Kantor Ariel, Haga Susanne B
Center for Applied Genomic & Precision Medicine, Duke University School of Medicine, Durham, NC 27708, USA.
Nuffield Laboratory of Ophthalmology, Nuffield Department of Clinical Neurosciences & NIHR Oxford Bio-Medical Research Centre, University of Oxford, Oxford OX3 9DU, UK.
J Pers Med. 2021 Jan 14;11(1):45. doi: 10.3390/jpm11010045.
Increased understanding of the molecular causes of disease has begun to fulfill the promise of precision medicine with the development of targeted drugs, particularly for serious diseases with unmet needs. The drug approval regulatory process is a critical component to the continued growth of precision medicine drugs and devices. To facilitate the development and approval process of drugs for serious unmet needs, four expedited approval programs have been developed in the US: priority review, accelerated approval, fast track, and breakthrough therapy programs.
To determine if expedited approval programs are fulfilling the intended goals, we reviewed drug approvals by the US Food and Drug Administration (FDA) between 2011 and 2017 for new molecular entities (NMEs).
From 2011 through 2017, the FDA approved 250 NMEs, ranging from 27 approvals in 2013 to 46 in 2017. The NME approvals spanned 22 different disease classes; almost one-third of all NMEs were for oncology treatments.
As these pathways are utilized more, additional legislative changes may be needed to re-align incentives to promote continued development of innovative drugs for serious unmet needs in a safe, efficacious, and affordable manner.
随着靶向药物的发展，尤其是针对尚未满足需求的严重疾病的靶向药物的发展，对疾病分子病因的深入了解已开始兑现精准医学的承诺。药物批准监管程序是精准医学药物和器械持续发展的关键组成部分。为了促进针对严重未满足需求的药物的开发和批准过程，美国制定了四项加速批准计划：优先审评、加速批准、快速通道和突破性疗法计划。
为了确定加速批准计划是否实现了预期目标，我们回顾了美国食品药品监督管理局（FDA）在2011年至2017年期间对新分子实体（NMEs）的药物批准情况。
从2011年到2017年，FDA批准了250个新分子实体，从2013年的27项批准到2017年的46项不等。新分子实体批准涵盖22种不同的疾病类别；所有新分子实体中近三分之一用于肿瘤治疗。
随着这些途径被更多地利用，可能需要进一步的立法变革来重新调整激励措施，以促进以安全、有效和可承受的方式持续开发针对严重未满足需求的创新药物。
