NIH TRND program: successes in preclinical therapeutic development.
NIH TRND program: successes in preclinical therapeutic development.
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NIH TRND 计划：临床前治疗开发的成功。
DOI：
10.1016/j.tips.2012.10.001
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发表时间：
2013
影响因子：
13.8
通讯作者：
Pilon,AndreM
中科院分区：
文献类型：
--
作者：
McKew,JohnC;Pilon,AndreM
罕见和被忽视疾病的治疗（TRND）计划（参见：http：//ncats.org/ncats.html）。nih。gov/trnd. html）寻求鼓励和加速对生物制药工业具有有限商业利益的疾病的新治疗的开发。面对有限的财政资源和开发过程中固有的失败风险，行业必须就治疗商业化做出战略决策[1，2]。通常情况下，“罕见”的低流行率的疾病，如定义的孤儿药法，修订[3，4]，以及高流行率的“被忽视”的疾病（见：http：//www. who.主要发生在发展中国家贫困人口中的疾病没有得到解决。TRND是美国国立卫生研究院（NIH）国家推进转化科学中心（NCATS）的一部分，代表了NIH致力于通过基础生物医学研究和再造转化科学来改善健康的重要方面[5]。TRND通过刺激NIH内部科学家和校外学术研究人员，非营利组织以及积极开发罕见或被忽视疾病（RND）适应症治疗候选药物的制药和生物技术公司之间的合作，为这一努力做出了贡献。
The Therapeutics for Rare and Neglected D iseases (TRND) program (see: http://ncats. nih. gov/trnd. html) seeks to encourage and speed the development of new treatments for diseases of limited commercial interest to the biopharmaceutical industry. Faced with constrained financial resources and the inherent risk of failure during development, industry must make strategic decisions regarding therapeutic commercialization [1, 2]. Often,“rare” diseases of low prevalence, as defined in the Orphan Drug Act, as amended [3, 4], as well as high-prevalence “neglected” diseases (see: http://www. who. int/neglected_diseases/diseases/en/) occurring chiefly among impoverished populations in the developing world go unaddressed. Part of the National Center for Advancing Translational Sciences (NCATS) at the US National Institutes of Health (NIH), TRND represents an important aspect of the NIH commitment to improving health through basic biomedical research and reengineering translational science [5]. TRND contributes to this effort by stimulating collaborations among intramural NIH scientists and extramural academic researchers, nonprofit organizations, and pharmaceutical and biotechnology companies actively developing therapeutic candidates for rare or neglected disease (RND) indications.