Chemical exposure : european citizens’ perspectives, trust, and concerns on human biomonitoring initiatives, information needs, and scientific results

Copyright: © 2021 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).Over the last few decades, citizen awareness and perception of chemical products has been a topic of interest, particularly concerning national and international policy decision makers, expert/scientific platforms, and the European Union itself. To date, few qualitative studies on human biomonitoring have analysed communication materials, made recommendations in terms of biomonitoring surveillance, or asked for feedback in terms of specific biomonitoring methods. This paper provides in-depth insight on citizens’ perceptions of knowledge of biomonitoring, impact of chemical exposure on daily life, and claims on how results of research should be used. Four semi-structured focus groups were held in Austria, Portugal, Ireland, and the United Kingdom (UK). The cross-sectional observational qualitative design of this study allows for better understanding of public concern regarding chemicals, application, and use of human biomonitoring. The main findings of this study include citizens’ clear articulation on pathways of exposure, the demand on stakeholders for transparent decision-making, and sensitivity in communication of results to the public. Validated and trustful communication is perceived as key to empowering citizens to take action. The results can be used to facilitate decision-making and policy development, and feeds into the awareness needs of similar and future projects in human biomonitoring. Furthermore, it also brings to light ideas and concepts of citizens’ in shaping collaborative knowledge between citizens’, experts, scientists, and policy makers on equal terms.The HBM4EU project received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 73303info:eu-repo/semantics/publishedVersio

The pediatric precision oncology INFORM registry: clinical outcome and benefit for patients with very high-evidence targets

INFORM is a prospective, multinational registry gathering clinical and molecular data of relapsed, progressive, or high-risk pediatric patients with cancer. This report describes long-term follow-up of 519 patients in whom molecular alterations were evaluated according to a predefined seven-scale target prioritization algorithm. Mean turnaround time from sample receipt to report was 25.4 days. The highest target priority level was observed in 42 patients (8.1%). Of these, 20 patients received matched targeted treatment with a median progression-free survival of 204 days [95% confidence interval (CI), 99-not applicable], compared with 117 days (95% CI, 106-143;P= 0.011) in all other patients. The respective molecular targets were shown to be predictive for matched treatment response and not prognostic surrogates for improved outcome. Hereditary cancer predisposition syndromes were identified in 7.5% of patients, half of which were newly identified through the study. Integrated molecular analyses resulted in a change or refinement of diagnoses in 8.2% of cases. SIGNIFICANCE: The pediatric precision oncology INFORM registry prospectively tested a target prioritization algorithm in a real-world, multinational setting and identified subgroups of patients benefiting from matched targeted treatment with improved progression-free survival, refinement of diagnosis, and identification of hereditary cancer predisposition syndromes

Current state-of-the-art and gaps in platform trials: 10 things you should know, insights from EU-PEARL

Summary: Platform trials bring the promise of making clinical research more efficient and more patient centric. While their use has become more widespread, including their prominent role during the COVID-19 pandemic response, broader adoption of platform trials has been limited by the lack of experience and tools to navigate the critical upfront planning required to launch such collaborative studies. The European Union-Patient-cEntric clinicAl tRial pLatform (EU-PEARL) initiative has produced new methodologies to expand the use of platform trials with an overarching infrastructure and services embedded into Integrated Research Platforms (IRPs), in collaboration with patient representatives and through consultation with U.S. Food and Drug Administration and European Medicines Agency stakeholders. In this narrative review, we discuss the outlook for platform trials in Europe, including challenges related to infrastructure, design, adaptations, data sharing and regulation. Documents derived from the EU-PEARL project, alongside a literature search including PubMed and relevant grey literature (e.g., guidance from regulatory agencies and health technology agencies) were used as sources for a multi-stage collaborative process through which the 10 more important points based on lessons drawn from the EU-PEARL project were developed and summarised as guidance for the setup of platform trials. We conclude that early involvement of critical stakeholder such as regulatory agencies or patients are critical steps in the implementation and later acceptance of platform trials. Addressing these gaps will be critical for attaining the full potential of platform trials for patients. Funding: Innovative Medicines Initiative 2 Joint Undertaking with support from the European Union’s Horizon 2020 research and innovation programme and EFPIA