Speaker Profile

M.D., Ph.D., Senior Scientific Director, Janssen Research & Development

Dr. Walton is co-Chair of QSTEPS at Janssen and advises drug development teams on clinical study design promoting coherent drug development across the breadth of Janssen’s clinical research. He has been named a Janssen Fellow and a Fellow of the Society for Clinical Trials.
With degrees from the University of Chicago and Neurology training he joined NIH researching neurotransmitter responses. Dr. Walton joined CBER at FDA in 1993 working on biological product development for neurology, adding additional clinical areas as Branch Chief. As Division Director he had clinical oversight for all non-oncology uses of biological proteins. Moving to the Office of Policy in the Commissioner’s Office gave involvement in agency-wide issues. He joined the Office of Translational Sciences in CDER as the Associate Director for Translational Medicine, fostering science and policies supporting innovations, including areas of biomarkers, clinical outcome assessments, clinical study design methods, and rare disease therapeutics.


Natural History: Essential for Developing Rare Disease Therapies
Studies in rare diseases often have no successful prior model. Poor design in endpoint selections, how and when to evaluate them, the specific study population, etc. can lead to a study failing to demonstrate efficacy. How deeply understanding the natural history is essential to strong study design will be illustrated.

Session Abstract – PMWC 2017 Silicon Valley

Session Synopsis: Rare Diseases are genetic, severe and life threatening; by definition they impact 200,000 patients or less. In 2015, 47% of the medications approved by the FDA were for rare diseases. We will explore how this trend has changed drug development: increasing collaboration, advancing personalized medications and personalized disease management tools.

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