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Rare Disease Conference 2024 - Rare Disease Summit
March 18-20, 2025
Pre-Conference Workshops on March 17, 2025Sheraton Philadelphia Downtown | Philadelphia, PA

Unite with the rare disease community to create powerful partnerships, advance orphan therapeutics and deliver life-changing breakthroughs

The Rare Disease Summit connects key stakeholders to drive therapeutic progress, propel commercial strategies and inspire impactful advocacy. You’ll generate solution-focused approaches surrounding patient access, reimbursement, commercialization, launch, partnering and more, as you unify in areas of unmet medical need.

A Thank You to All Rare Disease Summit 2024 Attendees

Informa Connect's Rare Disease Summit Team would like to take the opportunity to thank all of our speakers, sponsors and attendees for participating in this year's event. The 2024 event was a huge success and it was such an amazing experience to see the rare disease community gathering for interactive discussions and networking. Stay tuned for updates on what's in store for the 2025 event. We can't wait to welcome everyone back next year!

Have a profound impact on the Rare Disease Community

While the industry has seen growth and heightened focus around the pursuit of orphan products and rare therapies, we’ve only scratched the surface. 2024 Attendees benefitted from high-impact content from experts from across the rare disease industry:

Innovative Approaches to Amplify Collaboration and Advancement

Uncover innovative solutions to issues alongside the top industry experts committed to shaping the future of Rare Disease 

  • Navigate FDA Updates on Rare Disease Product Development  
  • Helpful or Harmful – Using AI in the Rare Disease Space 
  • Unlocking Rare Disease Investment Success through Valuation, Risk and Innovation
  • Innovation Called and Gene Therapy Answered 

Riveting Keynotes and Ground-Breaking Panels to Deliver Fresh Perspectives

Join market leaders setting the stage for game-changing dialogue  

  • The Importance of Funding and Partnering from the Perspective of an Early Stage BioPharma Company
  • Exploring the Terrain - Rare Disease Landscape from a Payer Perspective
  • Thinking Outside the Box – Forming Unique Partnerships within Rare Disease 
  • Rare Caregivers Report—Experiences Supporting Loved Ones Living with Rare Diseases

A Collaborative Think Tank to Benchmark and Strategize on the Top Challenges in Rare Disease

The all-plenary design of this event enables for more meaningful interaction, higher level networking and closer up learning 

  • Hear multiple keynote addresses featuring key stakeholder perspectives, including Industry, Advocacy and Regulatory 
  • Have dedicated time to brainstorm with your peers on innovative ideas surrounding the different phases of Rare Disease Drug Development (ie. diagnosis, preclinical, drug development, regulatory) 
  • Engage in our Innovation Station to hear rapid fire presentations from the key innovators within the Rare Disease space 
  • Be able to take home with you new connections, new ideas and new best practices

Vice President

Regulatory Affairs, North America

Sobi, Inc.

I thought this was one of the best conferences I’ve attended over the years. The focus was very practical and each speaker shared real world examples instead of theory. It was well organized and the passion each participant has for helping people with rare disease was very evident.

Rare Disease Innovation & Partnership Summit: Part of Access USA

Hundreds from the access community came together as three industry-leading events united under one roof for compelling discussions, industry benchmarking and extended networking. What happened? Click play to see event highlights and why you can't miss this pivotal access forum!

See Who You'll Meet at the Rare Disease Summit

You will benefit from attending this event if you work for a company that focuses on rare disease and orphan drugs and have responsibilities or involvement in the following areas:

  • Rare Disease
  • Orphan Drugs
  • Market Access
  • Commercial Strategy/Operations
  • Reimbursement
  • Early/Expanded Access
  • Launch Excellence
  • Product Launch
  • Portfolio Management/Strategy
  • Marketing
  • Development
  • Patient Advocacy
  • Payer Strategy
  • Patient Support/Services

This event will also benefit consultants and solution providers who provide services in the rare disease space.

Connect and Network with Key Decision-Makers at the Rare Disease Summit

Whether you’re looking to generate quality leads, showcase thought leaders, or reinforce your brand, collaborate with us to identify custom sponsorship opportunities to help you reach your business objectives.

For more information about custom opportunities to reach a qualified audience, please contact us here.