FDA启动神经科学推进与创新枢纽NAIN 首个专项行动聚焦阿尔茨海默病预防产品研发与临床试验路径创新
【中文译文】
美国食品药品监督管理局（FDA）今日宣布启动“神经科学推进与创新枢纽”（Neuroscience Advancement and Innovation Nexus，简称NAIN）。这是一项新的机构级计划，与政府加速医疗创新、加快突破性疗法交付的方向一致。NAIN旨在推进支撑脑与神经系统疾病医疗产品开发所需的科学、工具和监管方法。NAIN的首个专项行动“阿尔茨海默病预防加速器”（Alzheimer's Prevention Accelerator）将聚焦推动有助于延缓或预防阿尔茨海默病的医疗产品开发。两项工作均支持《国家阿尔茨海默病项目法案》（NAPA）——美国联邦政府针对阿尔茨海默病及相关痴呆症协调行动的综合性路线图。
FDA代理局长凯尔·迪亚曼塔斯（Kyle Diamantas，法学博士）表示：“NAIN的启动体现了FDA的承诺——支持政府推动医疗健康创新变革，确保我们的监管科学和跨中心协作能够跟得上神经科学领域的快速进展。通过NAIN和阿尔茨海默病预防加速器，我们将调动FDA全部专业能力，应对当今最紧迫的公共卫生挑战之一。”
FDA指出，针对神经科学适应症开发医疗产品面临复杂的科学和临床挑战，包括对潜在生物学机制认识有限、疾病表现差异大，以及难以证明有意义的临床获益，对病程长达多年的疾病尤其如此。不过，生物标志物、数字健康技术以及其他医疗产品开发工具的进步，为解决这些挑战提供了重要机会。
NAIN将汇集FDA药品评价与研究中心（CDER）、生物制品评价与研究中心（CBER）以及器械与放射健康中心（CDRH）的科学与监管专长，解决需要在药品、生物制品和器械之间协调考虑的科学与监管问题。NAIN覆盖影响脑与神经系统的广泛疾病，包括神经和精神障碍、物质使用障碍以及疼痛。NAIN还将支持与患者、产业界、学术界及其他利益相关方的深入互动。
作为首个专项行动，NAIN启动阿尔茨海默病预防加速器，汇集FDA各部门视角，并与患者和照护者、研究者、产业界、公私合作项目及其他政府机构合作。该行动将探索推进阿尔茨海默病预防和早期干预药物研发临床试验的创新路径，重点关注试验人群选择、与疾病阶段相适应的终点指标，以及证明可预防或延缓阿尔茨海默病所致认知衰退的证据方法。
FDA长期致力于支持针对脑与神经系统疾病的安全有效医疗产品开发。通过NAIN和阿尔茨海默病预防加速器，FDA正采取主动、协调的方式加速进展，整合科学与监管专长，发动更广泛的研发共同体，并与联邦伙伴协同应对这一公共卫生挑战的规模。
【英文原文】
The U.S. Food and Drug Administration today announced the launch of the Neuroscience Advancement and Innovation Nexus (NAIN), a new agency program aligned with the Trump Administration's efforts to accelerate medical innovation and deliver breakthrough treatments. NAIN is designed to advance the science, tools, and regulatory approaches needed to support the development of medical products for conditions affecting the brain and nervous system. NAIN's inaugural initiative, the Alzheimer's Prevention Accelerator, will focus on advancing medical products that help delay or prevent Alzheimer's disease. Both efforts support the National Alzheimer's Project Act (NAPA), the federal government's comprehensive roadmap for coordinated action on Alzheimer's and related dementias.
"The launch of NAIN reflects FDA's commitment — in support of the Trump Administration's drive to transform health care innovation — to making sure our regulatory science and cross-center collaboration keep pace with the rapid advances happening in neuroscience," said Acting FDA Commissioner Kyle Diamantas, J.D. "Through NAIN and the Alzheimer's Prevention Accelerator, we are bringing the full breadth of FDA's expertise to bear on one of the most urgent public health challenges of our time."
Developing medical products for neuroscience indications is associated with complex scientific and clinical challenges, including limited understanding of underlying biology, variable disease presentation, and difficulties in demonstrating meaningful clinical benefit, particularly for conditions that develop over many years. Advances in biomarkers, digital health technologies, and other medical product development tools, however, offer important opportunities to address these challenges.
NAIN will bring together scientific and regulatory expertise from the FDA's Center for Drug Evaluation and Research, Center for Biologics Evaluation and Research, and Center for Devices and Radiological Health to address scientific and regulatory questions that benefit from coordinated consideration across drugs, biologics, and devices. NAIN will encompass conditions that affect the brain and nervous system broadly, including neurologic and psychiatric disorders, substance use disorders, and pain. NAIN will also support robust engagement with patients, industry, academia and other stakeholders to advance these efforts.
As its first initiative, NAIN is launching the Alzheimer's Prevention Accelerator. This inaugural initiative will draw on perspectives from across the FDA and engage patients and caregivers, researchers, industry, public-private partnerships and other government agencies. The initiative will explore innovative approaches to advancing clinical trials for Alzheimer's disease prevention and early-intervention drug development with emphasis on trial populations, stage-appropriate endpoints, and evidentiary approaches for demonstrating prevention or delay of cognitive decline due to Alzheimer's disease.
The FDA has long been committed to supporting the development of safe and effective medical products that address diseases and conditions of the brain and nervous system. Through NAIN and the Alzheimer's Prevention Accelerator, the agency is taking a proactive, coordinated approach to accelerating progress, bringing together its scientific and regulatory expertise, engaging the broader research and development community, and working in concert with federal partners to meet the scale of this public health challenge.
