231
Views
0
CrossRef citations to date
0
Altmetric
Research Article

Performance of randomization-based causal methods with and without integrating external data sources for adjusting overall survival in case of extensive treatment switches in placebo-controlled randomized oncology phase 3 trials

, &
Pages 377-401 | Received 09 Feb 2019, Accepted 18 Nov 2019, Published online: 10 Dec 2019

References

  • Bowden, J., S. Seaman, X. Huang, and I. White. 2016. Gaining power and precision by using model-based weights in the analysis of late stage cancer trials with substantial treatment switching. Statistics in Medicine 35 (9):1423–1440. doi:10.1002/sim.6801.
  • Branson, M., and J. Whitehead. 2002. Estimating a treatment effect in survival studies in which patients switch treatment. Statistics in Medicine 21 (17):2449–2463. doi:10.1002/(ISSN)1097-0258.
  • Butler, D. 2012. Drug firm to share raw trial data: Full disclosure could improve health care and restore trust. Nature 490 (7420):322–323. doi:10.1038/490322a.
  • Clayton, D. G. 1978. A model for association in bivariate life tables and its application in epidemiological studies of familial tendency in chronic disease incidence. Biometrika 65:141–151. doi:10.1093/biomet/65.1.141.
  • ClinicalStudyDataRequest.com. ideaPoint, Inc. Accessed February 8 2019. https://clinicalstudydatarequest.com/
  • Cole, S. R., M. A. Hernán, J. M. Robins, K. Anastos, J. Chmiel, R. Detels, C. Ervin, J. Feldman, R. Greenblatt, and L. Kingsley. 2003. Effect of highly active antiretroviral therapy on time to acquired immunodeficiency syndrome or death using marginal structural models. American Journal of Epidemiology 158 (7):687–694. doi:10.1093/aje/kwg206.
  • Demetri, G. D., C. R. Garrett, P. Schöffski, M. H. Shah, J. Verweij, S. Leyvraz, H. I. Hurwitz, A. L. Pousa, A. Le Cesne, and D. Goldstein. 2012. Complete longitudinal analyses of the randomized, placebo-controlled, phase III trial of sunitinib in patients with gastrointestinal stromal tumor following imatinib failure. Clinical Cancer Research 18 (11):3170–3179. doi:10.1158/1078-0432.CCR-11-3005.
  • Eichler, H.-G., F. Pétavy, F. Pignatti, and G. Rasi. 2013. Access to patient-level trial data - a boon to drug developers. New England Journal of Medicine 369 (17):1577–1579. doi:10.1056/NEJMp1310771.
  • Frank, M. J. 1979. On the simultaneous associativity of F(x, y) and x + y − F(x, y). Aequationes Mathematicae 19 (1):194–226. doi:10.1007/BF02189866.
  • Freidlin, B., and E. Korn. 2011. Assessing causal relationships between treatments and clinical out- comes: Always read the fine print. Bone Marrow Transplantation 47:626–632. doi:10.1038/bmt.2011.119.
  • Green, A. K., K. E. Reeder-Hayes, R. W. Corty, E. Basch, M. I. Milowsky, S. B. Dusetzina, A. V. Bennett, and W. A. Wood. 2015. The project data sphere initiative: Accelerating cancer research by sharing data. The Oncologist 20 (5):464–e20. doi:10.1634/theoncologist.2014-0431.
  • Hernán, M. Á., B. Brumback, and J. M. Robins. 2000. Marginal structural models to estimate the causal effect of zidovudine on the survival of HIV-positive men. Epidemiology 11 (5):561–570. doi:10.1097/00001648-200009000-00012.
  • Hernán, M. A., S. R. Cole, J. Margolick, M. Cohen, and J. M. Robins. 2005. Structural accelerated failure time models for survival analysis in studies with time-varying treatments. Pharmacoepidemiology and Drug Safety 14 (7):477–491. doi:10.1002/(ISSN)1099-1557.
  • Hougaard, P. 1984. Life table methods for heterogeneous populations: Distributions describing the heterogeneity. Biometrika 71:75–83. doi:10.1093/biomet/71.1.75.
  • Hsu, L., and R. L. Prentice. 1996. On assessing the strength of dependency between failure time variates. Biometrika 83 (3):491–506. doi:10.1093/biomet/83.3.491.
  • Ibrahim, J. G., and M.-H. Chen. 2000. Power prior distributions for regression models. Statistical Science 15 (1): 46–60.
  • Ibrahim, J. G., M.-H. Chen, and D. Sinha. 2001. Bayesian survival analysis (springer series in statistics), . New York, NY: Springer. doi:10.1007/978-1-4757-3447-8.
  • Ishak, K. J., I. Proskorovsky, B. Korytowsky, R. Sandin, S. Faivre, and J. Valle. 2014. Methods for adjusting for bias due to crossover in oncology trials. PharmacoEconomics 32 (6):533–546. doi:10.1007/s40273-014-0145-y.
  • Jónsson, L., R. Sandin, M. Ekman, J. Ramsberg, C. Charbonneau, X. Huang, B. Jönsson, M. C. Weinstein, and M. Drummond. 2014. Analyzing overall survival in randomized controlled trials with crossover and implications for economic evaluation. Value in Health 17 (6):707–713. doi:10.1016/j.jval.2014.06.006.
  • Kilonzo, M., J. Hislop, A. Elders, C. Fraser, D. Bissett, S. McClinton, G. Mowatt, and L. Vale. 2013. Pazopanib for the first-line treatment of patients with advanced and/or metastatic renal cell carcinoma. Pharmacoeconomics 31 (1):15–24. doi:10.1007/s40273-012-0006-5.
  • Korhonen, P., E. Zuber, M. Branson, N. Hollaender, N. Yateman, T. Katiskalahti, D. Lebwohl, and T. Haas. 2012. Correcting overall survival for the impact of crossover via a rank-preserving structural failure time (RPSFT) model in the RECORD-1 trial of everolimus in metastatic renal-cell carcinoma. Journal of Biopharmaceutical Statistics 22 (6):1258–1271. doi:10.1080/10543406.2011.592233.
  • Korn, E., and B. Freidlin. 2010. Causal inference for definitive clinical end points in a randomized clinical trial with intervening nonrandomized treatments. Journal of Clinical Oncology 28 (24):3800–3802. doi:10.1200/JCO.2010.30.0442.
  • Korn, E. L., B. Freidlin, and J. S. Abrams. 2011. Overall survival as the outcome for randomized clinical trials with effective subsequent therapies. Journal of Clinical Oncology 29 (17):2439–2442. doi:10.1200/JCO.2011.34.6056.
  • Lagakos, S., L. Lim, and J. Robins. 1990. Adjusting for early treatment termination in comparative clinical trials. Statistics in Medicine 9 (12):1417–1424. doi:10.1002/(ISSN)1097-0258.
  • Latimer, N. 2015. Treatment switching in oncology trials and the acceptability of adjustment methods. Expert Review of Pharmacoeconomics & Outcomes Research 15 (4):561. doi:10.1586/14737167.2015.1037835.
  • Latimer, N., and K. R. Abrams (2014). NICE DSU technical support document 16: Adjusting survival time estimates in the presence of treatment switching. https://www.ncbi.nlm.nih.gov/books/NBK310374/pdf/Bookshelf_NBK310374.pdf accessed 8 Feb 2019.
  • Latimer, N. R., K. R. Abrams, P. C. Lambert, M. J. Crowther, A. J. Wailoo, J. P. Morden, R. L. Akehurst, and M. J. Campbell. 2014. Adjusting survival time estimates to account for treatment switching in randomized controlled trials-an economic evaluation context: Methods, limitations, and recommendations. Medical Decision Making 34 (3):387–402. doi:10.1177/0272989X13520192.
  • Law, M. G., and J. M. Kaldor. 1996. Survival analyses of randomized clinical trials adjusted for patients who switch treatments. Statistics in Medicine 15 (19):2069–2076. doi:10.1002/(ISSN)1097-0258.
  • Loeys, T., and E. Goetghebeur. 2003. A causal proportional hazards estimator for the effect of treatment actually received in a randomized trial with all-or-nothing compliance. Biometrics 59 (1):100–105. doi:10.1111/biom.2003.59.issue-1.
  • Longo, D., and J. Drazen. 2016. Data Sharing. New England Journal of Medicine 374 (3):276–277. doi:10.1056/NEJMe1516564.
  • Mello, M. M., J. K. Francer, M. Wilenzick, P. Teden, B. E. Bierer, and M. Barnes. 2013. Preparing for responsible sharing of clinical trial data. New England Journal of Medicine 369 (17):1651–1658. doi:10.1056/NEJMhle1309073.
  • Morden, J. P., P. C. Lambert, N. Latimer, K. R. Abrams, and A. J. Wailoo. 2011. Assessing methods for dealing with treatment switching in randomised controlled trials: A simulation study. BMC Medical Research Methodology 11 (1):4. doi:10.1186/1471-2288-11-4.
  • Motzer, R. J., B. Escudier, S. Oudard, T. E. Hutson, C. Porta, S. Bracarda, V. Grünwald, J. A. Thompson, R. A. Figlin, and N. Hollaender. 2010. Phase 3 trial of everolimus for metastatic renal cell carcinoma. Cancer 116 (18):4256–4265. doi:10.1002/cncr.25219.
  • National Institute for Health and Care Excellence (NICE). 2017. Everolimus for advanced renal cell carcinoma after previous treatment. Accessed February 8, 2019. https://www.nice.org.uk/guidance/ta432/resources/everolimus-for-advanced-renal-cell-carcinoma-after-previous-treatment-pdf- 82604720798917
  • O’shaughnessy, J., C. Osborne, J. E. Pippen, M. Yoffe, D. Patt, C. Rocha, I. C. Koo, B. M. Sherman, and C. Bradley. 2011. Iniparib plus chemotherapy in metastatic triple-negative breast cancer. New England Journal of Medicine 364 (3):205–214. doi:10.1056/NEJMoa1011418.
  • O’Shaughnessy, J., L. Schwartzberg, M. A. Danso, K. D. Miller, H. S. Rugo, M. Neubauer, N. Robert, B. Hellerstedt, M. Saleh, and P. Richards. 2014. Phase III study of iniparib plus gemcitabine and carboplatin versus gemcitabine and carboplatin in patients with metastatic triple-negative breast cancer. Journal of Clinical Oncology 32 (34):3840–3847. doi:10.1200/JCO.2014.55.2984.
  • Odondi, L., and R. McNamee. 2010. Performance of statistical methods for analysing survival data in the presence of non-random compliance. Statistics in Medicine 29 (29):2994–3003. doi:10.1002/sim.4070.
  • Pocock, S. J. 1976. The combination of randomized and historical controls in clinical trials. Journal of Chronic Diseases 29 (3):175–188. doi:10.1016/0021-9681(76)90044-8.
  • Raymond, E., L. Dahan, J.-L. Raoul, Y.-J. Bang, I. Borbath, C. Lombard-Bohas, J. Valle, P. Metrakos, D. Smith, and A. Vinik. 2011. Sunitinib malate for the treatment of pancreatic neuroendocrine tumors. New England Journal of Medicine 364 (6):501–513. doi:10.1056/NEJMoa1003825.
  • Robins, J. M. 1998. Correction for non-compliance in equivalence trials. Statistics in Medicine 17 (3):269–302. doi:10.1002/(ISSN)1097-0258.
  • Robins, J. M., and M. A. Hernán. 2009. Estimation of the causal effects of time-varying exposures. Eds. Fitzmaurice G, Davidian M, Verbeke G, and Molenberghs G, In Longitudinal data analysis, 553–599. Boca Raton, FL: Chapman and Hall
  • Robins, J. M., and A. A. Tsiatis. 1991. Correcting for non-compliance in randomized trials using rank preserving structural failure time models. Communications in Statistics-Theory and Methods 20 (8):2609–2631. doi:10.1080/03610929108830654.
  • Robins, J. M., M. Á. Hernán, and B. Brumback. 2000. Marginal structural models and causal inference in epidemiology. Epidemiology 11 (5):550–560. doi:10.1097/00001648-200009000-00011.
  • Rubinstein, L., M. Leblanc, and M. A. Smith. 2011. More randomization in phase II trials: Necessary but not sufficient. Journal of the National Cancer Institute 103 (14):1075–1077. doi:10.1093/jnci/djr238.
  • Rubinstein, L. V., E. L. Korn, B. Freidlin, S. Hunsberger, S. P. Ivy, and M. A. Smith. 2005. Design issues of randomized phase II trials and a proposal for phase II screening trials. Journal of Clinical Oncology 23 (28):7199–7206. doi:10.1200/JCO.2005.01.149.
  • Schlumberger, M., M. Tahara, L. J. Wirth, B. Robinson, M. S. Brose, R. Elisei, M. A. Habra, K. Newbold, M. H. Shah, and A. O. Hoff. 2015. Lenvatinib versus placebo in radioiodine-refractory thyroid cancer. New England Journal of Medicine 372 (7):621–630. doi:10.1056/NEJMoa1406470.
  • Schoenfeld, D. 1981. The asymptotic properties of nonparametric tests for comparing survival distributions. Biometrika 68 (1):316–319. doi:10.1093/biomet/68.1.316.
  • Schoenfeld, D. A. 1983. Sample-size formula for the proportional-hazards regression model. Biometrics 39:499–503. doi:10.2307/2531021.
  • Sternberg, C. N., R. E. Hawkins, J. Wagstaff, P. Salman, J. Mardiak, C. H. Barrios, J. J. Zarba, O. A. Gladkov, E. Lee, and C. Szczylik. 2013. A randomised, double-blind phase III study of pazopanib in patients with advanced and/or metastatic renal cell carcinoma: Final overall survival results and safety update. European Journal of Cancer 49 (6):1287–1296. doi:10.1016/j.ejca.2012.12.010.
  • Sugimoto, T., T. Sozu, T. Hamasaki, and S. R. Evans. 2013. A logrank test-based method for sizing clinical trials with two co-primary time-to-event endpoints. Biostatistics 14 (3):409–421. doi:10.1093/biostatistics/kxs057.
  • Walker, A. S., I. R. White, and A. G. Babiker. 2004. Parametric randomization-based methods for correcting for treatment changes in the assessment of the causal effect of treatment. Statistics in Medicine 23 (4):571–590. doi:10.1002/(ISSN)1097-0258.
  • Warren, E. 2016. Strengthening research through data sharing. New England Journal of Medicine 375 (5):401–403. doi:10.1056/NEJMp1607282.
  • Watkins, C., X. Huang, N. Latimer, Y. Tang, and E. J. Wright. 2013. Adjusting overall survival for treatment switches: Commonly used methods and practical application. Pharmaceutical Statistics 12 (6):348–357. doi:10.1002/pst.v12.6.
  • White, I. R. 2006. Estimating treatment effects in randomized trials with treatment switching. Statistics in Medicine 25 (9):1619–1622. doi:10.1002/(ISSN)1097-0258.
  • White, I. R., S. Walker, A. G. Babiker, and J. H. Darbyshire. 1997. Impact of treatment changes on the interpretation of the Concorde trial. Aids 11 (8):999–1006. doi:10.1097/00002030-199708000-00008.
  • Yamaguchi, T., and Y. Ohashi. 2004. Adjusting for differential proportions of second-line treatment in cancer clinical trials. Part I: Structural nested models and marginal structural models to test and estimate treatment arm effects. Statistics in Medicine 23 (13):1991–2003. doi:10.1002/(ISSN)1097-0258.
  • Zhang, J., and C. Chen. 2016. Correcting treatment effect for treatment switching in randomized on- cology trials with a modified iterative parametric estimation method. Statistics in Medicine 35 (21):3690–3703. doi:10.1002/sim.v35.21.

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.