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Background: In the European Union, the General Data Protection Regulation (GDPR) plays a central role in the complex health research legal framework. It aims to protect the fundamental right to the protection of individuals' personal data, while allowing the free movement of such data. However, it has been criticized for challenging the conduct of research. Existing scholarship has paid little attention to the experiences and views of the patient community. The aim of the study was to investigate 1) the awareness and knowledge of patients, carers, and members of patient organizations about the General Data Protection Regulation, 2) their experience with exercising data subject rights, and 3) their understanding of the notion of "data control" and preferences towards various data control tools. Methods: An online survey was disseminated between December 2022 and March 2023. Quantitative data was analyzed descriptively and inferentially. Answers to open-ended questions were analyzed using the thematic analysis method. Results: In total, 220 individuals from 28 European countries participated. The majority were patients (77%). Most participants had previously heard about the GDPR (90%) but had not exercised any of their data subject rights. Individual data control tools appeared to be marginally more important than collective tools. The willingness of participants to share personal data with data altruism organizations increased if patient representatives would be involved in the decision-making processes of such organizations. Conclusion: The results highlighted the importance of providing in-depth education about data protection. Although participants showed a slight preference towards individual control tools, the reflection based on existing scholarship identified that individual control holds risks that could be mitigated through carefully operationalized collective tools. The discussion of results was used to provide a critical view into the proposed European Health Data Space, which has yet to find a productive balance between individual control and allowing the reuse of personal data for research. [ABSTRACT FROM AUTHOR]

Background: Pragmatic clinical trials (PCTs) are designed to reflect how an investigational treatment would be applied in clinical practice. As such, unlike their explanatory counterparts, they measure therapeutic effectiveness and are capable of generating high-quality real-world evidence. However, the conduct of PCTs remains extremely rare. The scarcity of such studies has contributed to the emergence of the efficacy-effectiveness gap and has led to calls for launching more of them, including in the field of oncology. This analysis aimed to identify self-labelled pragmatic trials of antineoplastic interventions and to evaluate whether their use of this label was justified. Methods: We searched PubMed® and Embase® for publications corresponding with studies that investigated antitumor therapies and that were tagged as pragmatic in their titles, abstracts and/or index terms. Subsequently, we consulted all available source documents for the included trials and extracted relevant information from them. The data collected were then used to appraise the degree of pragmatism displayed by the PCTs with the help of the validated PRECIS-2 tool. Results: The literature search returned 803 unique records, of which 46 were retained upon conclusion of the screening process. This ultimately resulted in the identification of 42 distinct trials that carried the 'pragmatic' label. These studies examined eight different categories of neoplasms and were mostly randomized, open-label, multicentric, single-country trials sponsored by non-commercial parties. On a scale of one (very explanatory) to five (very pragmatic), the median PCT had a PRECIS-2 score per domain of 3.13 (interquartile range: 2.57–3.53). The most and least pragmatic studies in the sample had a score of 4.44 and 1.57, respectively. Only a minority of trials were described in sufficient detail to allow them to be graded across all domains of the PRECIS-2 instrument. Many of the studies examined also had features that arguably precluded them from being pragmatic altogether, such as being monocentric or placebo-controlled in nature. Conclusion: PCTs of antineoplastic treatments are generally no more pragmatic than they are explanatory. [ABSTRACT FROM AUTHOR]

Treatment optimisation studies (TOSs) are clinical trials which aim to tackle research questions that are often left unaddressed within the current drug development paradigm due to a lack of financial and regulatory incentives to undertake them. Examples include comparative effectiveness, therapeutic sequencing and dose de-escalation studies. Trials of this nature have historically been primarily carried out by academic institutions and not-for-profit organisations such as the European Organisation for Research and Treatment of Cancer (EORTC). Our objective was to conduct an in-depth analysis of the breast, lung and colorectal cancer TOSs that have been performed by the EORTC in the past four decades. We searched the EORTC clinical trials database for relevant studies and subsequently analysed them based on a number of predefined criteria relating to their design, organisation and scientific impact. The 113 EORTC TOSs examined in this analysis were mainly standard-sized, international, multicentre phase III trials using a relatively simple, randomised, open-label design and comparing pharmacological combination regimens against standard-of-care treatments in terms of their potential to improve overall survival of patients with cancer. Although they were typically financially and/or materially supported by the industry, their legal sponsor was nearly always an independent party that did not benefit monetarily from their outcomes. If meaningful findings were obtained, their results, regardless of whether positive or negative, were published in high-impact journals, and the corresponding articles usually received a considerable number of citations. Our analysis provides an empirical framework for setting up future TOSs based on the EORTC experience in oncology. • Treatment optimisation studies can address current evidence gaps in oncology. • Trials of this type remain poorly characterised in the existing literature. • The European Organisation for Research and Treatment of Cancer (EORTC) has decades of experience with conducting such studies. • An analysis of past EORTC trials offers insights into their key characteristics. • It yields an empirical framework for undertaking treatment optimisation studies. [ABSTRACT FROM AUTHOR]