Development of Gene Therapies: Open Questions on Design, Analysis, and Statistical Strategy

Abstract:

One of the recent advances in 21st-century medicine is the emergence of gene therapies, drugs that affect the basic biology of genetic disease. The field has seen some notable setbacks in the past, but in recent years has exploded as decades of basic science have been successfully translated into the most complex biologics ever constructed, leading to regulatory approval of several gene therapy products in oncology, hematology, neurology, and ophthalmology indications. These drugs are at the apex of biological manufacturing complexity, and have the potential to be disease modifying or even curative. Evidence-based and innovative quantitative clinical development and lifecycle management strategies will be required as fixtures in the development for these unique drugs in order to reach patients in need. In this webinar we provide an overview of the history and future of gene therapies, and discuss the crucial role of the statistician in the drug development process of these drugs, with a focus on innovative trial design and analysis techniques.

About the Speakers:

Avery McIntosh, PhD is a drug developer working in internal medicine and rare disease at Pfizer. He received his MSc and PhD in biostatistics from Boston University with a dissertation on Bayesian methods to model household tuberculosis transmission. He has managed teams of statisticians across study phases and in a variety of drug types and disease areas, including neurology, ophthalmology, infectious disease/global health, hematology, and oncology. He has published peer-reviewed articles on various topics in drug development and biostatistics, including development of cell and gene therapies and qualification of digital endpoints in neurological diseases.

Oleksandr Sverdlov, PhD is a Neuroscience Disease Area Statistical Lead at Novartis. He earned his BSc in Applied Mathematics from V.N. Karazin Kharkiv National University, Ukraine, MSc in Statistics from University of Maryland, Baltimore County (UMBC), and PhD in Information Technology with Concentration in Statistical Science from George Mason University. He has been actively involved in methodological research and applications of innovative statistical approaches in drug development. He has co-authored over forty refereed articles, edited two monographs, and co-authored a book “Mathematical and Statistical Skills in the Biopharmaceutical Industry: A Pragmatic Approach” (CRC Press/Chapman & Hall, 2019). His most recent work involves design and analysis of clinical trials evaluating novel digital technologies.

The webinar will be moderated by Kush Kapur (Webmaster Officer of Boston Chapter of the ASA; Argenx US, Inc.) and Wenting Cheng (President of Boston Chapter of the ASA; Biogen).

Host or Publisher Boston Chapter ASA