Considerations for demonstrating product comparability and process consistency with cell therapy products
View Presentation *Thomas Finn, FDA/CBER Keywords: cell therapy, product comparability, process consistency Cell therapies offer tremendous potential for treating a wide array of serious medical conditions, and may be the best strategy for some very difficult to treat diseases. Some cell therapies are personalized to the degree that product lots are manufactured for each individual patient. But challenges for manufacturing these products are numerous, and for some cell therapies demonstrating consistent product quality can be difficult. Two main areas of concern are 1) that a manufacturing process consistently generates a quality product; and 2) that product quality is being maintained after a manufacturing change. Addressing these concerns is not always easy, and approaches used for traditional drugs or biologics may have to be adjusted to accommodate the more complex nature of cell therapies. Some personalized biologics can vary 100 fold lot-to-lot due to inherent variability of source material from patients. The level of product testing feasible for lot release may be limited for many practical reasons. In some cases limited product quality testing data may need to be supplemented with additional testing in order to establish product comparability or manufacturing consistency. Despite these difficulties, personalized autologous and allogeneic cell therapies also present some unique statistical opportunities. The number of lots generated during the IND phase can be many hundreds or even thousands, and for commercial products thousands per year. One can leverage these large data sets to evaluate manufacturing data in ways that cannot be done for more traditional biologics where small numbers of lots are generated. Ways in which statistics can be very helpful for evaluating cell therapy manufacturing goes beyond questions about product consistency and manufacturing, and can include manufacturing and distribution logistics, data trending to look for patterns among routine manufacturing results, and conducting stability studies. In some cases a statistical analysis can also be part of a post-marketing commitment. In this presentation I will discuss some of the regulatory concerns with traditional approaches to analysis of CMC data for these types of products, and cover some common flaws in study design and analysis.
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Key Dates
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November 1 - December 17, 2013
Online proposal submission for a session, short course and Town Hall Open -
January 6 - March 11, 2014
Online proposal submission for Roundtables Open -
April 30 - May 28, 2014
Abstract Submission Open -
June 4, 2014
Online Registration Opens -
August 8 - August 22, 2014
Invited Abstract Editing -
August 11, 2014
Short Course materials due from Instructors -
September 1, 2014
Housing Deadline -
September 15, 2014
Cancellation Deadline and Registration Closes @ 11:59 pm EDT -
September 22 - September 24, 2014
Marriott Wardman Park, Washington, DC