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 accelerate therapeutic innovation and drive commercial success in areas of unmet medical need. You’ll generate solution-focused approaches surrounding launch planning, market access, reimbursement, data utilization and strategic partnerships, and gain actionable insights to optimize rare disease commercialization and elevate patient impact.

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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. Benefit from high-impact content from experts from across the rare disease industry:

Strategic Innovation for Rare Disease Commercialization Success

Explore the cutting-edge strategies and technologies that are transforming rare disease commercialization and ensuring long-term patient access. Learn from industry leaders on how to:

Launch rare disease therapies with precision using proven pre-launch and post-launch planning strategies
Leverage AI and advanced data analytics to improve patient identification engagement, and adherence
Navigate competitive and underserved markets with tailored go-to-market strategies for rare conditions
Align cross-functional teams across commercial, market access and patient support for scalable success
Drive innovation in patient services with high-touch models and personalized patient support programs

Whether you're leading a new product launch, refining your access strategy or scaling patient support, the Rare Disease Summit delivers the strategic insights and peer-tested approaches that you need to succeed in the rare disease space.

Operational Strategy and Market Access Alignment for Rare Disease Leaders

Gain strategic clarity on how to align internal teams, payer engagement and regulatory pathways to drive rare disease therapy success. Hone in on the operational execution and access strategies that matter most to commercial leaders:

Engage payers and regulators early to shape favorable access and reimbursement outcomes
Navigate global vs. U.S. market dynamics with tailored distribution and commercialization models
Design high-impact patient support programs that meet the unique needs of rare disease populations
Break down silos across commercial, medical and patient services teams to accelerate performance

Whether you're leading commercial strategy, market access or patient services, the Rare Disease Summit delivers the frameworks and insights needed to optimize execution and ensure therapies reach the right patients—at the right time.

Collaborative Leadership Across the Rare Disease Ecosystem

Experience a uniquely integrated summit that brings together the full spectrum of rare disease stakeholders—from industry leaders to patient advocates and regulatory experts. Understand the collective power of collaboration to drive innovation, access and impact:

Participate in sessions across the Rare Disease Summit as well as the Hub East Congress and Patient Assistance and Access event
Engage with the top pharmaceutical companies that shaping rare disease strategy and commercialization
Collaborate with patient advocacy organizations to amplify patient voice and improve support programs
Gain insights from FDA, NIH, and health systems on policy, access, and innovation in rare disease

The Rare Disease Summit is the only event on the market where you’ll hear directly from industry, advocacy, government representatives and thought leaders from across the patient access industry – giving you the holistic perspective needed to lead effectively in this complex space.

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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: