Data-driven strategies for increasing patient diversity in Bristol Myers Squibb-sponsored US oncology clinical trials.

BACKGROUND/AIMS: Determining whether clinical trial findings are applicable to diverse, real-world patient populations can be challenging when the full demographic characteristics of enrolled patients are not consistently reported. Here, we present the results of a descriptive analysis of racial and ethnic demographic information for patients in Bristol Myers Squibb (BMS)-sponsored oncology trials in the United States (US) and describe factors associated with increased patient diversity. METHODS: BMS-sponsored oncology trials conducted at US sites with study enrollment dates between 1 January 2013 and 31 May 2021 were analyzed. Patient race/ethnicity information was self-reported in case report forms. As principal investigators (PIs) did not report their own race/ethnicity, a deep-learning algorithm (ethnicolr) was used to predict PI race/ethnicity. Trial sites were linked to counties to understand the role of county-level demographics. The impact of working with patient advocacy and community-based organizations to increase diversity in prostate cancer trials was analyzed. The magnitude of associations between patient diversity and PI diversity, US county demographics, and recruitment interventions in prostate cancer trials were assessed by bootstrapping. RESULTS: A total of 108 trials for solid tumors were analyzed, including 15,763 patients with race/ethnicity information and 834 unique PIs. Of the 15,763 patients, 13,968 (89%) self-reported as White, 956 (6%) Black, 466 (3%) Asian, and 373 (2%) Hispanic. Among 834 PIs, 607 (73%) were predicted to be White, 17 (2%) Black, 161 (19%) Asian, and 49 (6%) Hispanic. A positive concordance was observed between Hispanic patients and PIs (mean = 5.9%; 95% confidence interval (CI) = 2.4, 8.9), a less positive concordance between Black patients and PIs (mean = 1.0%; 95% CI = -2.7, 5.5), and no concordance between Asian patients and PIs. Geographic analyses showed that more non-White patients enrolled in study sites in counties with higher proportions of non-White residents (e.g. a county population that was 5%-30% Black had 7%-14% more Black patients enrolled in study sites). Following purposeful recruitment efforts in prostate cancer trials, 11% (95% CI = 7.7, 15.3) more Black men enrolled in prostate cancer trials. CONCLUSION: Most patients in these clinical trials were White. PI diversity, geographic diversity, and recruitment efforts were related to greater patient diversity. This report constitutes an essential step in benchmarking patient diversity in BMS US oncology trials and enables BMS to understand which initiatives may increase patient diversity. While complete reporting of patient characteristics such as race/ethnicity is critical, identifying diversity improvement tactics with the highest impact is essential. Strategies with the greatest concordance to clinical trial patient diversity should be implemented to make meaningful improvements to the diversity of clinical trial populations.

Development of a Brief Screening Tool for Early Literacy Skills in Preschool Children.

BACKGROUND: Preschool children develop early literacy skills (ELS) needed for reading acquisition. Screening for delayed ELS could trigger interventions to prevent reading problems. OBJECTIVE: To develop a brief screening test for ELS delays, the Early Literacy Skills Assessment Tool (ELSAT). METHODS: This study included 4-year-old, typically developing, English language-predominant children attending preschool. The ELSAT comprised 63 items relating to 3 main ELS domains and was piloted with 21 children. After we excluded items that were nondiscriminatory, 57 items remained and were administered to 96 children. Items were compared with reference measures of ELS (Get Ready to Read-Revised), and language (Peabody Picture Vocabulary Test-4 and Phonological Awareness from the Comprehensive Test of Phonological Processing-2). Within-domain reliability was calculated for each of the 3 ELS domains and item correlations between all ELSAT items and the reference measures were calculated. RESULTS: A final set of 10 items was retained that represented all 3 ELS domains and that maximized correlations with reference measures. Cronbach alpha for the refined 10-item ELSAT was 0.868; correlations between individual items and a composite of the reference measures ranged from 0.409 to 0.617 (all Ps < .01). In a receiver operating characteristic curve analysis, a cut-off score of ≤5 predicted a below-average score for any of the reference measures with sensitivity of 90%, specificity of 71.4%, and area under the curve of 0.872. CONCLUSIONS: The 10-item ELSAT shows strong psychometric properties and with further validation may prove valuable in screening preschool children for ELS delays.

Are negative/unrealistic parent descriptors of infant attributes associated with physical abuse?

Parents' perceptions of child behavior influence their responses to the child and may be important predictors of physical abuse. We examined whether infants 12 months of age or younger who were described with negative or developmentally unrealistic words were more likely than other infants to have been physically abused. As part of a prospective observational multicenter study investigating bruising and familial psychosocial characteristics, parents were asked to (1) describe their child's personality, and (2) list three words to describe their child. Four independent raters coded parent responses using a qualitative content analysis, identifying descriptors of infants and classifying each as positive, neutral, or negative/unrealistic. A medical expert panel, blinded to the psychosocial data, separately categorized each case as abuse or accident. We then analyzed the potential association between negative/unrealistic descriptors and abusive injury. Of 185 children enrolled, 147 cases (79%) were categorized as accident and 38 (21%) as abuse. Parents used at least one negative/unrealistic descriptor in 35/185 cases (19%), while the remaining 150 cases (81%) included only positive or neutral descriptors. Of the infants described with negative/unrealistic words, 60% were abused, compared to 11% of those described with positive or neutral words (p < .0001; age group-adjusted OR = 9.95; 95% confidence interval [3.98, 24.90]). Though limited by sample-size, this pilot study informs future work to create a screening tool utilizing negative/unrealistic descriptors in combination with other predictive factors to identify infants at high risk for physical child abuse.

Childhood Poverty and Its Effect on Health and Well-being: Enhancing Training for Learners Across the Medical Education Continuum.

OBJECTIVE: Childhood poverty is unacceptably common in the US and threatens the health, development, and lifelong well-being of millions of children. Health care providers should be prepared through medical curricula to directly address the health harms of poverty. In this article, authors from The Child Poverty Education Subcommittee (CPES) of the Academic Pediatric Association Task Force on Child Poverty describe the development of the first such child poverty curriculum for teachers and learners across the medical education continuum. METHODS: Educators, physicians, trainees, and public health professionals from 25 institutions across the United States and Canada were convened over a 2-year period and addressed 3 goals: 1) define the core competencies of child poverty education, 2) delineate the scope and aims of a child poverty curriculum, and 3) create a child poverty curriculum ready to implement in undergraduate and graduate medical education settings. RESULTS: The CPES identified 4 core domains for the curriculum including the epidemiology of child poverty, poverty-related social determinants of health, pathophysiology of the health effects of poverty, and leadership and action to reduce and prevent poverty's health effects. Workgroups, focused on each domain, developed learning goals and objectives, built interactive learning modules to meet them, and created evaluation and faculty development materials to supplement the core curriculum. An editorial team with representatives from each workgroup coordinated activities and are preparing the final curriculum for national implementation. CONCLUSIONS: This comprehensive, standardized child poverty curriculum developed by an international group of educators in pediatrics and experts in the health effects of poverty should prepare medical trainees to address child poverty and improve the health of poor children.

Risk Evaluation and Mitigation Strategies With Elements to Assure Safe Use: Alignment of the Goals With the Tools to Manage Risk.

The Food and Drug Administration Amendments Act of 2007 gave the FDA the authority to require drug sponsors to submit a risk evaluation and mitigation strategies (REMS) program for those medicines with serious risks such that failure to effectively manage these risks would tip the benefit-risk balance. As of August 8, 2013, the 34 distinct individual and shared REMS programs that have specific elements to assure safe use (ETASU) were reviewed to ascertain the types of risks managed, the goals of the REMS, and the tools that were employed targeting prescribers, health care facilities, pharmacists, and particular conditions for safe use. Most REMS (65%) have a combination of risk mitigation and educational goals, but 4 REMS programs (12%) have exclusively educational goals. Preventing fetotoxicity (25%) is the most common risk managed by REMS with ETASU. Seventy-nine percent of ETASU REMS employ prescriber-based tools that include performing laboratory testing (eg, pregnancy, hepatic enzymes) or monitoring (eg, ophthalmologic examinations, documenting vaccination). The goals of REMS programs should focus on measurable behaviors that directly reduce risk. The tools that are employed should create the appropriate conditions for safe use. With the number of programs and the plethora of tools applied, serious consideration should be given to better integration of risk management into health care and pharmacy systems that are best equipped to manage such risks.