As recognized by the Council Recommendation 2009/C 151/02, rare diseases (RD) are a prime example of a research area that can strongly profit from coordination on a European and international scale. RD research should be improved to overcome fragmentation, leading to efficacious use of data and resources, faster scientific progress and competitiveness, and most importantly to decrease unnecessary hardship and prolonged suffering of RD patients. In the specific context of the massive generation, need for reuse and efficient interpretation of data, introduction of omics into care practice and the structuration of RD care centers in European Reference Networks, it appears crucial and timely to maximize the potential of already funded tools and programmes by supporting them further, scaling up, linking, and most importantly, adapting them to the needs of end-users through implementation tests in real settings. Such a concerted effort is necessary to develop a sustainable ecosystem allowing a virtuous circle between RD care, research and medical innovation. To achieve this goal, the European Joint Programme on RD (EJP RD) has two major objectives: (i) To improve the integration, the efficacy, the production and the social impact of research on RD through the development, demonstration and promotion of Europe/world-wide sharing of research and clinical data, materials, processes, knowledge and know-how; (ii) To implement and further develop an efficient model of financial support for all types of research on RD (fundamental, clinical, epidemiological, social, economic, health service) coupled with accelerated exploitation of research results for benefit of patients. To this end, the EJP RD actions will be organized within four major Pillars assisted by the central coordination: (P1): Funding of research; (P2): Coordinated access to data and services; (P3) Capacity building; (P4): Accelerated translation of research projects and improvement outcomes of clinical studies.

ERAMET will provide an integrated approach for developers and regulators’ decision-making for paediatric and orphan drugs, centred on the drug development questions. This will constitute a transparent ecosystem for drug development and assessment, that will facilitate the adoption of modelling and simulation (M&S) methods and related data types (including real word data such as registries and electronic healthcare data). The overall objective of ERAMET is to provide and implement a framework for establishing the credibility of M&S methods and related results as sources of evidence within regulatory procedures. The ecosystem proposed by ERAMET will be based on three pillars: (1) A repository connecting questions, data and methods. (2) The development and validation of high-quality standards for data and analytical methods (including M&S and hybrid approaches). These will cover computational M&S, digital twins, AI, hybrid approaches, standard statistics and pharmacometrics, as analytical methods and alternative data types and sources such as RWD, eHealth data, registries, historical regulatory submissions, scientific and (non)clinical trials). (3) An AI-based platform that will automate and optimise the data collection, formatting and modelling and simulation analysis and implement the credibility assessment. As part of ERAMET, the ecosystem will be applied to five use-cases including paediatric extrapolation and characterisation of drug benefit/risk in 4 groups of rare diseases, namely ataxia, transfusion dependent haemoglobinopathies, bronchopulmonary dysplasia, and degenerative neuromuscular. Each of the use-case is planned to lead to submission and regulatory approval of at least one validated M&S tool via the EMA qualification procedure. Training will be proposed to familiarise regulatory assessors, drug developers and clinical researchers with this new approach.

"Disorders of the brain, i.e. mental and neurological conditions, impose a tremendous social and economic burden on European society and are among the leading causes of disability and death. In 2010, it was estimated that approximately 179 million European citizens were living with a brain disease (Gustavsson et al, 2010). Recent figures provided by the OECD estimated that in 2015 ill-mental health alone costed the EU more than 600 billion euro (OECD, 2018). Data released by the JAMA Psychiatry in 2015 indicated that 14,3% of deaths worldwide were attributable to mental disorders (Walker et al, 2015). What is more, the Lancet recently estimated that neurological conditions were the cause of 16,8% of global deaths in 2015 (GBD 2015 Neurological Disorders Collaborator Group, 2017).In the light of the above, it is crucial that robust measures are jointly taken by EU governments, patient groups, caregivers and scientific communities across Europe in order to address the human burden caused by brain disorders. In December 2017, the Norwegian Government took an important step in this direction by launching a National Brain Health Strategy which constitutes a key tool for advancing knowledge about the diseased brain, enhancing brain health and supporting brain research.The “Share4Brain” project aims to follow the example of Norway by uniting patients, brain researchers, scientists, clinicians and carers at national level and providing them with the skills and knowledge necessary to promote brain research and raise awareness about the societal impact of brain diseases. Representatives of the above groups will meet on multiple occasions during the lifecycle of the project in order to share good practices, exchange perspectives on effective advocacy strategies and learn how to engage with decision-makers at national and EU levels. What is more, the project aims to bring them together within National Brain Councils (NBCs), thereby uniting all stakeholders involved in brain research within transversal platforms that operate at country level. This will enable them to speak with a single voice when addressing societal problems associated with brain conditions. Lastly, “Share4Brain” will aim to enhance cross-border cooperation between NBCs for the purpose of defining key priorities for National Brain Plans, which will ultimately benefit people living with mental or neurological conditions. Our goal is to draw the attention of the academic and the general public, as well as relevant state institutions, to the importance of the development of basic and clinical research in the field of neurosciences, as well as to ensure, by integrating all levels of research, better prevention, better diagnosis and better treatment of patients suffering from neurological and psychiatric disorders. During the second year, we will compose a report on ""Successful Campaigns and Strategies for Brain Awareness in Europe"". The European Brain Council (EBC) will share results of European level research for the Brain. We will create a project website in WordPress platform, linking to the partners’ own websites, where all project outcomes will be uploaded. Several Focus Groups organized by representatives of the partners aimed at familiarizing the participants with their work will be facilitated during each transnational meeting. Furthermore, a Webinar on relevant EU policies impacting brain research as well as on how to engage with the European Institutions will be organized as part of the project. Lastly, various round-table discussions will be held in order to enable the participants to share their perspectives on priorities that should be included in National Brain Plans.What is more, all stakeholders will be invited to share good practices and examples of brain advocacy strategies in which they themselves have been involved. By including in this Erasmus+ partnership the partner “SrBC” from Serbia we widen the collaboration in Brain Education not only between EU and Member states but also by including emerging Associated countries and regions. By inviting the partner Greek Carers Network we underline the significant contribution made by family carers of patients with brain disorders to health and the European economy as a whole. Patients will be included in the project in the form of a Patient Advisory Board (PAB). We will encourage the participation of the members of the PAB who will be people suffering from Brain disorders .The above goals will primarily be achieved by means of a series of project meetings, focus groups, workshops in Brussels and Bari, What is more, a main conference, constituting a key milestone in promoting brain health, will be held in Athens at the office of the European Parliament in Greece during September 2020 and a Brain Awareness event in Madrid. During these events, the participants will be introduced to various cases constituting effective brain health advocacy strategies."