Subgroup Analyses in Oncology Trials: Regulatory Considerations and Case Examples.

Clinical Cancer Research : an Official Journal of the American Association for Cancer Research
Anup K AmatyaShenghui Tang

Abstract

Subgroup analyses are assessments of treatment effects based on certain patient characteristics out of the total study population and are important for interpretation of pivotal oncology trials. However, appropriate use of subgroup analyses results for regulatory decision-making and product labeling is challenging. Typically, drugs approved by the FDA are indicated for use in the total patient population studied; however, there are examples of restriction to a subgroup of patients despite positive study results in the entire study population and also extension of an indication to the entire study population despite positive results appearing primarily in one or more subgroups. In this article, we summarize key issues related to subgroup analyses in the benefit-risk assessment of cancer drugs and provide case examples to illustrate approaches that the FDA Oncology Center of Excellence has taken when considering the appropriate patient population for cancer drug approval. In general, if a subgroup is of interest, the subgroup analysis should be hypothesis-driven and have adequate sample size to demonstrate evidence of a treatment effect. In addition to statistical efficacy considerations, the decision on what subgroups to include...Continue Reading

References

Feb 2, 2011·Clinical Trials : Journal of the Society for Clinical Trials·Hayley E JonesMichael Branson
Sep 15, 2015·Journal of Biopharmaceutical Statistics·Alex DmitrienkoIlya Lipkovich
May 4, 2016·Annals of Oncology : Official Journal of the European Society for Medical Oncology·J A Ledermann
Nov 29, 2016·Statistics in Medicine·Mohamed AloshRalph B D'Agostino
Mar 1, 2017·Clinical Cancer Research : an Official Journal of the American Association for Cancer Research·Christy L OsgoodRichard Pazdur
Dec 29, 2018·Pharmaceutical Statistics·Aaron DaneUNKNOWN PSI/EFSPI Working Group on Subgroup Analysis
Feb 1, 2020·Clinical Cancer Research : an Official Journal of the American Association for Cancer Research·Preeti NarayanJulia A Beaver

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