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Paediatric clinical studies

Work Package 4 - New methods for clinical studies in paediatrics 

 

GRiP clinical studies in paediatricsDrug development programs in children need to be considered on a case-by-case basis depending on the drug, the condition, and the target population as the approach to clinical drug development that has been developed in adults is not automatically applicable to them. Specific considerations, such as the relatively small populations with a condition and ethical responsibilities, need to be taken into account. Work Package 4 will address these concerns regarding methodology and study designs.

 

The aims of Work Package 4 are:

  • To produce an assessment of paediatric clinical research methods based on studies that have already been performed.
  • To identify innovative approaches that are likely to be accepted by the Regulatory Authorities and the scientific community.
  • To achieve a scientific consensus on: the role of PK/PD modeling in paediatric drug development to be proposed at the regulatory level, the use of simulation to reduce the number of children enrolled in trials and the number of samples for lab tests, and how the extrapolation methodology (bridging approach) can be used in regulatory procedures. 
  • To evaluate new approaches to clinical drug development based on innovative approaches.

 

LATEST RESULTS

 

Please see the latest results delivered by the GRiP Network under Work Package 4. Where possible, abstracts of the reports have been provided on this page. To obtain more detailed information, you can download the available materials by clicking on the links at the bottom of the page (only public documents will be made available).

 

 

• Tool for the preparation of a sample size calculation in paediatric randomized clinical trials with a superiority design

 

This tool has been developed to educate practitioners on how to calculate sample size in paediatric clinical trials.

Click here to access the tool - Sample size calculation in paediatric randomized clinical trials 

 

 

• Completed deliverables so far

 

WP4 – New Methods For Clinical Studies In Paediatrics          

 

First Report on the activities of the Expert Groups                                                                                                                          

 

Second report on the activities of the Expert Groups

 

Recommendations for an adequate sample size definition

 

Recommendations for modelling and simulation

Recommendations for an appropriate comparator definition

 

List of principles to be considered when evaluating extrapolation approach from adults data

 

Recommendations fro innovative design to be adopted in an agreed PIP

 

Development of training modules to be included in the GRiP training programme palette

 

GRiP Position paper on CTs

 

Models, tools and strategies for innovative approaches on CTs 

 

The work carried out within the WP4 has led to the development of tools and strategies to guide researchers and investigators in properly planning a paediatric clinical trial, focusing on key issues such as sample size calculation, choice of study design and comparator. In order to show how innovative methodologies can be applied in practice in real-life clinical trials, this document describes three examples of case studies supporting the developmental plans for the use of lamotrigine, deferiprone and gabapentin in still uncovered paediatric subsets.

 

Final report on the activities of the Expert Groups

 

GRiP WP4 aims at contributing to the improvement of paediatric research by covering methodological gaps not already ruled within reference documents, and in particular by providing scientifically proven indications on how and when applying innovative approaches to trial designs even considering simulation approaches, on the role of PK/PD modelling and extrapolation in avoiding trials or in optimising the way of proving scientific evidence.

To attain these objectives, Partners involved in this workpackage have set up a WP4 Managing Group and an Ad hoc Experts Group (EAG) whose activities will focus on the areas of modelling and simulation, innovative design, data extrapolation, sample size and comparators, each having a coordinator within the EAG.

This Deliverable summarises the main results obtained by the group as made available through ad hoc deliverables, tools and publications.