Background Public involvement (PI) in research is increasingly requested, yet systematic evaluation is limited. This study evaluates whether a panel advising an Alzheimer's Disease and Related Dementia's (ADRD) Cohort Study fulfills PI quality criteria and leads to perceived Cohort Study improvement and describe lessons learned. Methods A panel of 49 members of the public advised the Cohort Study through questionnaires and meetings. Panelists and Cohort Study researchers completed questionnaires based on PI quality criteria as formulated in the Patient Engagement Quality Guidance Framework, specifically developed to capture insights on the quality of PI. Findings were supplemented by reflections of the panel organizers and three panelists. Additionally, we outlined the panel's aim and design to provide a practical example of PI in ADRD research. Results Of 49 panelists, 43 consented for use of their data for evaluation purposes (72% female (31/43), mean age 62 years, 82% (35/43) in good health). Response rates to the evaluation questionnaire were 37% for panelists (16/43) and 67% for researchers (4/6). Most panelists reported that the panel's purpose (95%; 15/16) and communication were (very) clear (94%; 15/16), certainly adequate resources to provide input (75%; 12/16), and sufficient transparency (87%; 14/16). All felt respected and found it easy to participate in online panel questionnaires. However, 56% (9/16) found attending in-person meetings challenging due to time or travel. Researchers reported positive impact on Cohort Study quality, e.g., improved participant information letter, yet also highlighted the required time and effort to involve a panel and suggested to improve management of panelists' expectations. Among the lessons learned are the value of offering multiple ways to participate, and the need for more communication between all parties involved. Conclusions Engaging a PI panel was perceived to improve an ADRD Cohort Study and fulfilled most quality criteria from the Patient Engagement Quality Guidance Framework. However, results must be interpreted with caution due to potential consent bias.
Paediatric diarrhoea is associated with 500 000 childhood deaths each year in low- and middle-income countries (LMICs). Enteropathogenic Escherichia coli, Shigella spp., Salmonella spp. and Campylobacter spp. are among the most common causative bacterial pathogens, however, their burden and rates of antimicrobial resistance (AMR) are poorly quantified. Current treatment typically involves oral and intravenous rehydration fluids, nutritional support for malnourished children, antibiotics for severe bacterial infections, antiemetic and anti-diarrheal medications, and analgesics. The World Health Organization recommends first-line empirical treatment of ciprofloxacin, with alternatives of third-generation cephalosporins or azithromycin. At present, no new antibacterials are in development specifically to treat paediatric diarrhoea. Target product profiles (TPPs) are used typically in drug development and describe the desired characteristics of a new therapeutic. In this paper, we aim to define a TPP for the development of novel antimicrobials for the treatment of severe paediatric diarrhoea in LMICs. Oral and intravenous formulations are preferable. Safety, tolerability, resolution of clinical signs and symptoms must be equivalent or better than standard of care (SoC) therapy. Interactions with commonly used therapies such as zinc, milk and traditional cures should be minimal. In addition, formulations should be stable at elevated temperatures and for extended periods of time to facilitate access and distribution across LMICs.
In May 2022, the European Commission issued the Proposal for a Regulation on the European Health Data Space (EHDS), with the aims of granting citizens increased access to and control of their (electronic) health data across the EU, and facilitating health data re-use for research, innovation, and policymaking. As the first in a series of European domain-specific "data spaces", the EHDS is a high-stakes development that will transform health data governance in the EU region. As an international consortium of experts from health policy, law, ethics and the social sciences, we are concerned that the EHDS Proposal will detract from, rather than lead to the achievement of, its stated aims. We are in no doubt on the benefits of using health data for secondary purposes, and we appreciate attempts to facilitate such uses across borders in a carefully curated manner. Based on the current draft Regulation, however, the EHDS risks undermining rather than enhancing patient control over data; hindering rather than facilitating the work of health professionals and researchers; and eroding rather than increasing the public value generated through health data sharing. Therefore, significant adjustments are needed if the EHDS is to realize its promised benefits. Besides analyzing the implications for key groups and European societies at large who will be affected by the implementation of the EHDS, this contribution advances targeted policy recommendations to address the identified shortcomings of the EHDS Proposal.
