The public-private partnership, READI, seeks to help clinical studies (CS) to finally serve the complete general population, and therefore more patients. To date CS have struggled to recruit and retain participants from diverse backgrounds and communities, such as marginalized or disadvantaged groups (e.g., sexual, gender, age, cultural, and socioeconomic cohorts). The resulting knowledge gaps entrench or increase health disparities. The READI consortium strives to tackle these challenges by fostering a more cohesive and integrated CS ecosystem for underserved (US) and underrepresented (UR) communities. It will actively connect all key stakeholders who can facilitate access to a wide range of patient populations. It will provide these stakeholders with the necessary tools, training programs, and approaches essential for the recruitment and retention of US/UR patients in CS. In addition, it will design, build and implement a digital platform which is patient-centred, sustainable, open and innovative. This will foster improved access to CS information and READI tools, while also supporting patient connections with the created communities. Finally, at least 4 CS will be used for testing the effectiveness of the developed tools and approaches. READI has a three-fold objective: to help US/UR communities overcome CS participation barriers (e.g., lack of information/awareness, mistrust, poor communication, geographic limitations, prejudice), which in turn will improve research of many diseases and conditions, preventative care and treatment effectiveness in different demographic groups, and better serve society. READI’s success will draw from its interdisciplinary, multi-stakeholder, consortium composition of 73 organizations from 18 countries, with key expertise in drug development and CS (design and operations), engagement strategies for US/UR populations, digital platform development, training and capability building initiatives, effective communication and dissemination, long-term sustainability, ethics and regulatory affairs.

Cancer remains the leading cause of disease-related death in children. For the ~25% of children who experience relapses of their malignant solid tumors, usually after very intensive first-line therapy, curative treatment options are scarce. Preclinical drug testing to identify promising treatment options that match the molecular make-up of the tumor is hampered by the facts that i) molecular genetic data on pediatric solid tumors from relapsed patients and thus our understanding of tumor evolution and therapy resistance are very limited to date and ii) for many of the high-risk entities no appropriate and molecularly well characterized patient-derived models and/or genetic mouse models are currently available. Thus, quality-assured upfront preclinical testing of novel molecularly targeted compounds in a (saturated) repertoire of well-characterized models will establish the basis to increase therapeutic successes of these drugs in children with solid malignancies. Since these tumors are overall genetically much less complex than their adult counterparts, it is anticipated that it will be easier to identify powerful predictive biomarkers to allow for accurate matching of targets and drugs. To address this high, as yet unmet clinical need, we have formed the ITCC-P4 consortium consisting of academic and commercial partners from 8 European countries and covering the full spectrum of qualifications needed for quality-assured preclinical drug development including expertise in patient derived models, histopathology, in vivo pharmacology, bioinformatics and data management, centralized testing capabilities, medical expertise regarding the entities in question, regulatory knowledge, and project management of large consortia. With this consortium in a public-private partnership with the participating pharma companies we strongly believe to be ideally positioned to greatly expedite the development of more precise and efficacious drugs for children with malignant solid tumors

Europe generates real world health data (RWD) that has the potential to inform the development and evaluation of medicines and medical devices. However, multiple barriers remain that limit the access, analysis, interpretation, and use of RWD, hampering its use. Additionally, the limited uptake of existing guidelines for the generation and use of real world evidence (RWE) adds complexity in the use of RWD to inform decision making. The GREG consortium will leverage the learnings of previous and ongoing key RWE initiatives to fill these gaps by generating, pilot-testing, and disseminating evidence-based guidance and tools for the use of RWE to inform the development and evaluation of medicines, medical devices, and combinations. To achieve these goals, we will work with key stakeholders to iteratively test and improve our guidance and tools, backed by case studies from previous successful and unsuccessful examples. We will engage with the most relevant European RWE initiatives and access the largest network of RWD partners in Europe, namely the European Health Data and Evidence Network (EHDEN) through our partner, the EHDEN Foundation. Additionally, we will provide multi-stakeholder gatherings (including patient and public representatives) to promote the dissemination, adoption, and implementation of RWE for decision-making in Europe. Our public and private partners will co-create use cases working closely with key regulatory and health technology experts in bespoke fora. These will be used initially to evaluate existing guidelines, and later to pilot-test the GREG guidance and tools. Our outputs will include training for all involved stakeholders on the use of our guidance and tools, and practical templates to facilitate regulatory and health technology/payer submissions. Together, these will accelerate access to better medicines and medical devices for European citizens.

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

The burden of cardiovascular disease (CVD) on society is huge with >85 million people affected in Europe. The overall prevalence continues to grow due to unhealthy lifestyles and population aging. Heart failure (HF) is the final common pathway of all CVD and has a 5 year mortality rate of 20-50% despite significant advances in therapy. iCARE4CVD aims to address this burden by contributing to three essential steps to improve the current care pathways, covering all stages from early risk to established HF: 1) early diagnosis to identify patients at risk of CVD and divide them into clinically meaningful subgroups; 2) risk stratification for these subgroups to define the urgency for intervention; and 3) prediction of treatment response for each subgroup. This will be achieved by the following steps: clinical partners will provide a large set of cohorts including >1,000,000 patients with a wide range of biomarkers (e.g. digital, blood, imaging). Anonymous access to data will be enabled by using a blockchain-supported federated database. Artificial intelligence-based modeling also considering patient relevant factors will assess changes in risk and stratify patients according to their individual responses to therapy. Results will then be prospectively validated in new and ongoing large cohorts and a pilot trial to test the prediction of treatment response by using multiple biomarkers going beyond current risk prediction (such as SCORE) towards individualized therapy. Results will be used to provide novel decision tools for each step targeting newly identified subgroups and as a blueprint for innovative future trials to individualise prevention and therapy. Patient involvement is key in every part of iCARE4CVD (e.g. patient advisory board, country-specific Patient Panels) to build a motivational framework for self-care by patients. The project brings together an EU-wide consortium with the needed resources and expertise from the public and private side to bring iCARE4CVD to success.