Rare diseases, FedEmo: “Fine. Single text but no steps backwards on haemophilia”

“The approval of the Consolidated Law on rare diseases is good, but no step backwards on haemophilia”. After the go-ahead in the Senate in recent days, FedEmo, Federation of haemophilic associations, launches an appeal to the institutions: “It appears essential – he asks – a specific treatment for haemophilia, and in general for all congenital haemorrhagic diseases (Mec), In fact, if on the one hand the initiative to rationalize and unify the multiple rare diseases under a single care umbrella represents an important step in responding to the needs of many patients and caregivers, on the other hand the specificities that characterize the ‘haemophilia and the other Mecs require a differentiated classification “. The Mecs – explains a FedEmo note – although falling within the scope of rare diseases, currently affect a large number of patients, over 11 thousand in our country according to the data of the Register of the Higher Institute of Health, and enjoy their own care network dedicated, already present in the area for some time, structured in regional reference centers and centers with highly specialized medical staff, able to efficiently ensure the necessary assistance to all patients. To this end, the Agreement on Mecs is in force in Italy, drawn up on the initiative of FedEmo and signed by the Government, Regions and Autonomous Provinces of Trento and Bolzano in 2013, which governs and defines in an extremely detailed manner the organization of assistance networks specifically dedicated to patients. The Agreement has been implemented by all the Regions and is currently being implemented at the local level. “We consider the approval of the Consolidated Law on Rare Diseases to be a necessary tool, but not sufficient to give concrete answers to all rare disease patients and above all to people suffering from congenital hemorrhagic diseases – declares Cristina Cassone, FedEmo president – We have already asked the XII Commission of the Senate, during the parliamentary process for the approval of the law, the safeguarding and protection of existing welfare networks, which can also represent a virtuous example for other pathologies and not only for the treatment of people with haemophilia. For our specificity and for our history, we therefore ask to be audited as patients during the approval process of the implementing decrees of this Consolidated text, in order to guarantee the continuation of the Mec Agreement and the protection of the Mec centers and networks already in place “. obstructed over decades and proved highly effective – FedEmo claims – it 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 capable of ensuring effective and personalized therapy are already available today. for all patients. Paola Boldrini, vice president of the XII Permanent Commission on Hygiene and Health of the Senate agrees: “The approval by the Commission of the Consolidated Law on rare diseases is a very important step forward and awaited by many patients – she says – But for the pathologies that already of organizations and centers of reference with healthcare professionals and patients it will not be a question of starting from scratch. A specific agenda signed by me foresees in fact to treasure all the excellences already present in the area, such as for haemophilia and thalassemia. The existing networks will continue their valuable activity and will be able to coordinate with the new structure to benefit from concrete help “. For the peculiarities described, according to the Haemophilic Association Federation”, specific treatment is essential for the area of ​​Mec within of the Consolidated Law on Rare Diseases, as well as desirable to evaluate the appropriate measures to ensure the maintenance of one s standard of care adequate to meet all patient needs “.