Planning Split-Apheresis Designs for Demonstrating Comparability of Cellular and Gene Therapy Products

被引：0

作者：

Burdick, Richard ^{[1
]}

Hofer, Jeff ^{[2
]}

Karl, Andrew ^{[3
]}

Rushing, Heath ^{[3
]}

机构：

[1] Burdick Stat Consulting LLC, 7783 Renegade Hill Dr, Colorado Springs, CO 80923 USA

[2] JDH CMC Stats LLC, 628 Horatio Dr, Avon, IN 46123 USA

[3] Adsurgo LLC, 1141 N Loop E Suite 105 Unit 463, San Antonio, TX 78232 USA

来源：

AAPS JOURNAL | 2024年 / 26卷 / 06期

关键词：

Car T-Cell products; Cell and gene therapy products; Comparability; Equivalence testing of means; Matched pairs; Split-apheresis study;

D O I：

10.1208/s12248-024-00977-9

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

A recent FDA draft guidance discusses statistical considerations for demonstrating comparability of cell and gene therapy products and processes. One experimental study described in the guidance is the split-apheresis design. The FDA draft guidance recommends a paired data analysis for such a design. This paper demonstrates that the paired analysis is under powered for some quality attributes for practical sample sizes of three to five donors unless a significant portion of variability is attributed to donor. Addition of historic lots from the pre-change process can increase the power for these attributes. This paper provides appropriate statistical methods for including this information.

引用

页数：11

共 4 条

[1] [Anonymous], 2019, DEV THERAPEUTIC PROT
[2] Statistical Considerations for Comparative Assessment of Quality Attributes
Burdick, Richard K.
[J]. STATISTICS IN BIOPHARMACEUTICAL RESEARCH, 2021, 13 (03): : 297 - 302
[3] FDA CBER, 2023, Manufacturing changes and comparability for human cellular and gene therapy products
[4] A method for comparing two normal means using combined samples of correlated and uncorrelated data
Looney, SW
Jones, PW
[J]. STATISTICS IN MEDICINE, 2003, 22 (09) : 1601 - 1610

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