INSERM, Centre d'Investigation Clinique - 1433 and Unité 1116, CHU Nancy, Université de Lorraine and F-CRIN INI-CRCT, 54500 Nancy, France
Campbell University College of Pharmacy and Health Sciences, Buies Creek, NC, USA.
Eur Heart J Cardiovasc Pharmacother. 2016 Jul;2(3):200-5. doi: 10.1093/ehjcvp/pvw007. Epub 2016 Apr 3.
The Food and Drug Administration issued guidance for evaluating the cardiovascular risk of new diabetes mellitus drugs in 2008. Accumulating evidence from several completed trials conducted within this framework raises questions as to whether requiring safety outcome studies for all new diabetes mellitus therapies remains justified. Given the burden of cardiovascular disease in patients with diabetes, the focus should shift towards cardiovascular outcome studies designed to evaluate efficacy (i.e. to determine the efficacy of a drug over placebo or standard care) rather than demonstrating that risk is not increased by a pre-specified safety margin. All stakeholders are responsible for ensuring that new drug approvals occur under conditions of appropriate safety and effectiveness. It is also a shared responsibility to avoid unnecessary hurdles that may compromise access to useful drugs and threaten the sustainability of health systems. It is critical to renew this debate so that stakeholders can collectively determine the optimal approach for developing new drugs to treat type 2 diabetes mellitus.
美国食品和药物管理局于 2008 年发布了评估新型糖尿病药物心血管风险的指导意见。在此框架内进行的几项已完成试验积累的证据引发了质疑，即是否仍然有理由要求所有新型糖尿病治疗药物都进行安全性结局研究。考虑到糖尿病患者心血管疾病的负担，重点应转向旨在评估疗效的心血管结局研究（即确定药物相对于安慰剂或标准治疗的疗效），而不是证明风险未超过预定的安全裕度。所有利益相关者都有责任确保新药批准在适当的安全性和有效性条件下进行。避免可能会影响到有用药物的可及性并威胁卫生系统可持续性的不必要障碍也是一项共同的责任。重新展开这场辩论至关重要，以便利益相关者能够共同确定开发治疗 2 型糖尿病新药的最佳方法。
