426
Views
1
CrossRef citations to date
0
Altmetric
Research Article

Propensity score-integrated approach to survival analysis: leveraging external evidence in single-arm studies

, , ORCID Icon, , , , & show all
Pages 400-413 | Received 13 Jul 2021, Accepted 22 Feb 2022, Published online: 08 Jun 2022

References

  • Austin, P. C. 2011. A tutorial and case study in propensity score analysis: An application to estimating the effect of in-hospital smoking cessation counseling on mortality. Multivariate Behavioral Research 46 (1):119–151. doi:10.1080/00273171.2011.540480.
  • Austin, P. C., and T. Schuster. 2016. The performance of different propensity score methods for estimating absolute effects of treatments on survival outcomes: A simulation study. Statistical Methods in Medical Research 25 (5):2214–2237. doi:10.1177/0962280213519716.
  • Campbell, G., and L. Q. Yue. 2016. Statistical innovations in the medical device world sparked by the FDA. Journal of Biopharmaceutical Statistics 26 (1):3–16. doi:10.1080/10543406.2015.1092037.
  • Chen, W.-C., C. Wang, H. Li, N. Lu, R. Tiwari, Y. Xu, L. Q. Yue. 2020. Propensity score-integrated composite likelihood approach for augmenting the control arm of a randomized controlled trial by incorporating real-world data. Journal of Biopharmaceutical Statistics 30(3):508–520. doi:10.1080/10543406.2020.1730877.
  • D’Agostino, R. B., Jr. 1998. Propensity score methods for bias reduction in the comparison of a treatment to a non-randomized control group. Statistics in Medicine 17 (19):2265–2281. doi:10.1002/(SICI)1097-0258(19981015)17:19<2265::AID-SIM918>3.0.CO;2-B.
  • Franklin, J. M., J. A. Rassen, D. Ackermann, D. B. Bartels, S. Schneeweiss. 2014. Metrics for covariate balance in cohort studies of causal effects. Statistics in Medicine 33(10):1685–1699. doi:10.1002/sim.6058.
  • Galimberti, S., P. Sasieni, and M. G. Valsecchi. 2002. A weighted Kaplan–Meier estimator for matched data with application to the comparison of chemotherapy and bone‐marrow transplant in leukaemia. Statistics in Medicine 21 (24):3847–3864. doi:10.1002/sim.1357.
  • Inman, H. F., and E. L. Bradley Jr. 1989. The overlapping coefficient as a measure of agreement between probability distributions and point estimation of the overlap of two normal densities. Communications in Statistics - Theory and Methods 18 (10):3851–3874. doi:10.1080/03610928908830127.
  • Jay, M., and R. A. Betensky. 2021. Displaying survival of patient groups defined by covariate paths: Extensions of the Kaplan‐Meier estimator. Statistics in Medicine 40 (8):2024–2036. doi:10.1002/sim.8888.
  • Kaplan, E. L., and P. Meier. 1958. Nonparametric estimation from incomplete observations. Journal of the American Statistical Association 53 (282):457–481. doi:10.1080/01621459.1958.10501452.
  • Li, H., V. Mukhi, N. Lu, Y.-L. Xu, L. Q. Yue. 2016. A note on good practice of objective propensity score design for premarket nonrandomized medical device studies with an example. Statistics in Biopharmaceutical Research 8(3):282–286. doi:10.1080/19466315.2016.1148071.
  • Lu, N., C. Wang, W.-C. Chen, H. Li, C. Song, R. Tiwari, Y. Xu, L. Q. Yue. 2021. Leverage multiple real-world data sources in single-arm medical device clinical studies. Journal of Biopharmaceutical Statistics 1–17. doi:10.1080/10543406.2021.1897994.
  • Rosenbaum, P. R., and D. B. Rubin. 1983. The central role of the propensity score in observational studies for causal effects. Biometrika 70 (1):41–55. doi:10.1093/biomet/70.1.41.
  • Rosenbaum, P. R., and D. B. Rubin. 1984. Reducing bias in observational studies using subclassification on the propensity score. Journal of the American Statistical Association 79 (387):516–524. doi:10.1080/01621459.1984.10478078.
  • Rubin, D. B. 2008. For objective causal inference, design trumps analysis. The Annals of Applied Statistics 2 (3):808–840. doi:10.1214/08-AOAS187.
  • Turlach, B. A. 1993. Bandwidth selection in kernel density estimation: A review. s.l., Citeseer. Accessed June 1, 2022. http://citeseerx.ist.psu.edu/viewdoc/summary?doi=10.1.1.44.6770
  • US Food and Drug Administration, 2013. Design considerations for pivotal clinical investigations for medical devices - Guidance for industry, clinical investigators, Institutional Review Boards and Food and Drug Administration staff. Accessed June 1, 2022. https://www.fda.gov/media/87363/download
  • US Food and Drug Administration, 2017. Use of Real-World Evidence to Support Regulatory Decision-Making for Medical Devices. [ Online] Online: Accessed June 1, 2022. https://www.fda.gov/media/99447/download
  • Wang, C., H. Li, W.-C. Chen, N. Lu, R. Tiwari, Y. Xu, L. Q. Yue. 2019. Propensity score-integrated power prior approach for incorporating real-world evidence in single-arm clinical studies. Journal of Biopharmaceutical Statistics 29(5):731–748. doi:10.1080/10543406.2019.1657133.
  • Wang, C., N. Lu, W.-C. Chen, H. Li, R. Tiwari, Y. Xu, L. Q. Yue. 2020. Propensity score-integrated composite likelihood approach for incorporating real-world evidence in single-arm clinical studies. Journal of Biopharmaceutical Statistics 30(3):495–507. doi:10.1080/10543406.2019.1684309.
  • Yue, L. Q. 2007. Statistical and regulatory issues with the application of propensity score analysis to nonrandomized medical device clinical studies. Journal of Biopharmaceutical Statistics 17 (1):1–13. doi:10.1080/10543400601044691.
  • Yue, L. Q., N. Lu, and Y. Xu. 2014. Designing premarket observational comparative studies using existing data as controls: Challenges and opportunities. Journal of Biopharmaceutical Statistics 24 (5):994–1010. doi:10.1080/10543406.2014.926367.
  • Yue, L. Q., G. Campbell, N. Lu, Y. Xu, B. Zuckerman. 2016. Utilizing national and international registries to enhance pre-market medical device regulatory evaluation. Journal of Biopharmaceutical Statistics 26(6):1136–1145. doi:10.1080/10543406.2016.1226336.

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.