Chronic Kidney Disease (CKD) is one of the major causes of death in high income countries and its prevalence is growing rapidly. New therapies for the treatment of CKD have shown clear efficacy at a population level. Yet, individual patients respond differently with many patients showing suboptimal efficacy. This indicates that a ‘one size fits all’ approach is no longer sustainable. Currently there are no validated pharmacodynamic biomarkers in CKD that aid in guiding optimal therapy for individual patients. Furthermore, there remains a large gap between biomarker research and actual implementation of biomarker based care in daily clinical practice. Thus, there is an urgent need to validate and translate biomarkers as tools for daily clinical practice. PRIME-CKD has a unique advantage to address this challenge as it builds on recently discovered pharmacodynamic biomarkers for CKD treatment, and has access to a vast amount of clinical data, bio-samples. PRIME CKD will: 1) Provide a breakthrough in the pharmacotherapy of CKD with the validation and implementation of pharmacodynamic biomarkers which will advance personalized medicine. 2) Scientifically validate innovative clinical trial elements, pipelines and qualification procedures that can be used by researchers, diagnostics industries and regulatory authorities. 3) Establish a Stakeholder Network to truly engage patients, clinicians and other stakeholders in implementation of personalized medicines 4) Develop a roadmap for biomarker use in daily clinical practice for the optimal treatment of CKD. These results will enable clinicians to match patients with CKD with the most effective available drug within the shortest possible timeframe leading to less disease burden and progression of disease, enhanced quality of life and increased cost-efficiency. PRIME-CKD results will thus unlock the potential of biomarkers in healthcare and close the gap between biomarker research and clinical use in daily practice.
There is an urgent need for novel approaches assessing functional decline in early AD. The main goal of the RADAR-AD project is to develop a digital platform to detect subtle functional deficits in early Alzheimer’s disease (AD) individuals by integrating a meaningful combination of smartphone, wearable and/or home sensor based parameters. The system developed will be suitable for future longitudinal studies, including trials. The objectives are to 1) Identify the most relevant functional domains and the most promising remote measurement tools (RMTs) for these domains based on reviewing the literature and piloting of RMTs in small studies; 2) Optimise the RADAR-CNS platform for use in AD studies; 3) Test the platform and selected RMTs in a real world environment clinical study with 240 participants across the AD spectrum ranging from the preclinical AD to the dementia stage; 4) Perform statistical modelling to estimate longitudinal predictions; 5) Discuss results with regulatory agencies in order to obtain guidance about how to develop a path for formal qualification as outcome measurements in future therapeutic interventions. Considering the limited budget and project duration, we will rely upon already available technology platforms and on available longitudinal datasets where possible. The consortium includes experts in clinical dementia studies, computer science, bioinformatics, regulatory policies, ethics, and patient and public involvement (PPI). Additional strengths of the consortium are the deep and broad interface with RADAR-CNS and related IMI projects, and the access to large amount of patient-level data from key European cohort studies for modelling purposes.
Lack of specific relevant know-how in regulatory science delays the development of new treatment strategies or limits the chances that promising innovations will reach patients. STARS aims to improve the direct regulatory impact of results obtained in medical research. Seventeen European countries are represented in the consortium through their national competent authorities, alongside academic and industry representatives, and associations with relevant experience. The work plan includes the development of a Comprehensive Inventory of existing support activities based on a detailed analysis of the currently established programmes. This analysis is also the basis for development of a Common Strategy to strengthen regulatory sciences and two curricula, the Core Curriculum specifying essential knowledge for the professional training of clinical scientists and the Comprehensive Curriculum defining relevant knowledge for specific post-graduate programmes. Three pilot projects aim (i) to transfer an identified best practice example for training programmes to other EEA countries, (ii) to implement a new support activity addressing a gap in regulatory knowledge of significant relevance and (iii) to implement the Comprehensive Curriculum. STARS will deliver consensual recommendations ensuring sustainable support of academic research and will propose additional support mechanisms based on a comprehensive analysis of needs. STARS has the objective and the potential to complement, coordinate and harmonise regulatory efforts among Member States and at European level to support academic health research for the benefit of patients. The aim is to reach academic researchers very early in the planning of relevant grant applications. A further aim is to strengthen regulatory knowledge in general by reaching clinical scientists during professional training and qualification.
The aim of ROADMAP is to provide the foundation for a Europe-wide real world evidence (RWE) platform in AD by piloting multi-modal data integration tools and engaging with all key stakeholder groups for consensual definition of patient outcomes, tools and methods that are actionable and relevant. ROADMAP will leverage best practice and exploit synergies with other projects and initiatives at the national and European levels in pursue of scalable and transferable solutions for dataset characterisation, outcome classification, data standards, data sourcing, software application and guidelines on the handling and interpretation of RWE data. In parallel, the project will deepen understanding of the ethical, legal and social implications (ELSI) and health economics (HE) impact of a RWE approach for a meaningful contribution to the Big Data for Better Outcomes programme in IMI2. The ROADMAP programme will consist of 8 integrated work packages by which the Consortium will work to (1) identify relevant AD outcomes and progression markers; (2) identify and pool AD-related RWE across data sources; (3) develop and validate disease progression models combining diverse datasets and strategies; (4) develop risk/value-based costing and health economics models for HTA/regulators, service providers, industry and carers; (5) establish stakeholder-based guidelines for RWE; (6) develop a communication strategy focussing on the needs of patients and professionals; (7) develop an ELSI framework for the development and application of RWE in AD. The ROADMAP consortium brings together outstanding expertise, experience and traction in the field, including many of Europe’s top institutions, authorities, companies, experts in AD, biomedical informatics and analysis of epidemiologic and routinely collected health data sources, providing direct connections to key initiatives such as DPUK, EMIF, EPAD and others.
Lot release testing of established vaccines relies on using animal models. Many of these models were developed decades ago and reflect the level of product knowledge, production technology and quality management in those days. Current production techniques are optimised, in-process control is implemented and tight quality management systems are in place. These developments have brought testing for lot-to-lot consistency into reach. The consistency approach involves the use of a set of parameters obtained by using innovative in vitro technologies, to define the product along the manufacturing pathway and to ensure similarity to a lot of proven efficacy and safety. The ambition of the VAC2VAC project is a demonstrated proof of concept of the consistency approach for lot release testing. We will develop/optimise and validate innovative in vitro methods for the categories of established vaccines on the market. Product profiles will be defined using analytical methods, cell-based assays and bioinformatics. The project includes 7 WPs: 4 on R&D, and one each on validation, promotion to regulatory acceptance and consortium management. Consortium partners represent the vaccine stakeholder groups (OMCLs, Academia, Translational research institutes and Vaccinology alliances) and are complementary for the expertise ranging from method development and validation to facilitating and providing guidance to regulatory acceptance. Acceptance will be promoted by strategic guidance activities: a roadmap conference, workshops and training courses. Implementation of the consistency approach in lot release testing has multiple impact: 1) strengthening fundamental understanding of vaccine products resulting in enhanced understanding of vaccine quality 2) strengthening competitiveness of European Vaccine Industries resulting in reduced QC costs and shortened QC time. 3) strengthening European animal welfare policies resulting in a substantial reduction in animal numbers for vaccine QC