The Amyloidosis Forum: Cross Stakeholder Working Group Projects to Accelerate Drug Development in AL and ATTR Amyloidosis

Amyloidosis Research Consortium, Inc., Newton MA

Investigators

Abstract

Project Summary/Abstract The Amyloidosis Research Consortium (ARC), established in 2015, is a patient-led nonprofit organization. ARC’s mission 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 with the US Food and Drug Administration (FDA) known as the Amyloidosis Forum to bridge scientific gaps in drug development for the treatment of amyloidosis. The Amyloidosis Forum seeks to leverage the expertise and resources of diverse stakeholders for the conduct of mutually beneficial scientific activities in the precompetitive domain to support bringing new, safe and efficacious therapies to patients with amyloidosis. Collaborative efforts across stakeholders and innovative approaches are essential to propel the future development of effective therapies for multi-systemic diseases like immunoglobulin light chain (AL) and transthyretin (ATTR) amyloidosis. The proposed conference, to be held at the FDA White Oak campus, will provide an opportunity for prioritized working groups launched through the Amyloidosis Forum to share their research and engage with diverse stakeholders to seek feedback and insight that will enable them to strengthen their research. These collaborative working groups are focused on four priority areas: imaging standardization, identification of prognostic factors, development of clinical endpoints, and accelerating access to data-driven insights from completed trials in AL and ATTR amyloidosis. This meeting, The Amyloidosis Forum: Cross Stakeholder Working Group Projects to Accelerate Drug Development in AL and ATTR Amyloidosis, will aim to do the following: Specific Aim 1: Engage diverse stakeholders to inform and strengthen working group research strategies and projects that will address scientific and regulatory gaps required to advance drug development. • Critically evaluate and discuss research updates provided by the Amyloidosis Forum working groups. • Present diverse feedback on the design of proposed collaborative research studies. Specific Aim 2: Provide a collaborative opportunity for stakeholders to convey their perspectives on future priorities and research direction. • Evaluate and align stakeholder priorities for future research projects. Specific Aim 3: Enable early-career professionals with opportunities to network with experienced amyloidosis researchers. • Provide early career professionals with broad perspectives across the amyloidosis therapeutic development spectrum and an opportunity to engage with experienced research professionals.