The time from identifying a drug target to a new drug approval is often measured in decades and can take even longer for therapies to treat rare diseases. In fact, 95% of rare diseases do not have a specific therapy approved at all. Coordinated efforts to augment the drug development pipeline along with long-term and comprehensive support that enable scientific breakthroughs for rare diseases are possible, but it requires integration across multiple stakeholders. This article analyzes the coordinated funding efforts of four federal and philanthropic organizations to advance drug development for neurofibromatosis type 1-associated tumors and discusses how these organizations have been collaborating and evolved practices to optimize funding and research support.

中文翻译：

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资助社区合作开发针对 1 型神经纤维瘤病的有效疗法。


从确定药物靶点到新药批准的时间通常需要数十年，而治疗罕见疾病的疗法可能需要更长的时间。事实上，95% 的罕见病根本没有获得批准的具体疗法。协调一致地努力扩大药物开发渠道以及长期和全面的支持，以实现罕见疾病的科学突破是可能的，但这需要多个利益相关者的整合。本文分析了四个联邦和慈善组织为推进 1 型神经纤维瘤病相关肿瘤的药物开发而进行的协调资助工作，并讨论了这些组织如何通过合作和发展实践来优化资助和研究支持。