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Rethinking the nonprofit foundation: an emerging niche in the rare disease ecosystem

机译：重新思考非营利基金会：稀有疾病生态系统中的新兴利基市场

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In recent years, medical foundations have become increasingly influential, and now play an instrumental and integral role in the research and development of their disease area of interest. While some foundations have directly invested in taking drug candidates to the clinic, others have focused on creating specific tools for accelerating the identification and development of effective treatments. Here, we describe a new model, developed by the Children's Tumor Foundation (CTF), by which foundations may play a role in the rare disease ecosystem. On the one hand, the CTF uses its position to build bridges between academic scientists, biotech and pharmaceutical companies, and patients, to accelerate the development of treatments that really matter most to patients. On the other hand, it acts as a niche investor to fund an integrated platform for critical R&D endeavors—including elements such as a patient registry, biobank, and open data platforms—which smoothen the transition from basic discovery to clinical benefit. Currently, the Children's Tumor Foundation is launching a call to top finance experts to collaborate on building an innovative model that will guarantee long‐term sustainability of this integrated platform (Fig ).>Overview of the initiatives of the Children's Tumor Foundation for accelerating drug discovery and development (R&D) 
机译：近年来，医学基金会越来越有影响力，现在在其感兴趣的疾病领域的研究和开发中发挥着工具性和不可或缺的作用。虽然一些基金会直接投资于将候选药物带到诊所，但其他基金会则专注于创建特定工具以加速识别和开发有效疗法。在这里，我们描述了儿童肿瘤基金会（CTF）开发的一种新模型，该基金会可能在稀有疾病生态系统中发挥作用。一方面，CTF利用其地位在学术科学家，生物技术和制药公司以及患者之间架起桥梁，以加速开发对患者真正重要的治疗方法。另一方面，它作为利基投资者，为关键的R＆D努力（包括诸如患者登记，生物库和开放数据平台等要素）的集成平台提供资金，从而平滑了从基本发现到临床收益的过渡。目前，儿童肿瘤基金会正呼吁顶级金融专家合作建立一个创新模型，以保证该集成平台的长期可持续性（图）。<！-fig ft0-> <！-fig mode = article f1-> <！-caption a7-> <！-caption a8-> >儿童肿瘤基金会为加速药物发现和开发（R＆D）计划的概述

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