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

淀粉样变性论坛：在不断发展的治疗格局中推进 ATTR 淀粉样变性的药物开发。

• 批准号: 10683562
• 负责人: Isabelle R. Lousada
• 金额: $ 4万
• 依托单位: AMYLOIDOSIS RESEARCH CONSORTIUM, INC.
• 依托单位国家: 美国
• 财政年份: 2023
• 资助国家: 美国
• 起止时间: 2023-04-15 至 2024-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10683562
• 关键词: Academia; Acceleration; Address; African ancestry; Amyloid; Amyloidosis; Biological Markers; Cardiac; Cardiomyopathies; Clinical Trials Design; Collaborations; Combined Modality Therapy; Communities; Community Surveys; Coronary heart disease; Data; Development; Diagnosis; Discipline; Disease; Disease Progression; Disease remission; Drug Approval; Elderly; Ensure; Eye Development; Familial Amyloidosis; Foundations; Future; Generations; Government; Heart failure; Industry; Inherited; Measures; Mission; Nonprofit Organizations; Outcome; Pathway interactions; Patient Participation; Patient Preferences; Patients; Persons; Prealbumin; Privatization; Program Development; Research; Resources; Role; Series; Shapes; Surveys; Symptoms; Syndrome; Systemic Therapy; Systemic disease; Time; United States Food and Drug Administration; United States National Institutes of Health; Variant; age related; aging population; assessment application; autosome; clinical development; collaborative approach; design; drug development; drug discovery; health care disparity; interest; meetings; multidisciplinary; next generation; novel; novel strategies; novel therapeutics; patient engagement; public-private partnership; real world application; research and development; response; symposium; therapeutic development; therapy development; trial design; working group

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.

项目总结/摘要 淀粉样变性研究联盟（ARC）成立于2015年，是一个以患者为主导的非营利组织。 organization. ARC的使命是建立互利合作， 政府、学术界、工业界、患者和监管机构系统地解决 严重的障碍和挑战，减缓了新疗法的发展， 淀粉样变性 2019年，ARC与美国食品药品监督管理局（FDA）达成公私合作伙伴关系（PPP）。 美国食品药品监督管理局（FDA）填补药物开发的科学空白，称为淀粉样变性 论坛淀粉样变性论坛提供了一个独特的机会，多学科，跨- 利益相关者淀粉样变性社区走到一起，以确定和弥合科学差距 作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，以协调实地工作， 正式成立工作组，加快行动。2022年，PPP获得FDA批准 扩大淀粉样变性论坛的职权范围，将甲状腺素运载蛋白淀粉样变性（ATTR）纳入 认识到当前和未来治疗中普遍存在的未满足的需求和挑战， 发展 ATTR的第一代药物批准重塑了研究格局， 需要重新考虑如何有效地设计试验，以应对治疗缓慢的背景 疾病的进展。本次论坛会议将通过分享进展， 评估影响和目前存在的未满足的需求，并确定与 目的是利用跨利益攸关方的资源，就外地塑造优先事项采取协作行动。 解决与在不断发展的治疗中推进ATTR药物开发相关的主题 景观，我们的具体目标包括： 具体目标1：了解新型治疗方法的影响和当前未满足的需求， ATTR患者 回顾和讨论ATTR领域的进展，包括治疗方法， 新疗法的选择，联合疗法作用的评估，以及 如何在现实世界中衡量进展和反应。 通过患者参与，为上述主题带来更大的背景，特别是 持续存在的医疗不平等。 具体目标2：定义ATTR临床试验设计的新方法。 * 就毒品问题办公室目前和预期面临的挑战达成共识 在ATTR中为下一代临床开发项目进行开发。 评估生物标志物的潜在作用和证据缺口，评估 各种生物标志物、终点和试验设计，并定义协作方法， 评估这些用于试验设计。 具体目标3：概述监管审批和报销挑战，并定义 前瞻性的行动计划。 ·了解监管部门面临的当前和预期挑战 ATTR中的批准和报销。 概述应对下一代疗法这些挑战的行动。