Elsevier

Biologicals

Volume 39, Issue 5, September 2011, Pages 266-269
Biologicals

Statistical considerations for confirmatory clinical trials for similar biotherapeutic products

https://doi.org/10.1016/j.biologicals.2011.06.006Get rights and content
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Abstract

For the purpose of comparing the efficacy and safety of a Similar Biotherapeutic Product (SBP) to a Reference Biotherapeutic Product (RBP), the “Guidelines on Evaluation of Similar Biotherapeutic Products (SBPs)” issued by the World Health Organisation (WHO), states that equivalence or non-inferiority studies may be acceptable. While in principle, equivalence trials are preferred, non-inferiority trials may be considered if appropriately justified, such as for a medicinal product with a wide safety margin. However, the statistical issues involved in the design, conduct, analysis and interpretation of equivalence and non-inferiority trials are complex and subtle, and require that all aspects of these trials be given careful consideration. These issues are important in order to ensure that equivalence and non-inferiority trials provide valid data that are necessary to draw reliable conclusions regarding the clinical similarity of an SBP to an RBP.

Keywords

Statistical principles
Clinical trials
Equivalence design
Non-inferiority design

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