Wan Mandy, Al Hashimi Ali, Batchelor Hannah
National Institute for Health Research, Clinical Research Network: Children, London SE1 7EH, UK; Guy's and St Thomas' NHS Foundation Trust, Evelina London Children's Hospital, London SE1 7EH, UK.
Alder Hey Children's Hospital, Eaton Road, Liverpool, Merseyside L12 2AP, UK.
Int J Pharm. 2016 Sep 25;511(2):1163-8. doi: 10.1016/j.ijpharm.2016.03.059. Epub 2016 Mar 30.
Availability and sourcing of investigational drugs for paediatric clinical trials is known to be a challenge for investigator-led clinical trials. The National Institute of Health Research Clinical Research Network: Children (CRN: Children) provides support for formulations and pharmacy related issues to researchers planning and setting up paediatric clinical trials within England. This paper reviews pharmacy and formulation support provided to a consecutive series of investigator-led clinical studies supported by CRN:Children. Case studies are included to describe some of the unique pharmaceutical challenges encountered. 44 trials were reviewed and a total of 103 products were required to support these clinical trials. UK authorised products were suitable for use for 62 of these 103 products. In the remaining 41 cases, 4 could be sourced as an authorised product within the European Union and the remaining 37 required bespoke manufacture. Bespoke manufacture of an investigational drug or placebo is costly. Typical costs for the initial development and testing of a bespoke investigational drug or placebo were in the range of £30,000-100,000 per product. The estimated cost for 19 out of 45 trials was available; in summary, the costs on a per patient per day of therapy basis ranged from under £1 to almost £600; short studies involving multiple agents are obviously the most expensive. This range is dependent upon the need for bespoke manufacture and also the number of participants within the trial. The arrangements for investigational drug supply can greatly affect the study design, regulatory requirements, trial logistics, as well as the total cost of research. As investigational product related activities are often costly, necessitating months of advance planning, it is imperative that specialist inputs are sought from the very start of the study design and planning process.
对于研究者主导的儿科临床试验而言，获取用于试验的研究药物及其来源是一项挑战。英国国家卫生研究院临床研究网络：儿童（CRN：儿童）为在英格兰计划和开展儿科临床试验的研究人员提供制剂和药学相关问题的支持。本文回顾了由CRN：儿童支持的一系列连续的研究者主导的临床研究中所提供的药学和制剂支持。纳入了案例研究以描述所遇到的一些独特的药学挑战。共审查了44项试验，支持这些临床试验总共需要103种产品。这103种产品中有62种英国授权产品适用。在其余41种情况中，4种可作为欧盟内的授权产品获取，其余37种需要定制生产。研究药物或安慰剂的定制生产成本高昂。定制研究药物或安慰剂的初始开发和测试的典型成本为每种产品30,000 - 100,000英镑。45项试验中有19项的估计成本可用；总体而言，基于每日每位患者的治疗成本范围从不到1英镑到近600英镑不等；涉及多种药物的短期研究显然是最昂贵的。这个范围取决于定制生产的需求以及试验中的参与者数量。研究药物供应安排会极大地影响研究设计、监管要求、试验后勤以及研究总成本。由于与研究产品相关的活动通常成本高昂，需要提前数月进行规划，因此在研究设计和规划过程一开始就寻求专家意见至关重要。
