Pilot programs are integral to catalyzing and accelerating research at Clinical and Translational Science Award (CTSA) hubs. However, little has been published about the structure and operationalization of pilot programs or how they impact the translational research enterprise at CTSAs. The North Carolina Translational and Clinical Science Institute (NC TraCS), the CTSA hub at the University of North Carolina at Chapel Hill (UNC-CH) conducted an evaluation case study to describe the pilot program structure, assess process outcomes, and provide a framework for other institutions to utilize for the evaluation of their respective pilot programs.

We describe the operationalization of our pilot program, the evaluation framework utilized to evaluate the program, and how we analyzed available data to understand how our pilot funding opportunities were utilized by investigators. We calculated application volumes and funding rates by investigator position title and pilot application type. We also reviewed feedback provided by pilot Principal Investigators (PIs) to understand how many pilot projects were completed, NC TraCS service utilization, and barriers to research. Limited data on publications and subsequent funding was also reviewed.

Between 2009 and 2019 the NC TraCS Pilot Program received 2343 applications and funded 933 projects, ranging from $2000 to $100,000 in amount, with an overall funding rate of 39.8%. Utilization of NC TraCS services had positive impacts on both resubmission funding and project completion rates.

This process evaluation indicates that the program is being operationalized in a way that successfully fulfills the program mission while meeting the needs of a diverse group of researchers.

In 2015, the University of Pittsburgh partnered with several Minority Serving Institutions to develop the Leading Emerging and Diverse Scientists to Success (LEADS) Program. LEADS was designed to provide skills development, mentoring, and networking support to early career underrepresented faculty.

LEADS included three components: skills training (e.g., grant and manuscript writing and team science), mentoring, and networking opportunities. Scholars completed a pre- and post-test survey and an annual alumni survey that included measures on burnout, motivation, leadership, professionalism, mentoring, job and career satisfaction, networking, and an assessment of their research self-efficacy.

Scholars demonstrated a significant increase in their research self-efficacy having completed all the modules (t = 6.12; P < 0.001). Collectively, LEADS scholars submitted 73 grants and secured 46 grants for a 63% success rate. Most scholars either agreed or strongly agreed that their mentor was effective in helping to develop their research skills (65%) and provided effective counseling (56%). Scholars did experience increased burnout with 50% feeling burned out at the exit survey (t = 1.42; P = 0.16) and 58% reporting feelings of burnout at the most recent survey in 2020 (t = 3.96; P < 0.001).

Our findings support the claim that participation in LEADS enhanced critical research skills, provided networking and mentoring opportunities, and contributed to research productivity for scientists from underrepresented backgrounds.

The COVID-19 pandemic’s need for life-saving treatments and a "warp speed" vaccine challenged the National Institutes of Health’s Clinical and Translational Science Award (CTSA) recipients to improve their methods and processes in conducting clinical research. While CTSA recipient, New Jersey Alliance for Clinical and Translational Science (NJ ACTS), responded to this call to action with significant clinical research milestones, a comprehensive understanding of regulatory metrics during the COVID-19 pandemic is uncertain. The objective of this research is to identify, compare, and contrast metrics that illustrate the effectiveness of NJ ACTS’s research mobilization efforts during COVID-19.

Data were collected from the Institutional Review Board (IRB), the Clinical Research Units (CRUs), and the Office of Research and Sponsored Programs (ORSP). IRB data detailed the volume and types of protocols approved and turnaround time (TAT) for approval in 2020 vs. 2019. CRU data examined study metrics of adult and pediatric clinical trials across 2018-2020. ORSP data documented awards received in 2019 and 2020

Analysis revealed a 95% increase in IRB-approved studies in 2020, with a significant decrease in TAT for COVID-19 studies. All CRUs observed a median 5.2-fold increase in the enrollment of adult and pediatric participants for COVID-19-related research. Study income was 106% and 196% greater than 2019 and 2018, respectively, with more than half funded through federal sponsors and 89% for COVID-19 trials. ORSP data revealed that 9% of awards and 26% of 2020 funding were COVID-19 studies.

This study demonstrates that NJACTS effectively responded to challenges posed by the pandemic

Research participation during undergraduate years has a powerful influence on career selection and attitudes toward scientific research. Most undergraduate research programs in academic health centers are oriented toward basic research or address a particular disease focus or research discipline. Undergraduate research programs that expose students to clinical and translational research may alter student perceptions about research and influence career selection.

We developed an undergraduate summer research curriculum, anchored upon a clinical and translational research study developed to address a common unmet needs in neonatal nurseries (e.g., assessment of neonatal opioid withdrawal syndrome). Program topics reflected the cross-disciplinary expertise that contributed to the development of this “bedside to bench” study, including opioid addiction, vulnerable populations, research ethics, statistics, data collection and management, assay development, analytical laboratory analysis, and pharmacokinetics. The curriculum was delivered through three offerings over 12 months, using Zoom video-conferencing due to restrictions imposed by the COVID-19 pandemic.

Nine students participated in the program. Two-thirds reported the course enhanced their understanding of clinical and translational research. Over three-quarters reported the curriculum topics were very good or excellent. In open-ended questions, students reported that the cross-disciplinary nature of the curriculum was the strongest aspect of the program.

The curriculum could be readily adapted by other Clinical and Translational Science Award programs seeking to provide clinical and translational research-oriented programs to undergraduate students. Application of cross-disciplinary research approaches to a specific clinical and translational research question provides students with relevant examples of translational research and translational science.

Increasing interest in real-world evidence has fueled the development of study designs incorporating real-world data (RWD). Using the Causal Roadmap, we specify three designs to evaluate the difference in risk of major adverse cardiovascular events (MACE) with oral semaglutide versus standard-of-care: (1) the actual sequence of non-inferiority and superiority randomized controlled trials (RCTs), (2) a single RCT, and (3) a hybrid randomized-external data study.

The hybrid design considers integration of the PIONEER 6 RCT with RWD controls using the experiment-selector cross-validated targeted maximum likelihood estimator. We evaluate 95% confidence interval coverage, power, and average patient time during which participants would be precluded from receiving a glucagon-like peptide-1 receptor agonist (GLP1-RA) for each design using simulations. Finally, we estimate the effect of oral semaglutide on MACE for the hybrid PIONEER 6-RWD analysis.

In simulations, Designs 1 and 2 performed similarly. The tradeoff between decreased coverage and patient time without the possibility of a GLP1-RA for Designs 1 and 3 depended on the simulated bias. In real data analysis using Design 3, external controls were integrated in 84% of cross-validation folds, resulting in an estimated risk difference of –1.53%-points (95% CI –2.75%-points to –0.30%-points).

The Causal Roadmap helps investigators to minimize potential bias in studies using RWD and to quantify tradeoffs between study designs. The simulation results help to interpret the level of evidence provided by the real data analysis in support of the superiority of oral semaglutide versus standard-of-care for cardiovascular risk reduction.

The objective of this study was to systematically assess the literature regarding postnatal healthcare utilization and barriers/facilitators of healthcare in neonatal abstinence syndrome (NAS) children.

A systematic search was performed in PubMed, Cochrane Database of Systematic Reviews, PsychINFO, Cumulative Index to Nursing and Allied Health Literature (CINAHL), and Web of Science to identify peer-reviewed research. Eligible studies were peer-reviewed articles reporting on broad aspects of primary and specialty healthcare utilization and access in NAS children. Three investigators independently reviewed all articles and extracted data. Study bias was assessed using the Newcastle–Ottawa Assessment Scale and the National Institute of Health Study Quality Assessment Tool.

This review identified 14 articles that met criteria. NAS children have poorer outpatient appointment adherence and have a higher rate of being lost to follow-up. These children have overall poorer health indicated by a significantly higher risk of ER visits, hospital readmission, and early childhood mortality compared with non-NAS infants. Intensive multidisciplinary support provided through outpatient weaning programs facilitates healthcare utilization and could serve as a model that could be applied to other healthcare fields to improve the health among this population.

This review investigated the difficulties in accessing outpatient care as well as the utilization of such care for NAS infants. NAS infants tend to have decreased access to and utilization of outpatient healthcare following hospital birth discharge. Outpatient weaning programs have proven to be effective; however, these programs require intensive resources and care coordination that has yet to be implemented into other healthcare areas for NAS children.

We evaluate a CTSA program hub by applying bibliometrics, social network analysis (SNA), and altmetrics and examine the changes in research productivity, citation impact, research collaboration, and CTSA-supported research topics since our pilot study in 2017.

The sampled data included North Carolina Translational and Clinical Science Institute (NC TraCS)-supported publications produced between September 2008 and March 2021. We applied measures and metrics from bibliometrics, SNA, and altmetrics to the dataset. In addition, we analyzed research topics and correlations between different metrics.

1154 NC TraCS-supported publications generated over 53,560 citation counts by April 2021. The average cites per year and the relative citation ratio (RCR) mean of these publications improved from 33 and 2.26 in 2017 to 48 and 2.58 in 2021. The number of involved UNC units in the most published authors’ collaboration network increased from 7 (2017) to 10 (2021). NC TraCS-supported co-authorship involved 61 NC organizations. PlumX metrics identified articles with the highest altmetrics scores. About 96% NC TraCS-supported publications have above the average SciVal Topic Prominence Percentile; the average approximate potential to translate of the included publication was 54.2%; and 177 publications addressed health disparity issues. Bibliometric measures (e.g., citation counts, RCR) and PlumX metrics (i.e., Citations, Captures, and Social-Media) are positively correlated (p < .05).

Bibliometrics, SNA, and altmetrics offer distinctive but related perspectives to examine CTSA research performance and longitudinal growth, especially at the individual program hub level. These perspectives can help CTSAs build program foci.

Failure to achieve accrual goals is a common problem in health-related research. Electronic health records represent a promising resource, offering the ability to identify a precisely defined cohort of patients who meet inclusion/exclusion criteria. However, challenges associated with the recruitment process remain and institutional policies vary.

We interviewed researchers, institutional review board chairs, and primary care physicians in North Carolina and Tennessee. Questions focused on strategies for initiating contact with potentially eligible patients, as well as recruitment letters asking recipients to opt in versus opt out of further communication.

When we asked about initiating contact with prospective participants, qualitative themes included trust, credibility, and established relationships; research efficiency and validity; privacy and autonomy; the intersection between research and clinical care; and disruption to physician–researcher and physician–patient relationships. All interviewees said it was acceptable for researchers to contact patients through their physicians; most said it was acceptable for researchers to contact patients directly. Over half chose contact through physicians as more appropriate. Regarding recruitment letters, qualitative themes included the quality of the participant pool; privacy and control; research efficiency and representativeness; and patients’ opportunity to make their own decisions. All interviewees said asking recipients to opt in to further communication was acceptable; nearly all said opt out was acceptable. Similar proportions chose each approach as more appropriate.

Comparing these results to our previous research with patients reveals potential differences in stakeholder perspectives. We offer suggestions for developing balanced approaches that respect patients and facilitate the advancement of science.

Community engagement is important for advancing Clinical and Translational Science (CTS), but face-to-face engagement has limited reach and scale. We examined the feasibility of a novel virtual Facebook community platform for public engagement on health research statewide in Minnesota.

The Facebook platform, MN Research Link, was evaluated from June 19, 2019 to June 30, 2020. Facebook advertisements and boosts were used to recruit followers. Content, based on prior formative work, included health research information and interactive postings (e.g., live interviews with researchers). Standard metrics obtained from Facebook analytics included participation (followers), content reach (views), and engagement (likes, shares, comments, clicks).

During the 12-month period, we acquired 1406 followers (31% rural residents), with a retention of followers of 99.7%. Mean number of views per month was 9379.83 (Mdn = 2791, range 724–41,510). Engagement metrics indicated a mean of 535.2 likes, shares, comments, and/or clicks per month (Mdn = 296.5, range 55–1535). The page continued to acquire new followers, but a slight decrease in engagement was observed in the final months after state COVID-19 mitigation strategies were implemented.

As the complexity of CTS continues to grow, along with social distancing measures resulting from the COVID-19 pandemic, the availability of virtual digital platforms to reach and engage community stakeholders in conversations about health and research has increasing importance. Preliminary findings from this program evaluation indicate that a Facebook community platform is feasible to engage Minnesota residents in conversations around health and research topics. Future work will evaluate its potential for reach, scale, and sustainability.

We describe the effectiveness of community outreach and engagement in supporting recruitment for the US National Children’s Vanguard Study between 2009 and 2012.

Thirty-seven study locations used 1 of 4 strategies to recruit 18–49-year-old pregnant or trying to conceive women: (1) Initial Vanguard Study used household-based recruitment; (2) Direct Outreach emphasized self-referral; (3) Enhanced Household-Based Recruitment enhanced Initial Vanguard Study strategies; and (4) Provider-Based Recruitment recruited through healthcare providers. Outreach and engagement included advance letters, interactions with healthcare providers, participation in community events, contacts with community organizations, and media outreach.

After 1–2 years, 41%–74% of 9844 study-eligible women had heard about the National Children’s Vanguard Study when first approached. Women who heard were 1.5–3 times more likely to consent. Hearing via word-of-mouth or the media most frequently predicted consent. The more sources women heard from the higher the odds of consent.

We conclude that tailored outreach and engagement facilitate recruitment in cohort studies.