Paediatric drug development and evaluation: Existing challenges and recommendations

Kaguelidou, Florentia; Ouèdraogo, Maria; Tréluyer, Jean‐Marc; Jeunne, Claire Le; Annereau, Maxime; Blanc, P.; Bureau, Serge; Ducassou, Stéphane; Fiquet, Béatrice; Flamein, Florence; Gaillard, Ségolène; Hankard, R.; Laugel, Vincent; Laurent, Corinne; Lévy, Corinne; Marquet, Thierry; Polak, Michel; Portefaix, Aurélie; Vassal, Gilles

doi:10.1016/j.therap.2022.11.010

Cited by 2 publications

(3 citation statements)

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“…Until more data are provided, clinical decision-making should be guided by the best available scientific evidence and closely monitored while authorities, academics, and manufacturers collaborate to foster trials and authorize adequate drugs for this population. Therefore, we agree with Yamashiro et al [ 165 ], van der Zanden et al, [ 150 ], and Kaguelidou et al [ 164 ] that the most important thing is the compliance with the off-label prescription algorithm and the creation of a professional network of off-label prescriptions and their implications so that healthcare providers have a foundation to stand on when deciding whether to prescribe an off-label drug or not.…”

Section: Discussionsupporting

confidence: 89%

“…Hence, off-label use could be associated with increased safety concerns, and under or overdosing due to insufficient or unclear information for use in children [ 92 , 162 , 163 ]. Despite various international regulatory initiatives and achievements over the past 20 years, many challenges remain in the development and evaluation of the safety and efficacy of pediatric medicines [ 164 ]. The review showed the complexity of the matter.…”

Section: Discussionmentioning

confidence: 99%

“…The problem of pediatric clinical trials existed for many years. Although there were great achievements in the last two decades, clinical trials in children will always lag behind those in adults because of the enormous diversity and specificity of this category of patients [ 164 ]. Off-label use in pediatrics is ubiquitous and primarily based on physicians’ experience, collaboration, knowledge, and existing published scientific literature.…”

Section: Discussionmentioning

confidence: 99%

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Off-Label Prescribing in Pediatric Population—Literature Review for 2012–2022

Petkova,

Georgieva,

Dimitrov

et al. 2023

Pharmaceutics

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Off-label prescribing is widespread among pediatricians, and it is unlikely that this trend will soon be bound by a uniform legal framework. This is necessitated by the fact that there are four variables: the patient’s health condition, the physician’s experience and knowledge, the legislative measures (laws, directives, guidelines, and recommendations), and finally, the pharmaceutical industry. There is considerable concern worldwide about the use of off-label medicines in children. We may call it an enormous global problem that is much talked about and written about; however, we should not forget that the goal around which everyone should unite is the patient’s life. For healthcare providers, the most important thing will always be the health and preservation of the patient’s life, particularly when it comes to children with life-threatening conditions in neonatal and pediatric intensive care units (NICU and PICU). The study aimed to examine the prevalence of off-label drug use in pediatrics. Literature research was conducted, and we included studies from 2012 to 2022 that evaluated off-label drug prevalence in various pediatric patient populations.

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Section: Discussionsupporting

confidence: 89%