1st PanItalian Associations Meeting on New & Innovative Therapies for Haemoglobinopathies_Italy

The PanItalian Associations Meeting, born from the collaboration between TIF and UNITED Onlus, was held virtually on 18 December 2020 to represent a moment of in-depth study with regard to both the standard treatment and the innovative therapies, now present or in the pipeline, for thalassaemia and sickle cell disease (SCD).
TIF’s President, Mr Panos Englezos, stated the importance of the TIF.ACCESS initiative to help ameliorate the patients’ access to novel therapies in Italy, one of the countries with the highest incidence of haemoglobinopathies globally. The President of UNITED Onlus, Mr Raffaele Vindigni, pointed out the need to arrive soon at the approval of the Decree on the National Network of Thalassaemia and Haemoglobinopathies in Italy, because this would represent a milestone to make the treatment of these diseases as homogeneous as possible throughout the country.
Subsequently, Dr. Gianluca Forni, President of the Italian Society of Haematology (SITE), made a statistical analysis on patients with haemoglobin disorders in Italy, listing in detail the numbers of patients, the incidences of the pathologies region by region, and the treatment centers distributed throughout the territory. Dr Forni added that the nascent National Network of Thalassemia and Haemoglobinopathies would only improve both this knowledge and the centers themselves by offering patients specializing treatment as much as possible.
Dr. Antonia Gigante, President of the Foundation for Research on Anaemia & Haemoglobinopathies in Italy FOR ANEMIA, illustrated the regulatory process followed by the Italian Medicines Agency (AIFA) for the approval and introduction of new drugs and innovative therapies in the Italian market. In the interactive session of this meeting, Prof Maria D. Cappellini, Department of Medicine and Medical Specialties, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) Ca’ Granda Foundation Maggiore Policlinico Hospital, and Prof Lucia de Franceschi, Associated Professor of Inernal Medicine, University of Verona; Azienda Ospedaliera Universitaria Integrata, responded to questions submitted by participants on Reblozyl and Adakveo, two EMA-authorized innovative treatments for thalassaemia and SCD respectively, and on the gene therapy Zynteglo, approved for use in the EU for patients with β-thalassaemia major.