平台试验时代的随机化:未经探索的挑战和一些潜在的解决方案
Olga Kuznetsova1, Jennifer Ross2, Daniel Bodden3
1Merck & Co., Inc., Rahway, NJ, USA.
BMC medical research methodology
|November 22, 2025
概括
平台试验提供了适应性的好处,但面临随机化挑战. 本文探讨了复杂的分配比率和独特的试验特征的解决方案,强调了交互式响应技术系统设计的成功执行.
科学领域:
- 临床试验方法论 临床试验方法论
- 生物统计学 生物统计学
- 适应性试验设计
背景情况:
- 平台试验允许同时调查与共享控制对应的多种干预措施,并动态添加/删除治疗臂.
- 不平等的分配比率经常用于平台试验,以提高统计效率,参与者利益和入学控制.
- 适应性特征,如响应适应性随机化,可能需要改变分配比率,即使治疗臂数量恒定.
研究的目的:
- 解决平台试验设计和执行中固有的中央随机化挑战.
- 探索管理复杂的分配比率和特定平台试验特征的潜在解决方案.
- 突出交互响应技术 (IRT) 系统设计在成功随机化中的关键作用.
主要方法:
- 对适用于平台试验的随机化方法的审查,包括适用于不平等分配比率的方法.
- 讨论特殊的随机化技术,这些技术对于差异性资格,部分失明,动态平衡和有限的药物供应等特征是必要的.
- 强调主协议层面的交互响应技术 (IRT) 系统的设计,以纳入各种随机化需求.
主要成果:
- 保持接近目标比率的分配比例的随机化方法对于设计效率和预先确定的比率至关重要.
- 特定的平台试验特征需要定制或新的随机化方法,包括多因素动态随机化和部分盲目的两步随机化.
- 保持每次分配的无条件分配比率对于防止选择偏差,意外偏差和保持统计能力至关重要.
结论:
- 有效地管理随机化是实现平台试验的好处的关键.
- 交互响应技术 (IRT) 系统的专家设计对于成功实施复杂的随机化策略至关重要.
- 仔细考虑分配比率的保存是必要的,以避免偏见,并确保试验结果的完整性.
相关概念视频
01:13Randomized Experiments
8.8K
The randomization process involves assigning study participants randomly to experimental or control groups based on their probability of being equally assigned. Randomization is meant to eliminate selection bias and balance known and unknown confounding factors so that the control group is similar to the treatment group as much as possible. A computer program and a random number generator can be used to assign participants to groups in a way that minimizes bias.
Simple randomization
Simple...
Simple randomization
Simple...
8.8K
01:12Trial and Error and Algorithm
371
A problem-solving strategy is a plan of action used to find a solution. Different strategies have distinct action plans. Trial and error involves trying different solutions until one works. For instance, to fix a broken printer, you might check ink levels, ensure the paper tray isn't jammed, and verify the printer's connection to your laptop. This method can be time-consuming but is commonly used. Thomas Edison, for example, used trial and error to find a suitable filament for the light...
371
01:11Blinding
3.8K
Blinding is a commonly used method of not telling participants which treatment a subject is receiving. Blinding is a critical part of a randomized control trial or RCT. It reduces the bias that affects the results. In an RCT, blinding is used in the form of a placebo. A placebo effect occurs when untreated subjects falsely believe they have received the treatment and report improved symptoms. A placebo or a dummy treatment is administered to subjects to negate the bias caused by such an effect.
3.8K
01:15Bioequivalence Experimental Study Designs: Repeated Measures, Cross-Over, Carry-Over, and Latin Square Designs
166
Body:Bioequivalence experimental study designs play a pivotal role in testing the effectiveness of various treatments. Key among these are the repeated measures, cross-over, carry-over, and Latin square designs. In the repeated measures design, each subject receives all treatments, allowing for temporal comparisons. This type of design is useful in reducing variability but requires careful planning to avoid bias.The cross-over design, an economical method, involves sequential administration of...
166
01:20Random and Systematic Errors
14.3K
Scientists always try their best to record measurements with the utmost accuracy and precision. However, sometimes errors do occur. These errors can be random or systematic. Random errors are observed due to the inconsistency or fluctuation in the measurement process, or variations in the quantity itself that is being measured. Such errors fluctuate from being greater than or less than the true value in repeated measurements. Consider a scientist measuring the length of an earthworm using a...
14.3K
02:01Group Design
10.1K
The most basic experimental design involves two groups: the experimental group and the control group. The two groups are designed to be the same except for one difference— experimental manipulation. The experimental group gets the experimental manipulation—that is, the treatment or variable being tested—and the control group does not. Since experimental manipulation is the only difference between the experimental and control groups, we can be sure that any differences between...
10.1K