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A BLUE-PRINT FOR PERFORMING PAEDIATRIC PHARMACOLOGICAL RESEARCH
  1. Eric Vermeulen1,
  2. Debbie Nuytemans2,
  3. Timo de Haan2,
  4. Robert Flint3,
  5. Johanna van der Lee1
  1. 1Pediatric clinical Research Office, AMC Amsterdam
  2. 2Deptartment of Neonatology, AMC Amsterdam
  3. 3Deptartment of Neonatology, Erasmus MC Rooterdam and Deptartment of Pharmacy UMC Radboud Nijmegen

Abstract

Introduction In order to develop rational, patient tailored dosing schemes, population PK/PD studies in children and infants are needed. The emergence of new laboratory techniques (LC-MS/MS) and statistical tools (population PK/PD modeling) allows for the analysis of sparse and unbalanced data and has increased the possibilities to perform (observational) PK/PD studies in the paediatric clinic.

To improve the quality of future pediatric PK/PD investigations the experience and knowledge about these tools should be shared and a basic study template agreed upon.

Objective We aim to present a possible blue-print to assist paediatricians in the design and conduct of multicentre PK/PD studies in children and infants.

Methods Based on a review of the existing literature and multiple interviews with paediatricians and pharmacologists involved in multicenter paediatric PK/PD research a blue-print is drafted containing recommendations and examples for the design and implementation of PK/PD studies in children. The draft blue-print will be available at the conference and participants are asked for feed-back.

Results PK/PD research to determine dosing in children and infants is preferably performed in a multi-center infrastructure in order to include a sufficient number of subjects within a reasonable time, covering all covariates relevant for dosage. The multi-center collaboration may also enable the analysis of multiple drugs in one cohort increasing cost-efficiency. Formal monitoring is needed to guarantee the quality of collected data.

Discussion If performed well, the results of these studies will contribute to the evidence base underlying clinical guidelines and regulatory decisions concerning labeling adjustments.

  • ESDP

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