Biopharma (includes Biologics)

Orphan Precedent and the Continued Evolution of the Orphan Drug Framework

Monday, September 14, 2020

10:00 AM – 11:30 AM EDT

Session Leader(s)

Mark Rutter

Senior Director, Regulatory Affairs
Biogen - ENTERPRISE

Slides

Speaker(s)

KL

Kristina Larsson, MSc

Head of Orphan Drugs
European Medicines Agency

Slides
JM

Janet Maynard, MD, MHS

Director of Office of Orphan Products Development
US Food and Drug Administration
Martine Zimmermann, PharmD

SVP, Head of Global Regulatory Affairs
Alexion Pharmaceuticals

Slides

The framework for the development and incentives for rare disease research is never far from the spotlight! This session provides an opportunity to hear directly from and to interact with key FDA and EMA leaders in the orphan drug and rare disease space. Hear directly from Agency and Industry experts on the latest trends and initiatives relating to rare diseases in the US and Europe as we explore a range of topics from Rare Pediatric Disease Designation to the just published evaluation of EU Regulation 141/2000. Prior to an open discussion, we will explore how stakeholders can leverage regulatory dialogue in certain jurisdictions to advance orphan development plans, how to plan regulatory engagement effectively and how to address divergent advice from authorities, often with an absence of any disease specific guidance.

Learning Objectives:

Understand the evolving regulatory landscape, new and revised initiatives from the FDA and EMA.
Design regulatory strategies that benefit from adequate regulatory engagement in key regions, to support single global development plans in rare diseases.
Engage with key regulators and industry experts, share perspectives and foster dialogue in this ever-evolving space.