Rose Klaus, Neubauer David, Grant-Kels Jane M
Pediatric Drug Development & More, klausrose Consulting, Riehen, Switzerland.
Department of Child, Adolescent & Developmental Neurology, University Childrens' Hospital, Ljubljana, Slovenia.
Curr Ther Res Clin Exp. 2019 Jan 18;90:86-91. doi: 10.1016/j.curtheres.2019.01.002. eCollection 2019.
US and EU pediatric laws promote industry-sponsored pediatric studies, based on the therapeutic orphans concept that claims discrimination of children in drug treatment and drug development.
We investigated the medical validity of international pediatric studies with centers in Slovenia, an EU member state, and challenge their medical utility.
We analyzed international industry-sponsored pediatric studies with centers in Slovenia, listed in www.ClinicalTrials.gov, for their medical value.
Most pediatric studies triggered by the US Food and Drug Administration and by the European Medicines Agency were/are without medical or scientific value. They were/are formally and regulatorily justified, but lack medical sense and thus were/are unethical. Several even harm children and/or adolescents with serious diseases by exposing them to placebo or substandard treatment.
Pediatric studies triggered by US and EU regulatory demands are a serious abuse of nonneonatal children and adolescents in Slovenia and worldwide. They are medically redundant at best and often deter patients from effective innovative personalized therapy. They also exclude young patients from reasonable studies. Institutional review boards/ethics committees should be alerted, should critically review all ongoing pediatric studies, should suspend those found to be questionable, and should reject newly submitted questionable ones.
美国和欧盟的儿科法律基于治疗孤儿概念推动行业资助的儿科研究，该概念声称儿童在药物治疗和药物研发中受到歧视。
我们调查了在欧盟成员国斯洛文尼亚设有中心的国际儿科研究的医学有效性，并对其医学实用性提出质疑。
我们分析了在www.ClinicalTrials.gov上列出的、在斯洛文尼亚设有中心的国际行业资助的儿科研究的医学价值。
美国食品药品监督管理局和欧洲药品管理局发起的大多数儿科研究没有医学或科学价值。它们在形式上和监管上有正当理由，但缺乏医学意义，因此是不道德的。有几项研究甚至通过让患有严重疾病的儿童和/或青少年接受安慰剂或不合格治疗而对他们造成伤害。
美国和欧盟监管要求引发的儿科研究严重滥用了斯洛文尼亚乃至全球的非新生儿儿童和青少年。这些研究充其量在医学上是多余的，而且常常阻碍患者接受有效的创新个性化治疗。它们还将年轻患者排除在合理的研究之外。机构审查委员会/伦理委员会应提高警惕，应严格审查所有正在进行的儿科研究，应暂停那些被发现有问题的研究，并应拒绝新提交的有问题的研究。
