Statistical issues and challenges in immuno-oncology
Open Access
- 21 October 2013
- journal article
- Published by BMJ in Journal for ImmunoTherapy of Cancer
- Vol. 1 (1), 18
- https://doi.org/10.1186/2051-1426-1-18
Abstract
The development of immuno-oncologic agents poses unique challenges, namely that both efficacy and safety profiles differ from previously characterized cytotoxic and pathway-specific agents. In addition, exponential distribution is usually assumed in study designs with time-to-event endpoints such as overall survival or progression-free survival. This assumption might lead to wrong estimates of study duration and statistical power if the phenomena of long term survival and delayed clinical effects are present. The aim here was to evaluate the magnitude of the impact caused by the violation of this assumption, and to describe new ways of analyzing efficacy and safety of immuno-oncologic agents.This publication has 34 references indexed in Scilit:
- Phase III Randomized Clinical Trial Comparing Tremelimumab With Standard-of-Care Chemotherapy in Patients With Advanced MelanomaJournal of Clinical Oncology, 2013
- Very long‐term follow‐up results of imatinib mesylate therapy in chronic phase chronic myeloid leukemia after failure of interferon alpha therapyCancer, 2012
- gp100 Peptide Vaccine and Interleukin-2 in Patients with Advanced MelanomaThe New England Journal of Medicine, 2011
- Efficacy and safety of ipilimumab monotherapy in patients with pretreated advanced melanoma: a multicenter single-arm phase II studyAnnals of Oncology, 2010
- Improved Survival with Ipilimumab in Patients with Metastatic MelanomaThe New England Journal of Medicine, 2010
- Guidelines for the Evaluation of Immune Therapy Activity in Solid Tumors: Immune-Related Response CriteriaClinical Cancer Research, 2009
- New response evaluation criteria in solid tumours: Revised RECIST guideline (version 1.1)European Journal of Cancer, 2009
- Immunostimulatory monoclonal antibodies for cancer therapyNature Reviews Cancer, 2007
- Interim Monitoring of Group Sequential Trials Using Spending Functions for the Type I and Type II Error ProbabilitiesDrug Information Journal, 2001
- New Guidelines to Evaluate the Response to Treatment in Solid TumorsJNCI Journal of the National Cancer Institute, 2000