title: Statistical considerations for confirmatory clinical trials for similar biotherapeutic products.
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.
authors.affiliation: Biostatistics Unit, Centre for Vaccine Evaluation, Biologics and Genetic Therapies Directorate, Health Products and Food Branch, Health Canada, 251 Sir Frederick Banting Driveway, Tunney's Pasture, Ottawa, Ontario, Canada.
authors.forename: Catherine
authors.initials: C
authors.lastname: Njue
mesh_terms: D002986:Clinical Trials as Topic / Q000379:methods*; D004341:Drug Evaluation / Q000379:methods*; D017408:Guidelines as Topic; D012107:Research Design; D013223:Statistics as Topic / Q000379:methods*; D014944:World Health Organization