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Thursday, June 18 • 9:30am - 10:30am
#413 L: Orphan Drug Development in Personalized Medicine

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Component Type: Session
Level: Intermediate
CE: ACPE 1.00 Knowledge UAN: 0286-0000-20-739-L04-P; CME 1.00; IACET 1.00; RN 1.00

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 Objectives

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.


Philip (P.J.) Brooks, PhD


Rare Mutation Oncology Trials: Learnings Over Barriers and Enablers for Successful Precision Medicine Development
Angela Qu, MD, PhD

Innovative Study Designs for Better Outcomes
Richard Huckle, MSc

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

avatar for Philip (P.J.) Brooks

Philip (P.J.) Brooks

Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences (NCATS), NIH, United States
Philip John (P.J.) Brooks is a Program Director in the NCATS Office of Rare Diseases Research. He is also the Working Group Coordinator for the NIH Common Fund program on Somatic Cell Genome Editing. He earned his Ph.D. in neurobiology from the University of North Carolina at Chapel... Read More →
avatar for Richard Huckle

Richard Huckle

Regulatory and Development Consultant, Osiris Pharma
Richard is the regulatory and development Consultant at Osiris Pharma. In addition to running the day-to-day business operations of the company, Richard consults with clients on strategic regulatory issues facing their drug and biologic development programmes, with regularly stakeholder... Read More →
avatar for Angela Qu

Angela Qu

Vice President, Translational Medicine, Parexel, United States
Dr. Qu is Vice President in Parexel Translational Medicine, leading therapeutic strategy development and implementation of genomics and biomarker clinical studies, provision of scientific guidance, and consulting on partnerships across therapeutic areas. She serves as a core member... Read More →

Thursday June 18, 2020 9:30am - 10:30am EDT
TBD Virtual Event Horsham, PA 19044
  06: PreClin Dev-EarlyPhaseCR, Session