Evoluzione terapeutica: tradurre l'innovazione farmacologica in benefici tangibili per le persone con emofilia A. Una prospettiva multi-stakeholder

The advances that have marked the history of hemophilia represent a concrete example of therapeutic evolution. The progress made over the years has been able to revolutionize its treatment, bringing about a radical change both in the therapeutic algorithm and in life expectancy. Considering the recent milestones achieved, it would be anachronistic to maintain the same therapeutic goals as in the past; the normalization of hemostasis is the path leading to the ultimate therapeutic goal, which should be the achievement of health equity: a life comparable to that of a person without such a condition. This article highlights the need for a multidisciplinary approach to the management of hemophilia, as raised from the different perspectives of the stakeholders who contributed: pharmacologists, patients, clinicians, bioethicists, pain therapists, and pharmacoeconomists. This has made it possible to gain a comprehensive view that reflects the complexity of managing a chronic disease such as hemophilia.