Innovative study design for paediatric clinical trials

Paola Baiardi, Carlo Giaquinto, Silvia Girotto, Cristina Manfredi, Adriana Ceci

Research output: Contribution to journalArticlepeer-review


Purpose: Despite representing a fundamental step towards the efficacious and safe utilisation of drugs in the paediatric population, the conduct of clinical trials in children poses several problems. Methodological issues and ethical concerns represent the major obstacles that have traditionally limited paediatric research. The randomised clinical trial, mainstay of clinical studies to assess the effects of any therapeutic intervention, shows some weaknesses that make it scarcely applicable to the paediatric population. Alternative and innovative approaches to clinical trial design in small populations have been developed in the last few decades with the aim of overcoming the limits related to small samples and to the acceptability of the trial. Methods: This systematic review describes a variety of alternative designs to assess efficacy and safety in the paediatric population, including their applicability, advantages, disadvantages and real case examples. Approaches include sequential and adaptive designs, Bayesian methods and other innovative approaches. Results: By limiting the sample size and increasing acceptability, these methods may rationally limit the amount of experimentation in children to what is achievable, necessary and ethical Conclusion: Thanks to their features, these methods represent a reliable way of ultimately improving paediatric care.

Original languageEnglish
JournalEuropean Journal of Clinical Pharmacology
Issue numberSUPPL. 1
Publication statusPublished - May 2011


  • Clinical trial design
  • Clinical trials
  • Paediatrics

ASJC Scopus subject areas

  • Pharmacology (medical)
  • Pharmacology


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