Department of Medical Sciences, Clinical Diabetes and Metabolism, Uppsala University, Uppsala, Sweden.
Department of Medicine, Sahlgrenska University Hospital, Gothenburg, Sweden.
Diabetologia. 2022 Oct;65(10):1575-1586. doi: 10.1007/s00125-022-05762-x. Epub 2022 Jul 29.
This narrative review describes a new approach to navigation in a challenging landscape of clinical drug development in diabetes. Successful outcome studies in recent years have led to new indications and guidelines in type 2 diabetes, yet the number of clinical trials in diabetes is now declining. This is due to many environmental factors acting in concert, including the prioritisation of funding for other diseases, high costs of large randomised clinical trials, increase in regulatory requirements and limited entry of novel candidate drugs. There is a need for novel and cost-effective paradigms of clinical development to meet these and other challenges. The concept of registry-based randomised clinical trials (RRCTs) is an attractive option. In this review we focus on type 2 diabetes and the prevention of cardiovascular and microvascular comorbidities and mortality, using the Swedish SMARTEST trial as an example of an RRCT. We also give some examples from other disease areas. The RRCT concept is a novel, cost-effective and scientifically sound approach for conducting large-scale diabetes trials in a real-world setting.
这篇叙述性评论描述了一种在糖尿病临床药物开发充满挑战的领域中进行导航的新方法。近年来成功的结局研究导致了 2 型糖尿病的新适应症和新指南，但糖尿病临床试验的数量现在正在减少。这是由于许多环境因素协同作用的结果，包括为其他疾病优先提供资金、大型随机临床试验的高成本、监管要求的增加以及新型候选药物的进入受限。需要新的、具有成本效益的临床开发模式来应对这些和其他挑战。基于登记的随机临床试验（RRCT）的概念是一个有吸引力的选择。在这篇综述中，我们以瑞典 SMARTEST 试验为例，重点关注 2 型糖尿病及其对心血管和微血管合并症及死亡率的预防，并介绍了来自其他疾病领域的一些例子。RRCT 概念是一种新颖、具有成本效益和科学合理的方法，可在真实环境中进行大规模的糖尿病试验。
