To target helminth elimination, a drug research and development (R&D) pipeline is needed to provide new chemotherapeutics that effectively eliminate or sterilize adult worms, thus bringing about the paradigm shift necessary to reach the 2030 SDG goals on health. Our consortium proposes to establish a R&D pipeline for anthelminthics targeting nematodes. The focus will be on soil-transmitted helminthiasis and onchocerciasis, since these infections are among the leading neglected tropical diseases. Ground breaking characteristics of the drugs developed within our project are that they will have a unique mechanism of action that, at best, will target multiple nematodes (pan-nematode) with an excellent safety profile, including no efficacy against non-targeted co-endemic species. We will benefit from collaborations with our industrial partners Bayer and Celgene providing preselected compounds to populate the early preclinical stages of the R&D pipeline. Compounds with the best profile will be progressed through preclinical studies. Corallopyronin A, a compound with proven efficacy against essential Wolbachia endosymbionts in filariae that has superiority to the gold standard doxycycline, excellent bioavailability and promising exploratory safety data will undergo state-of-the-art toxicity profiling to advance towards phase 1 trials. We will also evaluate oxfendazol and oxantel pamoate in clinical trials. They have already proven efficacious in animals or humans and will only require clinical trials according to current regulatory guidelines to be implemented. With this strategy, the consortium will ensure that a pipeline of drug candidates is available for treating onchocerciasis, especially should current candidates fail in upcoming clinical trials. Moreover, we will establish a much-needed drug R&D pipeline to treat soil-transmitted helminth infections for which there is currently neither a drug with good efficacy against all species nor any prospects on the horizon.

Europe is generating huge amounts of patient-level information contained in Electronic Health Record (EHR) systems and other types of health databases. These include structured data in the form of diagnoses, medications, laboratory test results, etc., and unstructured data in clinical narratives. The Electronic Health Data in a European Network (EHDEN) Consortium leverages these vast volumes of data to improve future clinical practice and individual patient outcomes by increasing our understanding of disease and treatment pathways. EHDEN will galvanize transparent and reproducible analytics that will generate valid real-world evidence to improve patient care, and enable medical outcomes-based research at an unprecedented scale. The EHDEN Consortium provides the infrastructure and eco-system supporting disease-specific projects in the IMI Big Data for Better Outcomes (BD4BO) programme. The core of EHDEN is the use of a common data model (OMOP-CDM), standardised outcome assessment (ICHOM), and transparent open-source analytics (OHDSI). The objective of the EHDEN consortium is to provide all the necessary services that enable a distributed European data network to perform fast, scalable and highly reproducible research, while respecting privacy regulations, local data provenance and governance. This will include services and tools to perform data standardization, analytical pipelines, tools to share study results, and tools for stakeholder engagement and training. The EHDEN Consortium combines active participation of stakeholder representatives with proven experience in: a) integrating different data types, methods and technologies to utilize diverse clinical datasets; b) platform development to make methods and datasets Findable, Accessible, Interoperable and Reusable (FAIR); and c) engaging a wide variety of stakeholders, including health technology assessment agencies, regulators and patients.

Immune cells that are empowered by gene-engineering to seek and destroy cancer cells (engineered T cell therapy) constitute a transformative novel treatment that has the potential to cure cancer. Multiple new versions of this therapy are being developed for distinct types of cancer but their introduction into clinical practice is hampered by a lack of standardized and validated models to predict safety and efficacy, customized manufacturing and monitoring to scale up production and clinical use to industry standard, and strategies for optimal patient conditioning. The T2EVOLVE consortium unites scientists and physicians, regulators and policy makers, SMEs, and patient stakeholders to tackle these challenges in an orchestrated multi-disciplinary multi-stakeholder approach. A core feature of this approach will be the embedding of patient stakeholders as contributing members of the team across all levels of the R&D process. The overall aim is the development of an innovation ecosystem that will accelerate the process of developing engineered T cell therapy in the EU. The project will deliver novel tools for education and for improving the communication between healthcare providers and patients, optimized laboratory models that can help determine how safe and effective new therapies with engineered T cells are, standardized methods in which these therapies are produced and monitored during treatment. The consortium members are innovators and pioneers in this field that are dedicated to bringing the EU to the forefront of the global engineered T cell therapy movement. This effort will ensure that EU citizens will continue to have access to the most innovative and best-available medical care, provide guidance on how to implement this novel treatment into the EU health care system in a sustainable way, and secure a leading role for Europe in this emerging field in medicine and science, the economy and society.

The overall goal of the Big Data for Better Outcomes (BD4BO) programme is to facilitate the use of ‘big data’ to promote the development of value-based, outcomes-focused healthcare systems in Europe. To fully exploit the transformative potential of big data, consideration will need to be taken of the use of detailed personal and biological information across the spectrum of care delivery, starting from the development of innovative medicines and treatments, to market access and adoption, diffusion, and use in healthcare systems by providers and patients. This paradigm shift requires shared understanding and standards among healthcare stakeholders including patients, providers, payers, regulators, policy makers, pharmaceutical industry, and academia. OBJECTIVES The proposed Coordination and Support Action (CSA) will establish an enabling platform that brings together these stakeholder groups across the BD4BO programme to ensure quality and consistency of individual projects in line with the overarching programme objective. Our consortium therefore aims to promote the use of big Data for better Outcomes, policy Innovation and healthcare system Transformation (DO->IT). Accordingly, we will: • Define a programme strategy that ensures quality, consistency and sustainability of health outcomes related activities across individual BD4BO projects. • Integrate, synthesise, and manage knowledge from all BD4BO projects, making it easily accessible via a single knowledge exchange platform. • Act as pivotal point of collaboration, stakeholder engagement and communication for all BD4BO projects. • Provide transparency and enable the use of patient health data and human biological samples for research purposes by developing minimum data privacy standards for Informed Consent Forms (ICFs) and supporting materials for use by individual BD4BO disease-specific projects and more widely in the Research and Development (R&D) sector.

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 these 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.