By Chris Houchin
Dr. Peter Marks, Director of the Center for Biologics Evaluation and Research (CBER), U.S. Food and Drug Administration (FDA), speaks with Chris Houchin, Partner at Guidehouse, in a CDO Magazine video interview about the challenges and opportunities of using data sets for rare disease treatment, using decentralized trials, the possibility of leveraging real-world evidence for cell and gene therapies, and partnering with the FDA.
Speaking about the challenges and opportunities related to data sets for treatments affecting small population sizes, Marks says that small populations come with unique challenges. Traditional randomized trials are not acceptable here for a variety of reasons, often because of the lack of a large enough patient number or the small number of patients that exist would not accept traditional randomization.
Marks explains that such situations require devising novel trial designs and extracting as much information as possible. It means that the data from each individual has to be leveraged as much as possible to understand the effectiveness of a product.
Watch the full interview below.
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