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Letter to the Editor

Designing Dose-Optimization Studies in Cancer Drug Development: Discussions with Regulators

, , , , , , , , , , , , , , , , , , ORCID Icon, , , , , , , , , , & show all
Pages 697-703 | Received 30 Nov 2022, Accepted 20 Dec 2022, Published online: 14 Feb 2023

References

  • Bekele, B. N., and Thall, P. F. (2004), “Dose-Finding based on Multiple Toxicities in a Soft Tissue Sarcoma Trial,” Journal of the American Statistical Association, 99, 26–35.
  • Bornkamp, B., Bretz, F., and Pinheiro, J. (2006), “Design and Analysis of Dose Finding Studies Combining Multiple Comparisons and Modeling Procedures,” Journal of Biopharmaceutical Statistics, 16, 639–656. DOI: 10.1080/10543400600860428.
  • Bretz, F., Pinheiro, J. C., and Branson, M. (2005), “Combining Multiple Comparisons and Modelling Techniques in Dose-Response Studies,” Biometrics, 61, 738–748. DOI: 10.1111/j.1541-0420.2005.00344.x.
  • Cheung, Y. K., and Chappell, R. (2000), “Sequential Designs for Phase I Clinical Trials with Lateonset Toxicities,” Biometrics, 56, 1177–1182. DOI: 10.1111/j.0006-341x.2000.01177.x.
  • de Greef, R., Elassaiss-Schaap, J., Chatterjee, M., Turner, D. C., Ahamadi, M., Forman, M., Cutler, D., de Alwis, D. P., Kondic, A., and Stone, J. (2017), “Pembrolizumab: Role of Modeling and Simulation in Bringing a Novel Immunotherapy to Patients With Melanoma,” CPT: Pharmacometrics & Systems Pharmacology, 6, 5–7. DOI: 10.1002/psp4.12131.
  • Du, Y., Yin, J., Sargent, D. J., and Mandrekar, S. J. (2019), “An Adaptive Multi-Stage Phase I Dose-finding Design Incorporating Continuous Efficacy and Toxicity Data from Multiple Treatment Cycles,” Journal of Biopharmaceutical Statistics, 29, 271–286. DOI: 10.1080/10543406.2018.1535497.
  • Ezzalfani, M., Zohar, S., Qin, R., Mandrekar, S. J., and Deley, M.-C. L. (2013), “Dose-Finding Designs Using a Novel Quasi-Continuous Endpoint for Multiple Toxicities,” Statistics in Medicine, 32, 2728–2746. DOI: 10.1002/sim.5737.
  • Freshwater, T., Kondic, A., Ahamadi, M., Li, C. H., de Greef, R., de Alwis, D., and Stone, J. A. (2017), “Evaluation of Dosing Strategy for Pembrolizumab for Oncology Indications,” Journal for Immunotherapy of Cancer, 5, 43. DOI: 10.1186/s40425-017-0242-5.
  • Hobbs, B. P., Carlin, B. P., and Sargent, D. J. (2013), “Adaptive Adjustment of the Randomization Ratio using Historical Control Data,” Clinical Trials, 10, 430–440. DOI: 10.1177/1740774513483934.
  • Jin, I. H., Liu, S., Thall, P. F., and Yuan, Y. (2014), “Using Data Augmentation to Facilitate Conduct of Phase I-II Clinical Trials with Delayed Outcomes,” Journal of the American Statistical Association, 109, 525–536.
  • Lala, M., Li, T. R., de Alwis, D. P., Sinha, V., Mayawala, K., Yamamoto, N., Siu, L. L., Chartash, E., Aboshady, H., and Jain, L. (2020), “A Six-weekly Dosing Schedule for Pembrolizumab in Patients with Cancer Based on Evaluation using Modelling and Simulation,” European Journal of Cancer, 131, 68–75.
  • Le-Rademacher, J. G., Hillman, S., Storrick, E., Mahoney, M. R., Thall, P. F., Jatoi, A., and Mandrekar, S. J. (2020), “A Versatile Summary Measure for Cancer Clinical Trials,” Cancers, 12, 1–15.
  • Lin, R., Zhou, Y., Yan, F., Li, D., and Yuan, Y. (2020), “BOIN12: Bayesian Optimal Interval Phase I/II Trial Design for Utility-Based Dose Finding in Immunotherapy and Targeted Therapies,” JCO Precision Oncology, 4, 1393–1402.
  • Liu, S., Yin, G., and Yuan, Y. (2013), “Bayesian Data Augmentation Dose Finding with Continual Reassessment Method and Delayed Toxicity,” The Annals of Applied Statistics, 7, 1837–2457.
  • Marchenko, O., Sridhara, R., Jiang, Q., Barksdale, E., and Pazdur, R. (2021a), “Summary of ASA Virtual Discussion with Regulators on Designing Dose-Optimization Studies in Cancer Drug Development,” Biopharmaceutical Report, Summer Issue, 28, 26–27.
  • Marchenko, O., Sridhara, R., Jiang, Q., Barksdale, E., and Pazdur, R. (2021b), “Summary of ASA Virtual Discussion with Regulators on Designing Dose-Optimization Studies in Cancer Drug Development,” Biopharmaceutical Report, Fall Issue, 28, 16–20.
  • Mu, R., Yuan, Y., Xu, J., Mandrekar, S. J., and Yin, J. (2019), “gBOIN: A Unified Model-Assisted Phase I Trial Design Accounting for Toxicity Grades, and Binary or Continuous End Points,” Journal of the Royal Statistical Society, Series C, 68, 289–308.
  • Normington, J., Zhu, J., Mattiello, F., Sarkar, S., and Carlin, B. (2020). “An Efficient Bayesian Platform Trial Design for Borrowing Adaptively from Historical Control Data in Lymphoma,” Contemporary Clinical Trials, 89, 105890. DOI: 10.1016/j.cct.2019.105890.
  • Pinheiro, J., Bornkamp, B., Glimm, E., and Bretz, F. (2014), “Model-Based Dose Finding Under Model Uncertainty Using General Parametric Models,” Statistics in Medicine, 33, 1646–1661.
  • Shah, M., Rahman, A., Theoret, M. R., and Pazdur, R. (2021), “The Drug-Dosing Conundrum in Oncology – When Less Is More,” The New England Journal of Medicine, 385, 1445–1447.
  • Thall, P. F., and Cook, J. D. (2004), “Dose-Finding Based on Efficacy-Toxicity Trade-offs,” Biometrics, 60, 684–693. DOI: 10.1111/j.0006-341X.2004.00218.x.
  • Yin, J., Qin, R., Ezzalfani, M., Sargent, D. J., and Mandrekar, S. J. (2017), “A Bayesian Dose-Finding Design Incorporating Toxicity Data from Multiple Treatment Cycles,” Statistics in Medicine, 36, 67–80. DOI: 10.1002/sim.7134.
  • Zhou, Y., Lee, J. J., and Yuan, Y. (2019), “A Utility-Based Bayesian Optimal Interval (U-BOIN) Phase I/II Design to Identify the Optimal Biological Dose for Targeted and Immune Therapies,” Statistics in Medicine, 38, 5299–5316.
  • Zhou, Y., Lin, R., Lee, J. J., L. D., Wang, L., Li, R., and Yuan, Y. (2021), “TITE-BOIN12: A Bayesian Phase I/II Trial Design to Find the Optimal Biological Dose with Late-onset Toxicity and Efficacy,” Stat Med. Under revision.

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