Development of Gene Therapies: Strategic, Scientific, Regulatory, and Access Considerations
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Key Features:
- A collection of papers covering a wide spectrum of topics in gene therapies (GTx), written by leading subject matter experts.
- An exposition of the core principles of GTx product development, emerging business models, industry standards, best practices, and regulatory pathways.
- An exposition of statistical and innovative modeling tools for design and analysis of clinical trials of GTx.
- Insights into commercial models, access hurdles, and health economics of gene therapies.
- Case studies of successful GTx approvals from core team members that developed the first two FDA-approved AAV gene therapies: Luxturna and Zolgensma.
- A discussion of potential benefits and hurdles to be overcome for GTx in coming years from a multi-stakeholder perspective.
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Avery McIntosh, Ph.D. is a drug developer working in rare diseases at Pfizer. He received his M.Sc. and Ph.D. 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, Ph.D. is a Neuroscience Disease Area Statistical Lead at Novartis. He received B.Sc. in Applied Mathematics from V.N. Karazin Kharkiv National University, Ukraine, M.Sc. in Statistics from University of Maryland, Baltimore County (UMBC), and Ph.D. 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.
