The Amyloidosis Forum: Advancing Drug Development in ATTR Amyloidosis in an Evolving Treatment Landscape.

项目摘要/摘要 淀粉样变性研究联盟(ARC)成立于2015年，是一个由患者主导的非营利性组织 组织。ARC的使命是在两国之间建立互利合作 政府、学术界、产业界、患者和监管机构要系统地解决 阻碍系统性疾病新疗法开发的关键障碍和挑战 淀粉样变性。 2019年，ARC与美国食品和药物管理局建立了公私合作伙伴关系(PPP) 美国食品和药物管理局(FDA)弥合药物开发中的科学差距，即淀粉样变性 论坛。淀粉样变性论坛提供了一个独特的机会，多学科，交叉- 利益相关者淀粉样变性社区走到一起，识别和弥合科学鸿沟 成为药物发现和开发的障碍，用于治疗不同类型的 淀粉样变性。公私伙伴关系框架包括召开会议以协调外地工作，结果是 使工作组正规化，以加快行动。2022年，PPP获得了FDA的批准 扩大淀粉样变性论坛的范围，将转甲状腺蛋白淀粉样变性(ATTR)包括在 认识到当前和未来治疗中普遍存在的未得到满足的需求和挑战 发展。 ATTR的第一代药物审批重塑了研究格局，创造了 需要重新考虑如何在治疗缓慢的背景下有效地设计试验 疾病的发展。本次论坛会议将通过分享进步来搭建舞台， 评估当前存在的影响和未得到满足的需求，并确定关键计划 目的是利用跨利益攸关方的资源，就实地塑造的优先事项采取协作行动。 在不断发展的治疗中解决与推进ATTR药物开发有关的主题 在景观方面，我们的具体目标包括： 具体目标1：了解新疗法的影响和目前未得到满足的需求 患有ATTR的患者。 回顾和讨论ATTR领域的进展，包括治疗和治疗的方法 新疗法的选择，联合疗法作用的评估，以及 如何在真实环境中衡量进度和反应。 通过患者参与为上述主题带来了更大的背景，特别是在 尊重持续存在的医疗不平等现象。 具体目标2：确定设计ATTR临床试验的新方法。 建立了对药物面临的当前和预期挑战的共同理解 为下一代临床开发计划在ATTR中的开发。 评估生物标记物的潜在作用和证据差距，评估 各种生物标记物、终点和试验设计，并定义协作方法 评估这些在试验设计中的应用。 具体目标3：概述监管审批和偿还方面的挑战，并确定 前瞻性行动计划。 可帮助您了解当前和预期面临的监管挑战 在Attr中审批和报销。 概述了为应对下一代疗法的这些挑战而采取的行动。

Project Summary/Abstract The Amyloidosis Research Consortium (ARC), established in 2015, is a patient-led nonprofit organization. The mission of ARC is to build mutually beneficial collaborations between government, academia, industry, patients, and regulatory agencies to systematically address critical barriers and challenges that slow the development of new therapies for systemic amyloidosis. In 2019, ARC entered a public-private partnership (PPP) with the US Food and Drug Administration (FDA) to bridge scientific gaps in drug development, known as the Amyloidosis Forum. The Amyloidosis Forum provides a unique opportunity for the multidisciplinary, cross- stakeholder amyloidosis community to come together to identify and bridge the scientific gaps that act as barriers to drug discovery and development for the treatment different types of amyloidosis. The PPP framework includes convening meetings to align the field, resulting in formalized working groups to accelerate action. In 2022, the PPP obtained approval from the FDA to expand the remit of the Amyloidosis Forum to include transthyretin amyloidosis (ATTR) in recognition of the unmet needs and challenges that prevail in current and future therapeutic development. The first generation of drug approvals in ATTR has reshaped the research landscape creating the need to reconsider how to effectively design trials against a backdrop of treatments that slow progression of the disease. This Forum meeting will set the stage by sharing the advancements, evaluating the impact and unmet needs that currently exist, and identifying key initiatives with the aim of leveraging cross-stakeholder resources to collaboratively act on field-shaping priorities. Addressing themes related to Advancing Drug Development in ATTR in an Evolving Treatment Landscape, our Specific Aims include: Specific Aim 1: Understand the impact of novel treatments and the current unmet needs in patients with ATTR.  Review and discuss advances in the field of ATTR including approaches to treatment and the selection of new therapies, the assessment of the role of combination therapies, and how progression and response is measured in real-world settings.  Bring greater context to the above themes through patient participation, particularly with respect to persistent healthcare inequities. Specific Aim 2: Define new approaches to designing clinical trials in ATTR.  Establish a common understanding of the current and anticipated challenges facing drug development in ATTR for the next generation of clinical development programs.  Assess the potential role of biomarkers and evidence gaps, evaluate the strength of various biomarkers, endpoints, and trial designs, and define collaborative approaches to assessing these for application in trial design. Specific Aim 3: Outline regulatory approval and reimbursement challenges and define forward-looking action plans.  Provide an understanding of the current and anticipated challenges facing regulatory approvals and reimbursement in ATTR.  Outline actions to address these challenges for the next generation of therapies.