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。

基本信息

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
项目类别：
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

项目摘要

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.

项目概要/摘要淀粉样变性研究联盟 (ARC) 成立于 2015 年，是一家以患者为主导的非营利组织 ARC 的使命是建立互惠互利的合作关系。政府、学术界、工业界、患者和监管机构系统地解决减缓系统性新疗法开发的关键障碍和挑战淀粉样变性。 2019年，ARC与美国食品药品监督管理局建立公私合作伙伴关系（PPP）政府 (FDA) 旨在弥合药物开发中的科学差距，称为淀粉样变性淀粉样变性论坛为多学科、跨领域的研究提供了独特的机会。淀粉样变性利益相关者社区齐心协力，确定并弥合科学差距成为治疗不同类型疾病的药物发现和开发的障碍淀粉样变性 PPP 框架包括召开会议来协调该领域，从而导致正式工作组加速行动 2022 年，PPP 获得 FDA 批准。扩大淀粉样变性论坛的范围，将转甲状腺素蛋白淀粉样变性 (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 中的第一代药物批准重塑了研究格局，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本次论坛会议将通过分享进展奠定基础，评估当前存在的影响和未满足的需求，并与目标是利用跨利益相关者资源，就领域塑造优先事项开展协作行动。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：了解新疗法的影响以及当前未满足的需求 ATTR 患者。回顾并讨论 ATTR 领域的进展，包括治疗方法和新疗法的选择，联合疗法作用的评估，以及如何在现实环境中衡量进展和反应。  通过患者参与，特别是患者的参与，为上述主题提供更多背景信息尊重持续存在的医疗保健不平等。具体目标 2：定义 ATTR 中设计临床试验的新方法。  对药物面临的当前和预期挑战建立共识 ATTR 的开发用于下一代临床开发项目。  评估生物标志物的潜在作用和证据差距，评估生物标志物的强度各种生物标志物、终点和试验设计，并定义协作方法评估这些在试验设计中的应用。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面临的这些挑战的行动。