Day 2: Wednesday, March 18th, 2026Wednesday, 18 March 2026 - ET (Eastern Time, GMT-05:00)

This session explores innovative strategies for building meaningful, productive relationships with patient advocacy groups. Hear from patients and industry executives directly on creating partnerships that advance shared goals while respecting each organization's unique mission.

• Learn how to optimize patient advocacy partnerships through clear role definition and strategic alignment

• Explore innovative omnichannel approaches that amplify patient voices across digital platforms to build disease awareness and support communities in fragmented rare disease populations

• Understand how patient advocacy initiatives can serve as catalysts in commercialization efforts

• Discover models for creating collaborative spaces that advance research and patient care

This session explores critical pre-launch considerations that can make or break a rare disease therapy launch. Gain insights into timing, cross-functional alignment and go-to-market strategy.

• Discover how early involvement of market access, trade, patient services and field teams create a stronger launch foundation

• Learn strategies for determining optimal distribution models based on market and payer challenges

• Explore nuanced approaches for first-in-market versus competitive landscape scenarios

• Gain insights into building effective access programs and communication plans before launch

This session focuses on creating patient support programs specifically tailored to rare disease populations. Learn how to evaluate service models and implementation strategies.

• Learn how to craft a hub that effectively meets the unique needs of rare disease patients beyond traditional services

• Explore the decision framework for determining whether your hub should be mandatory

• Understand how to balance patient access liaisons, physician office support and other specialized services

• Discover strategies for maintaining program continuity and high-touch adherence support

This session tackles the unique challenges of launching in markets with established competitors versus pioneering new treatment areas. Learn targeted approaches for different competitive scenarios.

• Master strategies for differentiating your therapy in crowded rare disease spaces

• Explore education approaches when launching first-in-market versus entering competitive landscapes

• Learn how to ensure stakeholders have relevant clinical information and confidence in access/affordability

• Discover techniques for staying abreast of industry developments to continuously serve patient need

This session provides insights into current FDA approaches to rare disease and strategies for effective engagement. Hear from the FDA directly and learn how to adapt to evolving regulatory expectations.

• Explore strategies for engaging with FDA's Accelerating Rare Disease Cures (ARC) program

• Understand how to best engage with FDA CDER during the drug development process

• Learn about the role of the Rare Disease Innovation Hub between CBER and CDER

This session provides a roadmap for the critical first year after launch, focusing on monitoring, adaptation and maintaining momentum. Learn how to establish effective metrics and communication channels.

• Discover how to establish metrics and communication channels to monitor post-launch success, from early weeks to long-term milestones

• Learn why patience is crucial – and why you should wait at least six months before making major strategy adjustments

• Identify and implement KPIs and metrics specific to rare disease launch, including patient identification rates, time-to-treatment and adherence indicators

• Discover how cross-functional meetings and watchtower views can identify and address hurdles quickly

This session explores the practical differences between US and global markets and how they shape rare disease launch strategy. Drawing on experience across the US. EU, Japan, and LATAM, the discussion focuses on aligning global and local teams when the US is the primary revenue driver, without compromising ex-US access and execution.

Key takeaways:

• Key structural differences across the US, EU, Japan, and LATAM that influence launch decisions and sequencing
• Operating models for effective global organizations with US-led revenue dynamics
• How patient identification, access, and support models differ across regions
• Brand planning approaches that reflect region-specific success factors and optimize total brand value

This exclusive case study examines Sarepta Therapeutics' pivotal role at the intersection of regulatory pathways, patient advocacy and industry perspectives on accelerated approvals in rare disease treatment. Through the lens of Sarepta's experience, our distinguished panel—featuring former Sarepta leadership, patient advocacy voices, regulatory expertise and clinical insight—will unpack the profound ethical dilemmas and transparency challenges that define rare disease drug development. Attendees will gain critical insights into balancing innovation with risk, fostering authentic stakeholder trust and navigating the complex ecosystem where scientific advancement meets urgent patient need—lessons that extend far beyond a single company to shape the future of rare disease therapeutics.

This session addresses one of the fundamental challenges in rare disease—getting patients diagnosed quickly and accurately. Learn strategies for improving the diagnostic pathway.

• Explore innovations in screening, including genomic sequencing and newborn screening advances

• Learn approaches for educating HCPs about rare disease indicators and diagnostic pathways

• Understand strategies for patient identification when traditional claims data is limited or unavailable

• Discover how to optimize the patient journey to minimize diagnostic delays and improve outcomes