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DIA 2017 Annual Meeting

Serving the Patient Who Needs Early Access to Treatments and Achieving Meaningful Outcomes

Session Chair(s)

Stella Blackburn, MD, MA, MSc, FFPM, FISPE, FRCP

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)

Stella Blackburn, MD, MA, MSc, FFPM, FISPE, FRCP

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

  • Strategy
  • Consultant, United Kingdom
Richard Scheyer, MD

Aligning Objectives of Drug Development and Patient Access

Richard Scheyer, MD

  • Vice President, Medical
  • Medpace, United States
June Wasser, MA

Reagan-Udall Foundation for the FDA Expanded Access Navigator

June Wasser, MA

  • Executive Director
  • Reagan-Udall Foundation For the FDA, United States

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