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.

Request 2026 Agenda

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.

President & CEO

Canadian Organization of Rare Disorders

Most amazing were the voices from the patient community who are driving drug discovery and access. What some doing for a few benefits us all in this rare space. The Rare Disease Summit was a prime example of how the patients are critical to informing the research and development dialogue but also vice versa. We need to know the innovations planned and in progress.

President & CEO

Canadian Organization of Rare Disorders

Most amazing were the voices from the patient community who are driving drug discovery and access. What some doing for a few benefits us all in this rare space. The Rare Disease Summit was a prime example of how the patients are critical to informing the research and development dialogue but also vice versa. We need to know the innovations planned and in progress.

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. The 2025 attendees benefitted from high-impact content from experts from across the rare disease industry:

Innovative Approaches to Amplify Collaboration and Advance Commercialization

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

Unpack the factors shaping the Rare Disease pricing landscape and its respective implications on patient access
Strategize on best practices in overcoming hurdles, optimizing commercialization strategies and driving long term success for Rare therapeutics
Navigate the complexities of innovative therapies, including commercialization approaches and managing patient expectations
Take a practical look at using AI technology in this unique, underserved landscape

Dedicated Think Tank to Strategize on All Things Rare Disease Funding

Engage in dedicated sessions to intimately brainstorm with your peers on innovative ideas surrounding the different aspects of Rare Disease funding:

Mergers & Acquisitions in Rare Disease – Balancing Growth with Preservation
Cracking the Code – Investor Insights into Rare Disease Funding Opportunities
Funding Opportunities – A Panel Discussion with NIH and Other Organizations
Industry’s Stake in Rare Disease Investment
Funding the Fight – Patient Power in Driving Rare Disease Investment
Funding Think Tank

Bringing Together the Key Perspectives in Rare Disease

The all-plenary design of this event enables for more meaningful interaction, higher level networking and closer-up learning. Hear from different stakeholders within the Rare ecosystem for the most holistic landscape view.

Industry: Chiesi, UCB, Merck, Mahzi, Mirum Pharmaceuticals, Pharavis, Sumitomo, Vation Ventures, Oxford Angel Fund
Advocacy: TSC Alliance, Foundation for Sarcoidosis Research, Isabella Santos Foundation, National Eczema Association
Government/Academia/Health Systems: USC Schaeffer Center for Health Policy and Economics, OND/CDER/FDA, NIH, Baptist Health System

And more to come!

Request 2026 Agenda

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: