Barrow Paul C, Barbellion Stéphane, Stadler Jeanne
Ricerca Biosciences, Les Oncins, Saint-Germain sur l'Arbresle, France.
Methods Mol Biol. 2011;691:17-35. doi: 10.1007/978-1-60761-849-2_2.
A pediatric assessment is now a required component of every New Drug Application in North America or Marketing Authorization Application in Europe, unless a waiver has been granted previously. Nonclinical juvenile toxicity studies are usually required as part of this assessment. The protocols for juvenile toxicity studies are devised in consultation with the FDA or EMEA. It is important to approach the regulatory authority well in advance in order not to delay the marketing authorization of the drug and to confirm the need or not to perform a preclinical evaluation in juvenile animals. The choice of species and the design of juvenile studies are based on a series of complex considerations, including: the therapeutic use of the drug, the age at which children will be treated, the duration of treatment, and potential age- or species-specific differences in pharmacokinetics or toxicity.
在北美，儿科评估现已成为每份新药申请的必要组成部分；在欧洲，则是上市许可申请的必要组成部分，除非此前已获批豁免。非临床幼年毒性研究通常是该评估的一部分。幼年毒性研究方案是在与美国食品药品监督管理局（FDA）或欧洲药品管理局（EMEA）协商后制定的。提前与监管机构充分沟通很重要，以免延误药物的上市许可，并确认是否需要在幼年动物身上进行临床前评估。物种的选择和幼年研究的设计基于一系列复杂的考量因素，包括：药物的治疗用途、儿童接受治疗的年龄、治疗持续时间，以及药代动力学或毒性方面潜在的年龄或物种特异性差异。
