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Orphan Drug Development in Personalized Medicine
Session Chair(s)
Philip (P.J.) Brooks, PhD
Deputy Director, Division of Rare Diseases Research Innovation
National Center for Advancing Translational Sciences (NCATS), NIH, United States
For many years, drug development in oncology focused on treatments for tumors in specific parts of the body. However , with the advent of genomic sequencing, it has become clear that anatomically different tumors can have the same mutation that result in susceptibility to a drug, and that it is those mutations, rather than the anatomical site of the tumor, that should drive patient selection in clinical trials. This same approach, grouping patients based on molecular targets rather than clinical phenotypes, has broader implications for orphan drug development beyond oncology. In this session, the speakers will cover a variety of topics related to this issue, including rare mutation oncology trials, innovative study designs, and perspectives on orphan drug development in the age of personalized medicine.
Learning Objective : Discuss insights for overcoming barriers to successful precision medicine development in rare mutation oncology trials; Describe different types of study designs for orphan drug development in personalized medicine; Identify different perspectives on orphan drug development in the age of personalized medicine.
Speaker(s)
Rare Mutation Oncology Trials: Learnings Over Barriers and Enablers for Successful Precision Medicine Development
Angela Qu, MD, PhD
Parexel, United States
Vice President, Translational Medicine
Innovative Study Designs for Better Outcomes
Richard Huckle, MSc
Osiris Pharma, United Kingdom
Regulatory and Development Consultant
Adapting the Oncology Basket Trial Model to Rare Diseases: Grouping Patients by Underlying Etiology Rather Than Clinical Characteristics to Accelerate Clinical Trials
Philip (P.J.) Brooks, PhD
National Center for Advancing Translational Sciences (NCATS), NIH, United States
Deputy Director, Division of Rare Diseases Research Innovation
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