"Walking on both sides of the fence": A qualitative exploration of the challenges and opportunities facing emergent clinician-scientists in child health.

RATIONALE, AIMS, AND OBJECTIVES: While paediatric clinician-scientists are ideally positioned to generate clinically relevant research and translate research evidence into practice, they face challenges in this dual role. The authors sought to explore the unique contributions, opportunities, and challenges of paediatric clinician-scientists, including issues related to training and ongoing support needs to ensure their success. METHOD: The authors used a qualitative descriptive approach with thematic analysis to explore the experiences of clinician-scientist stakeholders in child health (n = 39). Semi-structured interviews (60 min) were conducted virtually and recorded. Thematic analysis was conducted according to the phases outlined by Braun and Clarke (2006). RESULTS: The analysis resulted in the creation of three themes: (1) "Walking on both sides of the fence": unique positioning of clinician-scientists for advancing clinical practice and research; (2) the clinician-scientist: a specialized role with significant challenges; and (3) beyond the basics of clinical and research training programmes: essential skill sets and knowledge for future clinician-scientists. CONCLUSIONS: While clinician-scientists can make unique contributions to the advancement of evidence-based practice, they face significant barriers straddling their dual roles including divergent institutional cultures in healthcare and academia and a lack of infrastructure to effectively support clinician-scientist positions. Training programmes can play an important role in mentoring and supporting early-career clinician-scientists.

Equity, diversity and inclusion of pediatric clinician-scientists in Canada: a thematic analysis.

BACKGROUND: Underrepresented voices and perspectives are missing from academic and clinical health sciences. We aimed to define the unique opportunities and challenges of pediatric clinician-scientists related to equity, diversity and inclusion; and to identify key components of training needed to support people from equity-seeking groups as emerging and early-career pediatric clinician-scientists to generate diverse health research leaders in knowledge generation, implementation and translation. METHODS: Using a qualitative descriptive approach, we examined the experiences of clinician stakeholders. Semistructured interviews were conducted with pediatric clinician-scientist stakeholders. Thematic analysis was performed. RESULTS: We interviewed a total of 39 individuals. Our analysis resulted in 4 interrelated themes: the pervasiveness and invisibility of sexism; the invisibility and visibility of racism; proposed individual-level solutions to the sexism and racism; and proposed institutional and system-level changes to address the porous and leaky pipeline. These themes acknowledged that, ultimately, system change is required for addressing equity, diversity and inclusion in clinical and academic training environments. INTERPRETATION: These findings highlight the importance of addressing systemic biases that limit the inclusion of women and racialized individuals in pediatric clinician-scientist careers. Further research is needed to explore the problem of exclusion, which will, in turn, inform education of pediatric clinician-scientists and inform better ways to promote equity, diversity and inclusivity; these steps are needed to foster systemic change in the cultures that perpetuate exclusivity in both academic and clinical communities.

Codevelopment and usability testing of Patient Engagement 101: a Patient-Oriented Research Curriculum in Child Health e-learning module for health care professionals, researchers and trainees.

BACKGROUND: Patient and family engagement is thought to improve the quality and relevance of child health research. We developed and evaluated the usability of Patient Engagement 101, an e-learning module designed to strengthen the patient-oriented research readiness of health care professionals, researchers, trainees and other stakeholders. METHODS: The development of Patient Engagement 101 was co-led by a parent and a researcher and overseen by a diverse multistake-holder steering committee. The module was refined and evaluated using a mixed-methods usability testing approach with 2 iterative cycles of semistructured interviews, observations and questionnaires. We collected module feedback by way of semistructured interviews, the validated System Usability Scale, and satisfaction, knowledge and confidence questionnaires. Thematic coding of transcripts and field notes, informed by team discussions, guided the module revisions. RESULTS: Thirty end-users completed usability testing (15 per cycle). In each cycle, we modified the module with respect to its content, learner experience, learner-centred design and aesthetic design. Participants were highly satisfied, and System Usability Scale scores indicated the module had the best imaginable usability. Substantial increases in the participants' knowledge test scores and the confidence to engage in patient-oriented research, but not self-rated knowledge, were observed after module completion. INTERPRETATION: Codevelopment with patients and caregivers, and refinement through comprehensive end-user testing, resulted in a training resource with exceptional usability that improved knowledge and confidence to engage in patient-oriented research in child health. Patient Engagement 101 is openly available online, and the methods used to develop and evaluate it may facilitate the creation and evaluation of similar capacity-building resources.

Curricula, Teaching Methods, and Success Metrics of Clinician-Scientist Training Programs: A Scoping Review.

PURPOSE: To describe the literature on clinician-scientist training programs to inform the development of contemporary and inclusive training models. METHOD: The authors conducted a scoping review, searching the PubMed/MEDLINE, CINAHL, and Embase databases from database inception until May 25, 2020. Studies presenting primary research that described and evaluated clinician-scientist training programs were identified for data abstraction. On the basis of deductive and inductive methods, information about program characteristics, curricula, teaching strategies, and success metrics was extracted. The extracted variables were analyzed using descriptive statistics. RESULTS: From the initial 7,544 citations retrieved and 4,974 unique abstracts screened, 81 studies were included. Of the 81 included studies, 65 (80.2%) were published between 2011 and 2020, 54 (66.7%) were conducted in the United States, and 64 (79.0%) described programs that provided broad clinician-scientist training. Few programs provided funding or protected research time or specifically addressed needs of trainees from underrepresented minority groups. Curricula emphasized research methods and knowledge dissemination, whereas patient-oriented research competencies were not described. Most programs incorporated aspects of mentorship and used multiple teaching strategies, such as direct and interactive instruction. Extrinsic metrics of success (e.g., research output) were dominant in reported program outcomes compared with markers of intrinsic success (e.g., career fulfillment). CONCLUSIONS: Although programs are providing clinician-scientists with practical skills training, opportunities exist for curricular and pedagogic optimization that may better support this complex career path. Training programs for clinician-scientists can address contemporary issues of wellness and equity by reconsidering metrics of program success and evolving the core tenets of their education models to include equity, diversity, and inclusion principles and patient-oriented research competencies.

Co-development and Usability Testing of Research 101: A Patient-Oriented Research Curriculum in Child Health (PORCCH) E-Learning Module for Patients and Families.

Background: Engaging patients and families as research partners increases the relevance, quality, and impact of child health research. However, those interested in research engagement may feel underequipped to meaningfully partner. We sought to co-develop an online learning (e-learning) module, "Research 101," to support capacity-development in patient-oriented child health research amongst patients and families. Methods: Module co-development was co-led by a parent and researcher, with guidance from a diverse, multi-stakeholder steering committee. A mixed-methods usability testing approach, with three iterative cycles of semi-structured interviews, observations, and questionnaires, was used to refine and evaluate the e-learning module. Module feedback was collected during testing and a post-module interview, and with the validated System Usability Scale (SUS), and satisfaction, knowledge, and self-efficacy questionnaires. Transcripts and field notes were analyzed through team discussion and thematic coding to inform module revisions. Results: Thirty participants fully tested Research 101, and another 15 completed confirmatory usability testing (32 caregivers, 6 patients, and 7 clinician-researchers). Module modifications pertaining to learner-centered design, content, aesthetic design, and learner experience were made in each cycle. SUS scores indicated the overall usability of the final version was "excellent." Participants' knowledge of patient-oriented research and self-efficacy to engage in research improved significantly after completing Research 101 (p < 0.01). Conclusions: Co-development and usability testing facilitated the creation of an engaging and effective resource to support the scaling up of patient-oriented child health research capacity. The methods and findings of this study may help guide the integration of co-development and usability testing in creating similar resources.

Development of the patient-oriented research curriculum in child health (PORCCH).

BACKGROUND: The Canadian Institutes for Health Research launched a national 'Strategy for Patient-Oriented Research' (SPOR) in 2011. Patient-oriented research is defined as a continuum of research that engages patients as partners, focuses on patient-identified priorities, and improves patient outcomes. Capacity development is a core element of SPOR. Barriers to patient-oriented research include unfamiliarity with the research process for patients and families and unfamiliarity with the methods of patient and family engagement for researchers. METHODS: The aim of the Patient-Oriented Research Curriculum in Child Health (PORCCH) is to build capacity in patient-oriented research in child health among patients and families, researchers, healthcare professionals, decision-makers, and trainees through a curriculum delivered via a series of interactive online modules (e-learning). A multi-disciplinary, multi-stakeholder steering committee, which included patients and families, guided the development of the curriculum and provided feedback on individual modules. The content, design, and development of each module were co-led by a parent and researcher in an equal partnership. RESULTS: PORCCH consists of a series of five modules. All modules are interactive and include video vignettes and knowledge comprehension questions. Access to the modules is free and each module takes approximately 30 min to complete. The five modules are: Research 101 (an Introduction to Patient-Oriented Research, parts 1 and 2), Patient Engagement 101 (an Introduction to Patient Engagement in Child Health Research, parts 1 and 2), and Research Ethics 101. CONCLUSIONS: PORCCH was developed specifically to overcome recognized barriers to the engagement of patients and families in child health research. The aim of the curriculum is to build capacity in patient-oriented research in child health. The goal is for PORCCH to be a useful resource for all stakeholders involved in patient-oriented research: patients and families, researchers, healthcare professionals, decision-makers, and trainees.

Canada launched a national 'Strategy for Patient-Oriented Research' in 2011. The aim of the strategy is to improve patients' health and well being by focusing on research that is important to patients, and by involving patients and families as equal partners in research. Our group developed the Patient-Oriented Research Curriculum in Child Health (PORCCH) to help patients and families, and other individuals involved in research, learn about patient-oriented research. Patients and families will learn about the research process, who is involved, and the timelines of a research study. Researchers will learn about the methods used in patient-oriented research and some practical ways to meaningfully engage patients and families in research. The online curriculum includes five modules. The design and development of each module was co-led by a parent and researcher in an equal partnership. Access to the modules is free ( www.porcch.ca ) and each module takes approximately 30 min to complete. The five modules are: a) Research 101 part 1, What is Health Research and Who is Involved? b) Research 101 part 2, Timeline of a Research Study, c) Patient Engagement 101 part 1, Foundations of Patient Engagement, d) Patient Engagement 101 part 2, Patient Engagement in Practice, and e) Research Ethics 101. It is hoped that PORCCH will be a useful resource for patients and families and other stakeholders involved in patient-oriented research in child health.

Defining combined immunodeficiency.

BACKGROUND: Although the extreme condition of typical profound T-cell dysfunction (TD), severe combined immunodeficiency (SCID), has been carefully defined, we are currently in the process of better defining less typical T-cell deficiencies, which tend to present with autologous circulating T-cell combined immunodeficiency (CID). Because autologous cells might interfere with the outcome of bone marrow transplantation, protocols usually include conditioning regimens. Therefore it is important to define the numbers of autologous cells usually detected in patients with CID versus those with SCID. OBJECTIVES: We sought to determine the number of circulating T cells in patients with SCID as opposed to those with CID, to study their function, and to evaluate their possible detection during newborn screening using T-cell receptor excision circle (TREC) analysis. METHODS: Numbers of circulating CD3(+) T cells (as determined by means of flow cytometry), in vitro responses to PHA, and TREC levels, all measured at presentation, were compiled from the research charts of the entire cohort of patients followed prospectively for T-cell immunodeficiency at the Hospital for Sick Children. Clinical data were ascertained retrospectively from the patient's hospital charts. RESULTS: One hundred three patients had CD3(+) determinations, and 80 of them had a genetic diagnosis. All patients considered to have typical SCID had CD3(+) T-cell counts of fewer than 500 cells/µL. Some variability was observed among different genotypes. In vitro responses to PHA were recorded in 88 patients, of whom 68 had a genetic diagnosis. All patients with low CD3(+) T-cell numbers (<500 cells/µL) also had markedly decreased responses to PHA (typical SCIDs). However, responses ranged widely in the groups of patients with TD who had more than 500 CD3(+) autologous circulating T cells per microliter. Although patients with Omenn syndrome and ζ chain-associated protein, 70 kDa (ZAP70), and purine nucleoside phosphorylase (PNP) deficiencies had low responses, patients with the p.R222C mutation in the IL-2 receptor γ(IL2RG) gene as well as IL-10 receptor and CD40 ligand deficiencies had normal or near-normal mitogen responses. Finally, 51 patients had TREC levels measured. All patients with typical SCID, Omenn syndrome, and ZAP70 deficiency had low TREC levels. In contrast, patients with mutations in forkhead box protein 3 (FOXP3), CD40 ligand (CD40L), and IL-10 receptor α(IL10RA), as well as patients with the p.R222C mutation in the IL2RG gene, had normal TREC levels. CONCLUSION: Patients with typical SCID can be defined as having fewer than 500 circulating CD3(+) T cells. Most patients with autologous T cells still have profound TD, as defined by reduced in vitro function and thymus output. Some patients with conditions including TD have normal TREC levels and will therefore not be detected in a TREC-based newborn screening program.