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.

Advanced Therapy Medicinal Products (ATMPs) are medicines based on genes, cells and tissues for human use. They can provide patients novel treatment alternatives to significantly improve or even cure diseases that currently have no or inadequate standard-of-care options. However, significant challenges remain for the scientific, clinical and patient communities as well as commercial entities and regulatory, policy-making and technology-assessing bodies. The JOIN4ATMP consortium unites international experts and stakeholders with the mission to accelerate and de-risk European ATMP development and ensure wide-spread equitable ATMP access for patients with common diseases, rare non-cancer diseases and rare cancers in Europe. It brings together all members of the European University Hospital Alliance with the existing EU-funded T2EVOLVE and RESTORE networks, and with active support from industry partners and patient representatives as our 14 core partners. The core partners are joined by supporting partners active in ATMP development, production, application and regulation. Our team will use real-world data and experiences to achieve the most rapid and holistic map of current hurdles and the best way to improve support and regulation of ATMP development and use in the EU and associated countries. The JOIN4ATMP end-product will be a state-of-the-art information package circumscribing (i) currently existing and potential future hurdles preventing rapid and streamlined ATMP development and clinical translation and (ii) a defined set of recommendations to overcome these hurdles in a manner guaranteeing equitable access for patients throughout Europe. We are committed to the long-term operation of our network and take steps to assure its sustainability in order to inform and coordinate activities in Europe for the ATMP field, which will only gain in importance with the advance of personalized medicine in the future.

While randomized controlled trials (RCTs) remain the mainstay in drug development, approval, and reimbursement, the potential of real world data (RWD) to contribute to the understanding of drug effects is increasingly realized. Evidence, based on RWD – real world evidence (RWE) - can contribute significantly to the evidence to support decision making throughout all phases of (clinical) drug development, as well as improve efficiency in design and conduct of clinical trial programs. The aim of this project is to develop, implement and establish evidentiary standards and methods to address the data and evidentiary needs of regulatory authorities and HTA bodies towards a more efficient use of RWD for the development, registration and assessment of medicinal products in Europe (More-EUROPA).

Progress in the life sciences and related technologies offer great potential for therapeutic benefits to patients in need. However, major adaptations to current paradigms of bringing medicines to patients are required in order to realize that potential and to address important challenges in the healthcare ecosystem. Against this background, several initiatives are exploring new pathways to market, collectively referred to as Medicines Adaptive Pathways to Patients (MAPPs). The ADAPT-SMART consortium is aligning a limited number of major stakeholders eager to progress towards MAPPs implementation. It will act as a neutral collaborative platform that will engage industry, SMEs, regulators, Health Technology Assessment bodies (HTAs), payers, governments, clinicians and patients. The ADAPT-SMART consortium will contribute to align understanding of the impact of MAPPs, to share learnings between all stakeholders, and to allow the field to actively work towards MAPPs implementation. The impact of the ADAPT-SMART CSA will be a result of the delivery of • actionable advice/recommendations to IMI on how to best leverage results from past/current projects; • concrete proposals for future (IMI) projects; • actionable advice/recommendations and information to other actors in the healthcare environment; • synthesis of learnings from pilot projects and case studies with relevance to MAPPs; • communication of CSA outcomes by way of publications and conference presentations. This CSA will increase the probability of successful implementation of MAPPs and accelerate access to crucial therapies, thus improving the position of both the patients in need of novel treatments and the research-based pharmaceutical industry.

The Genetics Clinic of the Future (GCOF) project aims to ensure that the clinical implementation of genome technologies is relevant and responsive to the needs of all. It offers a stepping stone approach towards the genetics clinic of the future, engaging all stakeholders involved in a process of mutual learning and information exchange. The GCOF project implements key Science with and for Society issues, ensuring that ethical reflection and stakeholder involvement do not occur in parallel, but are effectively integrated in the core of the project. It establishes a robust communication and implementation strategy that integrates the project’s outcomes and recommendations in research and clinical practices and policy processes, outlining opportunities for a more responsive health research and innovation system by: 1. Envisioning the Genetics Clinic of the Future (WP1) 2. Mapping out the concept of data control (WP2) 3. Considering ethical and legal dimensions in the consent framework (WP3) 4. Exploring novel models for use of clinical data in research and vice versa (WP4) 5. Initiating public engagement, mutual learning and dissemination (WP5) 6. Engaging policy makers (WP6) The consortium brings together 12 key partners from 10 countries across Europe who represent the breadth of stakeholders involved in the genetics clinic of the future: genomics research, clinical genetics, bioinformatics, public health, policy making, patient representation, education, commercial genetics and bioinformatics services, social research, communication, responsible innovation and ethics and law. The GCOF project connects to the major EU-initiatives in the field of personalised health and care. The consortium also represents a variety of organisation types, including research organisations, businesses, policy makers, civil society organisations, education establishments and science & society centres.