Work package leader:

Gilles Vassal (ITCC) and Silvia Chioato (PFZ)
Co-leads: Birgit Geoerger (IGR), Olaf Witt (DKFZ), Richard Vart (LILLY) and Louise Hayes (ROCHE)

Objectives of the work package:

Regulatory consensus on disease models and POC data package for PIP requirements

Description of the tasks:

ITCC-P4 aims to generate a science-based regulatory consensus endorsed by the EMA and PDCO on the biological and preclinical information required to support a pediatric investigation plan for an oncology medicinal product. This will eventually support the development of an EMA guideline.
ITCC-P4 will take advantage of the unique combination of distinct and synergistic expertises of its academic, CRO and EFPIA partners with preclinical proof-of-concept (POC) testing in cancer models and with Health Authority interactions and Parent’s organisations to establish the science-based regulatory consensus on POC data-packages and minimally-required datasets.
The project and then the platform, will generate biological and preclinical data for oncology medicinal products that are considered for pediatric development. This information along with the evaluation of the therapeutic needs of the young population will contribute to the rationale for a pediatric investigation plan. In addition, the PDX platform might generate comparative preclinical data for compounds targeting the same molecular pathway or share the same or similar mechanism of action. This could facilitate the identification of  the potentially best and more relevant oncology medicinal products.
Data from the platform could be used in pediatric drug development strategy forums to discuss how best to develop innovative compounds for pediatric malignancies.

WP4 will thus contribute to build the regulatory basis for the process of identifying drugs more likely to be effective in pediatric malignancies and which should be developed in children. It aims to provide the regulatory consensus guideline that will improve and streamline the efficiency of drug development for children with cancer.