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Rare diseases, FedEmo appeal for the protection of haemophilia patients and caregivers


To guarantee the continuation of the agreement on congenital haemorrhagic diseases (Mec), not to withdraw from the fundamental welfare principles contained in the Consolidated Law on rare diseases and participation in the National Committee for rare diseases provided for by the new legislative provision. This is the pressing appeal that the Federation of Haemophilia Associations (FedEmo) addresses to institutions on the occasion of World Hemophilia Day, which this year is celebrated on 11 April. According to the latest data released by the Higher Institute of Health in 2018 in Italy there are 10,554 patients affected by Mec present in the National Register of Congenital Coagulopathies. Of these 4,109 are affected by haemophilia A, 882 by haemophilia B, 3,245 by von Willebrand’s disease and 2,318 by defects in other coagulation factors. There are 54 haemophilia centers in Italy. A dedicated assistance network capable of efficiently ensuring the necessary assistance to all. “Consistent with its constant commitment to safeguarding the existing network of treatment centers for congenital hemorrhagic diseases – declares Cristina Cassone, FedEmo president – on this day the Federation asks politics and institutions for a concrete commitment to avoid the adoption of measures that can cause a reduction in the quality of care for people with rare coagulation defects. FedEmo proposes to this end as a reference for the National Committee envisaged by the Consolidated Law on rare diseases to help define and protect adequate care pathways for all Mec patients “.” The path undertaken with the Consolidated Law constitutes the fundamental starting point for responding to the needs of people with rare diseases. In this context, the Ministry will pay the utmost attention to the proposals made by FedEmo to effectively implement the Law recently approved – underlines Pierpaolo Sileri, undersecretary of the Health -. The collaboration of the Ministry of Health with the associations representing people with rare diseases and their families are precious in order to offer concrete answers to their needs and extend the best standards of care and the most effective assistance and therapeutic paths to the entire national territory ” The Federation with an official document ‘commits’ the Government and the institutions to “define relevant objectives and interventions, specific in the field of hereditary anemia, such as thalassemia and other hemoglobinopathies, and hereditary coagulation defects, such as congenital hemorrhagic diseases , already subject to specific sector regulations “. Furthermore, the Government is asked to provide for the autonomous and specialized organization of the welfare networks dedicated to congenital haemorrhagic diseases and of the specific coordination centers for these networks, which must necessarily link up with the coordination centers for rare diseases. If, therefore, it is important for patients and caregivers to unify the multiple rare diseases under a single umbrella, it is equally important to provide for different and specific frameworks for the treatments intended for MECs. The agreement drawn up at the instigation of FedEmo and signed by the Government, Regions and Autonomous Provinces of Trento and Bolzano in 2013, which governs the organization of care networks specifically dedicated to patients in Italy, responds to this purpose. “The care networks for diagnosis , care and research, in particular for rare diseases, represent an essential cornerstone for promoting, spreading and maintaining the quality of treatment standards in infrequent diseases – explains Giancarlo Castaman, director of the Hemorrhagic Diseases Center, Aou Careggi in Florence – In particular , for historical reasons, haemophilic syndromes have always represented a sensitive observatory of the attention of the health system towards rare diseases, and at the same time stimulating the creation and development of advanced diagnostic and therapeutic technologies. A model that remains a paradigm of the evolution of medical and scientific progress, a stimulus and inspiration for other rare genetic diseases, also for the important active role played by patient associations “. The Federation, as President Cristina Cassone reiterates, asked the XII Commission of the Senate to be heard during the process of approval of the implementing decrees of the Consolidated Law, in order to guarantee the continuation of the Mec agreement and the protection of the centers and already existing Mec networks, virtuous examples also for the treatment of other pathologies, not only for the treatment of people with haemophilia. These models, developed over decades and proven to be highly effective, cannot be dispersed within a general reorganization of rare diseases. The figure of expert haematologists within the reference centers cannot be replaced tout court by medical personnel without adequate training. Not to mention that a large number of drugs are currently available that can ensure effective and personalized therapy for all patients, for the correct management of which it is essential to make use of the clinical practice of hematologists expert in Mecs. he assistance and care of haemophilic patients is another focal point of the letter of commitment that FedEmo has sent to the Government. Precisely for this specificity, the Federation has asked to establish or maintain dedicated national and regional working tables for congenital hemorrhagic diseases, composed of expert doctors, representatives of scientific societies, the head of the National Rare Diseases Coordination Center for the national table. and from the regional one for the regional tables, by the representatives of the patient associations. “Hemophilia is a complex condition that generates clinical and social vulnerability and fragility, which requires appropriate and widespread clinical-assistance interventions throughout the national territory, equal access to care for those who suffer from it and a high level of institutional attention – says Andrea Lenzi, coordinator of the Technical Committee for Rare Diseases -. Hence the need to create a collaborative and interdisciplinary community of experts who discuss rare diseases, including haemophilia, in a multidimensional perspective, to analyze and find solutions to specific problems. A real research and assistance model that involves the clinical-assistance plan and the political, institutional, sociological, economic, organizational plan “. The document also asks for the commitment to provide that the national networks for Mec transmit their data to the Registries of pathology of national and regional importance, to the National Register of Congenital Coagulopathies, established at the ISS, which will then send the information to the National Center for Rare Diseases. In order for this synergy to function without obstacles and impediments, FedEmo’s collaboration with the National Committee for Rare Diseases provided for in the Consolidated Law cannot be ignored. A need, however, reaffirmed by Andrea Lenzi himself, according to whom “the role of patient associations, such as FedEmo, is strategic and indispensable, both in the process of education, information, but also in the preparation and co-management of the care pathway. Patient associations must also play a role in the process of dialogue with the ministries and departments involved, contributing, from the point of view of the patient and his family, to the provision of useful tools to combine the concepts of personalization of care and a PNMR that provides for shared standards at national level “. Many important voices, all agree on a single principle: the need for specific treatment of Mecs within the Consolidated Law on rare diseases and the importance of evaluating the appropriate measures that guarantee the maintenance of an adequate standard of care to respond to all the needs of patients.


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