Second Annual Ropes & Gray Rare Disease Forum

After a successful Forum in 2023, Ropes & Gray is excited to announce that we are returning to Cambridge, MA this May for our Second Annual Ropes & Gray Rare Disease Forum. Please join a cross-disciplinary group of Ropes & Gray attorneys and industry experts as they explore new developments in the regulatory framework, compliance risks, transactional considerations and other key topics related to the development and commercialization of rare disease products.

The Forum will take place in person at Mass Bio (700 Technology Square, Cambridge, MA). Networking reception at Sulmona to follow.

We look forward to seeing you in May!

For additional information, click here.

AGENDA

PANEL DESCRIPTIONS

Rare Disease 101: An Introduction to FDA Regulation and Healthcare Laws for Rare Disease Products
10:30AM-12:00PM
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

Lunch and Fireside Chat
12:15-1:00PM

Moderator:
Greg Levine, Partner, Ropes & Gray LLP

Speaker:
Pamela Gavin, Executive Vice President, NORD

Clinical Trials for Rare Disease Products
1:00-1:45PM
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

Drug Pricing & Market Access – Global Trends
2:00-2:45PM
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

Deal Trends and Considerations for Rare Disease Products and Companies
2:45-3:30PM
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

Compliance Risks for Rare Disease-Focused Life Sciences Companies
3:45-4:30PM
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

Advertising and Promotion for Rare Disease Products
4:30-5:15PM
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 at Alexion Pharmaceuticals, Inc. 
Lynn Zuchowski, Bluebird Bio, Vice President, Chief Counsel, Severe Genetic Disease

**Networking Reception at Sulmona**
5:30PM+