Modern therapies in hemophilia: an investment in children, an investment in the future of society

What are the social and economic effects of faster introduction of modern therapies for rare diseases such as hemophilia?

First and foremost, it gives a person a chance, along with a chronic disease that will accompany a person for many years, to make his or her life nevertheless have a chance for normalcy, to realize goals, dreams and ambitions. This is especially important with regard to children. I think no one who has any awareness of the importance and value of the young generation in any society should be in any doubt that an investment in the health, security, stability and quality of life of the youngest generation is really an investment in the future of a country.

Our Constitution has very strongly articulated that children, pregnant women and the elderly should be under special supervision, should receive care that is exceptionally effective, sensitive to experience, to the state's ability to secure the health effect that is made possible by scientific progress. Today we are witnessing great advances in medicine. Every now and then we learn about the registration of modern technologies, which very often can be considered breakthroughs, or even if not, those that radically change both the procedure and the treatment, giving such a radical change in the quality of life of patients. I will always defend a child's right to develop in health. I think we are missing the discussion about what should make up the realization of a child's right to develop in health. Children with hemophilia are children who have been affected by an extremely difficult, severe, acute disease, which today can be well taken care of. Let's think about this through the lens of our obligation as a state to take care of this generation and this social group. The fertility rate is declining. He is frighteningly low. So to say that we can't afford anything dedicated to children is like saying that we don't want the stable development of our state and our society. Hemophilia and children with hemophilia absolutely deserve the best care, standard care. I think this is the most important sentence in this statement. We are obligated to treat according to standards. If these standards define the technologies and diagnostic methods that are necessary for good care and guarantee an optimal therapeutic outcome, then we must find the funding to secure this standard-compliant care.

And just completely last sentence. Let's remember that a child, a youth turns 18 one day, and that we should not guarantee care that ends on this magical, important day for every young person. We already today, when designing solutions for children and adolescents, must remember and secure the continuation of their treatment when this magical date of 18 years of age appears on their calendar.