The FDA reported that most drugs are effective in only 25–62% of patients. Although many drugs require dose individualisation in clinical practice, dose-finding trials usually aim to identify an optimal dose for the patient population. Such a dose would be suboptimal for many patients. Simulations show that individualised dose titration, balancing efficacy against toxicity, can remarkably increase the response rate — doubling it in some situations. Dose titration in a clinical trial can efficiently establish the realistic expectations for the drug’s true utility in a trial setting that reflects clinical practice, as well as generate important knowledge to guide patient care through informative drug labels. This design answers key questions truly relevant to patient care that other designs cannot — will a patient benefit from a given therapy, to what extent and at what dose? Therefore, response-based dose titration should be considered for dose-finding trials, where appropriate, for drugs that will eventually be used this way in the clinic.

FDA报告说，大多数药物仅对25-62％的患者有效。尽管许多药物在临床实践中需要剂量个体化，但剂量寻找试验通常旨在为患者群体确定最佳剂量。对于许多患者来说，这样的剂量将不是最佳的。模拟表明，个性化的剂量滴定可以平衡毒性和毒性，可以显着提高响应速度，在某些情况下可以将响应速度提高一倍。在临床试验中进行剂量滴定可以有效地建立对药物在反映临床实践的试验环境中真正用途的现实期望，并产生重要知识以通过信息性药物标签指导患者护理。此设计回答了与其他设计真正无法解决的与患者护理真正相关的关键问题-患者将从给定的治疗中受益，到什么程度和剂量？因此，在适当的情况下，对于最终将在临床中使用的药物，应在剂量寻找试验中考虑基于反应的剂量滴定。