Centre d'Épidémiologie Clinique, Hôpital Hôtel-Dieu, Paris, France; Team METHODS, INSERM U1153, Paris, France; Faculté de Médecine, Université Paris Descartes, Paris, France.
Centre d'Épidémiologie Clinique, Hôpital Hôtel-Dieu, Paris, France; Team METHODS, INSERM U1153, Paris, France; Department of Epidemiology, Columbia University, Mailman School of Public Health, New York, NY, USA; School of Public Health, Boston University, MA, USA.
J Clin Epidemiol. 2018 Jun;98:123-132. doi: 10.1016/j.jclinepi.2018.01.006. Epub 2018 Jan 31.
Concerns exist as to whether the allocation of resources in clinical research is aligned with public health needs. We evaluated the alignment between the effort of clinical research through the conduct of randomized controlled trials (RCTs) and health needs measured as the burden of diseases for all regions and a broad range of diseases.
We grouped countries into seven regions and diseases into 27 groups. We mapped all RCTs initiated between 2006 and 2015 that were registered at the WHO International Clinical Trials Registry Platform to regions and diseases. The burden of diseases in 2005 was mapped as disability-adjusted life years (DALYs), based on the 2010 Global Burden of Diseases study. Within regions, we defined a research gap when the proportion of RCTs concerning a disease in the region was less than half the relative burden of the disease.
We mapped 117,180 RCTs planning to enroll 42.6 million patients and 2,220 million DALYs. In high- versus non-high-income countries, 130.9 versus 6.9 RCTs per million DALYs were conducted. We did not identify any research gap in high-income countries. We identified research gaps for all other regions. In particular, for Sub-Saharan Africa, we identified research gaps for common infectious diseases (CID) and neonatal disorders (ND): 5.8% (95% uncertainty interval 4.7-6.9) and 2.0% (0.9-4.5) of RCTs in Sub-Saharan Africa concerned CID and ND, although these diseases represented 22.9% and 11.6% of the burden in the region, respectively. For South Asia, we identified research gaps for the same two groups of diseases.
In non-high-income regions, the conduct of RCTs was misaligned with the distribution of major causes of burden, in particular infectious diseases and neonatal disorders in Sub-Saharan Africa and South Asia.
人们对临床研究资源的分配是否与公共卫生需求相一致存在担忧。我们评估了通过开展随机对照试验（RCT）进行临床研究的力度与所有地区和广泛疾病的疾病负担之间的一致性。
我们将国家分为七个地区，将疾病分为 27 组。我们将在 2006 年至 2015 年期间在世界卫生组织国际临床试验注册平台注册的所有启动的 RCT 分配给地区和疾病。2005 年的疾病负担以残疾调整生命年（DALY）表示，基于 2010 年全球疾病负担研究。在各地区内，当一个地区的 RCT 中涉及某一疾病的比例低于该疾病相对负担的一半时，我们就定义存在研究差距。
我们共绘制了 117180 项 RCT 计划，涉及 4260 万名患者和 22.2 亿 DALY。高收入国家与非高收入国家每百万 DALY 进行的 RCT 分别为 130.9 项和 6.9 项。我们没有发现高收入国家存在研究差距。我们发现了所有其他地区的研究差距。特别是在撒哈拉以南非洲，我们发现了常见传染病（CID）和新生儿疾病（ND）的研究差距：撒哈拉以南非洲的 RCT 中涉及 CID 和 ND 的比例分别为 5.8%（95%置信区间为 4.7-6.9）和 2.0%（0.9-4.5），而这两种疾病在该地区的负担分别占 22.9%和 11.6%。对于南亚，我们也发现了这两组疾病的研究差距。
在非高收入地区， RCT 的开展与主要负担原因的分布不一致，特别是撒哈拉以南非洲和南亚的传染病和新生儿疾病。
