HEcoPerMed responds to the demand for economic models that evaluate treatments made possible through innovations in personalised medicine and seeks to identify funding and reimbursement mechanisms that provide financial incentives for the rapid development and uptake of such innovations. HEcoPerMed goes beyond current assessment and payment models in order to serve the requirements of personalised medicine for more compre-hensive cost-effectiveness estimates that incorporate patient and societal perspectives and enhance sustainable affordability of cutting-edge health innovations. HEcoPerMed will provide an overview of and guidance on high-quality methodological approaches for model-based economic evaluations. In three case studies, HEcoPerMedwill apply state-of-the art economic modelling to demonstrate practical and methodological issues in evaluating personalised medicine innovations. The project team will also study existing shortcomings in stimulating the adoption of personalised medicine and pro-pose fin

COMPAR-EU aims to identify, compare, and rank the most effective and cost-effective self-management interventions (SMIs) for adults in Europe within four high-priority chronic conditions: type 2 diabetes, obesity, chronic obstructive pulmonary disease, and heart failure. This project addresses an important gap in current knowledge applying network meta-analysis, an extension of meta-analysis methodology that allows multiple (rather than pairwise) comparisons of intervention effectiveness, to randomised controlled trials (RCTs) that meet the study inclusion criteria. This centralised analysis of an estimated 4000 RCTs will substantially help to overcome current problems associated with the dispersion and duplication of evidence. The work will be based on a validated taxonomy of SMIs and will prioritise outcomes from the patients’ perspective. In addition, a cost-effectiveness of the most effective SMIs will be estimated to provide insights into the economic consequences of adopting SMIs for societies, healthcare budgets, and patients. Contextual factors associated with successful interventions will also be studied. Drawing on our results, we will develop and pilot decision-making tools to facilitate access to evidence-based information on the most effective SMIs to key users through a user-friendly interactive platform. A multiprong strategy for exploitation of the research findings will lead to clear business cases for implementing it in different contexts within the heterogeneous EU health system. The end goal of the project is to have an impact in supporting policy-makers, guideline developers, researchers, industry, professionals and patients to make informed decisions on the identification and implementation of the most suitable SMIs, therefore contributing to the diffusion of the knowledge, healthcare sustainability and equity and promoting EU competitiveness in a globally emerging market.

Current evidence from both randomized trials and real-world evidence studies suggests that older breast cancer patients derive clinical benefit from the addition of CDK 4/6-inhibitors to endocrine therapy but with higher risk for adverse events and treatment discontinuation compared to younger patients. The IMPORTANT project will design and conduct a pragmatic clinical study with a decentralised approach for older patients with advanced breast cancer aiming at high level of evidence (level I) with multi-layer results that can enable changes in current clinical practice. This study will be conducted across 6 (six) EU countries, and will include more than 500 female and male patients. The project will also perform a series of analyses over a) a wide range of clinical-relevant primary and secondary endpoints targeting to contribute to current clinical practice and b) the implementation of specific strategies aiming at more individualized treatment and follow-up approaches to a clinical trial setting that highly resembles real-world setting due to the trial's pragmatic design. The project will be developed in 60 months by a competitive consortium of 19 partners from 11 countries, which corresponds to a well-balanced structure, involving clinical sites, SMEs, universities and patient advocacy organizations. Despite the great diversity of entities within the proposal, IMPORTANT partners bring state-of-the-art complementary skills ensuring the ability of the consortium to successfully complete the proposed work.This action is part of the Cancer Mission cluster of projects on ‘Diagnosis and treatment’.

Parkinson’s Disease (PD) is a major neurodegenerative disorder with no established treatment modalities capable of modifying disease pathology, and no means of early diagnosis. Vaccines targeting aSyn aggregates are a promising route to disease-modifying therapy for PD, but the current generation of PD vaccines utilise conventional formulations, which are limited in their immunogenicity and require substantial quantities of adjuvant to achieve efficacy. NEXGEN’s proprietary WISIT vaccine platform is the first of the novel class of gluconeoconjugate vaccines (GNCVs), which are administered intradermally and specifically formulated to leverage skin dendritic cells (DCs) to generate substantially stronger and more specific immune responses than conventional vaccines. These stronger immune responses allow substantial reduction in adjuvants, while simultaneously increasing therapy efficacy. NEXGEN will identify and characterise candidate WISIT constructs targeting aSyn (PD-WISITs) and develop a novel extracellular vesicle (EV)-based biomarker assay that enables early diagnosis of PD using liquid biopsies, suitable for point of care use. Safety and efficacy of PD-WISITs will be demonstrated preclinically, before being translated to first-in-human Phase I/Ib clinical trials, along with the novel EV-based biomarker assay. The results of NEXGEN will be the extraordinary accomplishments of cheap and effective disease-modifying treatment of early PD and a novel biomarker assay to diagnose and guide prodromal/early PD treatment. Further still, GNCV technology will be clinically demonstrated, which has the potential to be transformative to the treatment of a wide range of additional diseases, resulting in far-reaching impacts to the health of millions.