Designing and conducting clinical trials are extremely difficult in rare diseases. Adapting tissue chips for rare disease therapy development is pivotal in assuring that treatments are available, especially for severe diseases that are difficult to treat. Thus far, the NCATS-led National Institutes of Health (NIH) Tissue Chip program has focused on deploying the technology towards in vitro tools for safety and efficacy assessments of therapeutics. However, exploring the feasibility and best possible approach to expanding this focus towards the development phase of therapeutics is critical to moving the field of CToCs forward and increasing confidence with the use of tissue chips. The working group of stakeholders and experts convened by NCATS and the Drug Information Association (DIA) addresses important questions related to disease setting, test agents, study design, data collection, benefit/risk, and stakeholder engagement-exploring both current and future best use cases and important prerequisites for progress in this area.

中文翻译：

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解决罕见疾病：芯片临床试验。

在罕见疾病中，设计和进行临床试验非常困难。使组织芯片适应罕见疾病治疗的发展对于确保可获得治疗至关重要，特别是对于难以治疗的严重疾病。迄今为止，由NCATS领导的美国国立卫生研究院（NIH）组织芯片计划已将重点放在将技术应用于体外工具上，以评估治疗剂的安全性和有效性。但是，探索将这种关注点扩展到治疗剂开发阶段的可行性和最佳可行方法对于推动CToC领域的发展并提高使用组织芯片的信心至关重要。由NCATS和药品信息协会（DIA）召集的利益相关者和专家工作组解决了与疾病背景相关的重要问题，