What to Expect

Calling all champions of innovation

Join over 900 patient leaders, researchers, healthcare advocates, pharma and biotech innovators, government agencies, and others to tackle the most pressing issues facing the rare disease community.

Pharma and Biotech

  • Stay ahead of emerging FDA regulatory priorities and trends impacting orphan product development.

  • Explore new partnership and business development opportunities.

  • Build meaningful relationships with patient advocacy leaders to strengthen patient engagement throughout the drug development lifecycle.

Government Officials and Regulators

  • Understand the evolving policy and regulatory landscape shaping orphan product development.

  • Learn about the latest scientific advances and emerging innovations in rare diseases.

  • Hear best-in-class examples of patient engagement in drug development from patient advocacy leaders.

CROS and Solutions Companies

  • Connect with current and prospective clients across the rare disease ecosystem.

  • Showcase your expertise in supporting clinical development, trials, evidence generation, and commercialization.

  • Generate new business opportunities through strategic networking and partnership development.

Patient Advocacy Leaders

  • Raise awareness of the unmet needs and priorities within your disease community .

  • Discover new resources, research, and collaborations that can benefit the patients and families you serve.

  • Share patient insights with researchers, industry, and regulators to help shape the development of future therapies.

Researchers and Scientists

  • Share cutting-edge research and innovative approaches advancing rare disease science.

  • Connect with industry, patient advocacy organizations, clinicians, and regulators to accelerate collaboration and translate discoveries into patient impact.