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.

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.

FACILITATE is a project built on a patient-centered, data-driven, technological platform where an innovative data sharing and re-use process allows the returning of clinical trial data to study participants within a GDPR compliant and approved ethical framework. FACILITATE starts-off by providing clear rules in a trusted ethical, legal and regulatory ecosystem before engaging patients as data generators. This avoids the current situation where clinical data are siloed in separate repositories without any possibility to be used beyond their original single-sided purpose. FACILITATE will provide the technological solutions to comply with GDPR in medical research by building on the empowered stakeholders' willingness to share and re-use their data. The FACILITATE Consortium was constituted by drawing from a broad range of capacities to tackle the ambitious challenges related to future clinical trials, such as preventive, long-term and real-world evidence trials. The Consortium took an innovative approach to the data return to study participants by asking them what they needed to be implemented to feel in a trusted ecosystem. This required all Consortium participants to leverage on their existing networks to bring together stakeholders at all levels in the decision-making chain, including patients, healthcare professionals, software designers, clinical trials repositories processors and controllers, ethicists, lawyers and other active regulators. Having obtained a consent on the data portability FACILITATE will re-use and cross-reference them with those contained in other repositories including RWE data captured across multiple settings and devices. FACILITATE will last 4 years and will participate in the extended Pilot on Open Research Data of Horizon 2020. Its strategy represents a unique and innovative opportunity for medicines drug development and regulation to better understand the clinics of diseases, and to evaluate the effectiveness of products in the healthcare system.

Worldwide, ~7 million women live with or beyond Breast Cancer (BC), as 10-year survival rates exceeding 80% for early-stage (I-III) BC. Premenopausal BC accounts for 25% in the EU and 55% in low/middle income countries. Most premenopausal women with BC have high risk of recurrence, therefore standard treatment includes adjuvant chemo- (CT) and endocrine therapy (ET). However, treatment has substantial physical, emotional and social burden, which is often more pressing for younger compared to older patients. Gene-expression assays are used for post-menopausal patients to identify women who can safely forego CT without detriment on clinical outcomes, preserving Quality of Life (QOL). However, a definitive study has yet to be conducted among premenopausal women with high-risk HR+HER2-BC. The primary objective of PATH-FOR-YOUNG is to conduct a pragmatic randomized controlled trial (RCT) with 5000 patients validating the use of a gene-expression assay to drive adjuvant treatment decisions in this target population. PATH-FOR-YOUNG aims to achieve its objective in 7 years. The project (i) builds on and complements an ongoing twin RCT to ensure timely recruitment, (ii) leverages on a large international consortium of oncologists, pathologists, patient representatives, psychologists, sociologists, ethics and communication experts, biostatisticians, health economists, and technology providers, to assure complementary and multidisciplinary expertise, and (iii) highlights patient-engagement, participatory care, and early involvement of end users. PATH-FOR-YOUNG will also study implementation of digital self-management to support patients throughout the cancer journey and particularly while on ET to improve QOL and medication adherence. A full HTA will ensure a path towards cross-country implementation. PATH-FOR-YOUNG has the ambition to improve BC care, fully integrating the predictive, personalized, preventive, and participatory principles of health management.

Drug repurposing can fill an important gap for rare disease patient groups with large unmet medical needs. In comparison to traditional drug development, drug repurposing reduces the time and costs for drug development, regulatory approval, and market authorization. Yet, we need to increase the efficiency of the drug repurposing pathway to provide broader access to new therapeutic modalities for larger groups of patients. SIMPATHIC’s main objective is to accelerate drug repurposing for rare neurological, neurometabolic and neuromuscular disorders. SIMPATHIC’s main accelerating innovation is the simultaneous drug development for groups of patients with different genetic diagnoses but overlapping neurological symptoms and molecular pathomechanisms. SIMPATHIC’s key outputs accelerating the drug repurposing pathway include: Standard operating procedures for culturing stem cell-derived neuronal cell models with proven relevance for clinical symptoms and amenable to high-throughput drug screens; New drug repurposing candidates with proven efficacy in advanced brain-on-a-chip and 3D brain organoid models, as demonstrated by reversal of molecular biomarker signatures and cellular readouts associated with clinical symptoms; Designs of innovative basket clinical trials to which patients with different disorders are recruited, utilizing and aggregating personalized clinical endpoints; A training module for patients and patient organizations to empower them as drivers of the drug repurposing pathway; Blueprints for intellectual property strategies, business models, regulatory dossiers and patient access strategies, developed in co-creation between all relevant stakeholders. SIMPATHIC’s proof-of-concept for the simultaneous development of repurposed drugs for multiple indications will show the path forward to development of personalized treatment opportunities for groups of rare disease patients in a cost- and time-efficient manner.