Imperial College London, Chelsea and Westminster Hospital, London, UK.
Lancet. 2013 Jan 5;381(9860):73-87. doi: 10.1016/S0140-6736(12)61818-2. Epub 2012 Nov 20.
Despite a general acknowledgment that research in children is necessary and ethical, the evidence base for child-specific treatments is still sparse. We investigated children's biomedical and health services research in the UK in relation to training, infrastructure and activity, research evidence, and visibility. We show that excellent opportunities for career researchers exist through a competitive, national integrated academic training programme, but that the number of academic paediatricians has decreased by 18% between 2000 and 2011, falling from 11·3% to 5·9% of the consultant workforce. The potential for rapid delivery of studies in children through the National Health Service (NHS) is not being realised: clinical trainees are poorly equipped with core research skills; most newly appointed consultant paediatricians have little or no research experience; less than 5% of contracted consultant time supports research; less than 2·5% of the 2 million children seen in the NHS every year are recruited to studies; and ten of the 20 UK children's hospitals do not have a clinical research facility. Support through National Institute for Health Research networks is good for studies into drugs, but inconsistent for non-drug research; less than 5% of registered studies involve children and only one children's biomedical research centre has been allocated funding from 2012. Of the UK annual public and charitable biomedical research expenditure of roughly £2·2 billion, about 5% is directed at child health research. The scant evidence base is impeding the development of clinical guidance and policy-less than 20% of the outputs of the National Institute for Health and Clinical Excellence are applicable to children. Paediatric representation on major research boards is weak. Parent and young people's advocacy is fragmented, and their views are insufficiently heeded by regulatory bodies. The strong UK Government commitment to biomedical research has not been translated fully to research for children. The power of research in children to turn the tide of the growing burden of non-communicable, chronic, adult diseases that have their origins in early life, to benefit the health of an ageing population and future generations, and to reduce health-care costs is inadequately recognised. On the basis of our findings, we make several recommendations to improve early-years research, including the formation of multidisciplinary, cross-institutional groups of clinical and non-clinical child health researchers and their access to diagnostic and laboratory facilities suitable for children; a unified Children's Research Network for drug studies and non-drug studies; regulatory assessment of research that is proportionate and based on consistent national criteria; an expansion of research posts; support for parents' and young people's advocacy; collaboration between children's research charities; improved research training for paediatric trainees; and closer integration of child health research with core NHS activities.
尽管人们普遍承认儿童研究是必要且合乎伦理的,但针对儿童特定治疗方法的证据基础仍然匮乏。我们调查了英国儿童的生物医学和健康服务研究,涉及培训、基础设施和活动、研究证据以及可见度。我们表明,通过竞争激烈的全国性综合学术培训计划,为职业研究人员提供了极好的机会,但在 2000 年至 2011 年间,学术儿科医生的数量减少了 18%,从顾问劳动力的 11.3%降至 5.9%。通过国民保健制度(NHS)快速开展研究的潜力尚未得到实现:临床培训生缺乏核心研究技能;大多数新任命的儿科顾问几乎没有或没有研究经验;不到 5%的签约顾问时间支持研究;每年在 NHS 就诊的 200 万儿童中,只有不到 2.5%被招募参加研究;20 家英国儿童医院中,有 10 家没有临床研究设施。国家卫生研究院网络的支持对药物研究很好,但对非药物研究不一致;不到 5%的注册研究涉及儿童,只有一个儿童生物医学研究中心从 2012 年开始获得资金。在英国约 22 亿英镑的年度公共和慈善生物医学研究支出中,约 5%用于儿童健康研究。证据基础匮乏,阻碍了临床指南和政策的制定——国家卫生与临床优化研究所的产出中只有不到 20%适用于儿童。在主要研究委员会中,儿科的代表性很薄弱。家长和年轻人的倡导是分散的,他们的意见没有得到监管机构的充分重视。英国政府对生物医学研究的大力支持并没有完全转化为针对儿童的研究。儿童研究的力量尚未得到充分认识,这种力量可以扭转非传染性、慢性、成人疾病负担不断增加的局面,这些疾病起源于生命早期,有利于人口老龄化和后代的健康,并降低医疗保健成本。基于我们的发现,我们提出了一些建议,以改善早期研究,包括形成多学科、跨机构的临床和非临床儿童健康研究人员团体,并为他们提供适合儿童的诊断和实验室设施;建立一个统一的儿童药物研究和非药物研究网络;根据一致的国家标准,对研究进行相称和基于一致国家标准的监管评估;扩大研究岗位;支持家长和年轻人的倡导;促进儿童研究慈善机构之间的合作;加强对儿科培训生的研究培训;并更紧密地将儿童健康研究与核心国民保健制度活动结合起来。