Chizoba Oparah

Clinical trials often enrol populations that differ from those who ultimately receive the interventions, raising concerns about external validity and health equity. Trial registries could provide an early opportunity to assess representativeness, but it is unclear whether registry data contain sufficient information to enable such assessments. This study evaluated the feasibility of using registry data to assess representativeness in Phase II and III pharmacological randomised controlled trials. A search of ClinicalTrials.gov (December 2024 to January 2025) identified trials with results posted after 1 January 2023 across cardiovascular disease (CVD) excluding stroke, diabetes mellitus, and selected mental health disorders. Of 1,328 records screened, 98 trials met inclusion criteria (51 Phase III, 47 Phase II). Reporting completeness was variable, particularly in Phase II studies. CVD and diabetes trials predominantly included middle-aged to older adults, while mental health trials recruited mainly individuals aged 36 to 50 years. Across CVD and mental health trials, participants were largely male. Reporting of BMI, contraception, and comorbidity criteria was inconsistent, though available data suggested these factors influenced sample composition. Fewer than 10% of trials reported equity-relevant characteristics beyond age and sex, and none addressed intersectionality. Assessing equity using registry data is feasible but constrained by incomplete and inconsistent reporting.

Background: Adaptive designs (ADs) are increasingly used, whereby outcome data accumulated during the trial may inform a trial’s course in accordance with pre-specified rules at interim analyses. The benefits however will only be realised if the approaches to conducting interim analyses are optimised. This study aims to identify existing literature highlighting best practice (and existing evidence gaps) with regard to conducting high-quality, timely interim analyses. Methods: Medline and Embase databases were searched for studies published between 2005 and July 2025 to identify best practice models and lessons learned for interim analyses in the delivery of adaptive trials. Specifically, we considered papers that discussed adaptive trial methodological approaches (Phases II–IV) in any clinical population and condition. A narrative synthesis focused on the following outcomes was conducted: design considerations, project/trial management, data management, statistical processes, trial committee processes, the implementation of interim decisions and patient and public involvement and engagement (PPIE). Results: We screened 6720 articles from databases and citation chaining and assessed 329 articles at full text. One hundred and one articles representing 92 unique studies were deemed eligible for inclusion. Key issues included the following: (a) Detailed planning of interim analyses set out in the protocol, and all patient information sheets for possible changes prepared in advance; (b) Effective communication and collaborative decision-making processes across stakeholders, effective staff training, and prompt site query resolutions; (c) Use of electronic data capture with automated data flow processes with integrated query processes to improve data quality; and (d) Use of secure databases with data transfer procedures to maintain data integrity. Conclusion: The currently available data suggest that although there is a considerable volume of evidence with regards to the conduct of adaptive trials and good trial management, the existing literature base offers limited specific guidance for interim analyses. There are significant gaps in the literature regarding best practice guidance related to PPIE and statistical considerations in adaptive trials. While there are examples of innovative methods speeding up the collation and analysis of data used for interim analyses, we advocate more robust research exploring the operational opportunities and challenges with regards to undertaking adaptive trials.

Over 500,000 people in England have a recorded diagnosis of dementia, of whom at least 40% have Alzheimer’s disease (AD) in isolation or in combination with other pathologies. Timely diagnosis ensures that people get the help and support they need. Mild cognitive impairment (MCI) is a term for subtle changes in memory, language and behaviour that do not impact on a person’s ability to function. People with MCI may develop AD or other dementias. This horizon scan, alongside an ultra-rapid evidence synthesis review, aimed to identify digital tests and devices which support the identification of MCI associated with early-stage AD. We searched clinical trial registries, news websites, patent databases, funding and regulatory agencies, and company websites, as well as three recently published systematic reviews. Eligible technologies were any digital tools to detect MCI or cognitive decline in adults without a current diagnosis of AD. 79 technologies were identified, classified as digitised versions of standard cognitive tests, newly developed digital cognitive tests, and digital devices that incorporate detection of MCI/cognitive decline. This work outlines digital technologies, at various development stages, that might complement emerging treatments in the care pathway by facilitating identification of AD at the earlier stages of clinical presentation.

Background: Adaptive designs are increasingly being used in clinical trials within diverse clinical areas. They can offer advantages over traditional non-adaptive approaches, including improved efficiency and patient benefit. The level of improvement observed in practice depends to a large degree on conducting interim analyses (at which adaptations can be made to the trial based on collected data) rapidly and to a high standard. Methods: The ROBust INterims for adaptive designs (ROBIN) project aimed to identify best practice for conducting high-quality and rapid interim analyses. This was done through evidence synthesis of published work, qualitative research with trial stakeholders working at public sector clinical trials units, engagement with patients and the public, and a meeting of trial stakeholders to discuss findings and agree recommendations. Results: This paper provides recommendations for teams that conduct adaptive trials about how to ensure interim analyses are done rapidly and to a high standard. We break down recommendations by stage of the trial. We also identify a lack of methodology on how best to involve patients in adaptive trials and related decision-making. A limitation of our recommendations is that the research was mostly focused on UK academic settings, although we believe much of the recommendations are relevant in other countries and to industry-sponsored trials. Conclusions: When following the recommendations outlined in this paper, the process of planning and executing interim analyses will be smoother; in turn, this will lead to more benefits from using adaptive designs.

Introduction: Children and young people comprise one quarter of the UK’s population. Neurological trauma (e.g., traumatic brain injury and spinal cord injury) is one of the most common causes of death and disability in this population group, yet health technology innovations for these patients lag behind those for adults.Methods: Using the horizon scanning methodologies developed by the Innovation Observatory, systematic searches were performed to identify registered clinical trials, published funding awards, and news articles that focused on innovative devices and digital and diagnostic health technologies developed for use in children and young people (from 28 days after birth up to and including 18 years of age) with actual or suspected neurological trauma. The search results were screened for relevance, and key information on the included technologies was extracted and summarized.Results: Twenty-nine technologies were identified, of which 10 were commercially available. The majority were developed in the UK or the USA. Overall, the development pipeline was evenly split amongst technologies considered to be a device (37%), digital (34%), or diagnostic (29%). Most technologies were intended for use across settings by healthcare professionals, either for initial onsite assessment, for in-hospital management, or for rehabilitation in hospital or in the community.Conclusions: Results from this horizon scan show that development of technologies for pediatric neurological trauma is currently limited, with only a small number of the technologies being developed covering an area of unmet need. To complement the horizon scan, we also sought stakeholder insights on medical technologies for this population group. The combined results and final conclusions will be shared in a future publication.

Background: There is an increasing focus on inequity in healthcare and health outcomes. Early awareness of potential sources of inequity in access to and outcomes from innovative health technologies can support system preparedness and allow implementation of mitigations. They may also be used to improve research inclusion. Objective: To explore methods used to integrate equality and equity into horizon scanning for healthcare innovations, focusing on acceptability, polypharmacy, and multiple long-term conditions (MLTC). Design: A scoping review followed Joanna Briggs Institute (JBI) guidelines to identify relevant methodologies for integrating equity into horizon scanning. Data sources: Searches were conducted in MEDLINE, Embase, ProQuest, and WHO Global Index Medicus up to May 24, 2024. Eligibility criteria: Studies were eligible if they presented methodologies for integrating equity and equality considerations into horizon scanning in health and care. Primary outcomes related to equity or equality, and secondary outcomes addressed acceptability, polypharmacy, and MLTC. Data extraction and synthesis: Data were extracted on study characteristics, equity frameworks, and the integration of equity-related factors, including socioeconomic status, gender, and geographic location. A narrative synthesis was used to present the findings. Results: Out of 951 records screened, three studies were included. The studies used varied horizon scanning methods, including scenario-building and foresight methodologies, and spanned multiple healthcare contexts such as precision oncology and complex paediatric care. Each study incorporated equity/equality by addressing the impact of emerging innovations on clinically vulnerable populations. Acceptability was found to be crucial for equitable implementation, particularly in precision oncology. However, managing complex health needs, especially in disadvantaged groups, is complicated by significant challenges such as polypharmacy and the presence of multiple long-term conditions. Conclusions: Limited evidence highlighted a lack of consistent approaches to integrating equity into horizon scanning. While methods such as stakeholder engagement and scenario analysis showed promise, further research is needed to refine frameworks that better detect early indicators of inequity in healthcare innovation. Article summary: Strengths and limitations of this study: Strengths: Followed a robust and transparent methodology using the Joanna Briggs Institute (JBI) guidelines for scoping reviews. Comprehensive search strategy developed in collaboration with an experienced information specialist, covering multiple databases without restrictions. Dual independent screening and data extraction enhanced the reliability and consistency of the review process. Limitations: Limited number of included studies and heterogeneity in methodologies and healthcare settings reduced the generalisability of findings. No critical appraisal of the quality of included studies, as the review focused on identifying methodologies rather than assessing study quality.