Regulating Rare Disease: Safely Facilitating Access to Orphan Drugs.

IF 1 3区 社会学 Q2 LAW
Fordham Law Review Pub Date : 2018-03-01
Julien B Bannister
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引用次数: 0

Abstract

While approximately one in ten Americans suffers from a rare disease, only 5 percent of rare diseases have a U.S. Food and Drug Administration (FDA) approved treatment. Congressional and regulatory efforts to stimulate the development of rare-disease treatments, while laudable, have not resolved the fundamental issues surrounding rare-disease treatment development. Indeed, small patient populations, incomplete scientific understanding of rare diseases, and high development costs continually limit the availability of rare-disease treatments. To illustrate the struggle of developing and approving safe rare-disease treatments, this Note begins by discussing the approval of Eteplirsen, the first drug approved for treating a rare disease called Duchenne muscular dystrophy. After exploring the current drug regulation system and how this impacts the availability of rare-disease treatments, this Note examines the 21st Century Cures Act's patient experience data provisions and the currently pending Trickett Wendler Right to Try Act. Ultimately, the unmet therapeutic needs of rare-disease patients can be met while protecting patient safety. This Note reasons that, if carefully implemented, the 21st Century Cures Act and the Trickett Wendler Right to Try Act could work in tandem to safely facilitate patient access to rare-disease treatments.

调节罕见病:安全促进孤儿药的获取。
虽然大约十分之一的美国人患有罕见疾病,但只有5%的罕见疾病得到了美国食品和药物管理局(FDA)的批准治疗。国会和监管机构为刺激罕见疾病治疗发展所做的努力虽然值得称赞,但并没有解决围绕罕见疾病治疗发展的根本问题。事实上,患者人数少、对罕见病的科学认识不完整以及高昂的开发成本不断限制罕见病治疗的可得性。为了说明开发和批准安全的罕见疾病治疗方法的困难,本文首先讨论批准Eteplirsen,这是第一种被批准用于治疗罕见疾病杜氏肌营养不良症的药物。在探讨了当前的药物监管体系及其如何影响罕见疾病治疗的可用性之后,本文研究了《21世纪治愈法案》的患者体验数据规定和目前待定的《特里克特·温德勒尝试权法案》。最终,可以在保护患者安全的同时满足罕见病患者未满足的治疗需求。本文认为,如果仔细实施,《21世纪治愈法案》和《特里克特·温德勒尝试权法案》可以协同工作,安全地促进患者获得罕见疾病的治疗。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
1.10
自引率
12.50%
发文量
0
期刊介绍: The Fordham Law Review is a scholarly journal serving the legal profession and the public by discussing current legal issues. Approximately 75 articles, written by students or submitted by outside authors, are published each year. Each volume comprises six books, three each semester, totaling over 3,000 pages. Managed by a board of up to eighteen student editors, the Law Review is a working journal, not merely an honor society. Nevertheless, Law Review membership is considered among the highest scholarly achievements at the Law School.
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