GSK Vaccines, Rixensart, Belgium.
Biom J. 2021 Oct;63(7):1434-1443. doi: 10.1002/bimj.202100015. Epub 2021 Jul 12.
The assurance of a future clinical trial is a key quantitative tool for decision-making in drug development. It is derived from prior knowledge (Bayesian approach) about the clinical endpoint of interest, typically from previous clinical trials. In this paper, we examine assurance in the specific context of vaccine development, where early development (Phase 2) is often based on immunological endpoints (e.g., antibody levels), while the confirmatory trial (Phase 3) is based on the clinical endpoint (very large sample sizes and long follow-up). Our proposal is to use the Phase 2 vaccine efficacy predicted by the immunological endpoint (using a model estimated from epidemiological studies) as prior information for the calculation of the assurance.
未来临床试验的保证是药物开发决策的关键定量工具。它源自对感兴趣的临床终点（通常来自先前的临床试验）的先验知识（贝叶斯方法）。在本文中，我们在疫苗开发的特定背景下研究了保证，其中早期开发（第 2 阶段）通常基于免疫学终点（例如，抗体水平），而确证试验（第 3 阶段）则基于临床终点（非常大的样本量和长期随访）。我们的建议是使用免疫终点预测的第 2 阶段疫苗效力（使用来自流行病学研究的模型进行估计）作为计算保证的先验信息。
