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Applied Drug Research, Clinical Trials and Regulatory Affairs

Editor-in-Chief

ISSN (Print): 2667-3371
ISSN (Online): 2667-338X

Review Article

Combating Childhood Cancer: Paediatric Patients Living with Neuroblastoma - Regulatory Ramifications and Roadblocks

Author(s): Manfred Kurz*

Volume 9, Issue 1, 2022

Published on: 27 December, 2022

Article ID: e071222211726 Pages: 9

DOI: 10.2174/2667337109666221207154414

Price: $65

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Abstract

This paper relates to Neuroblastoma (NBL), a rare, solid cancer affecting children and aims to describe regulatory obligations to adhere to during development, marketing authorisation application (MAA) and post-authorisation stage. The focus is on European Union (EU) paediatric legislation, although essential US Food and Drug Administration (FDA) elements are briefly outlined. Practical regulatory aspects and reporting requirements, players in the therapeutic area as well as clinical management are described. The feasibility and limitations of future harmonised clinical development are pointed out.

Keywords: Oncology, children, neuroblastoma, PDCO, SIOPEN, COG, COJEC, GPOH, compliance check, PIP, iPSP, EMA, FDA, orphan drug, induction chemotherapy, immunotherapy, paediatrics, RfM.

Graphical Abstract
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