Specific Populations

Symposium 7: Operational Considerations for Early-phase Pharmacokinetic/Pharmacodynamic/Safety Studies in Pediatric Patients

Co-Chair & Faculty(s)

• 

  Dionna Green, MD

  Slides
• 

  Susan McCune, MD (she/her/hers)

  Vice President, Pediatrics & Clinical Pharmacology
  Medical Science & Strategy
  PPD, Part of Thermo Fisher Scientific

Faculty(s)

• 

  John Van Den Anker, MD, PhD, FCP, FAAP

  Chief, Division of Clinical Pharmacology
  Children's National Hosp
• 

  Janelle Burnham, MD

  Senior Director
  Otsuka Pharmaceuticals

Target Audience:

The target audience for this Symposium is physicians, pharmacists and pharmaceutical scientists in academia, industry, regulatory, CRO and clinical settings interested in pharmacokinetic/pharmacodynamic/safety studies in pediatric patients.

Description:

In 1994, the National Inst of Child Health & Human Development (NICHD) established the Pediatric Pharmacology Research Units (PPRU) Network to promote pediatric labeling of new drugs or drugs already on the market by fostering collaboration between academia, industry and healthcare providers. The PPRU network ended in 2010. Since that time, there has been a substantial growth in the number of drugs being studied under the Pediatric Research Equity Act (PREA) and Best Pharmaceuticals for Children Act (BPCA) with more than 1,000 drugs that currently have labeling changes to include pediatric use information. Many rare diseases only occur in pediatric patients so that early phase pharmacokinetic (PK)/pharmacodynamic (PD)/safety First-in-Human studies are necessary in children. In addition, given the move toward increased extrapolation of efficacy from adult studies, PK/PD/safety studies may be all that is required for pediatric labeling, especially in the infectious diseases therapeutic area (an area with the highest number of pediatric labeling changes). Given the US Food & Drug Administration guidance with respect to population PK, sparse sampling, the use of micro assays and convenience sampling, it is important to understand the operational considerations for early phase PK/PD/safety studies in pediatric patients in order to be efficient and minimize the burden to the patients and their families.

Presentations:

• Introduction of Session & History of Pediatric Clinical Pharmacology Units
  

  Co-Chair & Faculty: Susan McCune, MD (she/her/hers) – PPD, Part of Thermo Fisher Scientific
• Considerations for Early-phase Pharmacokinetic/Pharmacodynamic/Safety Clinical Trials in Pediatrics
  

  Co-Chair & Faculty: Dionna J. Green, MD
• Current Status of Early-phase Pharmacokinetic/Pharmacodynamic/Safety Clinical Trials in Pediatrics
  

  Faculty: John Van Den Anker, MD, PhD, FCP, FAAP – Children's National Hosp
• Operational Considerations in Early-phase Pharmacokinetic/Pharmacodynamic/Safety Clinical Trials in Pediatrics
  

  Faculty: Janelle M. Burnham, MD – Otsuka Pharmaceuticals
• Discussion, Questions & Answers, Learner Feedback & Evaluations