Achieving Meaningful Regulatory and Clinical Outcomes for Patients: Strategies in Rare Disease Therapy Development

A collaborative workshop designed to examine practical issues in the structuring of rare diseases therapeutic development programs.

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Session 3: Impact of Early Patient Engagement

Session Chair(s)

Steven L. Roberds, PHD

Chief Scientific Officer, Tuberous Sclerosis Alliance, United States

In this session we will address both the value of and the challenges in engaging patients early-on in the clinical development process. Experts will review the specific concepts and principles relating to overcoming barriers and demonstrate how patient engagement can be successfully interwoven in regulatory and clinical programs.

Speaker(s)

Addressing the Challenges of Early Patient Engagement

President, Acid Maltase Deficiency Association (AMDA), United States

Putting it All Together

Director, Expanded Access Programs & Policy, Former FDA, Patient Liaison, GE2P2 Global Foundation, United States

Interactive Panel Discussion

Executive Director, Myotonic Dystrophy Foundation, United States

Joel Beetsch, PHD

Vice President, Global Patient Advocacy, Celgene Corporation, United States

Tiffany House, JD