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.

RealiseD implements a collaborative and compRehensive mEthodological and operational Approach to cLinical trIalS in rarE Diseases enabling timely drug development and approval centred on patients’ needs with high level of evidence reachable in a limited environment. The core of RealiseD operational approaches includes the systematic procedures for patients’ referral, the certification of clinical sites and filling the gaps for successful development of rare and ultra rare diseases[(U)RD]’ clinical trials using 4 (U)RD diseases from 4 European Research Networks (ERNs). RealiseD methodological teams will optimise innovative statistical and quantitative approaches for design and analysis of (U)RD-CTs developed in the past 10 years and refined in the last years to facilitate the regulatory pathway for innovative drug development. RealiseD is implemented in a public private partnership consortium including regulators and HTA bodies representatives to co-create operational and methodological approaches in an iterative procedure using a multistakeholders’ agreement process. RealiseD will increase the incentives and motivation for the pharmaceutical industry by reducing many uncertainties on the path of drug development in (U)RD that will follow widely accepted rules. To achieve these innovative approaches, RealiseD will capitalise on an international multistakeholders effort as the problem in (U)RD drug development cannot be solved in a restricted geographical area. With the development and deployment of playbooks arising from the co-created developments, RealiseD will ascertain visibility and readiness of the innovations. The overall ambition of RealiseD is to change the paradigm of CT design for U(RD) by enlarging the spectrum of methodological and operational approaches and to establish a sustainable, innovative, and optimised CT paradigm for U(RD) medicinal product development programs, while maximizing the acceptance by all stakeholders.

Drug safety assessment is a knowledge-intensive process that demands advancement in data handling methods and tools for facilitating data sharing, mining, analysis and predictive modelling. This need is not restricted to any specific type of data and real advancement requires integrating information of different types and from different sources (e.g. publicly available biomedical knowledge, proprietary preclinical and clinical data, evidence from post-marketing studies) for addressing drug safety assessment from a holistic perspective. The eTRANSAFE project will address these needs by developing a powerful data integration infrastructure (Knowledge Hub) and a collection of computational tools (exploitation modules) for making optimal use of these data. The proposed system will be a flexible and expandable framework, built on previous experience and applying state-of-the-art methods and technologies for providing the most advanced solutions for data sharing, interoperability and exploitation. An experienced honest broker will guarantee the confidentiality of any proprietary data. The project will develop in silico tools for data mining, visualisation, and prediction of potential toxicity, with specific attention to the assessment of the preclinical to clinical predictivity and the discovery of safety biomarkers. The CDISC-SEND format will be supported, facilitating the preparation of regulatory-compliant documentation. This infrastructure will be underpinned by development of open standards and guidelines recognised by regulatory agencies and international organisations. The eTRANSAFE consortium brings together 13 organizations that mobilize an unprecedented capacity and resources, uniquely placed to leverage the experience of eTOX and other EU projects and initiatives. The project partners are six prestigious academic institutions, six highly skilled small-medium enterprises (SME) and one European infrastructure (ELIXIR), bringing in this way a perfect balance.