Bannister Julien B
Fordham Law Rev. 2018 Mar;86(4):1889-921.
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.
虽然大约十分之一的美国人患有罕见病，但只有5%的罕见病有美国食品药品监督管理局（FDA）批准的治疗方法。国会和监管部门为刺激罕见病治疗方法的研发所做的努力虽然值得称赞，但尚未解决围绕罕见病治疗方法研发的根本问题。事实上，患者群体规模小、对罕见病的科学认识不全面以及研发成本高昂，持续限制着罕见病治疗方法的可及性。为说明研发和批准安全的罕见病治疗方法所面临的困难，本评论首先讨论首个获批用于治疗一种名为杜氏肌营养不良症的罕见病的药物依特立生的批准情况。在探究当前的药物监管体系及其如何影响罕见病治疗方法的可及性之后，本评论审视了《21世纪治愈法案》中关于患者体验数据的条款以及目前悬而未决的《特里克特·温德勒“尝试权”法案》。最终，在保护患者安全的同时，可以满足罕见病患者未得到满足的治疗需求。本评论认为，如果谨慎实施，《21世纪治愈法案》和《特里克特·温德勒“尝试权”法案》可以协同发挥作用，安全地帮助患者获得罕见病治疗方法。
