958
Views
1
CrossRef citations to date
0
Altmetric
Statistical Innovation in Healthcare: Celebrating the Past 40 Years and Looking Toward the Future - Special issue for the 2021 Regulatory-Industry Statistics Workshop

Clinical and Statistical Perspectives on the ICH E9(R1) Estimand Framework Implementation

ORCID Icon, , , ORCID Icon, , , , , & ORCID Icon show all
Pages 554-559 | Received 10 Jan 2022, Accepted 20 May 2022, Published online: 06 Jul 2022

References

  • Akacha, M., Branson, J., Bretz, F., Dharan, B., Gallo, P., Gathmann, I., Hemmings, R., Jones, J., Xi, D., and Zuber, E. (2020), “Challenges in Assessing the Impact of the COVID-19 Pandemic on the Integrity and Interpretability of Clinical Trials,” Statistics in Biopharmaceutical Research, 12, 419–426. no. DOI: 10.1080/19466315.2020.1788984.
  • Bornkamp, B., Rufibach, K., Lin, J., Liu, Y., Mehrotra, D. V., Roychoudhury, S., Schmidli, H., Shentu, Y., and Wolbers, M. (2021), “Principal stratum strategy: Potential role in drug development,” Pharmaceutical Statistics, 20, 737–751. DOI: 10.1002/pst.2104.
  • Goodman, P. J., Tangen, C. M., and Thompson, I. M. Jr. (2019), “More on Long-Term Effects of Finasteride on Prostate Cancer Mortality. Reply,” The New England Journal of Medicine, 380, e38. DOI: 10.1056/NEJMc1902700.
  • International Council for Harmonization (1998), “E9: Statistical Principles for Clinical Trials,” available at http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E9/Step4/E9_Guideline.pdf.
  • International Council for Harmonization (2017), “E9(R1): Addendum: Statistical Principles for Clinical Trials,” available at http://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E9/E9-R1EWG_Step2_Guideline_2017_0616.pdf.
  • International Council for Harmonization (2019), “E9(R1): Addendum: Statistical Principles for Clinical Trials,” available at https://database.ich.org/sites/default/files/E9-R1_Step4_Guideline_2019_1203.pdf.
  • LaVange, L. M., and Permutt, T. (2016), “A Regulatory Perspective on Missing Data in the Aftermath of the NRC Report,” Statistics in Medicine, 35, 2853–2864. DOI: 10.1002/sim.6840.
  • Lipkovich, I., Ratitch, B., and Mallinckrodt, C. H. (2020), “Causal Inference and Estimands in Clinical Trials,” Statistics in Biopharmaceutical Research, 12, 54–67. DOI: 10.1080/19466315.2019.1697739.
  • Ludvik, B., Giorgino, F., Jódar, E., Frias, J. P., Fernández Landó, L., Brown, K., Bray, R., and Rodríguez, Á. (2021), “Once-Weekly Tirzepatide Versus Once-Daily Insulin Degludec as add-on to Metformin with or without SGLT2 Inhibitors in Patients with Type 2 Diabetes (SURPASS-3): A Randomised, Open-Label, Parallel-Group, Phase 3 Trial,” The Lancet, 398, 583–598. no. 01443-4. DOI: 10.1016/S0140-6736(21).
  • Meyer, R. D., Ratitch, B., Wolbers, M., Marchenko, O., Quan, H., Li, D., Fletcher, C., Li, X., Wright, D., Shentu, Y., Englert, S., Shen, W., Dey, J., Liu, T., Zhou, M., Bohidar, N., Zhao, P. L., and Hale, M. (2020), “Statistical Issues and Recommendations for Clinical Trials Conducted During the COVID-19 Pandemic,” Statistics in Biopharmaceutical Research, 12, 399–411. no. DOI: 10.1080/19466315.2020.1779122.
  • National Research Council, Committee on National Statistics, Division of Behavioral and Social Sciences and Education (2010), The Prevention and Treatment of Missing Data in Clinical Trials. Panel on Handling Missing Data in Clinical Trials. Washington, DC: National Academies Press.
  • Permutt, T. (2015a), “A Taxonomy of Estimands for Regulatory Clinical Trials with Discontinuations,” Statistics in Medicine, 35, 2865–2875. no. DOI: 10.1002/sim.6841.
  • Permutt, T. (2015b), “Sensitivity Analysis for Missing Data in Regulatory Submissions,” Statistics in Medicine, 35, 2876–2879. doi:10. 1002/sim.6753.
  • Qu, Y., and Lipkovich, I. (2021), “Implementation of ICH E9 (R1): A Few Points Learned During the COVID-19 Pandemic,” Therapeutic Innovation & Regulatory Science, 55, 984–988. DOI: 10.1007/s43441-021-00297-6.
  • Rabe, K. F., Martinez, F. J., Ferguson, G. T., Wang, C., Singh, D., Wedzicha, J. A., Trivedi, R., St. Rose, E., Ballal, S., McLaren, J., Darken, P., Aurivillius, M., Reisner, C., and Dorinsky, P. (2020), “Triple Inhaled Therapy at Two Glucocorticoid Doses in Moderate-to-Very-Severe COPD,” New England Journal of Medicine, 383, 35–48. DOI: 10.1056/NEJMoa1916046.
  • Rubino, D., Abrahamsson, N., Davies, M., Hesse, D., Greenway, F. L., Jensen, C., Lingvay, I., Mosenzon, O., Rosenstock, J., Rubio, M. A., Rudofsky, G., Tadayon, S., Wadden, T. A., and Dicker, D. and Step Investigators. (2021), “Effect of Continued Weekly Subcutaneous Semaglutide vs Placebo on Weight Loss Maintenance in Adults With Overweight or Obesity: The STEP 4 Randomized Clinical Trial,” JAMA, 325, 1414–1425. no. DOI: 10.1001/jama.2021.3224.
  • Shepherd, B. E., Redman, M. W., and Ankerst, D. P. (2008), “Does Finasteride Affect the Severity of Prostate Cancer? A Causal Sensitivity Analysis,” Journal of the American Statistical Association, 103, 1392–1404. noDOI: 10.1198/016214508000000706.
  • Thompson, I. M., Goodman, P. J., Tangen, C. M., Lucia, M. S., Miller, G. J., Ford, L. G., Lieber, M. M., Cespedes, R. D., Atkins, J. N., Lippman, S. M., Carlin, S. M., Ryan, A., Szczepanek, C. M., Crowley, J. J., and Coltman, C. A. Jr. (2003), “The Influence of Finasteride on the Development of Prostate Cancer,” New England Journal of Medicine, 349, 215–224. DOI: 10.1056/NEJMoa030660.
  • U.S. Food and Drug Administration (2021a), “E9(R1) Statistical Principles for Clinical Trials: Addendum: Estimands and Sensitivity Analysis in Clinical Trials,” available at https://www.fda.gov/media/148473/download.
  • U.S. Food and Drug Administration (2021b), “Draft Guidance: Chronic Rhinosinusitis with Nasal Polyps: Developing Drugs for Treatment,” available at https://www.fda.gov/media/154724/download.

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.