Pediatric Research Participants Research Articles

Reporting and Representation of Participant Race and Ethnicity in National Institutes of Health–Funded Pediatric Clinical Trials

Enrolling racially and ethnically diverse pediatric research participants is critical to ensuring equitable access to health advances and generalizability of research findings. To examine the reporting of race and ethnicity for National Institutes of Health (NIH)-funded pediatric clinical trials and to assess the representation of pediatric participants from different racial and ethnic groups compared with distributions in the US population. This cross-sectional study included NIH-funded pediatric (ages 0-17 years) trials with grant funding completed between January 1, 2017, and December 31, 2019, and trial results reported as of June 30, 2022. National Institutes of Health policies and guidance statements on the reporting of race and ethnicity of participants in NIH-funded clinical trials. The main outcome was reporting of participant race and ethnicity for NIH-funded pediatric clinical trials in publications and ClinicalTrials.gov. There were 363 NIH-funded pediatric trials included in the analysis. Reporting of race and ethnicity data was similar in publications and ClinicalTrials.gov, with 90.3% (167 of 185) of publications and 93.9% (77 of 82) of ClinicalTrial.gov reports providing data on race and/or ethnicity. Among the 160 publications reporting race, there were 43 different race classifications, with only 3 publications (1.9%) using the NIH-required categories. By contrast, in ClinicalTrials.gov, 61 reports (79.2%) provided participant race and ethnicity using the NIH-specified categories (P < .001). There was racially and ethnically diverse enrollment of pediatric participants, with overrepresentation of racial and ethnic minority groups compared with the US population. This cross-sectional study of NIH-funded pediatric clinical trials found high rates of reporting of participant race and ethnicity, with diverse representation of trial participants. These findings suggest that the NIH is meeting its directive of ensuring diverse participant enrollment in the research it supports.

Assessing attitudes towards pediatric research participation across diverse populations: Psychometric properties of a novel tool

BackgroundClinical trials play a crucial role in advancing medical knowledge and improving health outcomes. However, there is a recognized need for greater representation of marginalized groups to ensure that research findings can be generalized and effectively applied to all individuals. While the Pediatric Research Participation Questionnaire (PRPQ) was developed to assist pediatric clinical trials research by identifying benefits and barriers to research participation among children with chronic medical conditions, there is still limited insight into the structure of the PRPQ when administered in diverse samples, including the general pediatric population. Therefore, the current study examined the factor structure of the PRPQ in a general pediatric population to investigate whether rural-urban differences exist in the PRPQ factor structure. MethodsCaregivers (N = 600) of children under age 18 completed the PRPQ in a population-based survey in Mississippi. Sampling was stratified to ensure equal representation in rural (n = 300) and urban areas (n = 300). Exploratory and confirmatory factor analyses were conducted to determine the factor structure of the PRPQ. ResultsA five-factor structure was identified, compromising: social pressure, direct benefit, reasons for participation, mistrust in research/researchers, reasons against participation. While results were similar among urban participants, a three-factor structure emerged for rural participants. ConclusionsThis study contributes to the broader understanding of research participation among underrepresented groups. The findings suggest that clinical researchers should consider tailoring recruitment strategies to increase clinical trial participation among children in rural areas. Understanding factors that influence pediatric research participation, particularly among marginalized communities, is crucial for developing effective recruitment and retention strategies.

Comparing Attitudes About Genomic Privacy and Data Sharing in Adolescents and Parents of Children Enrolled in a Genomic Research Repository

Background Sharing of genomic data aims to make efficient use of limited resources, which may be particularly valuable in rare disease research. Adult research participants and parents of pediatric research participants have shown support for data sharing with protections, but little is known about adolescent attitudes on genomic privacy and data sharing. Methods In-depth interviews were conducted with 10 adolescents and 18 parents of children enrolled in a pediatric genomic research repository. Interview transcripts were analyzed for themes on attitudes toward genomic privacy, restricted-access data sharing, and open-access data sharing. Findings in adolescent and parent participants were compared and contrasted. Results No adolescents endorsed privacy concerns for restricted-access data sharing. Both adolescents and parents saw value in data sharing for reaching the goals of research and discussed trust in institutions and researchers to protect their data and use it as intended. Adolescents were more likely than parents to accept open-access data sharing, including after risks were discussed. Conclusions In this exploratory study, adolescents and parents enrolled in a genomic research repository shared many attitudes about genomic data sharing, but adolescents were less concerned about privacy and more agreeable toward open-access data sharing. Future research is needed to investigate this hypothesis in expanded populations and settings, and to clarify whether adolescent attitudes change with age and experiences.

93 Empowering the Next Generation of Clinical &amp; Translational Scientists

OBJECTIVES/GOALS: Biomedical research fields are facing the challenges of demand for skilled workers as well as challenges related to diversity in that workforce. It is important that the healthcare workforce reflect the population it serves. The Exposures Internship seeks to address this by building pathways for youth to pursue careers in research and medicine. METHODS/STUDY POPULATION: In 2021, the Yale Cultural Ambassadors expressed concern about the lack of free high quality, educational offerings for youth that summer. They asked YCCI to consider developing a summer program for students aged 15 and older that focused on spurring interest in careers in healthcare, medicine, and clinical and translational research. The result was a 4-week virtual learning experience for 34 interns who met daily via Zoom and participated in course work, lectures, journal clubs, group projects, and virtual lunches with internationally renowned clinical research and healthcare leaders. Sessions were designed to help interns gain knowledge of and exposure to current topics in clinical and translational science and to observe the various steps of proposing, designing, undertaking, and analyzing clinical trials. RESULTS/ANTICIPATED RESULTS: YCCI received over 900 inquiries from around the world with more than 200 completed applications for participation in the internship for the pilot year. Since then, YCCI leadership has worked with community partners to engage young scholars from 17 different states, Canada, Mexico and Puerto Rico. Of those, we estimate 75% are minority, ~50% female and 20% from rural areas with limited similar opportunities. During the four weeks of the program these highly motivated students worked on projects aimed at increasing participation in pediatric research through a revised Informed consent and adolescent assent process and a youth centered awareness campaign. Interns were so inspired that they requested the program be continued beyond the initial four weeks. As such, YCCI continued to offer sessions throughout the year. DISCUSSION/SIGNIFICANCE: In evaluation of the pilot program 95% of respondents strongly agreed that the program exposed them to new information about clinical and translational research. One intern shared, This program has unquestionably made me consider becoming a researcher in the future with the goal of becoming a principal investigator within my interest in medicine.

Re-Consenting Pediatric Research Participants as Legal Adulthood Approaches: Lessons from the SPARK Autism Study

To explore issues surrounding re-consenting youth in longitudinal studies as they reach legal adulthood interviews were conducted with 46 parents plus 13 autistic teens enrolled in the Simons Foundation Powering Autism Research for Knowledge (SPARK) study. Qualitative analysis focused on family sensitivities regarding guardianship decisions, transition concerns, and the re-consenting process. Questions regarding guardianship were difficult for parents unsure of a teen’s future status. Mothers were key facilitators of re-consenting for soon-to-be-independent teens. As legal adulthood approached, parents were willing to assist teens with re-consenting but needed support, asking for multiple contacts, transition resources, and explanatory materials from the research team. Most teens were not cognizant of SPARK but willing to continue participation once made aware.

Perceived Barriers to Clinical Trials Participation: A Survey of Pediatric Caregivers.

IntroductionPediatric clinical trials are difficult to conduct, leading to off-label use of medication in children based on results of trials with adults. As a unique population, children deserve to have appropriately tested therapies. The purpose of this study was to evaluate pediatric caregivers’ beliefs and perceived barriers to participation in clinical trials.MethodsThe study was completed within the Sunflower Pediatric Clinical Trials Research Extension (SPeCTRE), an affiliate of the IDeA States Pediatric Clinical Trials Network (ISPCTN). This was a cross-sectional survey, adapted from the Pediatric Research Participation Questionnaire. A convenience sample of pediatric caregivers was recruited in three areas of a highly rural Midwestern state between 2017 and 2018.ResultsA total of 159 caregivers completed surveys; the majority (72.3%) were previously familiar with clinical trials, but less than 20% had ever been invited to participate. Caregivers were willing to consider enrolling their child if a physician in whom they had high trust recommended the trials (H = 10.1, p = 0.04) and if there were perceived benefits, such as access to tests and medications not covered by insurance (correlation coefficient [CC] = 0.4, p < 0.01) and compensation for time and travel (CC = 0.3, p = 0.04).ConclusionsTrust in their physician highly influences likelihood of a caregiver consenting to have their child participate in a clinical trial. Therefore, to facilitate opportunities for children to participate in clinical trials, physicians need to be trained so they can offer trials locally. In addition, trials need to offer benefits, such as increased access to tests and medications as well as appropriate compensation.

Improve Eczema/Asthma Research Study Enrollment and Retention Using Engaging, Participant Co-designed Graphic-Style Stories

Stories are powerful tools to increase understanding of complex information, encourage engagement and activate learning and positive behavior change. We hypothesized that sharing of participant co-designed graphic-style stories with caregivers of pediatric research participants wound improve study enrollment and retention and be more inclusive of minority and lower health literacy participants.

Factors Influencing US Hospital and Medical School Participation in Pediatric COVID-19 Research.

Literature suggests that funding for pediatric clinical trials is inequitably awarded. Furthermore, although coronavirus disease 2019 (COVID-19) affected all hospitals, institutions with already limited resources were more severely impacted. We hypothesized that there would be difference in schools and hospitals that were able to participate in the initial round of pediatric COVID-19 clinical research. We searched online databases for preregistered studies using the keywords "COVID-19," "COVID," "SARS-CoV-2," "2019-nCov," "2019 novel coronavirus," and "severe acute respiratory syndrome coronavirus 2." Search results were limited to studies enrolling participants from birth to 17 years, studies started in 2020, and studies originating in the United states. We calculated the proportion of institutions with active COVID-19 pediatric clinical studies in 2020 and compared institutional characteristics between institutions with and without at least one qualifying COVID-19 study, using rank-sum tests, χ2 tests, or Fisher's exact tests, as appropriate. We identified 150 allopathic medical schools, 34 osteopathic medical schools, and 178 children's hospitals meeting inclusion criteria. Among included institutions, 25% of medical schools and 20% children's hospitals participated in 1 of the registered pediatric COVID-19 studies the year before the study period. Institutions that participated in pediatric COVID-19 studies had more publications, more National Institutes of Health funding, and more studies registered on Clinicaltrials.gov in 2019. Despite the pandemic affecting everyone, participation in early clinical research on the impact of COVID-19 in pediatric populations was concentrated in a few well-resourced institutions that were highly experienced in research.

Incentive delivery timing and follow-up survey completion in a prospective cohort study of injured children: a randomized experiment comparing prepaid and postpaid incentives

BackgroundRetaining participants over time is a frequent challenge in research studies evaluating long-term health outcomes. This study’s objective was to compare the impact of prepaid and postpaid incentives on response to a six-month follow-up survey.MethodsWe conducted an experiment to compare response between participants randomized to receive either prepaid or postpaid cash card incentives within a multisite study of children under 15 years in age who were hospitalized for a serious, severe, or critical injury. Participants were parents or guardians of enrolled children. The primary outcome was survey response. We also examined whether demographic characteristics were associated with response and if incentive timing influenced the relationship between demographic characteristics and response. We evaluated whether incentive timing was associated with the number of calls needed for contact.ResultsThe study enrolled 427 children, and parents of 420 children were included in this analysis. Follow-up survey response did not differ according to the assigned treatment arm, with the percentage of parents responding to the survey being 68.1% for the prepaid incentive and 66.7% with the postpaid incentive. Likelihood of response varied by demographics. Spanish-speaking parents and parents with lower income and lower educational attainment were less likely to respond. Parents of Hispanic/Latino children and children with Medicaid insurance were also less likely to respond. We found no relationship between the assigned incentive treatment and the demographics of respondents compared to non-respondents.ConclusionsPrepaid and postpaid incentives can obtain similar participation in longitudinal pediatric critical care outcomes research. Incentives alone do not ensure retention of all demographic subgroups. Strategies for improving representation of hard-to-reach populations are needed to address health disparities and ensure the generalizability of studies using these results.

Identifying Research Priorities among Patients and Families of Children with Rheumatic Diseases Living in the United States.

To improve the quality and participation in pediatric rheumatology research, patient-prioritized studies should be emphasized. We collaborated with United States-based pediatric rheumatology advocacy organizations to survey patients and caregivers of children with rheumatic diseases to identify what research topics were most important to them. We conducted Web-based surveys and focus groups (FG) of patients and caregivers of children with juvenile myositis (JM), juvenile arthritis (JA), and childhood-onset systemic lupus erythematosus (cSLE). Surveys were emailed to listservs and posted to social media sites of JM, JA, and cSLE patient advocacy organizations. An initial survey asked open-ended questions about patient/caregiver research preferences. Responses were further characterized through FG. A final ranking survey asked respondents to rank from a list of research themes the 7 most important to them. There were 365 JM respondents, 44 JA respondents, and 32 cSLE respondents to the final ranking survey. The top research priority for JM was finding new treatments, and for JA and cSLE, the priority was understanding genetic/environmental etiology. The 3 prioritized research themes common across all disease groups were medication side effects, disease flare, and disease etiology. Patient-centered research prioritization is recognized as valuable in conducting high-quality research, yet there is a paucity of data describing patient/family preferences, especially in pediatrics. We used multimodal methodologies to assess current patient/caregiver research priorities to help frame the agenda for the pediatric rheumatology research community. Patients and caregivers from all surveyed disease groups prioritized the study of medication side effects, disease flares, and disease etiology.

Online Pediatric Research: Addressing Consent, Assent, and Parental Permission.

This article provides practical guidance for researchers who wish to enroll and collect data from pediatric research participants through online and mobile platforms, with a focus on the involvement of both children and their parents in the decision to participate.

Anticipatory Waivers of Consent for Pediatric Biobanking.

As pediatric biobank research grows, additional guidance will be needed about whether researchers should always obtain consent from participants when they reach the legal age of majority. Biobanks struggle with a range of practical and ethical issues related to this question. We propose a framework for the use of anticipatory waivers of consent that is empirically rooted in research that shows that children and adolescents are often developmentally capable of meaningful deliberation about the risks and benefits of participation in research. Accordingly, bright-line legal concepts of majority or competency do not accurately capture the emerging capacity for autonomous decision-making of many pediatric research participants and unnecessarily complicate the issues about contacting participants at the age of majority to obtain consent for the continued or first use of their biospecimens that were obtained during childhood. We believe the proposed framework provides an ethically sound balance between the concern for potential exploitation of vulnerable populations, the impetus for the federal regulations governing research with children, and the need to conduct valuable research in the age of genomic medicine.

Increased Recruitment and Retention of Pediatric Research Participants after Implementation of Novel Recruitment Strategies in Inner City Asthma Consortium Site.

Increased Recruitment and Retention of Pediatric Research Participants after Implementation of Novel Recruitment Strategies in Inner City Asthma Consortium Site.

East African Perceptions of Barriers/Facilitators for Pediatric Clinical Research Participation and Development of the Inclusive Research Model

PurposeThis study sought to gain a greater understanding of perceptions related to barriers/facilitators for pediatric (ages 0–17) clinical research participation among East African immigrant community members. Design and MethodsCommunity leader interviews (n = 6) and focus groups with lay members (n = 16) from the three largest East African communities in the Seattle area (Eritrean, Ethiopian and Somali) were conducted. Discussions were semi-structured based on existing barrier/facilitator research and analyzed using directed content analysis to identify major themes. ResultsAnalysis revealed two novel barrier sub-themes: inadequate interpretation and translation of information even when services were available and a lack of adequate vocabulary in preferred languages. Participants also confirmed previously identified logistical barriers/facilitators (lack of knowledge regarding clinical research; time, cost, transportation, and child care challenges; providing incentives) and psychosocial barriers/facilitators (mistrust of research; cultural and/or religious differences; connecting benefits to the community; involving religious/community leaders or organizations and including community members on the research team; transparency in the research process; presenting results to the community) for clinical research participation among underrepresented groups. ConclusionPerceptions of barriers/facilitators for pediatric clinical research participation among East African immigrants identified two novel sub-themes and confirmed those previously described by other underrepresented communities. Practice ImplicationsTo facilitate more inclusive research participation, researchers, nurses and other health care providers might consider ensuring adequate time for discussion of the research study and process, engaging the community in the research process, employing lay reviews of translated materials and/or oral consent processes, and other strategies outlined in the Inclusive Research Model.

Acceptability of In Utero Hematopoietic Cell Transplantation for Sickle Cell Disease.

In utero hematopoietic cell transplantation (IUHCT) has curative potential for sickle cell disease (SCD) but carries a risk of fetal demise. We assessed the conditions under which parents of children with SCD and young adults with SCD would consider IUHCT in a future pregnancy, given a 5% fixed risk of fetal demise. Participants were randomized to consider a hypothetical cure rate (20%, 40%, or 70%). Subsequently, cure rate was either increased or decreased depending on the previous answer to reveal the lowest acceptable rate. Participants also completed the Pediatric Research Participation Questionnaire (PRPQ) and an omission scale. Overall, 74 of 79 (94%) participants were willing to consider IUHCT, and 52 (66%) participants accepted IUHCT at a cure rate of 40%, the estimated rate of therapeutic mixed chimerism. Participants with higher scores on the PRPQ perceived benefits scale were more likely to participate at lower cure rates (OR 1.08, p=0.007) and participants with a greater degree of omission bias were less likely to participate at lower cure rates (OR 0.83, p=0.04). Demographics and SCD severity were not significantly associated with acceptability of IUHCT. This study suggests that the majority of parents >and young adults would consider IUHCT under expected therapeutic conditions.

Fathers' Perceived Reasons for Their Underrepresentation in Child Health Research and Strategies to Increase Their Involvement.

Purpose Examine fathers' perceived reasons for their lack of inclusion in pediatric research and strategies to increase their participation. Description We conducted expert interviews with researchers and practitioners (N=13) working with fathers to inform the development of an online survey. The survey-which measured fathers' perceived reasons for their underrepresentation in pediatric research, recommended recruitment venues, and research personnel and study characteristics valued by fathers-was distributed online and in-person to fathers. Assessment Respondents included 303 fathers. Over 80% of respondents reported that fathers are underrepresented in pediatric research because they have not been asked to participate. Frequently recommended recruitment venues included community sports events (52%), social service programs (48%) and the internet (60%). Compared with white fathers, more non-white fathers recommended public transit (19% vs. 10%, p=.02), playgrounds (16% vs. 6%, p=.007) and barber shops (34% vs. 14%, p<.0001) and fewer recommended doctors' offices (31% vs. 43%, p=.046) as recruitment venues. Compared with residential fathers (100% resident with the target child), more non-residential fathers recommended social services programs (45% vs. 63%, p=.03) and public transit (10% vs. 27%, p=.001) and fewer recommended the workplace (17% vs. 40%, p=.002) as recruitment venues. Study brevity, perceived benefits for fathers and their families, and the credibility of the lead organization were valued by fathers. Conclusion Fathers' participation in pediatric research may increase if researchers explicitly invite father to participate, target father-focused recruitment venues, clearly communicate the benefits of the research for fathers and their families and adopt streamlined study procedures.

6. Promoting Family Participation in Pediatric Palliative Care Research

To design a strategy to recruit parents of children with life-threatening illnesses (LTI) and their healthy siblings for a Pediatric Palliative Care (PPC) study.

Topical Review: Unique Contributions of Magnetic Resonance Imaging to Pediatric Psychology Research.

This review aims to provide a brief introduction of the utility of magnetic resonance imaging (MRI) methods in pediatric psychology research, describe several exemplar studies that highlight the unique benefits of MRI techniques for pediatric psychology research, and detail methods for addressing several challenges inherent to pediatric MRI research. Literature review. Numerous useful applications of MRI research in pediatric psychology have been illustrated in published research. MRI methods yield information that cannot be obtained using neuropsychological or behavioral measures. Using MRI in pediatric psychology research may facilitate examination of neural structures and processes that underlie health behaviors. Challenges inherent to conducting MRI research with pediatric research participants (e.g., head movement) may be addressed using evidence-based strategies. We encourage pediatric psychology researchers to consider adopting MRI techniques to answer research questions relevant to pediatric health and illness.

Lay attitudes toward trust, uncertainty, and the return of pediatric research results in biobanking

ABSTRACTBackground: Trust plays a role in participants’ reactions to clinical residual biobanks. The purpose of this study is to assess whether trust in medical researchers and negative reactions to uncertainty influence the attitudes of parents of pediatric research participants toward the return of genomic research results from biobanking. Methods: Focus groups were conducted in collaboration with two community-based organizations. Participants completed a demographic questionnaire and the trust in research and intolerance of uncertainty scales. The focus groups were then conducted according to a thematic focus-group guide; discussions were transcribed and analyzed by two trained coders. Results: Emerging themes included the importance of returning research results to both children and parents, sharing results with few limitations based on a child's age, and the desire for results even when researchers had concerns about analytic validity. Negative reactions to uncertainty appear to have influenced only one theme: the paradoxical claim by participants with stronger reactions to uncertainty that they had a “right to information.” Conclusion: Participants prefer to receive most or all of the results produced by genomic research, and they want their children, within variable age restrictions, to have access to that information as well.

Practical Guidance on Informed Consent for Pediatric Participants in a Biorepository

In the decade since the Human Genome Project was completed, the knowledge and technologies that this project enabled have led to a remarkable evolution in the way biorepositories are designed and operate. Early biobanks were often designed to facilitate the study of a single condition, while biobanks established in the last decade have more frequently been created with a broader research mission in mind.1 Accompanying this transition have come other changes in biobank practices, including the generation and storage of genome-scale sequencing data, frequent sharing of biosamples and data, pooling of resources among sample collections, and increased interest in returning genetic research results to sample donors. As biorepository practices have become more complex, the task of developing appropriate informed consent practices has become more challenging. There are at least three reasons for this. First, the regulations that govern research with human subjects in the U.S., known collectively as the Common Rule, were written at a time when many of the recent innovations in biobank practices were not anticipated. Second, Institutional Review Boards (IRBs) are tasked with evaluating whether research studies meet both federal regulations and local standards for acceptable research, yet IRB members are often unfamiliar with the complexities of biobanks. Third, it can be quite challenging to explain these practices in informed consent documents in a way that is easy for potential research participants to read and understand. Because of these challenges, several groups have developed practical guidance on informed consent. For example, the website of the National Human Genome Research Institute (NHGRI), Genome.gov, provides model informed consent language developed for genomic research studies, including biobanks.2 The NHGRI website also hosts a white paper developed by our group, the Electronic Medical Records and Genomics (eMERGE) Network.3 This document provides model language for informed consent documents that investigators may adapt for their own biorepository projects. One limitation of these resources, however, is their focus on adult research participants. There are currently no similar resources that address the unique issues that arise for biorepositories that aim to collect samples from pediatric participants. This is an important gap in the literature, since the challenges associated with biobanking are magnified in the setting of pediatric research. The ability of children to engage in informed decision-making varies according to their developmental level, so parental permission is usually required for pediatric research participation. However, a parent’s permission for a child to participate in research is quite different from an adult’s consent for his or her own research participation. A parent’s decision must account for the best interests of the child, while at the same time balancing the future autonomy of the child and the needs of the family. To be sure, there is a robust literature on these unique issues that arise in pediatric research,4,5 including a number of helpful papers that address pediatric biorepositories specifically.6,7 However, it can be difficult for investigators and IRB members to distill these empirical and analytical resources into concrete practices related to the informed consent process. This document is designed to address that need. Writing on behalf of the Consent, Education, Regulation, and Consultation (CERC) workgroup of the eMERGE Network, we provide pediatric-focused guidance for investigators and IRB members working in the U.S regulatory context on pediatric informed consent practices for biorepositories.