Please join a cross-disciplinary group of Ropes & Gray attorneys and industry experts as they explore the regulatory framework, compliance risks, transactional considerations, and other key topics related to the development and commercialization of rare disease products.
Sessions will cover:
- Go-to-market strategies, including regulatory incentives, market access, and drug pricing
- Clinical trials
- Licensing and collaboration considerations
- Post-market compliance risks
* We will be offering a pre-forum workshop for attendees interested in an introduction to FDA regulatory and health care issues relevant to rare disease products.
| DATE | June 14, 2023 |
| TIME |
10:30AM - 5:30PM |
| LOCATION | Royal Sonesta 40 Edwin H Land Blvd. Cambridge, MA 02142 |
PRE-FORUM REGISTRATION
► 10:00AM - 10:30AM
PRE-FORUM WORKSHOP
► 10:30AM - 12:00PM
Rare Disease 101: An Introduction to FDA Regulation and Health Care Laws for Rare Disease Products
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:
- FDA designations for rare diseases and orphan drugs
- Expedited development and approval pathways
- Product promotion and disease awareness communications
- Fraud and abuse
Speakers:
Alison Fethke, Counsel, Ropes & Gray LLP
Joshua Oyster, Partner, Ropes & Gray LLP
Rebecca Williams, Associate, Ropes & Gray LLP
CLE provided for CA, IL and NY.
FORUM | REGISTRATION AND LUNCH | 12:00PM - 1:00PM
PROGRAM* | 1:00PM - 5:30PM
► 1:00PM - 1:05PM
Welcome Remarks
Kellie Combs, Partner, Ropes & Gray LLP
Greg Levine, Partner, Ropes & Gray LLP
► 1:05PM - 2:15PM
Go-to-Market Strategies for Rare Disease Products
This panel will address strategies for the development and commercialization of rare disease products, including regulatory incentive programs, payer coverage, and drug pricing issues in the US and EU. This program will cover a range of hot topics including:
- Orphan drug designation and exclusivity
- Accelerated approval
- Market access and payer coverage considerations
- Drug pricing issues related to launch and commercial strategy
Moderator:
Kellie Combs, Partner, Ropes & Gray LLP
____________________________________
Speakers:
Margaux Hall, Partner, Ropes & Gray LLP
Lincoln Tsang, Partner, Ropes & Gray LLP
► 2:15PM - 3:00PM
Clinical Trials for Rare Disease Products
This panel will highlight key research and development issues for companies investigating products intended to treat rare diseases, including considerations for:
- Clinical trial design and execution
- Use of real-world evidence
- Clinical trial diversity
- Privacy concerns for study participants
Moderator:
Greg Levine, Partner, Ropes & Gray LLP
____________________________________
Speakers:
Mark Barnes, Partner, Ropes & Gray LLP
Jason Carter, Global Head of Legal at KITE Pharma
Edward Neilan MD, PhD, Chief Medical and Scientific Officer of NORD
► 3:30PM - 4:15PM
Considerations in Licensing and Collaborations Involving Rare Disease Products
This panel will address the key focus areas when considering any potential license or collaboration involving rare disease products, including gene therapy products, and review issues that have posed major concerns in such transactions. Our discussion will include:
- Collaboration structures, co-commercialization and royalty buy-ups
- Territorial licensing and treatment of arising IP
- Allocation of development, manufacturing, regulatory and commercialization responsibilities
- Regulatory issues related to product manufacturing and quality
- Development and approval of companion diagnostics
Moderator:
Joshua Oyster, Partner, Ropes & Gray LLP
____________________________________
Speakers:
Hannah England, Partner, Ropes & Gray LLP
Melissa Rones, Partner, Ropes & Gray LLP
Marc Rubenstein, Partner, Ropes & Gray LLP
Paige Goodwin, VP, Head of Transactions and Alliance Management at Vertex
► 4:30PM - 5:15PM
Compliance Risks for Rare Disease-Focused Life Sciences Companies: Patient Engagement, Patient Support, and Product Promotion
This panel will discuss the promotional activities that pose significant compliance and enforcement risk for companies with rare disease products, as well as unique challenges related to product promotion, including:
- Identifying patients for therapy
- Interacting with patient advocacy organizations
- Implementing patient support programs
- Product promotion and selection of distribution channels
Moderator:
Beth Weinman , Partner, Ropes & Gray LLP
____________________________________
Speakers:
Eve Brunts, Partner, Ropes & Gray LLP
Ryan Brown, Executive Vice President, Chief General Counsel at Sarepta Therapeutics
Sue Dyer, Head, Deputy General Counsel & Global Compliance Officer at Amylyx
NETWORKING RECEPTION TO FOLLOW
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