Scholarlink

<p>Although single-arm trials (SATs) can serve as a pivotal study design for evaluating drug efficacy in specific scenarios, they are accompanied by inherent limitations and challenges. This paper explores the challenges of validity, reliability, statistical power, and the use of external control in SATs from a statistical perspective. We reflect on the rationale for employing SATs to support efficacy assessments, and provide recommendations based on their limitations and potential biases. The objective is to offer guidance for the improved design and implementation of SATs in future research.</p>

错误：搜索内容不能为空，请输入英文关键词
错误：关键词超出字数限制，请精简
高级检索

Estimating comparative effectiveness using Single-Arm trials: A challenge in the field of agnosticism

  • 期刊论文

  • 出版模式：

    开放获取

  • 发表日期：

    2025年10月21日

查看全文
  • Shang Cao,
  • Hongwei Kan,
  • Mingliang Lu,
  • Jianhong Pan

摘要

Although single-arm trials (SATs) can serve as a pivotal study design for evaluating drug efficacy in specific scenarios, they are accompanied by inherent limitations and challenges. This paper explores the challenges of validity, reliability, statistical power, and the use of external control in SATs from a statistical perspective. We reflect on the rationale for employing SATs to support efficacy assessments, and provide recommendations based on their limitations and potential biases. The objective is to offer guidance for the improved design and implementation of SATs in future research.