Journal of Clinical and Translational Science最新文献

The quality by design (QbD) framework holds promise for improving success rates for completion of clinical studies, which often fail to complete on time. Initially used in manufacturing, the framework is now frequently applied to clinical trials to anticipate risks and avoid challenges that impact study completion or the credibility of results. The Southern California Clinical and Translational Science Institute created and implemented a program based on QbD to increase the success of research studies, including clinical trials and other study designs, being conducted by scholars in our Mentored Career Development Program and awardees in our Pilot Grant Programs. The program's three components are QbD Design Studios, project management, and team science support. The overall goal is to increase study quality and efficiency, thereby improving study completion success rates and, ultimately, driving innovations in healthcare and public health. The current article describes QbD program elements in detail, along with preliminary results from initial implementation, approaches for evaluating the program's implementation and impact on study success, and plans to disseminate the program widely.

Acquiring the skills to obtain extramural funding is a major challenge for early- and mid-career investigators. The Professional Development Core (PDC) of the NIH-funded Mountain West Clinical and Translational Research Infrastructure Network (MW CTR-IN) aims to support early-stage and mid-career investigators pursuing independent careers in clinical and translational science research. Since 2018, the PDC's Grant Writing Workshops (GWWs) have provided CTR investigators with didactic content and interactive feedback on their NIH grant applications, helping them reach this key career milestone. Four one-day GWWs were offered in person, and two half-day GWWs were offered virtually across two days during the COVID-19 pandemic. Evaluation data for each cohort revealed that participants' knowledge and confidence in the relevant sections of NIH R-series grants consistently improved following GWW attendance and resulted in notable enhancements in participants' feelings of positivity toward grant writing, regardless of delivery mode (virtual vs. in-person). Follow-up data showed that 12 GWW participants acquired external funding with a 21% success rate and $12,584,938 in total funding. This manuscript provides a roadmap for planning and implementing a successful virtual or in-person GWW that positively impacts the careers of early-stage and mid-career investigators.

Underrepresentation of people of color in clinical trials limits equity in research and treatment outcomes. This study evaluated the impact of a brief, community-focused educational intervention on perceptions and willingness to participate. Participants attended 30-minute sessions (9 virtual, 2 in-person). Identical pre- and post-surveys were analyzed using paired t-tests with Bonferroni correction. Eighty-three participants (90.5% Black, 88.4% female; mean age 46.3) showed significant improvements in comfort with participation, randomization, belief in protections, willingness to participate, and comfort in skin-related trials (all p < 0.05). Brief education may improve understanding and participation attitudes in underrepresented groups.

The Collaborating Network of Networks for Evaluating COVID-19 and Therapeutic Strategies (CONNECTS) was a novel network of networks created in response to the COVID-19 pandemic. This program brought together a large matrix of clinical research networks to swiftly design and/or implement concurrent clinical studies. Successful coordination of this large-scale collaboration required innovative solutions for timely and transparent centralized operations reporting. As the Administrative Coordinating Center (ACC) for CONNECTS, RTI International developed and maintained a web-based infrastructure that served as the central communication and reporting hub. This single-platform approach provided a robust collection of key topics to support daily operational oversight (e.g., enrollment and retention, site coverage, study milestones, financial tracking). Underlying data acquisition, harmonization, and portal reporting methods aimed to address nuances of the network of networks (e.g., disparate data sources, diverse user needs) while providing the necessary speed and agility to combat the COVID-19 pandemic. The resulting system was well received, readily adapted to changes, and successfully supported three years of early COVID-19 research. Although the CONNECTS central reporting methods arose from necessity during an urgent and dynamic public health emergency, they are a model for efficient and effective centralized operational reporting for any large-scale public health research effort.

Over 15 million children in the United States have been infected with COVID-19; nearly 2,000 have died. Approval of COVID-19 vaccines for children enabled reductions in disease severity and mortality. Disparities in vaccine adoption exist along racial, ethnic, and rural-urban lines, with lower uptake among medically underserved populations (e.g. Black, non-Hispanic White rural populations) compared to urban White populations. This study examined efforts to recruit and engage a diverse cohort as part of a vaccine communication randomized trial conducted across 15 states and compared demographic characteristics of the enrolled cohort to the broader US population. To enhance recruitment of diverse populations, eligible clinics had to serve a significant proportion of medically underserved individuals based on race, ethnicity, or geographic location. Coordinators used both traditional (in-person daily clinic schedule review) and retrospective (EHR and billing data review) recruitment methods adapted to enrich engagement with focus populations. Demographic characteristics were compared to national statistics obtained from the CDC's Household Pulse Survey. In total, 2999 parents/caregivers were screened; 725 were randomized (24.1%). Comparing enrolled subjects to the demographics of participating states, 17.3% vs 9.8% self-identified as Hispanic, 39.6% vs 13.0% as Black. Additionally, 34.3% self-described as living in a rural area. Of the 725 randomized, 512 (70.6%) completed the baseline survey. Of these 512, 422 (82.4%) also completed the final survey of the 24-week study. This analysis demonstrates the Institutional Development Award States Pediatric Clinical Trials Network can successfully recruit and engage populations from diverse and underrepresented populations in research.

Background: Acute rhinosinusitis is one of the most common conditions seen in primary care. One in seven adults are diagnosed with ARS annually, resulting in one in five of all antibiotic prescriptions. Yet there has been limited research comparing the effectiveness of widely used treatments such as antibiotics and nasal steroids. Conducting such a trial in the context of decades of established practice poses unique challenges.

Methods: A feasibility phase was conducted with continuing feedback to provide refinement and guidance regarding the design of a large-scale, pragmatic randomized controlled trial. The pilot trial assessed the ability to enroll, retain, and evaluate adherence to the intervention and assessment protocols.

Results: The feasibility phase allowed us to seek input from patients and experts. This resulted in changes pre and post pilot that will impact the full study. A priori enrollment targets for the pilot were achieved, and with high adherence rates. In total, 373 patients were pre-screened and 140 patients were enrolled participants. Adherence to data collection via the daily diary was 93% throughout the study, with 95% completing their diary on the day of the primary outcome, 3 post-randomization.

Conclusion: Expert panels and a patient advisory committee recommended critical changes to our study design. Stakeholder engagement is a key component of this funding source and was widely used throughout the 18-months. An achieved primary goal of the feasibility phase was to evaluate recruitment and study methods prior to implementing a large clinical trial that requires significant resources.

Collaboration across the Clinical and Translational Science Award (CTSA) consortium is essential for advancing translational science, yet institutional silos often hinder data-sharing and benchmarking efforts. This study examines the viability of a voluntary, multi-hub analysis of the CTSA education common metric on trainee and scholar engagement across five New York City-based sites or "hubs." Using a structured framework for collaboration and field-tested operational guidelines, a team of evaluators dubbed "The Gotham Group" pooled de-identified common education data to assess post-training research engagement and demographic representation. Their primary objective was to establish a sustainable model for independent data-sharing without national mandates or technical support. A secondary goal was to reassess the metric's usefulness as an impact benchmark. Results showed that NYC education engagement percentages remained stable despite institutional differences, suggesting the metric's viability for regional comparison. More importantly, the collaboration itself proved as valuable as its outcomes, fostering professional relationships, facilitating knowledge exchange, and strengthening evaluation capacity within and across the hubs. This study highlights the potential of voluntary data-sharing partnerships to overcome data silos and to create valuable networks driving continuous improvement in translational science.

Introduction: To successfully recruit and retain faculty members from underrepresented backgrounds (URBs), we need to understand the factors that attract them to research careers in the first place. However, scholarship in this area has focused largely on students who are contemplating research careers rather than faculty members who are currently in such careers.

Methods: This study explores the career motivations of early-career health researchers (faculty members and postdoctoral fellows) from URBs. It was conducted as part of a cluster randomized trial across 25 academic institutions investigating a support intervention. We conducted 1-hour semi-structured qualitative interviews with scholars from URBs in both the intervention and control arms of the trial. To our knowledge, this is the largest qualitative study of early-career faculty members from URBs to date.

Results: Seventy-eight individuals were interviewed. Our analysis revealed six key themes pertinent to participants' motivations to pursue research careers: (1) love of science; (2) making a larger impact; (3) happenstance and economic considerations; (4) family, community, and a path out of poverty; (5) the role of mentors and role models; and (6) support programs for scholars from URBs.

Conclusions: Our results align with prior studies while offering new insights into the motivations of URB faculty members in research careers. These insights can and should inform the design of programs to both recruit and retain URM faculty members in research careers.

Translational science methods often fall short due to the complexity of the healthcare delivery environment. We developed a methodology that involves multiple interest holders working within a pre-competitive consortium to develop solutions to translational barriers. The methodology supports innovative collaboration in a stepwise fashion: elucidating challenges, designing solutions, enabling implementation, monitoring, learning, disseminating, and catalyzing. Cases that benefit most from a structured collaborative methodology are those where diverse needs require elucidation and alignment. Application of the methodology to develop regulatory, clinical, and business innovations has shown the importance of an innovation facilitator and the capacity-building potential of collective skill enhancement.

Inclusion of uninsured and underinsured (UUI) individuals in clinical research (CR) is necessary to ensure data quality, diversity, generalizability and fairness. Yet, in the USA, UUI persons tend to be excluded from CR. We conducted an ethical analysis of: the regulatory and ethics literature related to protections of, and duty to care for, research participants from vulnerable groups; the nature and scope of the ancillary health care obligations of researchers, and the applicable laws, regulations and practices concerning the care for UUI participants. We consider six examples illustrating the challenges of including UUI persons in CR. We note that addressing fully the challenges of UUI participation in CR requires comprehensive legal and health care reforms. We maintain that even in the absence of such reforms, researchers, study sponsors and Institutional Review Boards can and ought to adopt an inclusive approach to the recruitment of UUI individuals to improve data quality, diversity, generalizability and social justice. We propose such a systematic, proactive and ethically sound approach. It considers the medical and ancillary care needs of UUI participants, addresses them in the study protocol and budget, and includes referral to community health resources, follow-up support, and noting assistance in the research records.