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Research Article

A pragmatic approach for dynamically incorporating predicate device data in prospective diagnostic test studies

ORCID Icon, , , &
Pages 77-89 | Received 16 Mar 2021, Accepted 13 Apr 2022, Published online: 01 Jun 2022

References

  • Abraham, W. T., K.-H. Kuck, R. L. Goldsmith, J. Lindenfeld, V. Y. Reddy, P. E. Carson, D. L. Mann, B. R. Saville, H. Parise, R. Chan, et al. 2018. A randomized controlled trial to evaluate the safety and efficacy of cardiac contractility modulation. JACC. Heart Failure. doi:10.1016/j.jchf.2018.04.010.
  • Altman, D. G., and J. M. Bland. 1994. Diagnostic tests 1: Sensitivity and specificity. BMJ 308 (6943):1552. doi:10.1136/bmj.308.6943.1552.
  • BD (2012), “BD MAX MRSA Assay [K120138],” 510(k) Premarket Notification, Available at.https://www.accessdata.fda.gov/cdrh_docs/reviews/K120138.pdf. (Accessed Mar 10, 2021).
  • BD (2013), “BD MAX MRSA XT Assay [K133605],” 510(k) Premarket Notification, Available at.https://www.accessdata.fda.gov/cdrh_docs/reviews/K133605.pdf. (Accessed Mar 10, 2021).
  • Berry, D. A. 2006. A guide to drug discovery: Bayesian clinical trials. Nature Reviews. Drug Discovery 5 (1):27–36. doi:10.1038/nrd1927.
  • Böhm, M., R. R. Townsend, K. Kario, D. E. Kandzari, F. Mahfoud, M. A. Weber, R. E. Schmieder, K. Tsioufis, G. L. Hickey, M. Fahy, et al. 2020. Rationale and design of two randomized sham-controlled trials of catheter-based renal denervation in subjects with uncontrolled hypertension in the absence (SPYRAL HTN-OFF MED Pivotal) and presence (SPYRAL HTN-ON MED Expansion) of antihypertensive medicat. Clinical Research in Cardiology 109 (3):289–302. doi:10.1007/s00392-020-01595-z.
  • Brard, C., S. Piperno-Neumann, J. Delaye, L. Brugières, L. V. Hampson, G. Le Teuff, M. C. Le Deley, and N. Gaspar. 2019. Sarcome-13/OS2016 trial protocol: A multicentre, randomised, open-label, phase II trial of mifamurtide combined with postoperative chemotherapy for patients with newly diagnosed high-risk osteosarcoma. BMJ Open 9 (5):1–9. doi:10.1136/bmjopen-2018-025877.
  • Broglio, K. R., J. T. Connor, and S. M. Berry. 2014. Not too big, not too small: A Goldilocks approach to sample size selection. Journal of Biopharmaceutical Statistics 24 (3):685–705. doi:10.1080/10543406.2014.888569.
  • Chen, W. C., C. Wang, H. Li, N. Lu, R. Tiwari, Y. Xu, and 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.
  • Chow, S.-C., and M. Chang. 2008. Adaptive design methods in clinical trials - A review. Orphanet Journal of Rare Diseases 3 (1):1–13. doi:10.1186/1750-1172-3-11.
  • Connor, J. T., B. R. Luce, K. R. Broglio, K. J. Ishak, C. D. Mullins, D. J. Vanness, R. Fleurence, E. Saunders, and B. R. Davis. 2013. Do Bayesian adaptive trials offer advantages for comparative effectiveness research? Protocol for the RE-ADAPT study. Clinical Trials 10 (5):807–827. doi:10.1177/1740774513497293.
  • de Santis, F. 2007. Using historical data for Bayesian sample size determination. Journal of the Royal Statistical Society. Series A: Statistics in Society 170 (1):95–113. doi:10.1111/j.1467-985X.2006.00438.x.
  • Drews, S. J., B. M. Willey, N. Kreiswirth, M. Wang, T. Ianes, J. Mitchell, M. Latchford, A. J. McGeer, and K. C. Katz. 2006. Verification of the IDI-MRSA assay for detecting methicillin-resistantStaphylococcus aureus in diverse specimen types in a core clinical laboratory setting. Journal of Clinical Microbiology 44 (10):3794–3796. doi:10.1128/JCM.01509-06.
  • Flahault, A., M. Cadilhac, and G. Thomas. 2005. Sample size calculation should be performed for design accuracy in diagnostic test studies. Journal of Clinical Epidemiology 58 (8):859–862. doi:10.1016/j.jclinepi.2004.12.009.
  • Haddad, T., A. Himes, L. Thompson, T. Irony, and R. Nair. 2017. Incorporation of stochastic engineering models as prior information in Bayesian medical device trials. Journal of Biopharmaceutical Statistics 27 (6):1089–1103. doi:10.1080/10543406.2017.1300907.
  • Hardy, K., C. Price, A. Szczepura, S. Gossain, R. Davies, N. Stallard, S. Shabir, C. McMurray, A. Bradbury, and P. M. Hawkey. 2010. Reduction in the rate of methicillin-resistant Staphylococcus aureus acquisition in surgical wards by rapid screening for colonization: A prospective, cross-over study. Clinical Microbiology and Infection 16 (4):333–339. doi:10.1111/j.1469-0691.2009.02899.x.
  • Hobbs, B. P., B. P. Carlin, S. J. Mandrekar, and D. J. Sargent. 2011. Hierarchical commensurate and power prior models for adaptive incorporation of historical information in clinical trials. Biometrics 67 (3):1047–1056. doi:10.1111/j.1541-0420.2011.01564.x.
  • Hobbs, B. P., D. J. Sargent, and B. P. Carlin. 2012. Commensurate priors for incorporating historical information in clinical trials using general and generalized linear models. Bayesian Analysis 7 (3):639–674. doi:10.1214/12-BA722.
  • Ibrahim, J. G., and M. H. Chen. 2000. Power prior distributions for regression models. Statistical Science 15:46–60. doi:10.1214/ss/1009212673.
  • Ibrahim, J. G., M. H. Chen, Y. Gwon, and F. Chen. 2015. The power prior: Theory and applications. Statistics in Medicine 34 (28):3724–3749. doi:10.1002/sim.6728.
  • Kopp-Schneider, A., S. Calderazzo, and M. Wiesenfarth. 2020. Power gains by using external information in clinical trials are typically not possible when requiring strict type I error control. Biometrical Journal 62 (2):361–374. doi:10.1002/bimj.201800395.
  • Lord, S. J., L. Irwig, and R. J. Simes. 2006. When is measuring sensitivity and specificity sufficient to evaluate a diagnostic test, and when do we need randomized trials? Annals of Internal Medicine 144 (11):850–855. doi:10.7326/0003-4819-144-11-200606060-00011.
  • Moons, K. G. M., G.-A. van Es, J. W. Deckers, J. D. F. Habbema, and D. E. Grobbee. 1997. Limitations of sensitivity, specificity, likelihood ratio, and Bayes’ theorem in assessing diagnostic probabilities: A clinical example. Epidemiology 8 (1):12–17. doi:10.1097/00001648-199701000-00002.
  • Morita, S., P. F. Thall, and P. Müller. 2008. Determining the effective sample size of a parametric prior. Biometrics 64 (2):595–602. doi:10.1111/j.1541-0420.2007.00888.x.
  • Neelon, B., and A. J. O’Malley. 2010. Bayesian analysis using power priors with application to pediatric quality of care. Journal of Biometrics & Biostatistics 01 (1):1–9. doi:10.4172/2155-6180.1000103.
  • Neuenschwander, B., M. Branson, and D. J. Spiegelhalter. 2009. A note on the power prior. Statistics in Medicine 28 (28):3562–3566. doi:10.1002/sim.3722.
  • Neuenschwander, B., S. Roychoudhury, and H. Schmidli. 2016. On the use of co-data in clinical trials. Statistics in Biopharmaceutical Research 8 (3):345–354. doi:10.1080/19466315.2016.1174149.
  • Psioda, M. A., and J. G. Ibrahim. 2019. Bayesian clinical trial design using historical data that inform the treatment effect. Biostatistics 20 (3):400–415. doi:10.1093/biostatistics/kxy009.
  • Russek-Cohen, E., T. Feldblyum, K. B. Whitaker, and S. Hojvat. 2011. FDA perspectives on diagnostic device clinical studies for respiratory infections. Clinical Infectious Diseases 52 (suppl_4):305–311. doi:10.1093/cid/cir056.
  • Spiegelhalter, D. J., K. R. Abrams, and J. P. Myles. 2004. Bayesian approaches to clinical trials and health-care evaluation. Chichester, UK: John Wiley & Sons, Ltd.
  • United States Food and Drug Administration. 2010. Guidance for industry and FDA staff: guidance for the use of Bayesian statistics in medical device clinical trials. Rockville, MD.
  • United States Food and Drug Administration. 2011. Draft guidance for industry and food and drug administration staff - establishing the performance characteristics of nucleic acid-based in vitro fiagnostic devices for the detection and differentiation of methicillin-resistant staphylococcus aureu. Rockville, MD.
  • United States Food and Drug Administration. 2015. Leveraging existing clinical data for extrapolation to pediatric uses of medical devices: guidance for industry and food and administration staff. Silver Spring, MD.
  • Vandenberg, O., D. Martiny, O. Rochas, A. van Belkum, and Z. Kozlakidis. 2020. Considerations for diagnostic COVID-19 tests. Nature Reviews. Microbiology 19 (3):171–183. doi:10.1038/s41579-020-00461-z.
  • Viele, K., S. M. Berry, B. Neuenschwander, B. Amzal, F. Chen, N. Enas, B. P. Hobbs, J. G. Ibrahim, N. Kinnersley, S. Lindborg, et al. 2014. Use of historical control data for assessing treatment effects in clinical trials. Pharmaceutical Statistics 13 (1):41–54. doi:10.1002/pst.1589.
  • Wang, C., H. Li, W. C. Chen, N. Lu, R. Tiwari, Y. Xu, and 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, and 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., 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.

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