Second Annual Ropes & Gray Rare Disease Forum

Mass Bio
700 Technology Square
Cambridge, MA 02139

The pre-Forum workshop will provide a primer on FDA regulation and healthcare laws relevant to the development and commercialization of rare disease products. Topics will include:

• FDA programs to support and advance the development of rare disease drugs
• Expedited development and approval pathways
• Product promotion and disease awareness communications
• Fraud and abuse laws

Panelists:

• Josh Oyster, Partner, Ropes & Gray LLP
• Alison Fethke, Counsel, Ropes & Gray LLP
• Becca Williams, Associate, Ropes & Gray LLP

Mass Bio
700 Technology Square
Cambridge, MA 02139

• Kellie Combs, Partner, Ropes & Gray LLP

Moderator:

• Greg Levine, Partner, Ropes & Gray LLP

Speaker:

• Pamela Gavin, CEO, NORD

This panel will explore both challenges and opportunities for clinical investigations of rare disease products, including considerations for:

• Patient recruitment
• Compassionate use
• Diversity, equity and inclusion in clinical trials
• Demonstrating effectiveness in rare diseases
• Research misconduct and data integrity issues

Moderator:

• Sarah Blankstein, Counsel, Ropes & Gray LLP

Panelists:

• David Peloquin, Partner, Ropes & Gray LLP
• Andrea DiFabio, Chief Legal Officer, Xenon Pharmaceuticals, Inc.
• Edward Neilan, M.D., Ph.D., Chief Medical and Scientific Officer, NORD

This panel will address drug pricing and market access considerations for rare disease products, highlighting recent trends and key issues impacting the US and EU markets. Panelists will explore a range of cross-border trends including:

• Growing government focus on cost-containment and access restrictions, including implications of the Inflation Reduction Act
• Evolving trends in pre-market and commercial strategy
• Market access and drug pricing across the product lifecycle, including potential role of real world evidence

Moderator:

• Greg Levine, Partner, Ropes & Gray LLP

Panelists:

• Margaux Hall, Partner, Ropes & Gray LLP
• Lincoln Tsang, Partner, Ropes & Gray LLP

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
• Key issues impacting valuation
• FTC enforcement trends

Moderator:

• Chris Comeau, Partner, Ropes & Gray LLP

Panelists:

• Amanda Austin, Partner, Ropes & Gray LLP
• Mike McFalls, Partner, Ropes & Gray LLP
• Joe Zenkus, Senior Vice President, Business Development & Strategic Alliances, Sarepta Therapeutics

This panel will focus on the compliance and enforcement risks faced by companies with rare disease products, including:

• Patient support programs
• Interactions with patients and patient advocacy groups
• Patient identification (including sponsored testing programs)
• Patient privacy considerations

Moderator:

• Beth Weinman, Counsel, Ropes & Gray LLP

Panelists:

• Eve Brunts, Partner, Ropes & Gray LLP
• Doug Hallward-Driemeier, Partner, Ropes & Gray LLP
• Shannon Kelley, Chief Compliance Officer, Madrigal Pharmaceuticals
• Sarah Whipple, Vice President, Global Chief Compliance Officer, Apellis Pharmaceuticals

The panel will discuss key regulatory risks and practical considerations for communications regarding rare disease products across the product life cycle. Panelists will cover a range of topics including:

• Disease awareness activities
• Use of patient ambassadors
• Proactive medical communications 

Moderator:

• Josh Oyster, Partner, Ropes & Gray LLP

Panelists:

• Kellie Combs, Partner, Ropes & Gray LLP
• Eric Rogers, Vice President, Ethics and Compliance, Alexion Pharmaceuticals, Inc. 
• Lynn Zuchowski, Vice President, Chief Counsel, Severe Genetic Disease, Bluebird Bio

Sulmona
608 Main St.
Cambridge, MA 02139