References
- Alosh, M., and Huque, M.F. (2009), “A Flexible Strategy for Testing Subgroups and Overall Population,” Statistics in Medicine, 28, 3–23.
- Biomarkers Definitions Working Group (2001), “Biomarkers and Surrogate Endpoints: Preferred Definitions and Conceptual Framework,” Clinical Pharmacology and Therapeutics, 69, 89–95.
- Carroll, R.J., Ruppert, D., Stefanski, L.A., and Crainiceanu, C.M. (2006), Measurement Error in Nonlinear Models: A Modern Perspective (2nd ed.), Boca Raton, FL: Chapman Hall/CRC.
- Efron, B., and Morris, C. (1973), “Stein's Estimation Rule and Its Competitors – An Empirical Bayes Approach,” Journal of the American Statistical Association, 68, 117–130.
- Food and Drug Administration (2008), “December 16, 2008 Oncologic Drugs Advisory Committee Meeting,” available at: http://www.fda.gov/ohrms/dockets/ac/08/briefing/2008-4409b1-01-FDA.pdf
- ——— (2010a), “Guidance for Industry: Non-Inferiority Trials (Draft Guidance),” available at: http://www.fda.gov/downloads/Drugs/Guidances/UCM202140.pdf
- ——— (2010b), “Guidance for Industry-Adaptive Design Clinical Trials for Drugs and Biologics (Draft Guidance),” available at: http://www.fda.gov/downloads/Drugs/Guidances/ucm201790.pdf
- ——— (2012a), “Draft Guidance for Industry: Enrichment Strategies for Clinical Trials to Support Approval of Human Drugs and Biological Products,” available at: http://www.fda.gov/downloads/drugs/guidancecomplianceregulatory/information/guidances/ucm332181.pdf
- ——— (2012b), Summary of Safety and Effectiveness Data, Cobas 4800 BRAF V600 Mutation Test, Roche Molecular Systems, Inc. Available at: http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfTopic/pma/pma.cfm?num=P110020
- ——— (2013a), Design Considerations for Pivotal Clinical Inves-tigations for Medical Devices: Guidance for Industry, Clinical Investigators, Institutional Review Boards and Food and Drug Administration Staff. Available at: http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM373766.pdf
- ——— (2013b), Labeling Revision, Erbitux (cetuximab), Imclone Inc. available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2013/125084s242lbl.pdf
- ——— (2013c), Labeling Revision, Ziagen (abacavir sulfate), ViiV Healthcare, available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2013/020977s026,020978s030lbl.pdf
- ——— (2014a), “FDA Action Plan to Enhance the Collection and Availability of Demographic Subgroup Data,” available at: http://www.fda.gov/downloads/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/FDASIA/UCM410474.pdf
- ——— (2014b), Labeling Revision, Vectibix (panitumumab), Amgen Inc. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2014/125147s194lbl.pdf
- ——— (2014c), Labeling Revision, Zelboraf (vemurafenib) Tablet, Hoffmann-La Roche Inc. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2014/202429s006lbl.pdf
- ——— (2014d), “Guidance for Industry and Food and Drug Administration Staff- In vitro Companion Diagnostic Devices,” available at: http://www.fda.gov/downloads/MedicalDevices/DeviceRegulationandGuidance/GuidanceDocuments/UCM262327.pdf
- ——— (2015), Labeling Revision. BiDil (Isosorbide Dinitrate/Hydralazine Hydrochloride), Arbor Pharmaceuticals. Available at: http://www.accessdata.fda.gov/drugsatfda_docs/label/2015/020727s005lbl.pdf
- Gail, M., and Simon, R. (1985), “Testing for Qualitative Interaction Between Treatment Effects and Patient Subsets,” Biometrics, 41, 361–372.
- Hughes, A.R., Spreen, W.R., Mosteller, M., Warren, L. L., Lai, E. H., Brothers, C. H., Cox, C., Nelsen, A. J., Hughes, S., Thorborn, D. E., Stancil, B., Hetherington, S. V., Burns, D. K. and Roses, A. D. (2008), “Pharmacogenetics of Hypersensitivity to Abacavir: From PGx Hypothesis to Confirmation to Clinical Utility,” Pharmacogenomics Journal, 8, 365–374.
- International Conference on Harmonization. (1998), “International Conference on Harmonization (ICH) Guidance, E9 Statistical Principles for Clinical Trials (ICH E9 Guidance),” available at: http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E9/Step4/E9_Guideline.pdf.
- Koch, G.G. (1997), Discussion of “P-Value Adjustments for Sub-Group Analyses,” Journal of Biopharmaceutical Statistics, 7, 323–331.
- Li, Z., Chuang-Stein, C., and Hoseyni, C. (2007), “The Probability of Observing Negative Subgroup Results When the Treatment Effect is Positive and Homogenous Across All Subgroups,” Drug Information Journal, 41, 47–56.
- Louis, T.A. (1984), “Estimating a Population of Parameter Values using Bayes and Empirical Bayes Methods,” Journal of the American Statistical Association, 79, 393–398.
- Maitournam, A., and Simon, R. (2005), “On the Efficacy of Targeted Clinical Trials,” Statistics in Medicine, 24, 329–339.
- Mallal, S., Phillips, E., Carosi, G., Molina, J-M., Workman, C., Tomažič, J., Jägel-Guedes, E., Rugina, S., Kozyrev, O., Flores Cid, J., Hay, P., Nolan, D., Hughes, S., Hughes, A., Ryan, S., Fitch, N., Thorborn, D., and Benbow, A. , for the PREDICT-1 Study Team. (2008), “HLA-B*5701 Screening for Hypersensitivity to Abacavir,” New England Journal of Medicine, 358, 568–579.
- Marschner, I. (2010), “Regional Differences in Multinational Clinical Trials: Anticipating Chance Variation, Clinical Trials, 7, 147–156.
- Molina, J.M., LaMarca, A., Andrade-Villanueva, J., Clotet, B., Clumeck, N., Liu, Y. P., Zhong, L., Margot, N., Cheng, A. K., Chuck, S. L., for the Study 145 Team. (2012), “Efficacy and Safety of Once Daily Elvitegravir Versus Twice Daily Raltegravir in Treatment-Experienced Patients With HIV-1 Receiving a Ritonavir-Boosted Protease Inhibitor: Randomised, Double-Blind, Phase 3, Non-Inferiority Study,” Lancet Infectious Diseases, 12, 27–35.
- Pennello, G. (2013), “Analytical and Clinical Evaluation of Biomarkers Assays: When are Biomarkers Ready for Prime Time?” Clinical Trials, 10, 666–676.
- Permutt, T. (2007), “A Note on Stratification in Clinical Trials,” Drug Information Journal, 41, 719–722.
- Russek-Cohen, E., and Simon, R.M. (1997), “Evaluating Treatment When a Gender by Treatment Interaction May Exist,” Statistics in Medicine, 16, 455–464.
- Scott, J.G., and Berger, J.O. (2006), “An Exploration of Aspects of Bayesian Multiple Testing,” Journal of Statistical Planning and Inference, 136, 2144–2162.
- Simon, R. (2002), “Bayesian Subset Analysis: Application to Studying Treatment-by-Gender Interactions,” Statistics in Medicine, 21, 2909–2916.
- Song, Y., and Chi, G.Y. (2007), “A Method for Testing a Prespecified Subgroup in Clinical Trials,” Statistics in Medicine, 26, 3535–3549.
- The European Agency for the Evaluation of Medicinal Products, Committee for Proprietary Medicinal Products. (2002), “Points to Consider on Multiplicity Issues in Clinical Trials London, UK,” available at: http://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2009/09/WC500003640.pdf
- Wiens, B.L., and Heyse, J.F. (2003), “Testing for Interaction in Studies of Noninferiority,” Journal of Biopharmaceutical Statistics, 13, 103–115.
- Zhao, Y.D., Dmitrienko, A., and Tamura, R. (2010), “Design and Analysis Considerations in Clinical Trials With a Sensitive Subpopulation,” Statistics in Biopharmaceutical Research, 2, 72–83.