4th Annual Ropes & Gray Rare Disease Forum | News & Events

Please join a cross-disciplinary group of Ropes & Gray attorneys and industry experts as we explore the latest developments and challenges in the regulatory, compliance, and transactional landscape for the development and commercialization of rare disease products.

We are honored to feature Dr. Richard Pazdur—former Director of FDA’s Center for Drug Evaluation and Research and Oncology Center of Excellence—as our keynote speaker. Over the course of his 26-year FDA career, Dr. Pazdur helped to transform modern cancer drug development by streamlining and strengthening approval processes for innovative therapies. Dr. Pazdur will share insights regarding the evolving FDA policies and practices that are top of mind for rare disease product developers and the broader biopharmaceutical industry.

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

Agenda

11:00 AM-12:00 PM - Registration & Lunch

12:00 PM-12:05 PM - Welcome & Opening Remarks

12:05 PM-12:50 PM - Panel 1: R&D Challenges in Rare Disease

This panel will address current legal and regulatory topics in the clinical development of rare disease products, including recent efforts to modernize the regulation of clinical trials, artificial intelligence (“AI”) and digital tools in clinical development, types of support provided to study participants in clinical trials, and challenges in gene therapy product development.

Moderator:

Sarah Blankstein
Counsel, Ropes & Gray

Speakers:

David Peloquin
Partner, Ropes & Gray

Lakshmi Nayak
MD, Director of the Center for CNS Lymphoma at Dana-Farber Cancer Institute

Ed Neilan
MD, PhD, Chief Medical and Scientific Officer, NORD

1:00 PM-2:00 PM - FDA Rare Disease Policy and Regulatory Priorities: A Conversation with Dr. Richard Pazdur

This fireside chat with Dr. Richard Pazdur, former Director of the FDA Center for Drug Evaluation and Research and the Oncology Center of Excellence, and Greg Levine, Chair of Ropes & Gray’s Life Sciences Regulatory & Compliance Practice Group, will explore current perspectives on FDA rare disease policy and the agency’s evolving regulatory priorities.

Interviewer:

Greg Levine
Partner, Ropes & Gray

Keynote:

Dr. Richard Pazdur
Former Director of FDA’s Center for Drug Evaluation and Research and Oncology Center of Excellence

2:10 PM-3:25 PM - Panel 2: Regulatory Outlook for Rare Disease

This panel will address the latest FDA regulatory and leadership developments, policy and program updates impacting the rare disease community, considerations for navigating the shifting landscape under the current FDA, and an overview of what may be on the horizon in 2026 and beyond.

Moderator:

Josh Oyster
Partner, Ropes & Gray

Speakers:

Kara Berasi
PharmD, MS, CEO, Haystack Project

Douglas Hallward-Driemeier
Partner, Ropes & Gray

Beth Weinman
Counsel, Ropes & Gray

3:30 PM-4:15 PM - Panel 3: A Global Approach to Drug Pricing and Market Access

The panel will address drug pricing and market access considerations for rare disease products, highlighting recent trends and key issues impacting the US, EU, and China markets. Topics include regulatory implications of most favored nation drug pricing, evolving trends in pre-market and commercial strategy, and market access across the product lifecycle.

Moderator:

Rebecca Williams
Senior Associate, Ropes & Gray

Speakers:

Lincoln Tsang
Partner, Ropes & Gray

Katherine Wang
Partner, Ropes & Gray

4:20 PM-5:00 PM - Panel 4: Bridging Personal Experience and Precision Medicine in Rare Disease

This panel will explore how personal experience with rare disease can shape professional pathways and influence broader efforts to advance genomic-based approaches to diagnosis and treatment. The discussion will highlight the role of patient perspectives in driving innovation, the evolving landscape of genomic medicine, and how emerging approaches are informing more precise and individualized care. Panelists will also reflect on opportunities and challenges in translating scientific progress into meaningful impact for rare disease communities.

Moderator:

Evan Tallmadge
Senior Associate, Ropes & Gray

Speakers:

Allyson Berent
Chief Development Officer, AS2 Bio
Co-Director, Angelman Syndrome Biomarker and Outcome Measure Consortium (ABOM)
Chief Science Officer, Foundation for Angelman Syndrome Therapeutics (FAST)

Hilary Eaton
PhD, Chief Business Officer, Profluent Bio