Agenda for Day 3 – Thursday, March 25, 2027Thursday, 25 March 2027 - ET (Eastern Time, GMT-05:00)

• Hear from the FDA directly to understand the evolving rare disease landscape, recent rare disease drug approvals and how manufacturers can best work with FDA

• Gain insights into current FDA approaches to rare disease, including strategies for effective engagement and updates on recent guidance’s

• Learn more about hot topics at FDA such as AI, advisory committees, the Rare Disease Innovation Hub and biomarkers

• Address the unique challenges of data scarcity in rare diseases and provides strategies for maximizing available information and best practices in data acquisition and utilization.

• Explore innovative data sets and partnerships that can help identify and understand rare disease patients

• Learn techniques for processing and linking disparate data sources in a commercial environment

• Understand the legal and ethical considerations around de-identified versus non-de-identified patient data

• Discover how to leverage data for patient identification, market sizing and commercial strategy development

• Understand the strategic rationale for simultaneous market entry versus sequential launches, including the commercial, ethical and competitive considerations that drive parallel access strategies

• Learn the operational infrastructure required to execute simultaneous launches across diverse regulatory environments, including resource allocation, cross-market coordination and risk mitigation strategies

• Explore the unique regulatory pathways, pricing dynamics and market access requirements in mid-sized and emerging markets, and how to address these challenges without compromising major market strategies

• Discover practical approaches to dramatically reduce the time between regulatory approval and patient access in underserved markets, addressing distribution, reimbursement and healthcare provider education barrier

• Identify the natural acquirers in rare disease and understand how acquisition targets vary by therapeutic area, modality and stage of development

• Discover what business development teams at large pharma are actively seeking in rare disease acquisitions and how to position your company to meet these requirements

• Explore how emerging modalities and technologies are reshaping the M&A landscape

• Gain insights into deal structures, valuation drivers and timing strategies that maximize acquisition potential

• Learn about alternative strategic options that move beyond acquisition, such as partnerships, licensing and co-development

Join your peers for Access USA 2027's most candid session yet, alongside all ecosystem stakeholders. In this interactive closing conversation, join focused roundtables organized by vendor type – PAP providers, Hub services, AI solutions, specialty pharmacy partners and more.

Whether you're evaluating new partnerships, troubleshooting existing relationships or simply curious about what others are experiencing, this peer-to-peer exchange offers the unvarnished insights you won't find in any RFP response.

Come ready to listen, share and leave with actionable intelligence from those who've been in the trenches.