Green Dionna J, Park Kyunghun, Bhatt-Mehta Varsha, Snyder Donna, Burckart Gilbert J
Office of Pediatric Therapeutics, Office of the Commissioner, US Food and Drug Administration, Silver Spring, MD, United States.
Office of Clinical Pharmacology, Center for Drug Evaluation and Research, US Food and Drug Administration, Silver Spring, MD, United States.
Front Pediatr. 2021 Jul 26;9:698611. doi: 10.3389/fped.2021.698611. eCollection 2021.
The regulatory framework for considering the fetal effects of new drugs is limited. This is partially due to the fact that pediatric regulations (21 CFR subpart D) do not apply to the fetus, and only US Health and Human Service (HHS) regulations apply to the fetus. The HHS regulation 45 CFR Part 46 Subpart B limits research approvable by an institutional review board to research where the risk to the fetus is minimal unless the research holds out the prospect of a direct benefit to the fetus or the pregnant woman (45 CFR 46.204). Research that does not meet these requirements, but presents an opportunity to understand, prevent, or alleviate a serious problem affecting the health of pregnant women, fetuses, or neonates, may be permitted by the Secretary of the HHS after expert panel consultation and opportunity for public review and comment (45 CFR 46.407). If the product is regulated by the US Food and Drug Administration (FDA), FDA may get involved in the review process. The FDA does however have a Reviewer Guidance on Evaluating the Risks of Drug Exposure in Human Pregnancies from 2005 and this guidance does discuss the intensity of drug exposure. Estimation of that exposure using physiologically based pharmacokinetic (PBPK) modeling has been suggested by some investigators. Given that drug exposure during pregnancy will impact the fetus, a number of new guidances in the last 2 years also address inclusion of pregnant women in clinical drug trials. Therefore, the drug-specific information on fetal pharmacology will increase dramatically in the next decade due to interest in drugs administered in pregnancy and with the assistance of model-informed drug development.
考虑新药对胎儿影响的监管框架有限。部分原因在于儿科法规(《联邦法规汇编》第21编D子部分)不适用于胎儿,仅有美国卫生与公众服务部(HHS)的法规适用于胎儿。HHS法规《联邦法规汇编》第45编第46部分B子部分将机构审查委员会可批准的研究限制为对胎儿风险最小的研究,除非该研究有望给胎儿或孕妇带来直接益处(《联邦法规汇编》第45编第46.204条)。不符合这些要求,但有机会了解、预防或缓解影响孕妇、胎儿或新生儿健康的严重问题的研究,经专家小组咨询并给予公众审查和发表意见的机会后,HHS部长可能会批准(《联邦法规汇编》第45编第46.407条)。如果该产品受美国食品药品监督管理局(FDA)监管,FDA可能会参与审查过程。不过,FDA确实有一份2005年的《评估人类孕期药物暴露风险的审评员指南》,该指南确实讨论了药物暴露的强度。一些研究人员建议使用基于生理的药代动力学(PBPK)模型来估计这种暴露。鉴于孕期药物暴露会影响胎儿,过去两年的一些新指南也涉及将孕妇纳入临床药物试验。因此,由于对孕期用药的关注以及模型指导药物研发的助力,未来十年关于胎儿药理学的特定药物信息将大幅增加。