DIA 2017 Annual Meeting

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.

Chair

Stella C.F. Blackburn

Speaker

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