Day 2, Wednesday, March 19th, 2025Wednesday, 19 March 2025 - ET (Eastern Time, GMT-05:00)

The title description can be changed to “Hear from the FDA directly on current rare disease initiatives, such as the Innovation Hub, START Program, and the Rare Disease Endpoint Advancement Pilot Program.”

The pricing of rare disease therapies has become increasingly complex and controversial. Unpack the factors shaping the evolving pricing landscape, including the impact of patient access initiatives, payer pressures and the role of value-based pricing models.

• Strategize on the best approaches to commercialize innovative therapies, such as regenerative medicines, including the evolving reimbursement landscape and challenges surrounding research, pricing and access

• Discuss strategies for defining and managing patient expectations and delve into the importance of advocacy groups and partnerships

• Highlight successful examples of patient-focused commercialization initiatives and unique techniques in engaging the patient community

Launching a rare disease product requires a unique and strategic approach. Dive into critical lessons learned from both successful and challenging launches as industry experts

share their insights on overcoming hurdles, optimizing commercialization strategies and driving long-term success.

• Adress common pitfalls in rare disease commercialization

• Unpack the evolving reimbursement landscape and learn strategies for ensuring patient access

• Understand the importance of leveraging real-world data to optimize value and outcomes

• Identify the key metrics and insights to measure and optimize commercial performance

The rare disease landscape is often associated with rapid innovation and strategic partnership. Mergers and acquisitions can offer significant opportunities for growth and expansion, but they can also present complex challenges.

• Explore the critical factors needed for a successful company integration

• Benchmark on ways to preserve the unique culture and expertise of smaller companies whilst leveraging the resources and reach of larger organizations

• Discover strategies for maintaining patient relationships amidst the transition, further accelerating your drug development and building a sustainable future for the combined entity

Gain invaluable insights into the minds of those who fund rare disease innovation. Hear from venture capitalists and manufacturer business development executives on the critical factors that drive investment decisions in the rare disease space. Learn about the key criteria they look for in companies, the importance of clinical and non-clinical data and the strategic partnerships that can accelerate development and commercialization. Discover how to position your rare disease organization for investment success by understanding the challenges and opportunities from an investor and business development point of view.

Uncover open funding opportunities and resources offered by various organizations for rare disease investigators of all career stages and backgrounds.

• Understand the role of patient advocacy groups in funding, including the economic burden of rare disease on patients and families

• Discuss development strategies to assist in funding, including grassroots efforts and unique partnerships

• Dissect the challenges and opportunities associated with funding and how to build a sustainable development model

After hearing from all the above stakeholders, take this opportunity to end your day with a funding brainstorming session. Engage in a facilitated discussion to not only expand upon the funding challenges that you may be facing but also the potential solutions to solve them.