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Overview


The research fellowship program in Pediatric Clinical Pharmacology at CMH provides didactic education in subject areas germane to the discipline, and training in both translational and clinical research.

The overarching goals of this program are to provide each trainee with:

  • A firm conceptual understanding of research methods that are considered to represent the fundamentals of pediatric clinical/developmental pharmacology.
  • Direct application of knowledge, skills and critical abilities to identify problems and design effective plans to conduct the research necessary to solve them.
  • The ability to effectively communicate through scientific writing and grant proposal development information to be gained and information attained through their research endeavors.
  • An ability to effectively organize a career development plan that will insure a successful transition to independent research support.
  • Values consistent with the ethical conduct of science, a commitment to lifelong learning and the education of others as part of a professional career.

Core didactics


Principles of Clinical Pharmacology (course facilitator – T. Sandritter) is a general clinical pharmacology course (30 hr total) sponsored by the NIH Clinical Center. This weekly lecture series covers the fundamentals of clinical pharmacology as a translational scientific discipline which spans the continuum of drug discovery through therapeutic use. Faculty for this course include NIH staff and distinguished scientists from the pharmaceutical industry, FDA and academic institutions. The most recent lectures will be viewed online and the companion text will be purchased for each trainee. Fellows will take a 10-question self-assessment test prepared by the course facilitator) for each lecture and will convene with the facilitator, or a faculty member, to discuss each topic area.

Developmental Pharmacology (course director – N/A) The original 12-hour didactic course developed for fellows at has been replaced by the NIH T32 Pediatric Clinical Pharmacology webinar series. This weekly series address the fundamentals of clinical pharmacology, including drug metabolism and transport, assessment of drug effects, pharmacotherapy, and pediatric drug formulations from a pediatric perspective. The course is taught by faculty from leading academic institutions (including Children’s Mercy), the NIH, and the FDA.

Introduction to Pharmacokinetics/Pharmacodynamics (course director – J.S. Leeder), over a 3 month time frame, provides trainees with a comprehensive, functional understanding of the processes (physiologic and mathematical) used to quantitate drug disposition (e.g. drug liberation, absorption, distribution, metabolism, excretion) and response.

Applied Pharmacokinetics I and II (course director – S.M. Abdel-Rahman) represent two experiential, trainee-paced courses that introduce fellows to both classical and population-based approaches for analyzing PK/PD data. Applied PK I focuses on bedside or clinical PK applications and is entirely case-based. Applied PK II is also case-based, examining larger PK-PD datasets derived from investigator initiated and industry sponsored studies. Software applications introduced in this course include Kinetica, Phoenix, WinNonLin, NONMEM, and Simcyp.

Survey in Pharmacogenomics (course director – A. Gaedigk) provides trainees with a conceptual and factual basis regarding the role of pharmacogenomics as a determinant of drug disposition and action. The course consists of two modules; the first focuses on genomic determinants of PK and PD, the role of pharmacogenomics in pediatric drug development and relevant contemporary bioethical issues, the second provides a comprehensive overview of the tools and technologies that are currently used to support research in the field and contemporary issues in genomic research (e.g. epigenetics, epistasis, applied statistical genetics). This module also introduces trainees to the emerging field of translational bioinformatics where computational and biostatistical methods are integrated to explore genomic data which can define the expression of disease and its response to treatments. Concepts germane to the field such as GWAS and DNA sequence analysis, SNP and haplotype analysis, next-generation DNA sequencing, meta-analysis of “-omics” (genomics, proteomics, metabolomics) data, expression quantitative trait loci analysis and the generation of data-driven hypothesis are covered. The experiential portion of this course will consist of six guided laboratory experiences (3 hr each) intended to provide “hands-on” training in techniques germane to PG (e.g., PCR-RFLP, qPCR, sequencing, microarray). A separate “hands-on” application introduces trainees to commonly used bio-computing tools.

Pediatric Drug Development (course director – R.E. Kauffman; 9 hour didactic) is an interactive, bi-monthly, seminar intended to introduce trainees to the aspects of the drug development process which includes drug discovery, pre-clinical testing, human clinical trials and bioethical issues. The format for the course includes interactive didactic presentations, assigned readings and case presentations.

Pharmacoepidemiology (course director – D. Yin), This mini-series is intended to provide trainees with a conceptual, working understanding of pharmacoepidemiology-based research from the conceptual (developing pharmacoepidemiology-related hypotheses) to the operational (data cleaning, data queries, compliant data management, outcomes research data management).

Elective rotations


Simcyp/Certara Corporation (Karen Rowland-Yeo, PhD)
Trainees will initially participate in one of several workshops offered by Certara, followed by an intensive onsite rotation. Using a hands-on approach, this rotation would introduce the trainee to the strategies by which existing in vitro and in vivo data are evaluated for inclusion in modeling tools and involve them in the process of identifying, vetting, and prioritizing data to refine these models. They should leave this rotation with; 1) a solid conceptual understanding of the principles of modeling and simulation along with the strengths and limitations inherent to PBPK-based modeling and simulation tools, 2) the knowledge of how to apply, and validate, PBPK-based modeling and simulation to virtual sets of children for the purposes of clinical trial design, and 3) an introduction on how to explore PBPK-driven approaches to generate hypothesis about drug disposition pathways when existing physiologic data are unavailable.

Office of Clinical Pharmacology, Center for Drug Evaluation and Research, FDA (Gil Burckart, PharmD)
Trainees will have an opportunity to join the Pediatric Clinical Pharmacology group led by Dr. Burckart under the competitive ORISE program. The experience is a 2-month commitment, and activities include review of IND, NDA and Phase II/III Pediatric plans submitted to the FDA with any pediatric studies planned, followed by attendance at the weekly Pediatric Review Committee (PeRC) meetings where the projects are discussed with representatives from the individual Divisions (e.g. GI, Neuro, Onc). Trainees will have the opportunity to work with the Office of Pediatric Clinical Pharmacology to discuss neonatal studies, and pharmacokinetic and pharmacodynamic studies with the specialty divisions. Trainees will be expected to prepare and deliver a presentation to members of the Office of Clinical Pharmacology on topic related to their area of expertise.

Pharmacogenomics KnowledgeBase (Russ Altman, MD, PhD and Teri Klein, PhD)
Under the supervision Drs. Klein and Altman, trainees may elect to spend one month at the Pharmacogenomics Knowledge Base (PharmGKB) at Stanford University. Trainees will be exposed to the curation process that has been developed, as well as the role of PharmGKB in the development of the Clinical Pharmacogenetics Implementation Consortium (CPIC) guidelines. To individualize the experience, the trainees have an opportunity to work on VIP gene summaries or pathways for drug response of interest to them, which are potentially postable on the site and publishable as summaries in Pharmacogenetics & Genomics.

Formulary Management
Interested fellows will be introduced to the process of promoting rational and appropriate pediatric medication use through two opportunities. Within the context of a health care delivery organization, fellows will undertake a participatory experience with the institutions Pharmacy & Therapeutics Committee (Chair: D. Blowey). Fellows will receive an initial didactic session followed by participation in two meetings. Meeting dates will be selected where discussion is anticipated to be centered around the therapeutic class most closely related to the fellow’s medical subspecialty. Fellows that are interested in gaining experience from the perspective of a health care management organization will join Dr. Abdel-Rahman at a quarterly meeting of the Missouri Medicaid Drug Utilization Review Board at the state capital. Dr. Abdel-Rahman serves as Chair for the Board and will provide the fellow with an informal discussion session about the process of formulary management for a government payor prior to the meeting.


 

Contact

Susan Abdel-Rahman, PharmD
Fellowship Director

  (816) 855-1759


Jennifer Goldman, MD, MS-CR
Fellowship Associate Director

  (816) 234-3061

  Email