Clinical trials increase in size, complexity and costs. This is fuelled with the need to demonstrate effects in more complex therapeutic areas, and to detect subgroups with different benefit and safety responses. Complexities, rigid clinical control, physical distance and (perceived) burden put patient engagement under pressure. (S)low recruitment and retention compromise efficiency, generalisability and validity of traditional, site-centred trials. Remote Decentralized Clinical Trials (RDCTs) and hybrid approaches address these challenges. RDCTs are an operational strategy for technology-enhanced clinical trials, which enable (semi-)continuous data collection and real-world evidence generation, increase patient recruitment and retention and decrease patient and investigator burden and costs. Trials brought to the home of patients. Paradigmatic changes in EU clinical trial design are required to fully benefit from the digital era. Yet, the feasibility of running RDCTs needs to be rigorously demonstrated together with guidance and support measures for their execution. Trials@Home brings together a very strong consortium and will reshape clinical trial design, conduct and operations, by analysing, developing and piloting standards, recommendations and tools to define and operationalize RDCTs. Trials@Home will design and run a pan-European RDCT pilot based on: a. best practices of trials with RDCT elements, b. assessment of latest technological tools, c. the regulatory and ethical framework and potential changes required to facilitate RDCTs and d. stakeholder perspectives on the change from classical RCTs to RDCTs with strong patient involvement. The results of these assessments and the pilot will drive the formulation and dissemination of recommendations and tools for the implementation of RDCTs in Europe with the ultimate goal to improve the speed, quality and efficiency of clinical trials, and improving patients’ access to innovative treatment strategies.

Pharmaceutical industry, as well as basic science, depend on robust data and scientific rigor as key drivers for decision making, patent strength and time-to-market, which in turn will determine knowledge gain and availability of new treatments to patients. Recent publications report challenges with the robustness, rigor, and/or validity of research data, which may impact decisions about whether to proceed to preclinical and clinical testing as well as conclusions on the predictability of preclinical models. There is a need for simple, sustainable solutions that facilitate data quality. We will propose simple, sustainable solutions that facilitate data quality without impacting innovation and freedom of research. Our consortium will pool resources from both academia and industry to pilot this action in Neuroscience and Safety, but with applicability beyond these R&D areas. The European Quality In Preclinical Data (EQIPD) consortium will (i) define those variables in study design and data analysis that influence outcome in pre-clinical neuroscience (focus on Alzheimer’s disease and psychosis) and (neuro-)safety studies conducted in industry; and establish whether these are the same variables which influence outcome in academia; (ii) define the components which will make up the EQIPD quality management system; (iii) define consensus quality management recommendations for non-regulated R&D; (iv) validate the feasibility of the quality management system in prospective studies; (v) deliver an online educational platform providing certified education and training in the principles and application of quality and rigour. We will use systematic review and meta-analysis of historical data sets from industry and academia to identify variables of study design which determine outcomes in preclinical studies. Informed by the outcome of these analyses, we will use a Delphi approach to reach consensus around core principles for preclinical robustness, validate these approaches in cross site experiments and establish ring testing experiments in non-regulated research. We will develop a quality system framework to attest to compliance and a governance system to ensure sustainability and relevance. We will develop an educational platform to ensure research community-wide expansion of knowledge on criteria and principles necessary to address robustness and quality. Junior researchers are involved in many of the tasks and are enrolled in an academia/industry joint exchange scheme. They will form the core from where a cultural change should emerge in form of a ripple effect. Consortium members have made pivotal scientific and policy contributions relating to the robustness, rigor and validity of pre-clinical research in the past and together have the know-how and infrastructure necessary to succeed in this highly ambitious project.

THERACAT is an international and multidisciplinary consortium aiming at the training of 13 ESRs on the innovative topic of novel bio-orthogonal catalysis-based tools for cancer therapy. This ETN comprises 6 academic partners, 3 industrial partners active in the pharmaceutical market (1 large pharmaceutical company, Teva, and 2 SMEs, BiogelX and Tagworks) and 3 partners with focus on science communication (Cancer research UK), gender and minorities (UAB-Observatory for Equality) and management and entrepreneurship (ESADE business school). The combination of academic, private and society-involved organisations will provide a broad training for the 13 ESRs recruited, equipping them with the necessary skills to succeed as scientists, industrial researchers and entrepreneurs. The development of novel cancer therapies is a major challenge for academic research and pharmaceutical industries. THERACAT aims to establish a training programme focused on the development of THERApeutic CATalysts. In this strategy, materials bearing a catalytic unit are delivered to the tumour and subsequently non-active prodrugs are administered. The prodrugs are non-toxic and therefore generate limited side effects. Only at the tumour site the catalytic particles convert the prodrugs into active compounds that generate a therapeutic effect. This approach presents several advantages on the classical drug delivery paradigm including limited side effects and prolonged efficacy. This multidisciplinary research programme will be the setting for the training of 13 ESRs. The combination of the research expertise, the cutting-edge facilities and the complementary skills present in the consortium holds a great promise for the advancement of their careers, as well as of the knowledge of catalysis-based anticancer therapies and the development of marketable technologies and products.

RealHOPE will create an understanding of the real-life handling of protein drugs in hospital pharmacy, clinics and in the hands of patients by applying smart tag technologies. Different parameters will be logged and combined with protein characterisation at different stages, as well as with information from EFPIA partners on their drugs in use. Statistical evaluation of the data will be used to identify patterns in handling that are linked to protein destabilisation occurrence and type of protein degradation. Focus interviews with personnel in hospital pharmacies, clinics and with patients/care givers will be used to understand current handling practice and what the desired handling instructions and limitations are. These insights will be used to design in-use mimicking stability protocols for the protein drugs in the project. Ultra-scaled-down devices for stability assessment will be designed to be used in early phase for efficient development cost effective and safe future protein therapies. Protocols and devices will be validated towards the collected handling data base. Interventions in hospital pharmacies using e.g. compounding robots will be investigated. Techniques to assess e.g. aggregate formation in the final drug preparation situation will be evaluated together with SMEs producing analytical tools and hospital pharmacists. The collected data and interviews will form the base of development of teaching materials directed towards different target groups: hospital pharmacists, nurses and patients/care givers. App developers will be active in this part to design attractive and efficient apps for teaching and collecting therapy performance data.

There is a very high need for improving the management of pain. Acute and persistent pain of different origins represent a common medical, social, and economic burden, and its pharmacotherapy is often inadequate. To advance management of pain patients and support decision making in clinical practice, more predictive assessments of treatment success are needed. The development of analgesics is onerous because promising preclinical data often do not translate into the clinic. Improved pharmacodynamic biomarkers could define whether nociceptive signalling is adequately modulated by a new drug, so increasing the chance of successful translation and greatly reducing the risk in initiating clinical development. Further, the pathophysiology of chronic pelvic pain indications is poorly understood and no adequate preclinical models are available, precluding focused preclinical research and leaving affected patients with little hope of relief. IMI-PainCare aims at making advances in these three pain areas in a complementary manner. Three subprojects will address specific scientific challenges. Subproject PROMPT will identify Patient Reported Outcome Measures as tools to standardise assessments of treatment success of acute and chronic pain in Real World conditions and controlled trials, and so improve its management; subproject BioPain will validate the translatability of pharmacodynamic biomarkers and PK-PD modelling in pain pathways of healthy subjects and preclinical species, thereby offering tools to improve drug development; subprojectTRiPP will identify biomarkers and novel therapeutic pathways of clinical phenotypes of patients with chronic pelvic pain, which after back-translation, can improve how preclinical models reflecting human diseases. The goal of IMI-PainCare is to improve the care of patients with acute or chronic pain by providing a toolbox to streamline the development process for novel analgesic drugs and to improve treatment quality in clinical practice.