447
Views
1
CrossRef citations to date
0
Altmetric
Articles

Quantitative decision-making in randomized Phase II studies with a time-to-event endpoint

ORCID Icon, , &
Pages 189-202 | Received 22 Oct 2016, Accepted 11 Jun 2018, Published online: 03 Jul 2018

References

  • Armitage, P. (1989). Inference and decision in clinical trials. Journal of Clinical Epidemiology 42:293–299.
  • Armitage, P., Berry, G., Matthews, J. N. S. (2002). Statistical Methods in Medical Research, 4th ed. Oxford: Blackwell Science.
  • Carroll, K. (2013). Decision making from Phase II to Phase III and the probability of success: Reassured by assurance? Journal of Biopharmaceutical Statistics 23(5):1188–1200. doi:10.1080/10543406.2013.813527
  • Chuang-Stein, C. (2006). Sample size and the probability of a successful trial. Pharmaceutical Statistics 5:305–309. doi:10.1002/pst.232
  • Chuang-Stein, C., Kirby, S., French, J., Kowalski, K., Marshall, S., Smith, M., Bycott, P., Beltangady, M. (2011a). A quantitative approach for making Go/No-Go decisions in drug development. Drug Information Journal 45(2):187–202. doi:10.1177/009286151104500213
  • Chuang-Stein, C., Kirby, S., Hirsch, I., Atkinson, G. (2011b). The role of the minimum clinically important difference and its impact on designing a trial. Pharmaceutical Statistics 10:250–256. doi:10.1002/pst.459
  • FDA Approves Palbociclib for Metastatic Breast Cancer. OncLive. 3 Febuary 2015. https://www.onclive.com/web-exclusives/fda-approves-palbociclib-for-metastatic-breast-cancer.
  • Fisch, R., Jones, I., Jones, J., Kerman, J., Rosenkranz, G. K., Schmidli, H. (2014). Bayesian design of proof-of-concept trials. Therapeutic Innovation and Regulatory Science Section 49(1):155–162. doi:10.1177/2168479014533970
  • Frewer, P., Mitchell, P., Watkins, C., Matcham, J. (2016). Decision-making in early clinical drug development. Pharmaceutical Statistics 15(3):255–263. doi:10.1002/pst.1746
  • Gsponer, T., Gerber, F., Bornkamp, B., Ohlssen, D., Vandemeulebroecke, M., Schmidli, H. (2014). A practical guide to Bayesian group sequential designs. Pharmaceutical Statistics 13(1):71–80. doi:10.1002/pst.1593
  • Hobbs, B., Carlin, B., Mandrekar, S., Sargent, D. (2011). Hierarchical commensurate and power prior models for adaptive incorporation of historical information in clinical trials. Biometrics 67:1047–1056. doi:10.1111/j.1541-0420.2011.01564.x
  • Jiang, K. (2011). Optimal sample sizes and Go/No-Go decisions for Phase II/III development programs based on probability of success. Statistics in Biopharmaceutical Research 3:463–475. doi:10.1198/sbr.2011.10068
  • Kieser, M., Hauschke, D. (2005). Assessment of clinical relevance by considering point estimates and associated confidence intervals. Pharmaceutical Statistics 4(2):101–107. doi:10.1002/(ISSN)1539-1612
  • King, M. (2009). Evaluating probability of success in oncology clinical trials. BASS XVI, November. http://www.bassconference.org/PDFs/Bass20200920MartinKing.pdf.
  • Kirby, S., Burke, J., Chuang-Stein, C., Sin, C. (2012). Discounting phase 2 results when planning phase 3 clinical trials. Pharmaceutical Statistics 11(5):373–385. doi:10.1002/pst.1521
  • Kirby, S., Chuang-Stein, C. (2017). A comparison of five approaches to decision-making for a first clinical trial of efficacy. Pharmaceutical Statistics 16(1):37–44. doi:10.1002/pst.1775
  • Lachin, J. M., Foulkes, M. A. (1986). Evaluation of sample size and power for analyses of survival with allowance for nonuniform patient entry, losses to follow-up, noncompliance, and stratification. Biometrics 42:507–519.
  • Neuenschwander, B., Branson, M., Spiegelhalter, D. (2009). A note on the power prior. Statistics in Medicine 28:3562–3566. doi:10.1002/sim.3722
  • Neuenschwander, B., Rouyrre, N., Hollaender, N., Zuber, E., Branson, M. (2011). A proof of concept phase II non-inferiority criterion. Statistics in Medicine 30(13):1618–1627. doi:10.1002/sim.3997
  • O’Hagan, A., Stevens, J. W., Campbell, M. (2005). Assurance in clinical trial design. Pharmaceutical Statistics 4:187–201. doi:10.1002/pst.175
  • Paul, S. M., Mytelka, D. S., Dunwiddie, C. T., Persinger, C. C., Munos, B. H., Lindborg, S. R., Schacht, A. L. (2010). How to improve R&D productivity: The pharmaceutical industry’s grand challenge. Nature Reviews Drug Discovery 9(3):203–214. doi:10.1038/nrd3078
  • Sabin, T., Matcham, J., Bray, S., Copas, A., Parmar, M. (2014). A quantitative process for enhancing end of phase 2 decisions. Statistics in Biopharmaceutical Research 6:67–77. doi:10.1080/19466315.2013.852617
  • Spiegelhalter, D. J., Freedman, L. S. (1986). A predictive approach to selecting the size of a clinical trial, based on subjective clinical opinion. Statistics in Medicine 5:1–13.
  • Stallard, N. (1998). Sample size determination for Phase II clinical trials based on Bayesian decision theory. Biometrics 54:279–294.
  • Sylvester, R. J. (1988). A Bayesian approach to the design of Phase II clinical trials. Biometrics 44:823–836.
  • Thall, P. F., Simon, R. (1994). A Bayesian approach to establishing sample size and monitoring criteria for Phase II clinical trials. Controlled Clinical Trials 15:463–481.
  • Wang, Y., Fu, H., Kulkarni, P., Kaiser, C. (2013). Evaluating and utilizing probability of study success in clinical development. Clinical Trials 10(3):407–413. doi:10.1177/1740774513478229
  • Walley, R., Smith, C., Gale, J., Woodward, P. (2015). Advantages of a wholly Bayesian approach to assessing efficacy in early drug development: A case study. Pharmaceutical Statistics 14(3):205–215. doi:10.1002/pst.1675

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.