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Expanding Innovative Master Protocol Trial Designs to Cell and Gene Therapies for Rare Diseases
Session Chair(s)
Nancy Bradish Myers, JD
CEO and Founder
Catalyst Healthcare Consulting, United States
Master Protocols, including basket trials and umbrella trials, enable efficiencies in investigating multiple conditions or multiple treatments, respectively, under a single trial design. Applying Master Protocol trial designs to Cell & Gene therapies (CGTs) can streamline clinical and regulatory processes to speed the delivery of treatments to patients with rare diseases.
Learning Objective : Identify barriers to and solutions for leveraging Master Protocol clinical trial designs to study Cell & Gene Therapies for rare diseases.
Speaker(s)
Thought Leader Perspective
Philip (P.J.) Brooks, PHD
National Center for Advancing Translational Sciences (NCATS), NIH, United States
Deputy Director, Division of Rare Diseases Research Innovation
Innovator Perspective
Kiran Musunuru, MD, PHD, MPH
University of Pennsylvania, United States
Professor of Medicine
Researcher Perspective
Jeff Allen, PHD
Friends of Cancer Research, United States
President and Chief Executive Officer
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