3rd Annual Ropes & Gray Rare Disease Forum

Kimpton Marlowe Hotel
25 Edwin H. Land Blvd
Cambridge, MA 02141

The pre-forum workshop will provide a high-level introduction to FDA regulation and health care laws relevant to the development and commercialization of rare disease products and provide a primer for topics that will be substantively addressed during the panels. Topics will include:

• Orphan drug regulation
• Expedited development and approval pathways
• Product promotion
• Fraud and abuse

Kimpton Marlowe Hotel
25 Edwin H. Land Blvd
Cambridge, MA 02141

We will kick off this year’s Forum with a discussion of the latest legal and regulatory developments, legislative proposals, and policy and program updates impacting the rare disease community, plus an overview of what may be on the horizon in 2025.

Details will be announced soon.

This panel will address drug pricing and market access considerations for rare disease products, highlighting recent trends and key issues impacting the U.S., UK/EU and APAC markets.

This panel will highlight deal trends and considerations for companies focused on the development and commercialization of rare disease products. The panel will cover:

• Notable transactions in the rare disease space;
• Considerations for deal structures.

This panel will discuss current and potential future uses of AI-based tools in the rare disease product lifecycle from therapeutic candidate identification, to nonclinical and clinical development, diagnosis, and treatment. Panelists will discuss emerging use cases for AI in this area as well as the evolving legal and regulatory landscape.

This panel will address current legal and regulatory topics in the clinical development and approval of rare disease products, including decentralized clinical trials, real-world evidence, clinical trial diversity, companion diagnostics, laboratory developed tests, and accelerated approval reforms.

This panel will explore opportunities, risks, and best practices associated with a variety of patient engagement tactics and patient support initiatives, including:

• Patient identification, including sponsored testing programs;
• Communications with patients and patient advocacy organizations, including prior to FDA approval;
• Use of patient-focused field roles;
• Hub services.

Geppetto
100 N First Street
Cambridge, MA 02141