社论:早期创新——适应加速药物审批的新时代

IF 1.8 4区 医学 Q3 PHARMACOLOGY & PHARMACY
Amalia M. Issa
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引用次数: 0

摘要

在科学创新和全球越来越多地采用加速审批途径的推动下,早期药物开发的前景正在迅速发展。美国食品和药物管理局(FDA)最近宣布的局长国家优先代金券(CNPV)计划标志着美国药物开发的潜在变革时刻。1通过大幅缩短审查时间,将传统的10-12个月压缩到1-2个月的合格申请,FDA既表明了对创新的承诺,也表明了重新思考其监管范式的意愿,以满足迫切的国家健康需求。随着FDA公布其新的CNPV项目,值得考虑的是如何改写早期药物开发的传统剧本。对于临床药理学家和药物开发人员来说,CNPV项目可以从几个方面从根本上改变早期阶段的情况。尽管潜在的好处是巨大的,但仍存在几个问题。该计划最初的推出是有限的,为期一年的试点项目,只发放少量代金券,因此其更广泛的影响将取决于未来的扩展和解决实施中的不确定性,如选择标准和透明度。还有一个长期关注的问题是,确保速度不会以牺牲科学严谨性或患者安全为代价,FDA坚持通过多学科审查和持续的赞助商沟通来维持这种平衡。3,4 CNPV项目是朝着监管科学现代化和将药物开发激励机制与国家卫生重点相结合迈出的大胆一步。对于处于早期阶段的开发人员来说,这既提供了机遇,也提供了挑战:做好准备,灵活应变,并与不断变化的公共卫生优先事项保持一致。如果成功,该项目可能成为未来监管创新的蓝图,在保持最高安全性和有效性标准的同时,促进更快地获得变革性疗法。随着试验的展开,临床药理学社区将密切关注,渴望看到这种“肿瘤板”模式是否能够实现其承诺,也许重新定义早期药物开发的可能性。作者声明无利益冲突。这项工作没有获得资金。
本文章由计算机程序翻译,如有差异,请以英文原文为准。

Editorial: Early Phase Innovation—Adapting to the New Age of Accelerated Drug Approvals

Editorial: Early Phase Innovation—Adapting to the New Age of Accelerated Drug Approvals

Editorial: Early Phase Innovation—Adapting to the New Age of Accelerated Drug Approvals

Editorial: Early Phase Innovation—Adapting to the New Age of Accelerated Drug Approvals

The landscape of early-phase drug development is evolving rapidly, driven by both scientific innovation and the increasing adoption of accelerated approval pathways worldwide. The US Food and Drug Administration's (FDA) newly announced Commissioner's National Priority Voucher (CNPV) program marks a potentially transformative moment for drug development in the United States.1 By offering a radically shortened review timeline—compressing the traditional 10-12 months to just 1-2 months for qualifying applications—the FDA signals both a commitment to innovation and a willingness to rethink its regulatory paradigms to meet urgent national health needs.

As the FDA unveils its new CNPV program, it is worth considering how the traditional playbook for early-phase drug development might be rewritten. For clinical pharmacologists and drug developers, the CNPV program could fundamentally alter the early-phase landscape in several ways.

Although the potential benefits are substantial, several questions remain. The program's initial rollout is limited —a 1-year pilot with a small number of vouchers—so its broader impact will depend on future expansion and the resolution of implementation uncertainties, such as selection criteria and transparency. There is also the perennial concern of ensuring that speed does not come at the expense of scientific rigor or patient safety, a balance the FDA insists will be maintained through multidisciplinary review and ongoing sponsor communication.3, 4

The CNPV program represents a bold step toward modernizing regulatory science and aligning drug development incentives with national health priorities. For early-phase developers, it offers both an opportunity and a challenge: to be ready, nimble, and aligned with the evolving priorities of public health. If successful, the program may serve as a blueprint for future regulatory innovation, catalyzing faster access to transformative therapies while upholding the highest standards of safety and efficacy.

As the pilot unfolds, the clinical pharmacology community will watch closely, eager to see whether this “tumor board” model can deliver on its promise and perhaps redefine what is possible in early-phase drug development.

The author declares no conflicts of interest.

No funding was obtained for this work.

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来源期刊
CiteScore
3.70
自引率
10.00%
发文量
154
期刊介绍: Clinical Pharmacology in Drug Development is an international, peer-reviewed, online publication focused on publishing high-quality clinical pharmacology studies in drug development which are primarily (but not exclusively) performed in early development phases in healthy subjects.
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