Are better endpoints and better design of clinical trials needed?
Are better endpoints and better design of clinical trials needed?
复制标题
DOI：
10.1016/j.berh.2003.09.011
复制
发表时间：
2004-02-01
影响因子：
5.2
通讯作者：
van Riel, PLCM
中科院分区：
文献类型：
--
作者：
Fransen, J;van Riel, PLCM
对于临床医生来说，重要的是要知道一种新药是否有效，以及这种新药对其他药物的作用如何。然而，新药通常在安慰剂对照试验中进行测试，而没有积极的比较。新药通常在疾病活动水平高的人群中进行测试。然而，临床医生也可能为持续中度疾病活动的患者寻求最佳治疗方法，答案可能来自临床试验，比较两种有效药物对中度疾病活动患者的治疗效果。然而，一个主要的结果是，需要试验终点，可以检测到小的，但相关的疗效差异。风湿性疾病试验的理想终点可能是持续跟踪疾病过程中短期变化的标志物。该标志物的水平应与长期结果密切相关，从而预测未来。当它可以被真实和可行地测量时，标记物将在试验中用作终点，并用于支持临床实践中的治疗决策。
For the clinician it is important to know whether a new drug works, and how the new drug performs against other drugs. However, new drugs are typically tested in placebo-controlled trials without active comparison. New drugs are often tested in a population with high levels of disease activity. Clinicians, however, may also seek the optimal treatment for patients with persistent moderate levels of disease activity.An answer may come from clinical trials that compare two effective drugs in patients with moderate disease activity. A main consequence, however, is that trial endpoints are needed that can detect small but relevant differences in efficacy. An ideal endpoint for trials in rheumatic diseases may be a marker that continually follows short-term changes in the disease process. The levels of the marker should be strongly associated with long-term outcome, thus prognosticating the future. When it can be measured truly and feasible, the marker would be useful as an endpoint in trials and for supporting treatment decisions in clinical practice.