Phase 2 Design Considerations from a Program Perspective

Tobias Mielke

doi:10.1080/19466315.2026.2621850

2026-02-01

PRE

Tobias Mielke *

Fredrik Öhrn

Vladimir Dragalin

DOI：10.1080/19466315.2026.2621850

原文链接

摘要

En 中文

新药开发中第二阶段试验的一个关键目标是降低对昂贵第三阶段试验的投资风险。这种风险降低带来了额外的成本、延长了药物开发周期，以及基于偶然发现而中断真正有效的干预措施的可能性。因此，第二阶段样本量和决策规则的选择对开发项目的价值具有重大影响。然而，第二阶段试验设计通常仅基于对I型错误和效能的定性讨论，而没有直接讨论财务影响。本文介绍了一个模型，该模型允许快速评估预期净现值（eNPV），并优化第二阶段样本量和决策规则。在增加有限假设的情况下，该模型生成了有助于理解项目设计操作特征的见解。我们讨论了仅由eNPV优化驱动的设计的局限性，并提出了使用约束优化来优化候选第二阶段研究设计的替代方法。

Keyword:

Assurance

eNPV

Go/No-Go

Group-sequential designs

Phase 2 designs

期刊

Statistics in Biopharmaceutical Research

IF:

1.3

论文数:

被引数:

机构

johnson & johnson germany

学者数:

论文数: 3

被引数: 0

Johnson & Johnson

学者数:

197

论文数: 54

被引数: 0

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