The May 2022 proposal from the European commission for a ‘European health data space’ envisages advantages for health from exploiting the growing mass of health data in Europe. However, key stakeholders have identified aspects that demand clarification to ensure success. Data will need to be freed from traditional silos to flow more easily and to cross artificial borders. Wide engagement will be necessary among healthcare professionals, researchers, and the patients and citizens that stand to gain the most but whose trust must be won if they are to allow use or transfer of their data. This paper aims to alert the wider scientific community to the impact the ongoing discussions among lawmakers will have. Based on the literature and the consensus findings of an expert multistakeholder panel organised by the European Alliance for Personalised Medicine (EAPM) in June 2022, it highlights the key issues at the intersection of science and policy, and the potential implications for health research for years, perhaps decades, to come.

Background: Neglected tropical diseases (NTDs) represent a diverse group of 20 communicable diseases that occur in tropical and subtropical areas in 149 countries, affecting over 1 billion people and costing developing economies billions of dollars every year. Within these diseases, those that present lesions on the skin surface are classified as skin NTDs (sNTDs). Mobile health interventions are currently being used worldwide to manage skin diseases and can be a good strategy in the epidemiological and clinical management of sNTDs.Objective: We aimed to analyze existing evidence about mobile health interventions to control and manage sNTDs in low- and middle-income countries (LMICs) and make recommendations for what should be considered in future interventions.Methods: A systematic review was conducted of the MEDLINE, Embase, and Scopus databases over 10 years up to April 30, 2020. All types of clinical studies were considered. Data were synthesized into evidence tables. Apps were selected through a comprehensive systematic search in the Google Play Store and Apple App Store conducted between March 20 and April 15, 2020.Results: From 133 potentially relevant publications, 13 studies met our criteria (9.8%). These analyzed eight different interventions (three SMS text messaging interventions and five app interventions). Six of the 13 (46%) studies were community-based cross-sectional studies intended to epidemiologically map a specific disease, mainly lymphatic filariasis, but also cutaneous leishmaniasis, leprosy, and NTDs, as well as sNTDs in general. Most of the studies were considered to have a high (5/13, 39%) or moderate (4/13, 31%) risk of bias. Fifteen apps were identified in the Google Play Store, of which three were also in the Apple App Store. Most of the apps (11/15, 73%) were targeted at health care professionals, with only four targeted at patients. The apps focused on scabies (3/15, 20%), lymphatic filariasis (3/15, 20%), cutaneous leishmaniasis (1/15, 7%), leprosy (1/15, 7%), yaws and Buruli ulcer (1/15, 7%), tropical diseases including more than one sNTDs (3/15, 20%), and NTDs including sNTDs (2/15, 13%). Only 1 (7%) app focused on the clinical management of sNTDs.Conclusions: All mobile health interventions that were identified face technological, legal, final user, and organizational issues. There was a remarkable heterogeneity among studies, and the majority had methodological limitations that leave considerable room for improvement. Based on existing evidence, eight recommendations have been made for future interventions.

Objectives: The main objective of this work was to explore and characterize the current landscape of mobile applications available to treat mood disorders such as depression, bipolar disorder, and dysthymia. Methods: We developed a tool that makes both the Apple App Store and the Google Play Store searchable using keywords and that facilitates the extraction of basic app information of the search results. All app results were filtered using various inclusion and exclusion criteria. We characterized all resultant applications according to their technical details. Furthermore, we searched for scientific publications on each app’s website and PubMed, to understand whether any of the apps were supported by any type of scientific evidence on their acceptability, validation, use, effectiveness, etc. Results: Thirty apps were identified that fit the inclusion and exclusion criteria. The literature search yielded 27 publications related to the apps. However, these did not exclusively concern mood disorders. 6 were randomized studies and the rest included a protocol, pilot-, feasibility, case-, or qualitative studies, among others. The majority of studies were conducted on relatively small scales and 9 of the 27 studies did not explicitly study the effects of mobile application use on mental wellbeing. Conclusion: While there exists a wealth of mobile applications aimed at the treatment of mental health disorders, including mood disorders, this study showed that only a handful of these are backed by robust scientific evidence. This result uncovers a need for further clinically oriented and systematic validation and testing of such apps.

Real‐world data (RWD)‐derived external controls can be used to contextualize efficacy findings for investigational therapies evaluated in uncontrolled trials. As the number of submissions to regulatory and health technology assessment (HTA) bodies using external controls rises, and in light of recent regulatory and HTA guidance on the appropriate use of RWD, there is a need to address the operational and methodological challenges impeding the quality of real‐world evidence (RWE) generation and the consistency in evaluation of RWE across agencies. This systematic review summarizes publicly available information on the use of external controls to contextualize outcomes from uncontrolled trials for all indications from January 1, 2015, through August 20, 2021, that were submitted to the European Medicines Agency, the US Food and Drug Administration, and/or select major HTA bodies (National Institute for Health and Care Excellence (NICE), Haute Autorité de Santé (HAS), Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG), and Gemeinsamer Bundesausschuss (G‐BA)). By systematically reviewing submissions to regulatory and HTA bodies in the context of recent guidance, this study provides quantitative and qualitative insights into how external control design and analytic choices may be viewed by different agencies in practice. The primary operational and methodological aspects identified for discussion include, but are not limited to, engagement of regulators and HTA bodies, approaches to handling missing data (a component of data quality), and selection of real‐world endpoints. Continued collaboration and guidance to address these and other aspects will inform and assist stakeholders attempting to generate evidence using external controls.

Among stakeholders and decision-makers in advanced breast cancer, the demand for insights from real-world data (RWD) is increasing. Although RWD can be used to support decisions throughout different stages of a breast cancer drug's life cycle, barriers exist to its use and acceptance. We propose a collaborative approach to generating and using RWD that is meaningful to multiple stakeholders, and encourage frameworks toward international guidelines to help standardize RWD methodologies to achieve more efficient use of RWD insights.

Objectives: We conducted a multi-stakeholder survey to determine key areas where a joint European health technology assessment (HTA) could provide ‘additional benefit’ compared to the status quo of many parallel independent national and subnational assessments. Methods: Leveraging three iterative Delphi cycles, a semiquantitative questionnaire was developed covering evidence challenges and heterogeneity of value drivers within HTAs across Europe with a focus on hematology/oncology. The questionnaire consisted of five sections: i) background information; ii) value drivers in HTA assessments today; iii) evolving evidence challenges; iv) heterogeneity of value drivers across Europe; v) impact of Europe’s Beating Cancer Plan (EBCP). The questionnaire was circulated across n = 189 stakeholder institutions comprising HTA and regulatory bodies, clinical oncology associations, patient representatives, and industry associations. Results: N = 30 responses were received (HTA bodies: 9; regulators: 10; patients’ and physicians’ associations: 3 each; industry: 5). Overall, 17 countries and EU level institutions were represented in the responses. Consistency across countries and stakeholder groups was high. Most relevant value drivers in HTAs today (scale 1, low to 5, high) were clinical trial design (mean 4.45), right endpoints (mean 4.40), and size of comparative effect (mean 4.33). Small patient numbers (mean 4.28) and innovative study designs (mean 4.1) were considered the most relevant evolving evidence challenges. Heterogeneity between regulatory and HTA evidence requirements and heterogeneity of the various national treatment standards and national HTA evidence requirements was high. All clinical and patient participants stated to have been with EBCP initiatives. Conclusions: For a European HTA to provide an ‘additional benefit’ over the multitude of existing national assessments key methodological and process challenges need to be addressed. These include approaches to address uncertainty in clinical development; comparator choice; consistency in approaching patient-relevant endpoints; and a transparent and consistent management of both HTA and regulatory procedures as well as their interface, including all involved stakeholder groups.