Avery McIntosh (Pfizer) and Oleksandr Sverdlov (Novartis)
This webinar will help listeners gain a broader understanding of a recent and rapidly advancing core area of pharmaceutical drug development. The content will be broad, but will focus on clinical design and analysis strategies for advanced therapeutics such as cell/gene therapies, and strategic options to advance candidate drugs in this space.
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.
A list of useful references from the presentation can be found below:
- McIntosh, Avery, and Oleksandr Sverdlov, eds. Development of Gene Therapies: Strategic, Scientific, Regulatory, and Access Considerations. CRC Press, 2024.
- Rohde, Maximilian, et al. "Practical and Statistical Considerations for the Long Term Follow‐Up of Gene Therapy Trial Participants." Clinical Pharmacology & Therapeutics 115.1 (2024): 139-146.
- McIntosh, Avery, et al. "Clinical design and analysis strategies for the development of gene therapies: considerations for quantitative drug development in the age of genetic medicine." Clinical Pharmacology & Therapeutics 110.5 (2021): 1207-1215.
- Mueller, Arne, et al. "Digital endpoints for self‐administered home‐based functional assessment in pediatric Friedreich’s ataxia." Annals of Clinical and Translational Neurology 8.9 (2021): 1845-1856.
- Hudry, Eloise, and Luk H. Vandenberghe. "Therapeutic AAV gene transfer to the nervous system: a clinical reality." Neuron 101.5 (2019): 839-862.
- Cox, Gerald F. "The art and science of choosing efficacy endpoints for rare disease clinical trials." American Journal of Medical Genetics Part A 176.4 (2018): 759-772