FDA's Expedited Approval Mechanisms for New Drug Products
FDA's Expedited Approval Mechanisms for New Drug Products
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DOI：
10.1089/blr.2015.9999
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发表时间：
2015-02-01
影响因子：
0.2
通讯作者：
Kepplinger, Erin E.
中科院分区：
文献类型：
--
作者：
Kepplinger, Erin E.
现代医学和科学在改善和延长生命方面取得了令人难以置信的进步。然而，许多疾病和病症仍然缺乏适当的治疗方法。根据总统科技顾问理事会（PCAST）2012年9月发布的一份关于推进药物发现、开发和评估创新的报告，大约有3000万美国人患有7,000种罕见疾病，但只有350种疗法被批准作为治疗方法。事实上，“96%的孤儿疾病，包括罕见的癌症，缺乏有效的治疗方法。2其他疾病也需要改进治疗方法。例如，“心脏病和中风仍然是死亡的主要原因。”3.继续研究和开发治疗重大疾病的新药和生物制品的公共卫生需求是明确和迫切的。但是，除了这些重要的健康原因刺激新化合物的研究和开发，还有辅助和支持性的经济考虑，以推动创新的研究和开发。根据美国药物研究与制造商协会（PhRMA）的数据，4美国生物制药行业对美国经济做出了巨大贡献。PhRMA报告说，该行业直接雇用了80多万名工人，从事高薪工作和不同领域的工作，并在全国范围内支持了250万个就业岗位。5此外，PhRMA声称它支持超过7890亿美元的经济总产出。然而，几年来，开发新化合物所需的时间和资金增加，潜在产品的失败率以及风险资本投资的减少，以及该行业的其他压力，促使人们担心美国的创新研究可能会萎缩，停止或转移到其他国家或地区，减少美国患者短期获得一些新产品的可能性。可能会让其他国家完全没有得到开发，并损害美国经济的一个重要部分。因此，国会、食品和药物管理局（FDA）和制药行业一直在寻求培育一个有利于在美国开发创新、安全和有效的新化合物的“生态系统”。在开发的机制中，有四种快速审批机制，其中最新的一种--突破性治疗指定--国会于2012年通过
Modern medicine and science have made in-credible strides in improving and extending lives. Nonetheless, many diseases and conditions still lack adequate therapies. According to a report issued by the President’s Council of Advisors on Science and Technology (PCAST) in September 2012 on Propelling Innovation in Drug Discovery, Development, and Evaluation, roughly 30 million Americans suffer from 7,000 rare diseases, but only 350 therapies are approved as treatments. 1 Indeed,‘‘[96] percent of orphan diseases, including rare cancers, lack effective therapies.’’2 Other conditions demand improved therapies as well. For example,‘‘[h] eart disease and stroke remain leading causes of mortality.’’3 The public health need for continued research and development of new drug and biologic products for significant diseases is clear and compelling. But beyond these important health reasons for stimulating research and development of new compounds, there are ancillary and supportive economic considerations for propelling innovative research and development. According to the Pharmaceutical Research and Manufacturers of America (PhRMA), 4 the United States’ biopharmaceutical industry contributes substantially to the US economy. PhRMA reports that the industry directly employs over 800,000 workers in well-paid jobs and diverse fields, and supports an additional 2.5 million jobs across the country. 5 Moreover, PhRMA asserts that it supports over $789 billion in total economic output. 6 For several years, though, the increased time and money necessary to develop a new compound, the failure rate of prospective products, and a decrease in venture capital investments, among other strains on the industry, have propelled concerns that innovative research in the US might wither, stop, or move to other nations or regions, decreasing the potential short term access for US patients to some new products, potentially leaving others unexplored entirely, and hurting a significant segment of the US economy. As a result, Congress, the Food and Drug Administration (FDA), and the pharmaceutical industry have sought to nurture an ‘‘ecosystem’’conducive to the development of innovative, safe, and effective new compounds in the US Among the mechanisms developed are four expedited approval mechanisms, the most recent of which—the Breakthrough Therapy designation—Congress created in 2012 through