Workshop 10: 2018 Scientific Workshop

Conceptualizing an FDA Rare Disease Center of Excellence

Thursday, September 13^th, 2018
8:30 a.m. until 4:00 p.m.
Willard Intercontinental Hotel in Washington, DC
1401 Pennsylvania Ave NW, Washington, DC 20004

Presentations

Julia Jenkins – Introduction

Christina Hartman – EveryLife Foundation Programs

Janet Woodcock – FDA CDER Re-Organization and Impact on Rare Diseases

Amy McKee, MD – FDA Oncology Center of Excellence Successes

Wilson Bryan, MD – Gene Therapy for Rare Diseases

Annetta Beauregard, MS, MBA – Innovative Approaches for Rare Disease: Cystic Fibrosis Case Study

Annie Kennedy – Integrating PFDD Data within Regulatory Review: Progress & Existing Opportunities

Emil Kakkis, MD, PhD – The Challenges of Managing Heterogeneity in Rare Diseases

Ronald Crystal, MD – Regulatory Challenges for Batten’s Disease Treatments

Rosa Bacchetta, MD – IPEX Syndrome Gene Therapy Case Study

Brad Glasscock – PKU Case Study: Use of Surrogate Endpoints

Ralph Kern, MD, MHSc – ALS Case Study: Clinical Trial Designs for Small Patient Populations

Alison Skrinar, PhD – X-Linked Hypophosphatemia Case Study

Mathias Schmidt, PhD – MPS-1 Case Study: Clinical Trial Designs for FDA & EMA Approvals

Dunni Odumosu, MS – Application of Current FDA Statute: A Fabry Case Study

Frank Sasinowski, MS, MPH, JD – FDA Center of Excellence for Rare Diseases Potential Model

Panel Discussion

The rapid pace of innovation in the biotechnology industry has created a number of opportunities to bring cutting edge treatments to patients with rare diseases. Despite the increased interest and investment in rare disease product development, many roadblocks in the regulatory process still threaten the pipeline of lifesaving therapies.

The EveryLife Foundation for Rare Diseases will convene its 10^th annual Scientific Workshop to explore this topic. The goal of the workshop will be to gather key thought-leaders from industry, the FDA, and patient organizations to discuss potential models, best practices and the pathway forward.

The Scientific Workshop, with input from stakeholders across the rare disease community, will prioritize finding actionable takeaways to the following questions:

• How do we leverage the expertise across FDA review divisions so that they can harmonize regulatory approaches related to the complexities of clinical trial designs and endpoints for small heterogeneous patient populations?
• How can we increase collaboration with international regulatory agencies to allow for clinical trial designs to be accepted across multiple agencies, especially when no established regulatory pathways to approval exists?
• What can be learned from the successes and challenges of the FDA Oncology Center of Excellence?

When possible, companies will be invited to share their thoughts and expertise. Senior leadership from FDA will also be invited to present an overview of possible resources and guidance available to industry and patient organizations.

Sponsorships are available with guaranteed seating. Our sponsors make it possible for us to open this event to the public free of charge. Reservations are limited to 125 attendees to provide for productive collaboration and discussion. 

Thank you to our 2018 Sponsors