. The drug program as a concept for organizing comprehensive care, coordinated care dedicated to very specific centers that have the competence to use these modern drug technologies for optimal treatment outcomes. This is because the drug program, which is often criticized, has very specific eligibility criteria for patient inclusion and exclusion, but through this we can guarantee that the optimal clinical outcome is achieved in relation to cost.

Today, this duality of paths, that is, guiding patients who obtain clotting factors through intravenous therapies and a drug program that guarantees access to subcutaneous therapies, are such two paths that we should not consider as competing, but as complementary paths, creating a system of patient care that is oriented to use technological advances to guarantee Polish patients, both pediatric and adult, the most effective and optimal treatment effects, and this treatment effect is to avoid bleeding and improve the quality of life.

Improved quality of life as measured by the fact that therapeutic intervention does not have to happen so often, because modern treatment has a long effect, but also gives the patient the opportunity to live a high quality of life. I think that at this stage where we are in the Polish health care system we already know that the organization of health care should not be oriented only to treatment. The health care organization should be oriented towards achieving optimal results, which is the clinical outcome, but also the patient's quality of life. The WHO, as it gives the modern definition of hemophilia prevention, emphasizes that it is not only about the maximum reduction of bleeding, but it is primarily about using such treatment regimens that the hemophilia patient has the best possible quality of life.

This high quality of life of the patient translates into systemic benefits, because the lower the need for therapeutic intervention over a given group of patients, the lower the burden on the health care system is also, the higher the state revenues from the professional activity, social activity of the patient with a chronic disease.

For we know that as of today, of course, hemophilia is not a curable disease, it is still a chronic disease.

So we don't count the unit price of the drug?

Thinking through the unit price of a drug is a road to nowhere. She has long been negatively verified by clinical practice, but also by system practice and health economics. We already know that we must look at health spending as an investment, seeing not only the unit cost, but more importantly how that investment will reduce other direct costs to the public payer and the indirect costs we incur when a patient is excluded from full activity, for example. We understand that we need to look through the lens of the patient's overall treatment in the pathway of his or her diagnosis and treatment, and compare the total costs of the disease to the total benefits and revenues we achieve. These revenues are measured in the patient's quality of life, but these revenues are also counted in the income he generates as an active person.

Even if we are talking about children, if these children can go to school, go to kindergarten with peace of mind, become active in the social environment and build their perception as a person with full rights to develop in health to live and develop in such equal opportunities with respect to the population of healthy children, this is not a discussion about whether, it is a decision about how quickly we will provide our patients with access to treatment that is in line with current medical knowledge, but also treatment with effectiveness. We no longer have doubts today, because the scientific evidence expressed by pharmacoeconomic analyses speaks clearly: The sooner we incorporate modern treatment, the higher the clinical, economic and social benefits are for a given system.

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