Build a Watertight Go-to-Market Strategy for Rare Disease Therapies that Demonstrates Value to all Stakeholders, Improves Patient Access, and Closes the Gap Between Product Approval & Commercial Success

Welcome to the Commercial Excellence in Rare Disease Summit

Uniting to Increase Commercial Success & Access in Rare Disease

The Commercial Excellence in Rare Disease Summit is the definitive meeting point for drug developers focused on bridging the gap between scientific breakthroughs and successful commercialization. As rare diseases account for nearly half of all new FDA approvals, the need to turn these innovations into real-world viable, and accessible therapies has never been more urgent.

Following a transformative year marked by major acquisitions and increased regulatory support from the FDA’s Rare Disease Innovation Hub, the summit offers a timely platform to connect, collaborate, and advance commercialization strategies.

Join the leading minds across market access, pricing, reimbursement, commercial success, new product planning, launch planning, medical affairs, patient services and everyone across the commercial team. Dive into critical conversations around forecasting, evidence generation, and value demonstration, designed to maximize patient access and uptake.

Whether you’re launching your first orphan therapy or scaling established programs, this summit brings together the expertise needed to shape the future of rare disease commercialization and improve patient access.

2025 Event Guide

Most Recent Brochure Cover -Commercial Excellence in Rare Disease

World-Class Speaker Faculty Includes:

Andrew Komjathy - speaker - Commercial Excellence in Rare Disease

Andrew Komjathy

Chief Commercial Officer

Astria Therapeutics

Jeffrey Yu - speaker - Commercial Excellence in Rare Disease

Jeffrey Yu

Associate Director - HEOR Rare Disease Neurology

Alexion Pharmaceuticals

Kathleen Gilluly - speaker - Commercial Excellence in Rare Disease

Kathleen Gilluly

Patient Education & Rare Disease Liaison

Amicus Therapeutics, Inc.

Marsha Pelletier - speaker - Commercial Excellence in Rare Disease

Marsha Pelletier

Global Pipeline & Business Development Commercial Strategy

BioMarin Pharmaceutical

Companies attending in other rare disease event series:

Feeback from other rare disease events in this series:

“The collaborative and honest space was refreshing. No one did a commercial for their company. People wanted to learn and ask their peers questions. It’s a rare opportunity to put people together who are working in this nuanced space”

- Bayer

Bayer - White -Logo

“This was quite possibly one of the best conferences I have ever attended. The calibre of the presentations combined with the smaller conference size made it very easy to interact with speakers and attendees”

- Barth Syndrome Foundation

Barth Syndrome Foundation

Other Events in the Series:

Rare Disease Events:

Commercial Events: