Day 3: Thursday, March 19th, 2026Thursday, 19 March 2026 - ET (Eastern Time, GMT-05:00)

Hear from CHOP (Children's Hospital of Philadelphia) as they reveal the complex infrastructure and significant investments that hospitals make to enroll patients in cutting-edge cell and gene therapy clinical trials.

• Gain insight into the hidden operational challenges that hospitals face when navigating health policy and coverage barriers that directly impact patient enrollment and access

• Learn practical strategies for closing the knowledge gap between manufacturers, hospitals and patients, including actionable frameworks for developing more efficient clinical trial pathways

This session explores business development approaches specifically tailored to rare disease opportunities. Learn frameworks for evaluating and executing deals in this unique space.

• Learn how to assess targets that fit within manufacturer focus and capabilities

• Explore approaches for quick commercial assessment to support leadership recommendations

• Understand how to determine fit in terms of evaluations and deal terms

• Discover what investors are looking for in rare disease opportunities in today's market

This dynamic session explores the groundbreaking partnership between TSC Alliance and Upsher Smith Laboratories, LLC, revealing how patient advocacy groups, manufacturers and specialty pharmacies are creating powerful synergies to transform the rare disease patient journey. Uncover practical insights on integrating patient support services, designed by patient advocacy groups in collaboration with a medication’s manufacturer, with specialty pharmacy operations, resulting in a more seamless experience for those navigating complex treatment journeys. Discover how this evolving relationship model is setting new standards for patient-centered care while addressing critical gaps in the rare disease ecosystem—ultimately delivering better outcomes through collaboration rather than siloed approaches.

This session provides a comprehensive update on the latest policy developments impacting the rare disease community.

• Discuss emerging policy trends and key legislative and regulatory changes

• Understand the respective implications for patients, caregivers, researchers and industry stakeholders

In this closing session, come back to the core of our conference with a focus on the patient journey, experience and outcomes. Here and see the direct impact of what we have learned and discussed over the course of the conference and be inspired for the year ahead.