Estimands in a Chronic Pain Trial: Challenges and Opportunities

References

• Akacha, M., Bretz, F., Ohlssen, D., Rosenkranz, G., and Schmidli, H. (2017), “Estimands and Their Role in Clinical Trials,” Statistics in Biopharmaceutical Research, 9, 268–271. DOI: 10.1080/19466315.2017.1302358.
   Web of Science ®Google Scholar
• Carpenter, J., Roger, J., and Kenward, M. (2013), “Analysis of Longitudinal Trials With Protocol Deviation: A Framework for Relevant, Accessible Assumptions, and Inference via Multiple Imputation,” Journal of Biopharmaceutical Statistics, 23, 1352–1371. DOI: 10.1080/10543406.2013.834911.
   PubMed Web of Science ®Google Scholar
• Chaparro, L. E., Wiffen, P. J., Moore, R. A., and Gilron, I. (2012), “Combination Pharmacotherapy for the Treatment of Neuropathic Pain in Adults,” Cochrane Database of Systematic Reviews, 7, CD008943.
   PubMed Web of Science ®Google Scholar
• Dworkin, R. H., Turk, D. C., Peirce-Sandner, S., Baron, R., Bellamy, N., Burke, L. B., Chappell, A., Chartier, K., Cleeland, C. S., Costello, A., Cowan, P., Dimitrova, R., Ellenberg, S., Farrar, J. T., French, J. A., Gilron, I., Hertz, S., Jadad, A. R., Jay, G. W., Kalliomäki, J., Katz, N. P., Kerns, R. D., Manning, D. C., McDermott, M. P., McGrath, P. J., Narayana, A., Porter, L., Quessy, S., Rappaport, B. A., Rauschkolb, C., Reeve, B. B., Rhodes, T., Sampaio, C., Simpson, D. M., Stauffer, J. W., Stucki, G., Tobias, J., White, R. E., and Witter, J. (2010), “Research Design Considerations for Confirmatory Chronic Pain Clinical Trials: IMMPACT Recommendations,” Pain, 149, 177–193. DOI: 10.1016/j.pain.2010.02.018.
   PubMed Web of Science ®Google Scholar
• Dworkin, R., Turk, D., Peirce-Sandner, S., Burke, L., Farrar, J., Gilron, I., Jensen, M., Katz, N., Raja, S., Rappaport, B., Rowbotham, M., Backonja, M., Baron, R., Bellamy, N., Bhagwagar, Z., Costello, A., Cowan, P., Fang, W., Hertz, S., Jay, G., Junor, R., Kerns, R., Kerwin, R., Kopecky, E., Lissin, D., Malamut, R., Markman, J., McDermott, M., Munera, C., Porter, L., Rauschkolb, C., Rice, A., Sampaio, C., Skljarevski, V., Sommerville, K., Stacey, B., Steigerwald, I., Tobias, J., Trentacosti, A., Wasan, A., Wells, G., Williams, J., Witter, J., and Ziegler, D. (2012), “Considerations for Improving Assay Sensitivity in Chronic Pain Clinical Trials: IMMPACT Recommendations,” Pain, 153, 1148–1158. DOI: 10.1016/j.pain.2012.03.003.
   PubMed Web of Science ®Google Scholar
• International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use (1998), “ICH Harmonised Tripartite Guideline E9: Statistical Principles for Clinical Trials,” available at https://www.ich.org/fileadmin/Public_Web_Site/ICH_Products/Guidelines/Efficacy/E9/Step4/E9_Guideline.pdf.
   Google Scholar
• International Conference on Harmonization (2017), “Draft ICH E9 (R1) Addendum on ‘Estimands and Sensitivity Analysis in Clinical Trials to the Guideline on Statistical Principles for Clinical Trials (EMA/CHMP/ICH/436221/2017)’,” available at https://www.ema.europa.eu/docs/en_GB/document_library/Scientific_guideline/2017/08/WC500233916.pdf.
   Google Scholar
• Hernán, M., Hernández-Díaz, S., and Robins, J. (2013), “Randomized Trials Analyzed as Observational Studies,” Annals of Internal Medicine, 159, 560–562, DOI: 10.7326/0003-4819-159-8-201310150-00709.
   PubMed Web of Science ®Google Scholar
• IASP Task Force on Taxonomy (1994), “Part III: Pain Terms—A Current List With Definitions and Notes on Usage,” in Classification of Chronic Pain (2nd ed.), eds. H. Merskey and N. Bogduk, Seattle: IASP Press, available at https://www.iasp-pain.org/Education/Content.aspx?ItemNumber=1698.
   Google Scholar
• Mehrotra, D., Hemmings, R., Russek-Cohen, E., and ICH E9/R1 Expert Working Group (2016), “Seeking Harmony: Estimands and Sensitivity Analyses for Confirmatory Clinical Trials,” Clinical Trials, 13, 456–458. DOI: 10.1177/1740774516633115.
   PubMed Web of Science ®Google Scholar
• Permutt, T. (2016), “Sensitivity Analysis for Missing Data in Regulatory Submissions,” Statistics in Medicine, 35, 2876–2879. DOI: 10.1002/sim.6753.
   PubMed Web of Science ®Google Scholar
• Phillips, A., Abellan-Andres, J., Andersen, S., Bretz, F., Fletcher, C., France, L., Garrett, A., Harris, R., Kjaer, M., Keene, O., Morgan, D., O’Kelly, M., and Roger, J. (2017), “Estimands: Discussion Points From the PSI Estimands and Sensitivity Expert Group,” Pharmaceutical Statistics, 16, 6–11. DOI: 10.1002/pst.1745.
   PubMed Web of Science ®Google Scholar
• Rubin, D. (2008), Multiple Imputation for Nonresponse in Surveys, Wiley Series in Probability and Statistics, New York: Wiley, DOI: 10.1002/9780470316696.
   Google Scholar
• Treede, R., Rief, W., Barke, A., Aziz, Q., Bennett, M., Benoliel, R., Cohen, M., Evers, S., Finnerup, N., First, M., Giamberardino, M., Kaasa, S., Kosek, E., Lavand’homme, P., Nicholas, M., Perrot, S., Scholz, J., Schug, S., Smith, B., Svensson, P., Vlaeyen, J., and Wang, S. (2015), “A Classification of Chronic Pain for ICD-11,” Pain, 156, 1003–1007. DOI: 10.1097/j.pain.0000000000000160.
   PubMed Web of Science ®Google Scholar