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Innovative study design for paediatric clinical trials

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Abstract

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.

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Acknowledgements

This report is part of the Task-force in Europe for Drug Development for the Young (TEDDY) Network of Excellence supported by the European Commission’s Sixth Framework Program (Contract n. 0005216 LSHBCT- 2005-005126).

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Authors and Affiliations

  1. Consorzio per Valutazioni Biologiche e Farmacologiche, Via Luigi Porta 14, 27100, Pavia, Italy

    Paola Baiardi, Cristina Manfredi & Adriana Ceci

  2. Dipartimento di Pediatria, Azienda Ospedaliera di Padova, Via Giustiniani 3, 35128, Padova, Italy

    Carlo Giaquinto

  3. Azienda U.L.S.S. n° 12 Veneziana, Pl. Zendrini 28, 30030, Venezia, Italy

    Silvia Girotto

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  1. Paola Baiardi
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  2. Carlo Giaquinto
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  4. Cristina Manfredi
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  5. Adriana Ceci
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on behalf of the TEDDY Network of Excellence

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Correspondence to Paola Baiardi.

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Part of the TEDDY supplement

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Baiardi, P., Giaquinto, C., Girotto, S. et al. Innovative study design for paediatric clinical trials. Eur J Clin Pharmacol 67 (Suppl 1), 109–115 (2011). https://doi.org/10.1007/s00228-011-0990-y

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  • DOI: https://doi.org/10.1007/s00228-011-0990-y

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