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Speaker 

Emily Morris, PhD
Mathematical Statistician
Office of Biostatistics
Center for Drug Evaluation and Research 
U.S. Food and Drug Administration

About the Speaker

Dr. Emily Morris is a mathematical statistician in the Division of Biometrics IV in the Office of Biostatistics at the FDA’s Center for Drug Evaluation and Research. She provides statistical support for the Division of Rare Diseases and Medical Genetics, which reviews drugs and biologics intended to treat inborn errors of metabolism. Prior to joining the FDA, Dr. Morris received her PhD in Biostatistics from the University of Michigan.  

About the Presentation

The first portion of this talk will focus on introducing some of the key challenges of designing a phase 3 trial in a rare disease setting and the ways statistics may be used to inform design decisions, and the second portion of the talk will focus on the ways adaptive design may be used to address the lack of information often faced at the design phase.

Learning Objectives

1. Identify at least three key challenges with the design of a pivotal study in a very rare disease.
2. Recognize areas in which statistical expertise can inform decisions related to the challenges with designing a pivotal trial in a rare disease.
3. Recognize what type of adaptive design may be useful to address challenges in rare disease trials

To Register

Non-FDA employees must register in:  

After MS Teams Webinar registration, you will receive a link via email to access the live webinar. You must log in with the username and password created when you registered.  Please pre-register at least two days before the event to ensure you receive the access link and outlook invitation for the session. 

For technical assistance please contact: Rakesh.Raghuwanshi@fda.hhs.gov

Continuing Education (CE) Credit

Participants wishing to obtain CE credit must first register for the Grand Rounds using the MS Teams Webinar Registration link above. As CE credit is being offered through the Johns Hopkins University School of Medicine, those wishing to obtain CE credit must create a user profile on the Hopkins Cloud CME platform. If you have never had a CME certificate/credits awarded and need a user profile created, please log onto https://hopkinscme.cloud-cme.com and click the “Sign Up Now” button. DO NOT create a new account if you have already received credit for Johns Hopkins CME activities at any time. Click on Forgot Password if you already have an email on file with the JHU OCME office. Instructions on how to claim the CE credit will be provided during the Grand Rounds presentation, but participants must already have a Hopkins Cloud CME user profile before the presentation.