226
Views
0
CrossRef citations to date
0
Altmetric
Research Articles

Probability of Study Success (PrSS) Evaluation Based on Multiple Endpoints in Late Phase Oncology Drug Development

, &
Pages 675-688 | Received 30 Jun 2021, Accepted 22 Aug 2022, Published online: 28 Oct 2022

References

  • Barlesi, F., Vansteenkiste, J., Spigel, D., Ishii, H., Garassino, M., de Marinis, F., Özgüroğlu, M., Szczesna, A., Polychronis, A., Uslu, R., Krzakowski, M., Lee, J.-S., Calabrò, L., Frontera, O. A., Ellers-Lenz, B., Bajars, M., Ruisi, M., and Park, K. (2018), “Avelumab versus Docetaxel in Patients with Platinum-Treated Advanced Non-Small-Cell Lung Cancer (JAVELIN lung 200): An Open-Label, Randomised, Phase 3 Study,” The Lancet Oncology, 19, 1468–1479. DOI: 10.1016/S1470-2045(18)30673-9.
  • Borghaei, H., Langer, C. J., Gadgeel, S., Papadimitrakopoulou, V. A., Patnaik, A., Powell, S. F., Gentzler, R. D., Martins, R. G., Stevenson, J. P., Jalal, S. I., Panwalkar, A., Chih-Hsin Yang, J., Gubens, M., Sequist, L. V., Awad, M. M., Fiore, J., Saraf, S., Keller, S. M., and Gandhi, L. (2019), “24-month Overall Survival from KEYNOTE-021 Cohort G: Pemetrexed and Carboplatin with or without Pembrolizumab as First-Line Therapy for Advanced Nonsquamous Non-Small Cell Lung Cancer,” Journal of Thoracic Oncology, 14, 124–129. DOI: 10.1016/j.jtho.2018.08.004.
  • Borghaei, H., Paz-Ares, L., Horn, L., Spigel, D. R., Steins, M., Ready, N. E., Chow, L. Q., Vokes, E. E., Felip, E., Holgado, E., Barlesi, F., Kohlhäufl, M., Arrieta, O., Burgio, M. A., Fayette, J., Lena, H., Poddubskaya, E., Gerber, D. E., Gettinger, S. N., Rudin, C. M., Rizvi, N., Crinò, L., Blumenschein, Jr., G. R., Antonia, S. J., Dorange, C., Harbison, C. T., Finckenstein, F. G., AND Brahmer, J. R. (2015), “Nivolumab versus Docetaxel in Advanced Nonsquamous Non-Small-Cell Lung Cancer,” New England Journal of Medicine, 373, 1627–1639. DOI: 10.1056/NEJMoa1507643.
  • Brahmer, J., Reckamp, K. L., Baas, P., Crinò, L., Eberhardt, W. E., Poddubskaya, E., Antonia, S., Pluzanski, A., Vokes, E. E., Holgado, E., Waterhouse, D., Ready, N., Gainor, J., Frontera, O. A., Havel, L., Steins, M., Garassino, M. C., Aerts, J. G., Domine, M., Paz-Ares, L., Reck, M., Baudelet, C., Harbison, C. T., Lestini, B., and Spigel, D. R. (2015), “Nivolumab versus Docetaxel in Advanced Squamous-Cell Non-Small-Cell Lung Cancer,” New England Journal of Medicine, 373, 123–135. DOI: 10.1056/NEJMoa1504627.
  • Carbone, D. P., Reck, M., Paz-Ares, L., Creelan, B., Horn, L., Steins, M., Felip, E., van den Heuvel, M. M., Ciuleanu, T.-E., Badin, F., Ready, N., Hiltermann, T. J. N., Nair, S., Juergens, R., Peters, S., Minenza, E., Wrangle, J. M., Rodriguez-Abreu, D., Borghaei, H., Blumenschein, G. R., Villaruz, L. C., Havel, L., Krejci, J., Jaime, J. C., Chang, H., Geese, W. J., Bhagavatheeswaran, P., Chen, A. C., and Socinski, M. A. (2017), “First-Line Nivolumab in Stage IV or Recurrent Non-Small-Cell Lung Cancer,” New England Journal of Medicine, 376, 2415–2426. DOI: 10.1056/NEJMoa1613493.
  • Cheung, Y. K., and Chappell, R. (2000), “Sequential Designs for Phase I Clinical Trials with Late-Onset Toxicities,” Biometrics, 56, 1177–1182. DOI: 10.1111/j.0006-341x.2000.01177.x.
  • Cho, M., Qiu, Z., Ye, J., Quan, H., and Zhao, P.-L. (2018), “Considerations for Pediatric Trial Designs and Analyses,” Journal of Biopharmaceutical Statistics, 28, 413–436. DOI: 10.1080/10543406.2017.1372771.
  • Fehrenbacher, L., Spira, A., Ballinger, M., Kowanetz, M., Vansteenkiste, J., Mazieres, J., Park, K., Smith, D., Artal-Cortes, A., Lewanski, C., Braiteh, F., Waterkamp, D., He, P., Zou, W., Chen, D. S., Yi, J., Sandler, A., Rittmeyer, A., and POPLAR Study Group. (2016), “Atezolizumab versus Docetaxel for Patients with Previously Treated Non-Small-Cell Lung Cancer (POPLAR): A Multicentre, Open-Label, Phase 2 Randomised Controlled Trial,” The Lancet, 387, 1837–1846. DOI: 10.1016/S0140-6736(16)00587-0.
  • Gandhi, L., Rodríguez-Abreu, D., Gadgeel, S., Esteban, E., Felip, E., De Angelis, F., Domine, M., Clingan, P., Hochmair, M. J., Powell, S. F., Cheng, S. Y.-S., Bischoff, H. G., Peled, N., Grossi, F., Jennens, R. R., Reck, M., Hui, R., Garon, E. B., Boyer, M., Rubio-Viqueira, B., Novello, S., Kurata, T., Gray, J. E., Vida, J., Wei, Z., Yang, J., Raftopoulos, H., Catherine Pietanza, M., and Garassino, M. C. (2018), “Pembrolizumab Plus Chemotherapy in Metastatic Non-Small-Cell Lung Cancer,” New England Journal of Medicine, 378, 2078–2092. DOI: 10.1056/NEJMoa1801005.
  • Herbst, R. S., Baas, P., Kim, D.-W., Felip, E., Pérez-Gracia, J. L., Han, J.-Y., Molina, J., Kim, J.-H., Arvis, C. D., Ahn, M.-J., Majem, M., Fidler, M. J., de Castro Jr, G., Garrido, M., Lubiniecki, G. M., Shentu, Y., Im, E., Dolled-Filhart, M., and Garon, E. B. (2016), “Pembrolizumab versus Docetaxel for Previously Treated, PD-L1-Positive, Advanced Non-Small-Cell Lung Cancer (keynote-010): A Randomised Controlled Trial,” The Lancet, 387), 1540–1550. DOI: 10.1016/S0140-6736(15)01281-7.
  • Hong, S., and Shi, L. (2012), “Predictive Power to Assist Phase 3 go/no go Decision based on Phase 2 Data on a Different Endpoint,” Statistics in Medicine, 31, 831–843. DOI: 10.1002/sim.4476.
  • Ibrahim, J. G., and Chen, M.-H. (2000), “Power Prior Distributions for Regression Models,” Statistical Science, 15, 46–60.
  • Imai, H., Kaira, K., and Minato, K. (2017), “Clinical Significance of Post-Progression Survival in Lung Cancer,” Thoracic Cancer, 8, 379–386. DOI: 10.1111/1759-7714.12463.
  • Jotte, R., Cappuzzo, F., Vynnychenko, I., Stroyakovskiy, D., Rodríguez-Abreu, D., Hussein, M., Soo, R., Conter, H. J., Kozuki, T., Huang, K.-C., Graupner, V., Sun, S. W., Hoang, T., Jessop, H., McCleland, M., Ballinger, M., Sandler, A., and Socinski, M. A. (2020), “Atezolizumab in Combination with Carboplatin and Nab-paclitaxel in Advanced Squamous NSCLC (IMpower131): Results from a Randomized Phase III Trial,” Journal of Thoracic Oncology, 15, 1351–1360. DOI: 10.1016/j.jtho.2020.03.028.
  • Kaciroti, N. A., Raghunathan, T. E., Taylor, J. M., and Julius, S. (2012), “A Bayesian Model for Time-to-Event Data with Informative Censoring,” Biostatistics, 13, 341–354. DOI: 10.1093/biostatistics/kxr048.
  • Kaufman, H. L., Schwartz, L. H., William, W. N., Sznol, M., Fahrbach, K., Xu, Y., Masson, E., and Vergara-Silva, A. (2018), “Evaluation of Classical Clinical Endpoints as Surrogates for Overall Survival in Patients Treated with Immune Checkpoint Blockers: A Systematic Review and Meta-Analysis,” Journal of Cancer Research and Clinical Oncology, 144, 2245–2261. DOI: 10.1007/s00432-018-2738-x.
  • Mok, T. S., Wu, Y.-L., Kudaba, I., Kowalski, D. M., Cho, B. C., Turna, H. Z., Castro Jr, G., Srimuninnimit, V., Laktionov, K. K., Bondarenko, I., Kubota, K., Lubiniecki, G. M., Zhang, J., Kush, D., and Lopes, G. (2019), “Pembrolizumab versus Chemotherapy for Previously Untreated, PD-L1-Expressing, Locally Advanced or Metastatic Non-Small-Cell Lung Cancer (keynote-042): A Randomised, Open-Label, Controlled, Phase 3 Trial,” The Lancet, 393, 1819–1830. DOI: 10.1016/S0140-6736(18)32409-7.
  • Mushti, S. L., Mulkey, F., and Sridhara, R. (2018), “Evaluation of Overall Response Rate and Progression-Free Survival as Potential Surrogate Endpoints for Overall Survival in Immunotherapy Trials,” Clinical Cancer Research, 24, 2268–2275. DOI: 10.1158/1078-0432.CCR-17-1902.
  • Papadimitrakopoulou, V., Cobo, M., Bordoni, R. A., Szalai, Z., and Ursol, G. (2018), “IMpower132: PFS and Safety Results with 1L Atezolizumab + Carboplatin/Cisplatin + Pemetrexed in Stage IV Non-Squamous NSCLC,” Journal of Thoracic Oncology, 13, 332–333. DOI: 10.1016/j.jtho.2018.08.262.
  • Paz-Ares, L., Ciuleanu, T., Yu, X., Salman, P., Pluzanski, A., Nagrial, A., Havel, L., Kowalyszyn, R., Audigier-Valette, C., Wu, Y. L., Borghaei, H., Hellmann, M., Brahmer, J., Reck, M., Ramalingam, S., Zhang, L., Bhagavatheeswaran, P., Nathan, F. E., and O’Byrne1, K. J. (2019), “LBA3 Nivolumab (nivo)+ Platinum-Doublet Chemotherapy (chemo) vs Chemo as First-Line (1l) Treatment (tx) for Advanced Non-Small Cell Lung Cancer (aNSCLC): Checkmate 227-Part 2 Final Analysis,” Annals of Oncology, 30, xi67–xi68. DOI: 10.1093/annonc/mdz453.004.
  • Paz-Ares, L., Luft, A., Vicente, D., Tafreshi, A., Gümüş, M., Mazières, J., Hermes, B., Çay, Ş. F., Csőszi, T., Fülöp, A., Rodr iguez-Cid, J., Wilson, J., Sugawara, S., Kato, T., Lee, K. H., Cheng, Y., Novello, S., Halmos, B., Li, X., Lubiniecki, G. M., Piperdi, B., and Kowalski, D. M. (2018), “Pembrolizumab Plus Chemotherapy for Squamous Non-Small-Cell Lung Cancer,” The New England Journal of Medicine, 379, 2040–2051. DOI: 10.1056/NEJMoa1810865.
  • Planchard, D., Reinmuth, N., Orlov, S., Fischer, J., Sugawara, S., Mandziuk, S., Marquez-Medina, D., Novello, S., Takeda, Y., Soo, R., Park, K., McCleod, M., Geater, S. L., Powell, M., May, R., Scheuring, U., Stockman, P., and Kowalski, D. (2020), “Arctic: Durvalumab with or without Tremelimumab as Third-Line or Later Treatment of Metastatic Non-Small-Cell Lung Cancer,” Annals of Oncology, 31, 609–618. DOI: 10.1016/j.annonc.2020.02.006.
  • Reck, M., Rodríguez-Abreu, D., Robinson, A. G., Hui, R., Csőszi, T., Fülöp, A., Gottfried, M., Peled, N., Tafreshi, A., Cuffe, S., O’Brien, M., Rao, S., Hotta, K., Leiby, M. A., Lubiniecki, G. M., Shentu, Y., Rangwala, R., and Brahmer, J. R. (2016), “Pembrolizumab versus Chemotherapy for PD-L1–Positive Non–Small-Cell Lung Cancer,” The New England Journal of Medicine, 375, 1823–1833. DOI: 10.1056/NEJMoa1606774.
  • Rittmeyer, A., Barlesi, F., Waterkamp, D., Park, K., Ciardiello, F., von Pawel, J., Gadgeel, S. M., Hida, T., Kowalski, D. M., Dols, M. C., Cortinovis, D. L., Leach, J., Polikoff, J., Barrios, C., Kabbinavar, F., Frontera, O. A., De Marinis, F., Turna, H., Lee, J.-S., Ballinger, M., Kowanetz, M., He, P., Chen, D. S., Sandler, A., and Gandara, D. R. (2017), “Atezolizumab versus Docetaxel in Patients with Previously Treated Non-small-cell Lung Cancer (oak): A Phase 3, Open-Label, Multicentre Randomised Controlled Trial,” The Lancet, 389, 255–265. DOI: 10.1016/S0140-6736(16)32517-X.
  • Sabin, T., Matcham, J., Bray, S., Copas, A., and Parmar, M. K. (2014), “A Quantitative Process for Enhancing End of Phase 2 Decisions,” Statistics in Biopharmaceutical Research, 6, 67–77. DOI: 10.1080/19466315.2013.852617.
  • Saint-Hilary, G., Barboux, V., Pannaux, M., Gasparini, M., Robert, V., and Mastrantonio, G. (2019), “Predictive Probability of Success using Surrogate Endpoints,” Statistics in Medicine, 38, 1753–1774. DOI: 10.1002/sim.8060.
  • Schmidli, H., Gsteiger, S., Roychoudhury, S., O’Hagan, A., Spiegelhalter, D., and Neuenschwander, B. (2014), “Robust Meta-Analytic-Predictive Priors in Clinical Trials with Historical Control Information,” Biometrics, 70, 1023–1032. DOI: 10.1111/biom.12242.
  • Shukuya, T., Mori, K., Amann, J. M., Bertino, E. M., Otterson, G. A., Shields, P. G., Morita, S., and Carbone, D. P. (2016), “Relationship between Overall Survival and Response or Progression-Free Survival in Advanced Non–Small Cell Lung Cancer Patients Treated with Anti–pd-1/pd-l1 Antibodies,” Journal of Thoracic Oncology, 11, 1927–1939.
  • Socinski, M. A., Jotte, R. M., Cappuzzo, F., Orlandi, F., Stroyakovskiy, D., Nogami, N., Rodríguez-Abreu, D., Moro-Sibilot, D., Thomas, C. A., Barlesi, F., Finley, G., Kelsch, C., Lee, A., Coleman, S., Deng, Y., Shen, Y., Kowanetz, M., Lopez-Chavez, A., Sandler, A., and Reck, M. (2018), “Atezolizumab for First-Line Treatment of Metastatic Nonsquamous NSCLC,” New England Journal of Medicine, 378, 2288–2301. DOI: 10.1056/NEJMoa1716948.
  • Stallard, N. (2010), “A Confirmatory Seamless Phase II/III Clinical Trial Design Incorporating Short-Term Endpoint Information,” Statistics in Medicine, 29, 959–971. DOI: 10.1002/sim.3863.
  • Wang, Y., Fu, H., Kulkarni, P., and Kaiser, C. (2013), “Evaluating and Utilizing Probability of Study Success in Clinical Development,” Clinical Trials, 10, 407–413. DOI: 10.1177/1740774513478229.
  • Wellner, J. A., and Zhan, Y. (1997), “A Hybrid Algorithm for Computation of the Nonparametric Maximum Likelihood Estimator from Censored Data,” Journal of the American Statistical Association, 92, 945–959. DOI: 10.1080/01621459.1997.10474049.
  • Yu, J., Hodge, J. P., Oliva, C., Neftelinov, S. T., Hubbard-Lucey, V. M., and Tang, J. (2019), “Trends in Clinical Development for pd-1/pd-l1 Inhibitors,” Nature Reviews Drug Discovery, 19, 163–164.

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.