References
- Bauer, P. (1989), “Multistage Testing with Adaptive Designs,” Biometrie und Informatik in Medizin und Biologie, 20, 130–148.
- Bauer, P., and Koenig, F. (2006), “The Reassessment of Ttrial Perspectives from Interim Data–A Critical View,” Statistics in Medicine, 25, 23–36. DOI: 10.1002/sim.2180.
- Bauer, P., and Kohne, K. (1994), “Evaluation of Experiments with Adaptive Interim Analyses,” Biometrics, 50, 1029–1041.
- Beckman, R. A., and Chen, C. (2019), “Informational Designs and Potential Applications to Rare Disease,” in Handbook of Biomarkers and Precision Medicine, eds. C. Carini, M. Fidock, and A. van Gool, pp. 183–187, New York: Chapman and Hall/CRC.
- Brannath, W., Posch, M., and Bauer, P. (2002), “Recursive Combination tests’, Journal of the American Statistical Association, 97, 236–244. DOI: 10.1198/016214502753479374.
- Bretz, F., Koenig, F., Brannath, W., Glimm, E., and Posch, M. (2009), “Adaptive Designs for Confirmatory Clinical Trials,” Statistics in Medicine, 28, 1181–1217. DOI: 10.1002/sim.3538.
- Bretz, F., Schmidli, H., König, F., Racine, A., and Maurer, W. (2006), “Confirmatory Seamless Phase ii/iii Clinical Trials with Hypotheses Selection at Interim: General Concepts,” Biometrical Journal: Journal of Mathematical Methods in Biosciences, 48, 623–634. DOI: 10.1002/bimj.200510232.
- Chen, C., Li, N., Shentu, Y., Pang, L., and Beckman, R. A. (2016), “Adaptive Informational Design of Confirmatory Phase iii Trials with an Uncertain Biomarker Effect to Improve the Probability of Success,” Statistics in Biopharmaceutical Research, 8, 237–247. DOI: 10.1080/19466315.2016.1173582.
- Chen, L. M., Ibrahim, J. G., and Chu, H. (2014), “Flexible Stopping Boundaries When Changing Primary Endpoints After Unblinded Interim Analyses,” Journal of Biopharmaceutical Statistics, 24, 817–833. DOI: 10.1080/10543406.2014.901341.
- Congress, U. (1983), “Orphan Drug Act: To Amend the Federal Food, Drug, and Cosmetic Act to Facilitate the Development of Drugs for Rare Diseases and Conditions, and for other Purposes,” Public Law, 97–414.
- FDA (2019a), “Adaptive Designs for Clinical Trials of Drugs and Biologics,” Center for Biologics Evaluation and Research (CBER).
- FDA (2019b), “Rare Diseases: Common Issues in Drug Development; Draft Guidance for Industry,” Pharmaceuticals Policy and Law, 11, 345–52.
- Finner, H., and Strassburger, K. (2002), “The Partitioning Principle: A Powerful Tool in Multiple Decision Theory,” Annals of Statistics, 30, 1194–1213.
- Griggs, R. C., Batshaw, M., Dunkle, M., Gopal-Srivastava, R., Kaye, E., Krischer, J., Nguyen, T., Paulus, K., Merkel, P. A. et al. (2009), “Clinical Research for Rare Disease: Opportunities, Challenges, and Solutions,” Molecular Genetics and Metabolism, 96, 20–26. DOI: 10.1016/j.ymgme.2008.10.003.
- Hellmich, M., and Hommel, G. (2004), “Multiple Testing in Adaptive Designs–A Review,” in Recent Developments in Multiple Comparison Procedures, eds. Y. Benjamini, F. Bretz, and S. Sarkar, pp. 33–47, Beachwood, OH: Institute of Mathematical Statistics.
- Hung, J. H., Wang, S.-J., and O’Neill, R. T. (2006), “Methodological Issues with Adaptation of Clinical Trial Design,” Pharmaceutical Statistics, 5, 99–107. DOI: 10.1002/pst.219.
- Lan, K. K. G., and Wittes, J. (1988), “The B-Value: A Tool for Monitoring Data,” Biometrics, 44, 579–585. DOI: 10.2307/2531870.
- Lehmacher, W., and Wassmer, G. (1999), “Adaptive Sample Size Calculations in Group Sequential Trials,” Biometrics, 55, 1286–1290. DOI: 10.1111/j.0006-341x.1999.01286.x.
- Liu, Y., and Xu, H. (2021), “Sample Size Re-estimation for Pivotal Clinical Trials,” Contemporary Clinical Trials, 102, 106215. DOI: 10.1016/j.cct.2020.106215.
- Mehta, C., Schäfer, H., Daniel, H., and Irle, S. (2014), “Biomarker Driven Population Enrichment for Adaptive Oncology Trials with Time to Event Endpoints,” Statistics in Medicine, 33, 4515–4531. DOI: 10.1002/sim.6272.
- Müller, H.-H., and Schäfer, H. (2004), “A General Statistical Principle for Changing a Design Any Time during the Course of a Trial,” Statistics in Medicine, 23, 2497–2508. DOI: 10.1002/sim.1852.
- Sarkar, S. K. (1998), “Some Probability Inequalities for Ordered mtp2 Random Variables: A Proof of the Simes Conjecture,” Annals of Statistics, 26, 494–504.
- Stallard, N., and Todd, S. (2011), “Seamless Phase ii/iii Designs,” Statistical Methods in Medical Research, 20, 623–634. DOI: 10.1177/0962280210379035.
- Stefansson, G., Kim, W., and Hsu, J. (1988), “On Confidence Sets in Multiple Comparisons,” Statistical Decision Theory and Related Topics IV, 2, 89–104.
- Van der Ploeg, A. T., Clemens, P. R., Corzo, D., Escolar, D. M., Florence, J., Groeneveld, G. J., Herson, S., Kishnani, P. S., Laforet, P., Lake, S. L., et al. (2010), “A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe’s Disease,” New England Journal of Medicine, 362, 1396–1406. DOI: 10.1056/NEJMoa0909859.