The pandemic, in addition to the serious health and economic damage, seems to have overshadowed important diseases and also overshadowed treatments. Among these pathologies there is haemophilia.
There are over 32,000 haemophilic people in Europe. According to the latest data (2017) released by the National Register of Congenital Coagulopathies of the Istituto Superiore di Sanità, in Italy there are 10,627 patients with rare congenital hemorrhagic diseases. Of these, 4,179 have haemophilia A and 898 have haemophilia B.
We talked about it with Alfonso Gentile, Medical & Regulatory Director of Takeda Italy, one of the groups most committed to the treatment of this pathology.
What are the main complications of this disease?
“Among the main complications of this disease are hemarthrosis, ie bleeding that occurs inside the joints and which, if not treated properly, can lead to chronic arthropathy and disability.
Rare diseases are multi-systemic pathologies characterized by a certain complexity of management, and require a multidisciplinary therapeutic approach.Due to this scenario, it is evident that, in order to have effective therapeutic responses, it is necessary to invest in the study of pathophysiological mechanisms that underlie the pathological conditions and from which the therapeutic options derive “.
How should the country system move on this issue?
“To understand the importance of research in the field of this type of pathology, that is rare diseases, it is necessary to underline their common characteristics, in particular the difficulty and delay in diagnosis. For this reason, the country system must support the establishment of an ‘alliance’ between the scientific community, the pharmaceutical industry, patients and their associations, aimed at improving the lives of patients together by speeding up diagnostic approaches and making therapies possible for all affected people. from rare diseases “.
How is your Group moving on this front?
“Takeda is a global biopharmaceutical company that actively contributes to raising awareness about haemophilia, and rare diseases in general, through a steady stream of next-generation therapies. In the field of haemophilia, in particular, Takeda research is committed to guaranteeing the total absence of bleeding, trying to adapt the treatment scheme based on the characteristics of each individual haemophilia patient, as emerges from recent published studies, with the result of a significant improvement in the patient’s quality of life and burden. As far as rare diseases are concerned, our Group’s aspiration is to transform the treatment of these diseases both in clinical terms and with a view to offering patients a better quality of life. Our pipeline boasts 40 new molecules in different stages of clinical development, of which 14 for rare diseases, with investments of approximately $ 4.5 billion per year in R&D ”.
What are your support programs for haemophiliacs?
“We confirm the commitment to simplify the life of haemophilia patients in this unprecedented historical phase in which the difference is still the containment of travel, especially for fragile populations. Support programs range from home infusion to home blood sampling for PK assessment up to home physiotherapy rehabilitation. For 10 years we have been active in supporting home services in various therapeutic areas, including, in addition to haemophilia, lysosomal storage diseases, immunodeficiencies, hereditary angioedema. The emergency situation has not stopped our vocation and action of service ”.