Can I read Performing Clinical Drug Trials in Children with a Rare Disease on EtoBox?

Performing Clinical Drug Trials in Children with a Rare Disease by Hedley, Victoria; Leary, Rebecca; Sen, Anando; Irvin, Anna; Heslop, Emma; Straub, Volker is a scholarly article available to read on EtoBox.

What is Performing Clinical Drug Trials in Children with a Rare Disease about?

Over the past 50 years, the advancements in medical and health research have radically changed the epidemiology of health conditions in neonates, children, and adolescents; and clinical research has on the whole, moved forward. However, large sections of the pediatric community remain vulnerable and underserved, by clinical research. One reason for this is the fact that most pediatric diseases are also rare diseases (i.e., they fit the EU definition of a rare condition, by affecting no more than 5 in 10,000 individuals), and indeed the majority of conditions under this umbrella heading are in fact much rarer, affecting fewer than 1 in 100,000. Rare pediatric diseases incur particular challenges, both in terms of actually conducting clinical trials but also planning trials (and indeed, stimulating the preclinical research and knowledge generation necessary to embark on clinical trials in the first place). The pediatric regulation and orphan regulation (covering rare diseases) were introduced to address the complexities in research and development of medicines specifically for children and for people living with a rare disease, respectively. The regulations have been reasonably effec

Author

Hedley, Victoria; Leary, Rebecca; Sen, Anando; Irvin, Anna; Heslop, Emma; Straub, Volker

Published

2024

Language

EN