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.

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:

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

See All High-Impact Topics Planned

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

Browse the Full 2025 Agenda

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!

View Full List of Inspiring Speakers

Request the 2025 Agenda

Their Passion is RARE: Meet the 2025 Rare Disease Conference Thought Leaders

Attendees will hear directly from those who have designed and led innovative approaches to complex challenges in driving curative progress

Larry Bressler

Senior Vice President, Global Value& Access, Rare Diseases

Chiesi

Aman Khera

Innovation Advisory Council Member

Vation Ventures

Alycia Shilton Lloyd

Executive Director, Hematology-Oncology, Patient Innovation & Engagement, Global Medical & Scientific Affairs

Merck

Kari Luther Rosbeck

President & CEO

TSC Alliance

View Complete Speaker Lineup

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

Tuberous Sclerosis Alliance

The Rare Disease Innovation Summit provided the opportunity for cross communication between industry, nonprofits and vendors who bridge the gap between the two. From presentations to panels, every attendee walked away with new information and resources to help improve the quality of life for the rare disease community. Thank you for making this possible CBI!

Senior Vice President

Global Business Operations & External Affairs

Horizon Pharma

The inaugural Rare Disease Innovation Conference brought together multidisciplinary stakeholders to discuss important issues challenging the rare disease ecosystem. The contributions by the audience accelerated thinking how to address and eventually resolve key access related issues impacting people in desperate need of therapeutic options.

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. CBI’s 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

Tuberous Sclerosis Alliance

The Rare Disease Innovation Summit provided the opportunity for cross communication between industry, nonprofits and vendors who bridge the gap between the two. From presentations to panels, every attendee walked away with new information and resources to help improve the quality of life for the rare disease community. Thank you for making this possible CBI!

Senior Vice President

Global Business Operations & External Affairs

Horizon Pharma

The inaugural Rare Disease Innovation Conference brought together multidisciplinary stakeholders to discuss important issues challenging the rare disease ecosystem. The contributions by the audience accelerated thinking how to address and eventually resolve key access related issues impacting people in desperate need of therapeutic options.

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. CBI’s 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.

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.

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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.

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