Principles of Pediatric Clinical Pharmacology and Therapeutics Course – schedule

Request for Approval under the “Generic Clearance for the Collection of Qualitative Feedback on Agency Service Delivery (NICHD)”

(OMB#: 0925-0648 ExpDate: 6/30/2024)

2021-22 Principles of Pediatric Clinical Pharmacology – Learning Objectives

10/6/2021: Career Development/ Educational Opportunities (Pediatric Trials Network)

Lecture Speaker: Kanecia Zimmerman

Learning Objectives:

• Understand potential career paths in clinical pharmacology

• Identify opportunities for additional training and education

10/13/2021: Leveraging Mentorship for a Productive Fellowship

Lecture Speakers: Kim Brouwer and Sonya Tang Girdwood

Learning Objectives:

• Recognize the need for mentors and a mentoring team

• State the qualities of a good mentor and a good mentee

• Reflect on challenging situations in mentorship through case studies

• Identify strategies to leverage their mentors to maximize productivity in their fellowship

10/20/2021: Influence of Physiologic Changes in The Developing Gastrointestinal Tract on Drug Absorption & Disposition in Children

Lecture Speaker: Valentina Shakhnovich

Learning Objectives:

• Review developmental changes in the maturing pediatric digestive tract 

  • Extrahepatic focus

  • Influence on peroral drug absorption/disposition 

  • Implications for rectally-administered drugs

• Discuss influences of diet, disease and the microbiome on drug absorption/disposition

• Provide clinical examples and identify gaps in knowledge 

10/27/2021: Ontogeny of Drug Biotransformation

Lecture Speaker: Jean Dinh

Learning Objectives:

• Review basic concepts relevant to a discussion of drug biotransformation in a pediatric context

  • Developmental trajectories

  • Regulation of endogenous molecules during growth and development

• Discuss specific Phase 1 and Phase 2 pathways

  • CYP2D6

  • CYP3A4

  • CYP2C19

  • CYP2B6

  • UGT2B7

• Present a basic strategy for investigating a specific drug or drug-related event in children

11/3/2021: Innovative Clinical Trial Design in Pediatric Drug Studies

Lecture Speaker: Stephen Balevic

Learning Objectives:

• Evaluate barriers to enrollment in pediatric drug studies

• Determine solutions to enrollment in pediatric drug studies

11/10/2021: Pharmacokinetics and Pharmacodynamics of Protein Therapeutics

Lecture Speaker: Bernd Meibohm

Learning Objectives:

• Understand the mechanistic basis for drug disposition of therapeutic proteins and how it differs from traditional small molecule drugs

• Appreciate the impact and challenges of drug-target interaction on drug disposition

• Explain the impact of immunogenicity on the pharmacokinetics (PK) and pharmacodynamics (PD) of therapeutic proteins

• Identify factors in the PK and PD of therapeutic proteins that are modulated by childhood development

11/17/2021: Model-Informed Pediatric Drug Development: Application of Pharmacometrics to Define the Right Dose for Children

Lecture Speaker: Sander Vinks

Learning Objectives:

• Appreciate the FDA’s Pediatric Study Decision Tree as a framework that can be a helpful starting point in determining the pediatric studies necessary for labeling based on the ability to extrapolate efficacy from adult or other data.

• Have a better understanding of how MIDD approaches are suited to address knowledge gaps including data leveraging to increase the success of pediatric studies.

• Highlight examples of successful pediatric model-informed drug development programs.

12/1/2021: Developmental Pharmacodyamics: Knowledge to be Gained in Pediatric Therapeutics

Lecture Speaker: Bridgette Jones

Learning Objectives:

• Understand the current knowledge base of pharmacodynamics in children

• Understand some areas of approach to increased knowledge of pharmacodynamics in children

• Understand research opportunities and needs to create more knowledge in the area

12/8/2021: High Frequency Clinical Data for AI/Machine Learning-Aided Biomarker Discovery and Enhancement of Clinical Trials: The Promise and Pitfalls

Lecture Speaker: Joseph Frassica

Learning Objectives:

• Understand the nature of available high frequency physiologic data

• Understand the possible benefits the use of large-scale data to clinical trials

• Recognize the pitfalls of the use of inadequately vetted and erroneous data in physiologic biomarker discovery

12/15/2021: Combining “Bedside" and Clinical Research Data to Inform Disease Progression and Outcomes/Biomarker Selection – Learning and Confirming

Lecture Speaker: Diane Mould

Learning Objectives:

• Understand the concept of disease progression versus disease activity

• Review the pharmacology of biologic agents and some of the issues associated with dosing metrics as applied to pediatric patients

• Understand the relationship between PK and PD commonly seen with biologic agents used to treat inflammatory disease

• Understand the concept of using Bayesian adaptive dosing and monitoring of individual PK parameters as a metric of disease activity for biologics

1/5/2022: Pharmaco-omics: Implications for Clinical Pharmacology

Lecture Speaker: Richard Weinshilboum

Learning Objectives:

• Briefly introduce the “omics” techniques being applied to clinical pharmacology

• Provide examples of the application of multiple omics to study drug response phenotypes

• Outline the use of multiple omics data to “guide” or “inform” genomic studies

1/12/2022: Systems Pharmacology and Translational Therapeutics

Lecture Speaker: Garret FitzGerald

Learning Objectives:

• Provide a view of clinical pharmacology that extends beyond modelling drug exposure and kinetics

• Incorporate contemporary technologies to understand mechanism of action

• Parse variability of drug response in an effort to achieve a more personalized approach to therapeutics.

1/19/2022: Pharmacotherapy of Pediatric Asthma

Lecture Speaker: Theresa Guilbert

Learning Objectives:

• Review national and international asthma guidelines and medications recommended by these groups.

• Present evidence for use of these recommended medications.

• Discuss future steps

1/26/2022: Pharmacotherapy of Viral Infections in Children

Lecture Speaker: Scott James

Learning Objectives:

• Describe clinical pharmacology principles of infectious disease agents

• List the drug development steps for infectious disease agents in children

2/2/2022: The Power of PK/PD and Monte Carlo Simulation (MCS): Pediatric Antimicrobial Drug Development and Recommendations for Clinical Use

Lecture Speaker: John Bradley

Learning Objectives:

• Review the various pharmacodynamic metrics for antibiotic activity

• Understand the rationale behind High/Infrequent dosing (aminoglycosides) and Low/Frequent dosing (beta-lactams)

• The antibiotic exposure needs to occur at the site of infection, and can vary tremendously from the serum concentration (e.g., CSF concentrations vs urine concentrations for aminoglycosides)

• Realize the “all susceptibilities are local” and the dosing/exposure need to match the susceptibilities in the institution of the clinician

• Consider how important, in children, it is to achieve the desired exposure, and the risk of failure you are willing to accept for a particular infection.

2/9/2022: Pharmacotherapy of Pediatric Cancer: Targeted New Agents

Lecture Speaker: Michael Ferguson

Learning Objectives:

• Review genetics of tumors

• Explain Precision Medicine (Precision Oncology/Precision Genomics/Personalized Medicine)

• Learn about different sequencing companies/techniques

• Garner knowledge about newer targeted therapy

• Apply new knowledge to case examples

2/16/2022: FDA Roundtable on Systems Pharmacology

Lecture Speaker: Gilbert Burckart + Team

Learning Objectives:

• Provide a current, and evolving overview of systems pharmacology

• Discuss specific research/case studies in systems pharmacology and drug development

• Engage a conversation in career opportunities as well as support for training in systems pharmacology