Corbacioglu S
Department of Paediatrics, University Children's Hospital, Ulm, Germany.
Bone Marrow Transplant. 2008 Jun;41 Suppl 2:S30-4. doi: 10.1038/bmt.2008.51.
In contrast to adults, 50% or more of medicines used in children have never been actually studied in the paediatric population in the European Union community (EU). Under the impression that compliance with good clinical practice (GCP) requirements will lead to an improved quality of clinical trials, the ratification of the EU Directive 2001/20/EG now imposes the same GCP regulations demanded for commercial clinical trials on non-commercial trials or so-called investigator-initiated trials (IITs). Although it is desirable that all clinical trials comply with ICH-GCP, ensuring that an IIT conforms creates a significant burden for the principal investigator, turning an IIT into a substantial logistic, administrative and financial enterprise. This can only be achieved with a multidisciplinary approach, including physicians, statisticians, data managers, administrators and others. In particular, 'treatment optimization studies'--the most important clinical trials in paediatric oncology--are affected by this new law, potentially resulting in significant delays in the implementation of new and innovative treatment strategies in the paediatric population. This significant drawback was not foreseen but is now recognized and lead to measures to improve the situation for both non-commercial and paediatric clinical trials. Draft guidance on 'specific modalities for non-commercial trials', posted for comment last October, attempts to redress some of the research-crippling problems caused by the initial legislation; however, major problems remain. The EU regulation (EC) no. 1901/2006 'on medicinal products for paediatric use' was enacted in January 2007. This new regulation is a promising step in the right direction, as it will facilitate the development and accessibility of medicinal products specifically for use in children. To adapt to and benefit from this new situation and encourage IIT, a coordinated approach of high expertise is necessary to support and guide the novice in the field of IIT to successfully launch, conduct and complete clinical trials especially in children.
与成人用药情况不同的是，欧盟儿童使用的药品中有50%或更多从未在欧盟儿科人群中进行过实际研究。在认为遵守良好临床实践（GCP）要求将提高临床试验质量的印象下，欧盟第2001/20/EG号指令的批准现在将商业临床试验所需的相同GCP法规强加于非商业试验或所谓的研究者发起的试验（IIT）。虽然所有临床试验都应符合国际人用药品注册技术协调会（ICH）-GCP是理想的，但确保IIT符合要求给主要研究者带来了巨大负担，使IIT变成了一个庞大的后勤、行政和金融企业。这只能通过多学科方法来实现，包括医生、统计学家、数据管理人员、行政人员等。特别是“治疗优化研究”——儿科肿瘤学中最重要的临床试验——受到这项新法律的影响，可能导致儿科人群新的和创新治疗策略的实施出现显著延迟。这一重大缺陷未曾预料到，但现在已得到认识，并促使采取措施改善非商业和儿科临床试验的状况。去年10月发布供征求意见的“非商业试验特定模式”指南草案试图纠正最初立法造成的一些阻碍研究的问题；然而，主要问题仍然存在。欧盟第1901/2006号“儿科用药品”法规于2007年1月颁布。这项新法规是朝着正确方向迈出的有希望的一步，因为它将促进专门用于儿童的药品的开发和可及性。为了适应并从这种新情况中受益，鼓励开展IIT，需要一种高度专业的协调方法来支持和指导IIT领域的新手成功开展、进行和完成临床试验，尤其是针对儿童的临床试验。
