Tuesday, June 20 • 10:30am - 11:45am
#222: Serving the Patient Who Needs Early Access to Treatments and Achieving Meaningful Outcomes

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Component Type: Session
Level: Intermediate
CE: ACPE 1.25 Knowledge UAN: 0286-0000-17-545-L04-P; CME 1.25; IACET 1.25; RN 1.25

As most rare diseases have no approved therapies, these patient populations are frequently desperate for any kind of treatment. As a result, they request earlier and more extensive access to investigational drugs before many of the safety and dosing questions have been addressed. This could result in erroneously high adverse event profiles or subjective benefits that skew the development program. How do we better define how to broaden access while simultaneously maintaining rigid control of the data?

Learning Objectives

Discuss the importance of collaboration between key stakeholders in the for profit and non-profit sectors in the development of Early Access therapeutic approaches for rare disease.


Stella C.F. Blackburn


So Near Yet So Far: How Do We Get Early Access for Patients with Unmet Medical Needs and Life-limiting Diseases?
Stella C.F. Blackburn

Aligning Objectives of Drug Development and Patient Access
Richard Scheyer, MD

Reagan-Udall Foundation for the FDA Expanded Access Navigator
June S. Wasser, MA

avatar for Stella Blackburn

Stella Blackburn

Vice President, Global Head of Early Access & Risk Management, RWI, QuintilesIMS
Dr Stella Blackburn is currently VP, Global Head of Early Access & Risk Management at QuintilesIMS. She joined QI in April 2014 after more than 25 years working in PhV and pharmacoepi in industry and the European Medicines Agency. Stella is a Fellow of the International Society of... Read More →

avatar for Richard Scheyer

Richard Scheyer

Vice President, Medical, Medpace
Dr. Richard Scheyer is VP Medical at Medpace. Prior to Medpace, he led Experimental Medicine, BM, and PGx functions at Daiichi Sankyo. He served in leadership roles at Sanofi-Aventis and CMO at Neurotrope Bioscience. Dr. Scheyer received his BS Physics from Stanford, MD from SUNY... Read More →
avatar for June Wasser

June Wasser

Executive Director, Reagan-Udall Foundation For the FDA
June Wasser is the Executive Director of the Reagan-Udall Foundation for the FDA and in this role leads the direction of the Foundation and implementation of its programs. She is responsible for building strategic relationships throughout the public-private sector with the goal of... Read More →

Tuesday June 20, 2017 10:30am - 11:45am
S505ab McCormick Place 2301 South Martin Luther King Jr. Drive, Gate 4 Chicago, IL 60616