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Serving the Patient Who Needs Early Access to Treatments and Achieving Meaningful Outcomes
Session Chair(s)
Stella Blackburn, MD, MA, MSC, FFPM, FISPE, FRCP
Strategy
Consultant, United Kingdom
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 Objective : 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.
Speaker(s)
So Near Yet So Far: How Do We Get Early Access for Patients with Unmet Medical Needs and Life-limiting Diseases?
Stella Blackburn, MD, MA, MSC, FFPM, FISPE, FRCP
Consultant, United Kingdom
Strategy
Aligning Objectives of Drug Development and Patient Access
Richard Scheyer, MD
Medpace, United States
Vice President, Medical
Reagan-Udall Foundation for the FDA Expanded Access Navigator
June Wasser, MA
Reagan-Udall Foundation For the FDA, United States
Executive Director