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Consequences of Directive 2001/20/EC for investigator-initiated trials in the paediatric population—a field report

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Abstract

On 4 April 2001, the European Parliament and Council enacted Directive 2001/20/EC, which had to be implemented in the national law of the European Union member states by May 2004. Its aim was to improve the quality of clinical trials and to assure the safety and well-being of trial subjects. We recently initiated the first paediatric investigator-initiated trial (IIT) at the University Hospital of Cologne according to Directive 2001/20/EC. This field report demonstrates the consequences and implications of the directive for paediatric IITs. Based on our experience, we agree that Directive 2001/20/EC improves the quality of clinical trials and assures the safety and well-being of trial subjects. However, at the same time, performing an IIT according to the new requirements is nearly impossible for clinicians and academic researchers without cooperating with expensive specialised experts, such as project managers, statisticians, data managers, pharmacists and monitors. Therefore, it is absolutely mandatory that financial support for paediatric IITs be adapted and increased in order to be able to meet the new requirements and obligations. Regulation (EC) No 141/2000 on orphan medicinal products and the recently adopted regulation on medicinal products for paediatric use (Paediatric Regulation) are important steps in improving clinical research in children. However, both regulations mainly encourage clinical research carried out by the pharmaceutical industry, whereas paediatric IITs are not in the scope of this legislation. We need to develop new concepts for funding to ensure future paediatric IITs, for example through specific grants from the European Union or member states.

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Abbreviations

IIT:

investigator-initiated trial

GCP:

good clinical practice

CCTC:

Coordinating Centre for Clinical Trials, Cologne

CRF:

case report form

SmPC:

summary of product characteristics

TMF:

trial master file

ISF:

investigator site file

AE:

adverse event

SAE:

serious adverse event

SUSAR:

suspected unexpected serious adverse reaction

DMSC:

Data monitoring and safety committee

EMEA:

European Medicines Agency

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Authors and Affiliations

  1. Department of Neonatology and Paediatric Intensive Care, University of Cologne, Children’s Hospital, Kerpener Str. 62, 50937, Cologne, Germany

    Lars Welzing & Bernhard Roth

  2. Coordinating Centre for Clinical Trials Cologne (CCTC), University of Cologne, Cologne, Germany

    Urs Harnischmacher

  3. Coordinating Centre for Clinical Trials Cologne (CCTC), Paediatric Unit, University of Cologne, Cologne, Germany

    Annic Weyersberg

Authors
  1. Lars Welzing
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  2. Urs Harnischmacher
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  3. Annic Weyersberg
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  4. Bernhard Roth
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Corresponding author

Correspondence to Lars Welzing.

Additional information

This field report was developed without any funding or grants. No author has any conflict of interest.

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Welzing, L., Harnischmacher, U., Weyersberg, A. et al. Consequences of Directive 2001/20/EC for investigator-initiated trials in the paediatric population—a field report. Eur J Pediatr 166, 1169–1176 (2007). https://doi.org/10.1007/s00431-007-0434-y

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  • DOI: https://doi.org/10.1007/s00431-007-0434-y

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