免疫系统的靶向在治疗癌症中显示出成功的疗法，通过免疫检查点抑制剂（ICI）或T细胞结合剂（TCE）的发展。由于免疫肿瘤治疗药物改变免疫系统以攻击癌细胞并不直接作用于致癌易感性，因此临床开发过程中应考虑这些化合物的特定特征。在本综述中，我们将讨论相关概念，包括临床前模型限制、特殊药理学边界、临床开发策略（如临床适应症选择、治疗线和基础伴侣）、以及不同阶段药物开发所需终点和预期疗效大小。此外，我们将回顾早期和晚期试验设计的未来方向。我们将讨论已获批药物或目前处于临床开发中的药物，并提供克服这些限制的选择。版权所有©2023年Pandiella, Calvo, Moreno, Amir, Templeton and Ocana.

Targeting of the immune system has shown to be a successful therapeutic approach in cancer, with the development of check point inhibitors (ICI) or T-cell engagers (TCE). As immuno-oncology agents modulate the immune system to attack cancer cells and do not act directly on oncogenic vulnerabilities, specific characteristics of these compounds should be taken in consideration during clinical development. In this review we will discuss relevant concepts including limitations of preclinical models, special pharmacologic boundaries, clinical development strategies such as the selection of clinical indication, line of treatment and backbone partner, as well as the endpoints and expected magnitude of benefit required at different stages of the drug development. In addition, future directions for early and late trial designs will be reviewed. Examples from approved drugs or those currently in clinical development will be discussed and options to overcome these limitations will be provided.Copyright © 2023 Pandiella, Calvo, Moreno, Amir, Templeton and Ocana.