Innovative Clinical Trial Designs

Nontraditional trial designs are being used across therapeutic areas to address heterogeneity, small sample sizes, and evolving scientific understanding. This session will provide an overview of nontraditional clinical trial structures, such as umbrella, basket, and pragmatic trial designs, highlighting their applicability to rare disease research and their potential to improve efficiency, generalizability, and evidentiary yield.

Moderator: Annette Bakker, CEO, Children’s Tumor Foundation

Panelists:

• Jaishri Blakeley, MD, Professor of Neurology, Neurosurgery and Oncology, Johns Hopkins University, a NORD Rare Disease Center of Excellence
• Gwen Nichols, MD, EVP, Chief Medical Officer, Blood Cancer United
• Amy Nicole Nayar, PharmD, MS, Vice President, Medical Affairs, Lundbeck

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