Haematological malignancies (HM), also known as blood cancers, are a heterogeneous and complex group of multicausal diseases that can’t be easily diagnosed nor treated. Nowadays most treatments are extremely complex, and advances in patient diagnosis and therapies slow due to the low number of patients per centre. Thus, there is a need to harmonise, store, and analyse the current HM information to speed-up and support the decision-making process for patients’ access to new therapies. HARMONY PLUS takes advantage of the capabilities of the HARMONY Big Data platform to match this unmet needs by expanding its scope to incorporate myeloproliferative neoplasms, including chronic myeloid leukemia, polycythaemia vera, essential thrombocythaemia, and myelofibrosis; and lymphoproliferative disorders, including Hodgkin’s lymphoma, Waldenström macroglobulinemia and all the other rare HMs not covered by HARMONY Project. In parallel, HARMONY PLUS will continue to refine and define the Core Outcome Sets (COS), especially for these new HMs to ensure the use by researchers of useful common outcomes relevant to all stakeholders. As previously accomplished in HARMONY, HARMONY PLUS is committed to pursue the maximum ethical and legal requirements, particularly to ensure patient’s right to privacy. Data-driven research within Europe will be enhanced by converting the current HARMONY platform into an Integrated Services Platform to serve as a valuable tool to support clinical trial design and use of available data as a control arm. This platform, combined with a HaemoDatabank repository with information about HMs patient biological samples across Europe, will facilitate a more efficient research and clinical trial design, and consequently will promote collaborations with recognised databases outside Europe. From the regulatory point of view, HARMONY PLUS will be a valuable technology tool during the evaluation of new treatments and drugs by also considering the patients’ needs.

Disease interception is a novel concept referring to treatment of a disease before the disease fully develops by removing altered cells. To make disease cell interception a reality we will need to overcome two key challenges. First, we will need to be able to identify few altered disease cells among many healthy ones. Second, we need to develop strategies that allow to specifically target disease cells but do not affect healthy cells. In the INTERCEPT-MDS ITN we propose to approach these challenges through research and the shared multidisciplinary and multisectorial training of 12 Early Stage Researchers (ESRs). As a result we will build and present some of Europe’s first experts in the novel field of disease cell interception. We will take advantage of single-cell omics methods that have reached a level of maturity to be applied on a broad-scale. For interception, we will explore and exploit the epigenetic regulatory space and use our privileged access to tool compounds and our capacity to perform genetic

Despite significant recent progress in the field of hematological malignancies (HMs), with increasing survival rates and improvement in quality of life, many children and adults with HMs still die from these disorders or experience disabling complications. Therefore, improvement of health care of HMs is an unmet medical need. Thus, it is important to define and align standard and efficient sets of HMs outcomes to measure and evaluate HM data for clinical decisions, long term risk/benefit profile, reimbursement, value analysis, and clinical trials design. Improving outcome measures and endpoint definitions by taking into account “real-life” data and differences in cross-national healthcare practice will undoubtedly result in an optimized, sustainable and effective treatment delivery, as well as in desirable and innovative accelerated pathways for novel drug availability. All these challenges will be addressed within a pan-EU perspective by HARMONY (Healthcare Alliance for Resourceful Medicines Offensive against Neoplasms in HematologY), a comprehensive public-private European consortium of excellence. HARMONY consortium is made up of 51 partners: 44 participants from 10 European countries and 7 pharmaceutical companies from the EFPIA. HARMONY aims to assemble, assess, connect, and analyze heterogeneous HM patient derived Big Data sets to define sets of outcome indicators that can be used for decision-making by key healthcare stakeholders. The consortium will orchestrate leading experts and working cooperative groups in HMs, European study alliances, pharmaceutical market leaders, patient advocacy groups, HTA and regulatory agencies, to: (i) optimize Europe-wide data collection and create a high-quality HM data repository for further explorative studies; (ii) establish a clinical data-sharing platform that empowers clinicians, patients and policy stakeholders to improve decision-making procedures and identify appropriate treatments to patients with HMs

GENOMED4ALL will support the pooling of genomic, clinical data and other “-omics” health data (data EHR, PET, MRI and CT , Next Generation Sequencing, Microarray, Genome Wide Association, Copy Number Variations, DNA sequencing, RNA sequencing, including single cell, etc.) through a secure and privacy respectful data sharing platform based on the novel Federated Learning scheme, to advance research in personalised medicine in haematological diseases thanks to advanced novel AI models and standardized sharing of cross-border data. GENOMED4ALL will make use of the existing infrastructures and initiatives, including powerful High Performance Computing facilities, hospital registries, data processing tools, and pre-existing repositories, starting from 10 clinical partners repositories to be enlarged especially by the resources provided by ERN-EuroBloodNet where GENOMED4ALL clinical partners have a leading position, which contain 66 relevant clinical sites providing repositories and knowledge, for the successful exploitation of genomics, clinical and other related “-omics” data to facilitate personalised medicine in common, rare and ultrarare haematological diseases to demonstrate the versatility and utility of the solutions, and 20 external of this network. GENOMED4ALL will demonstrate the potential and benefits of trustable and explainable AI technologies, with a novel approach to AI models and algorithms using AI advanced deep learning, variational autoencoders, generative models, besides combining with advanced statistical and Machine learning processes approaches to exploit the powerful set of “-omics” data which will be at researchers’ disposal. This will allow for identifying new knowledge, to support clinical research and decision making by linking Europe's relevant genomic repositories in haematological diseases, while ensuring full compliance with data protection legislation and ethical principles, and increasing the AI trust for personalized medicine.