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.

Myelodysplastic syndromes (MDS) are chronic bone marrow malignancies of the elderly, complicated by severe anaemia. MDS significantly affects quality-adjusted survival and it imposes an increasing financial burden on patients and healthcare (HC) systems. The burden of disease is expected to worsen in the future, given the aging EU population and newly identified MDS cases among those diagnosed with ‘Anaemia of the Elderly’. EUMDS - a unique registry with prospective, observational data on 1600 lower-risk MDS patients from 16 EU countries - enables comparison of existing MDS HC interventions (HCI). Objectives 1) Comparison of outcomes and costs of existing HCI, using established and new core outcome sets. This, alongside health technology assessment, will provide robust evidence to underpin sustainable use of HC resources; 2) Enhanced compliance with diagnostic procedures in MDS by introducing new minimally-invasive diagnostic methods. This will increase the number of correctly and timely diagnosed MDS patients; 3) Raised awareness of the relevance of obtaining the right diagnosis in elderly by comparing HRQoL between EUMDS and against a non-MDS cohort. This will improve HRQoL of individual patients through better tailoring of HCI; 4) Better outcome predictability of available HCI by refining classification of cases using molecular characterisation to incorporate response to HCI and to provide evidence for personalised medicine; 5) Improved, evidence-based, guidelines supporting the regulatory process, and providing information to patients and physicians to promote personalised decisions in MDS care; 6) Establishment of a European MDS competence network encompassing all stakeholders in the MDS field. Relevance Better treatment compliance, a more evidence-based use of HCI and an increasing tailored use of existing HC options will contribute to an efficient and cost effective (personalised) use of HC resources for elderly patients with MDS and their co-morbidities.

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