The vision of the PMPC (“Centre of excellence for precision medicine in pediatric care”) project is to position the Children’s Clinical University Hospital (CCUH) in Latvia at the leading-edge of pediatric precision medicine research and care in Europe. Precision medicine has proven its potential to revolutionize healthcare by tailoring treatments to individual patients based on their unique genetic makeup, environment and lifestyle factors. While it has shown promise and delivers results in adult populations, its potential in pediatric care is largely untapped. Our goal is to improve pediatric patient care and outcomes by establishing a new research Centre of Excellence (CoE) at CCUH’s campus for research on and adoption of innovative precision medicine approaches in an integrated and collaborative RESEARCH +INNOVATION+CARE+EDUCATION (“R+I+C+E”) environment. To this end, CCUH will team-up with Europe’s leading institute in pediatric precision medicine: the Princess Maxima Center, The Netherlands. Maxima has an extensive precision medicine program and uses advanced translational research and treatment expertise in many areas, including CAR-T cell therapies and pediatric clinical trials. The CoE will provide an environment for transdisciplinary team science with international visibility in the field of pediatric precision medicine. The center will leverage the latest advances in genomics, cell therapies, data science, and computational biology. CCUH has secured 20 million EUR from private donors as a complementary part of funding. Together with the Horizon Europe grant, the valuable knowledge/technology transfer and active collaboration with the Maxima team, PMPC’s unique value will be brought forward resulting in benefits for patients, their caregivers, researchers, healthcare professionals and society. It will strengthen CCUH’s scientific and technological capacity in precision medicine, enabling it to attain a competitive position in this promising field.

Every year 16.000 children in Europe are diagnosed with cancer, and 2000 children (approximately 70 school classes) die from this illness. New therapeutic solutions are advancing rapidly however the optimization and implementation into clinical trials accessible for these children is lacking behind. New therapeutic interventions often don’t make it through the valley of death between preclinical development and clinical implementation. In addition, the accessibility between countries of new treatments varies largely. Therefore, the GRASSHOPPER consortium aims to create a multidisciplinary network of stakeholders in pediatric cancer development to boost implementation of new therapeutic interventions for children with cancer across Europe and beyond. GRASSHOPPER consists of 10 beneficiaries and 7 associated partners located in 16 countries: 6 academic European Comprehensive Childhood Cancer Centers (CCCC’s) and 3 non-European CCCC’s, 5 non-academic national leading Childhood Cancer Centers, 2 leading pharmaceutical companies and 1 non-for-profit drug developer. The consortium has 4 objectives: identification and preclinical validation of new immune interventions (1) and new molecular interventions (2), stimulating repurposing of available therapeutic interventions (3) and improving clinical validation of high potential therapeutic interventions for pediatric cancer (4). These objectives will be achieved by intersectoral secondments between the various beneficiaries and partners. The consortium expects that these objectives will lead to a strong international and multidisciplinary network, exchange of knowledge and expertise between sectors and countries and lead to highly skilled researchers, trained in pediatric and transferable skills, and with a large network for future career perspectives, that will jump over the valley of death and accelerate new treatments.

Cross-border collaboration can tackle the challenges in accessing relevant health data essential for international collaboration between scientists and clinicians, researchers, and health industry. Privacy concerns and regulations on personal data have made the sharing of health data increasingly complex and time-consuming for data controllers, thus severely limiting the access of SMEs, researchers, and innovators to health data. Further complications in cross-border collaboration arise from differences in interpreting the EU GDPR, national regulations, and heterogenous and changing data permit processes at hospital sites. The PHEMS project will provide European children’s hospitals with a decentralized and open health data ecosystem concept consisting of technical components and governance frameworks. The objective is to facilitate access to health data, advance federated health data analysis and build services for the on-demand generation of shareable, synthetized, and anonymized datasets. To achieve this, the project will focus on bridging the gaps in data access and use, especially in the integration of ethical, legal, and technical requirements, including the responsibilities of data controllers and the rights of data subjects. This will allow health data controllers to engage in collaboration without losing control on compliance with respect to GDPR, national legislation or internal policies of their organization. The techniques and tools for generating algorithmically anonymized and synthetic datasets will undergo robust validation processes through three clinical use cases conducted by the European Children’s Hospitals Organisation (ECHO) community. The goal is to assess the usage of custom-generated synthetic data with real-life questions. Data users, such as researchers, SMEs, innovators and the pharmaceutical and MedTech industry, will be engaged through community building, hackathons, and interaction with relevant European large-scale initiatives.

The European Rare Diseases Research Alliance (ERDERA) aims to improve the health and well-being of the 30 million people living with a rare disease in Europe, by making Europe a world leader in Rare Disease (RD) research and innovation, to support concrete health benefits to rare disease patients, through better prevention, diagnosis and treatment. This Partnership will deliver a RD ecosystem that builds on the successes of previous programmes by supporting robust patient need-led research, developing new diagnostic methods and pathways, spearheading the digital transformational change connecting the dots between care, patient data and research, while ensuring strong alignment of strategies in RD research across countries and regions. Structuring goal-oriented public-private collaborations targeted at interventions all along the R&D value chain will ensure that the journey from knowledge to patient impact is expedited, thereby optimising EU innovation potential in RD. To support its ambition and missions ERDERA has been designed as a comprehensive and integrated ecosystem of which structure can be compared to an institute encompassing three main parts: (i) funding, (ii) internal (in house) Clinical Research Network that implements research activities targeting clinical trial readiness of RDs and accelerating diagnosis and translation of research discovery into improved patient care, and (iii) related supporting services (Data, Expertise, Education and Training) as well as an acceleration hub that serve external and internal RD community, all supported by all-embracing coordination and strategy and foundational (inter)national alignment.

Innovation in paediatric health is critical to ensure that children, regardless of their age, health condition, socio-economic situation or place of birth, have access to the life-changing healthcare solutions they need to grow-up stronger and contribute to a better society. Children are not just small adults and they need targeted innovative solutions. However, paediatric innovation is still elusive. There are two main causes for this: i) Stakeholders perceive paediatric innovation as risky and they just try to adapt adult solutions to children. ii) Paediatric experts, innovators and other relevant stakeholders are scattered in a fragmented ecosystem across Europe failing to identify opportunities or deliver effective and harmonized action. Thus, the unexploited innovation potential is large, particularly in emerging and moderate innovator countries. To reduce barriers and exploit these opportunities, while ensuring equality across different countries, the establishment of a European Paediatric Innovation Ecosystem is of paramount importance. The i4KIDS-EUROPE project builds on an existing successful initiative (i4KIDS), and it will expand its activities towards Europe in order to enlarge the participation of diverse European paediatric stakeholders and territories. The project will allow synergies, avoid duplications, reduce differences between strong and emerging/moderate innovator territories and strengthen competitiveness to optimise the innovation potential of the European paediatric sector. To achieve this, we will analyse the landscape of paediatric innovation in Europe as well as the barriers that emerging/moderate innovator countries face. i4KIDS-EUROPE will offer an interconnected paediatric innovation ecosystem that will capacitate its underrepresented actors and make them participate in the design of the future European Paediatric Innovation Ecosystem.