Chair: Elena Rantou, PhD (FDA)
Co-Chair: Shein-Chung Chow, PhD (Duke)
Instructors:
Michelle Zhang, PhD (AMMed)
Shein-Chung Chow, PhD (Duke)
Course Description:
This course delves into the complexities and unique challenges in the development of therapies for rare diseases, where small patient populations and limited data present significant hurdles. It provides an in-depth overview of the regulatory frameworks, including orphan drug designations, expedited approval processes like fast-tracking and breakthrough therapy designations, and pathways specifically designed to encourage innovation for rare diseases.
From a statistical perspective, the course will cover advanced methods tailored to small sample sizes, and how to address statistical challenges such as heterogeneity in patient populations, limited endpoints, and the interpretation of data from small cohorts.
Practical applications will be a core focus of the course, with real-world case studies illustrating how regulatory and statistical strategies have been successfully applied in rare disease drug development. The course will explore examples of therapies that navigated the regulatory process, overcoming obstacles in clinical trial design and data interpretation, and achieving market approval.
Instructors:
Michelle Zhang, Ph.D.
Executive Director
Stealth Biotherapeutics
Dr. Michelle Zhang has over a decade of experience in clinical trial design, regulatory sciences, and statistical methodologies, with a career that spans both the regulatory and pharmaceutical industries. She has been involved in clinical development programs across therapeutic areas such as Oncology, Neurology, Psychiatry, Ophthalmology, and Biosimilars. During her time at the FDA, she gained valuable expertise in trial design, regulatory review, and international collaboration, including contributing to initiatives like Project Orbis.
Dr. Zhang is a co-author of Advanced Statistics in Regulatory Critical Clinical Initiatives and serves on the editorial boards of scientific journals. She chairs the Statistician in Small Biotech Committee, fostering collaboration within the biotech sector, and is a member of the Board of Directors for the FDA Alumni Association, where she helps connect former FDA colleagues and supports the organization’s consumer protection mission.
Shein-Chung Chow, Ph.D.
Professor
Department of Biostatistics & Bioinformatics
Duke University
Shein-Chung Chow is a Professor at the Department of Biostatistics and Bioinformatics, Duke University School of Medicine, Durham, North Carolina. Dr. Chow was also a special government employee (SGE) appointed by the United States Food and Drug Administration (FDA) as an Oncologic Drug Advisory Committee (ODAC) voting member and Statistical Advisor to the FDA (2015-2017, 2019-2022). Between 2017-2019, Dr. Chow was on-leave for the FDA as an Associate Director at the Office of Biostatistics, Center for Drug Evaluation and Research (CDER), FDA. Dr. Chow was the Editor-in-Chief of the Journal of Biopharmaceutical Statistics (1992-2020) and is the Editor-in-Chief of the Biostatistics Book Series at Chapman and Hall/CRC Press of Taylor & Francis Group. He was elected Fellow of the American Statistical Association in 1995 and an elected member of the ISI (International Statistical Institute) in 1999.
Dr. Chow is the author or co-author of over 350 methodology papers and 33 books including Design and Analysis of Bioavailability and Bioequivalence Studies, Biosimilars: Design and Analysis of Follow-on Biologics, Biosimilar Product Development, Analytical Similarity Assessment in Biosimilar Drug Development, Advanced Statistics in Biosimilar Product Development, Advanced Statistics in Regulatory Critical Clinical Initiatives, and most recently, Innovative Methods for Rare Disease Drug Development.
