CORE COMPETENCIES IN CLINICAL & TRANSLATIONAL RESEARCH: The Child Health Perspective I. Clinical & Translational Research Questions: Extract information from the scientific literature that yields scientific insight for translational research innovation Describe how a translational research hypothesis might be modified to be testable in a pediatric population. (e.g., pediatric rare diseases, small numbers of patients) Analyze scientific papers and evaluate their strengths and limitations; check for inconsistencies in reported data, and re-examine in the context of newer information for inconsistencies I. Clinical & Translational Research Questions: Extract information from the scientific literature that yields scientific insight for translational research innovation Describe how a translational research hypothesis might be modified to be testable in a pediatric population. (e.g., pediatric rare diseases, small numbers of patients) Analyze scientific papers and evaluate their strengths and limitations; check for inconsistencies in reported data, and re-examine in the context of newer information for inconsistencies

Study Design Establish the specific aims and approaches to address them Prepare an application to an IRB Describe study design modifications and methods appropriate for small sample sizes Describe study design modifications or controls that would be appropriate for a vulnerable population Describe special considerations for biospecimen collection from children (e.g., age-appropriate sampling techniques, quantity/volume of specimen, subject assent/ informed consent) Establish the specific aims and approaches to address them Prepare an application to an IRB Describe study design modifications and methods appropriate for small sample sizes Describe study design modifications or controls that would be appropriate for a vulnerable population Describe special considerations for biospecimen collection from children (e.g., age-appropriate sampling techniques, quantity/volume of specimen, subject assent/ informed consent)

Research Implementation Integrate elements of translational research into given study designs that could provide the bases for future research, such as the collection of biological specimens, nested studies, and the development of community-based interventions Describe special considerations in collecting biological specimens from children for the purpose of biobanking and future studies. Describe the team and expertise needed that would accomplish the goals of the study. Evaluate the challenges of working with lower income communities and ways to improve participation in community based interventions Integrate elements of translational research into given study designs that could provide the bases for future research, such as the collection of biological specimens, nested studies, and the development of community-based interventions Describe special considerations in collecting biological specimens from children for the purpose of biobanking and future studies. Describe the team and expertise needed that would accomplish the goals of the study. Evaluate the challenges of working with lower income communities and ways to improve participation in community based interventions

Biomedical Informatics Collaborate with bioinformatics specialists in the design, development, and implementation of data collection for research projects. Identify unique or describe age-appropriate modifications to data fields for the conduct of studies involving children Describe how quality control/quality assurance standards for data entry might be modified for studies involving children (e.g., height/weight data fields appropriate for a 3 year old) Collaborate with bioinformatics specialists in the design, development, and implementation of data collection for research projects. Identify unique or describe age-appropriate modifications to data fields for the conduct of studies involving children Describe how quality control/quality assurance standards for data entry might be modified for studies involving children (e.g., height/weight data fields appropriate for a 3 year old)

Responsible Conduct of Research Explain how the structural arrangement of science and the research industry may influence the behavior of scientist and the production of scientific knowledge Summarize the US regulatory and historical framework to the conduct of clinical research involving children and how it differs from adults (e.g., section 407 and minimal risk; age-appropriate ascent procedures ) Analyze the ethical problems inherent in pediatric research and indicate ways to ensure protection of pediatric subjects. (eg. issues with both vs single parental consent etc) Identify one ethical dilemma and explain/defend viewpoint and course of action. Identify emerging challenges in the application of informed consent in the field of genetic studies (eg. how long to keep specimens, problems with de-identification of samples, how to deal with positive results) Explain how the structural arrangement of science and the research industry may influence the behavior of scientist and the production of scientific knowledge Summarize the US regulatory and historical framework to the conduct of clinical research involving children and how it differs from adults (e.g., section 407 and minimal risk; age-appropriate ascent procedures ) Analyze the ethical problems inherent in pediatric research and indicate ways to ensure protection of pediatric subjects. (eg. issues with both vs single parental consent etc) Identify one ethical dilemma and explain/defend viewpoint and course of action. Identify emerging challenges in the application of informed consent in the field of genetic studies (eg. how long to keep specimens, problems with de-identification of samples, how to deal with positive results)

Scientific Communication Describe the unique regulatory considerations for development and approval of pediatric medical devices and pharmaceuticals intended for use in children Involving a pediatric community in research is very difficult given the already existing lack of studies with children. Children are physiologically different than adults and are growing and changing- therefore the same rules can’t apply. This however makes progress in research difficult. There are ethical dilemmas as well as.physiologic/biological dilemmas with using medical devices and medications that have never been used/approved in children. Furthermore, children do not have the legal capacity to consent. If a guardian is to consent then they must have a full and clear understanding of what it is the research devices are intended for and how this can positively and adversely affect their children. Describe the unique regulatory considerations for development and approval of pediatric medical devices and pharmaceuticals intended for use in children Involving a pediatric community in research is very difficult given the already existing lack of studies with children. Children are physiologically different than adults and are growing and changing- therefore the same rules can’t apply. This however makes progress in research difficult. There are ethical dilemmas as well as.physiologic/biological dilemmas with using medical devices and medications that have never been used/approved in children. Furthermore, children do not have the legal capacity to consent. If a guardian is to consent then they must have a full and clear understanding of what it is the research devices are intended for and how this can positively and adversely affect their children.

Translational Teamwork Manage a clinical and /or translational research study A multidisciplinary team is key- However, it is also important to have a long -term commitment. This is especially difficult when there are trainees involved. The transitioning studies, lifespan studies need to have physicians, researchers, etc.. that have made this their life work leading the way. Describe special considerations in assembling and managing a multidisciplinary team to conduct longitudinal, lifespan studies (e.g., transitioning of child to adult care) Manage a clinical and /or translational research study A multidisciplinary team is key- However, it is also important to have a long -term commitment. This is especially difficult when there are trainees involved. The transitioning studies, lifespan studies need to have physicians, researchers, etc.. that have made this their life work leading the way. Describe special considerations in assembling and managing a multidisciplinary team to conduct longitudinal, lifespan studies (e.g., transitioning of child to adult care)

Community Engagement Specify how cultural and linguistic competence and health literacy have an impact on the conduct of community-engaged research There must be a clear understanding amongst researcher and community being researched. Information may be skewed and misinterpreted if those involved in research are not able to express their needs/ research design and goals to the community. It would be wise to engage physicians with different ethnicities and language skills to be involved in community research projects. Describe differences in community views on children and how this might impact community-engaged research. Evaluate areas of improvement in research participation of minority/non-English speaking communities and ways to adapt research design to be culturally sensitive. Specify how cultural and linguistic competence and health literacy have an impact on the conduct of community-engaged research There must be a clear understanding amongst researcher and community being researched. Information may be skewed and misinterpreted if those involved in research are not able to express their needs/ research design and goals to the community. It would be wise to engage physicians with different ethnicities and language skills to be involved in community research projects. Describe differences in community views on children and how this might impact community-engaged research. Evaluate areas of improvement in research participation of minority/non-English speaking communities and ways to adapt research design to be culturally sensitive.

Child Health Oversight Committee (CC-CHOC) Initiatives Create a multi-center IRB for pediatric studies Create metrics of success in child health research Create prioritization model for pediatric drugs and devices Create a virtual biorepository for study of rare disease Contribute to creation of guidelines related to core competencies for trainees in child health research. Create a multi-center IRB for pediatric studies Create metrics of success in child health research Create prioritization model for pediatric drugs and devices Create a virtual biorepository for study of rare disease Contribute to creation of guidelines related to core competencies for trainees in child health research.