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Statistical Innovation in Healthcare: Celebrating the Past 40 Years and Looking Toward the Future - Special issue for the 2021 Regulatory-Industry Statistics Workshop

A Comparison of Estimand and Estimation Strategies for Clinical Trials in Early Parkinson’s Disease

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Pages 491-501 | Received 07 Dec 2021, Accepted 17 Aug 2022, Published online: 03 Oct 2022

References

  • Armstrong, M. J., and Okun, M. S. (2020), “Diagnosis and Treatment of Parkinson Disease: A Review,” JAMA, 323, 548–560. DOI: 10.1001/jama.2019.22360.
  • Bartlett, J. W. (2021), “Reference-based Multiple Imputation—What is the Right Variance and How to Estimate it,” Statistics in Biopharmaceutical Research. DOI: 10.1080/19466315.2021.1983455..
  • 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.
  • Carpenter, J. R., Roger, J. H., and Kenward, M. G. (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.
  • Chen, Y.-F., Ni, X., Fleisher, A. S., Zhou, W., Aisen, P., and Mohs, R. (2018), “A Simulation Study Comparing Slope Model with Mixed-Model Repeated Measure to Assess Cognitive Data in Clinical Trials of Alzheimer’s Disease,” Alzheimer’s & Dementia: Translational Research & Clinical Interventions, 4, 46–53.
  • Committee for Medicinal Products for Human Use (CHMP) (2012), “Guideline on Clinical Investigation of Medicinal Products in the Treatment of Parkinson’s Disease (revision 2 – adopted guideline),” available at https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-clinical-investigation-medicinal-products-treatment-parkinsons-disease_en-0.pdf.
  • Committee for Medicinal Products for Human Use (CHMP) (2018), “Guideline on the Clinical Investigation of Medicines for the Treatment of Alzheimer’s Disease (revision 2 – adopted guideline),” available at https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-clinical-investigation-medicines-treatment-alzheimers-disease-revision-2_en.pdf.
  • Cro, S., Morris, T. P., Kenward, M. G., and Carpenter, J. R. (2020), “Sensitivity Analysis for Clinical Trials with Missing Continuous Outcome Data using Controlled Multiple Imputation: A Practical Guide,” Statistics in Medicine, 39, 2815–2842. DOI: 10.1002/sim.8569.
  • Darken, P., Nyberg, J., Ballal, S., and Wright, D. (2020), “The Attributable Estimand: A New Approach to Account for Intercurrent Events,” Pharmaceutical Statistics, 19, 626–635. DOI: 10.1002/pst.2019.
  • Goetz, C. G., Tilley, B. C., Shaftman, S. R., Stebbins, G. T., Fahn, S., Martinez-Martin, P., Poewe, W., Sampaio, C., Stern, M. B., Dodel, R., and Dubois, B. (2008), “Movement Disorder Society-Sponsored Revision of the Unified Parkinson’s Disease Rating Scale (MDS-UPDRS): Scale Presentation and Clinimetric Testing Results,” Movement Disorders: Official Journal of the Movement Disorder Society, 23, 2129–2170. DOI: 10.1002/mds.22340.
  • Gower-Page, C., Noci, A., and Wolbers, M. (2022), “rbmi: A R package for standard and reference-based multiple imputation methods,” Journal of Open Source Software, 7, 4251. DOI: 10.21105/joss.04251.
  • Guizzaro, l., Pétavy, F., Ristl, R., and Gallo, C. (2021), “The Use of a Variable Representing Compliance Improves Accuracy of Estimation of the Effect of Treatment Allocation Regardless of Discontinuation in Trials with Incomplete Follow-up,” Statistics in Biopharmaceutical Research, 13, 119–127. DOI: 10.1080/19466315.2020.1736141.
  • Horvath, K., Aschermann, Z., Acs, P., Deli, G., Janszky, J., Komoly, S., Balázs, É., Takács, K., Karádi, K., and Kovács, N. (2015), “Minimal Clinically Important Difference on the Motor Examination Part of MDS-UPDRS,” Parkinsonism & Related Disorders, 21, 1421–1426.
  • Horvath, K., Aschermann, Z., Marton, K., Makkos, A., Mark, H., Janszky, J., Komoly, S., Karádi, K., and Kovács, N. (2017), “Minimal Clinically Important Differences for the Experiences of Daily Living Parts of Movement Disorder Society—Sponsored Unified Parkinsonś Disease Rating Scale,” Movement Disorders, 32, 789–793. DOI: 10.1002/mds.26960.
  • ICH E9 working group (2019), “ICH E9 (R1): Addendum on Estimands and Sensitivity Analysis in Clinical Trials to the Guideline on Statistical Principles for Clinical Trials,” available at https://database.ich.org/sites/default/files/E9-R1_Step4_Guideline_2019_1203.pdf.
  • Little, R. J. A., and Rubin, D. B. (2002), Statistical Analysis with Missing Data (2nd ed.), New York: Wiley.
  • Mallinckrodt, C. H., Bell, J., Liu, G., Ratitch, B., O’Kelly, M., Lipkovich, I., Singh, P., Xu, L., and Molenberghs, G. (2020), “Aligning Estimators with Estimands in Clinical Trials: Putting the ich e9 (r1) Guidelines into Practice,” Therapeutic Innovation & Regulatory Science, 54, 353–364.
  • Mallinckrodt, C. H., Lane, P. W., Schnell, D., Peng, Y., and Mancuso, J. P. (2008), “Recommendations for the Primary Analysis of Continuous Endpoints in Longitudinal Clinical Trials,” Drug Information Journal, 42, 303–319. DOI: 10.1177/009286150804200402.
  • Marek, K., Jennings, D., Lasch, S., Siderowf, A., Tanner, C., Simuni, T., Coffey, C., Kieburtz, K., Flagg, E., Chowdhury, S., and Poewe, W. “The Parkinson Progression Marker Initiative (PPMI),” Progress in Neurobiology, 95, 629–635. DOI: 10.1016/j.pneurobio.2011.09.005.
  • Parra, C. O., Daniel, R. M., and Bartlett, J. W. (2021), “Hypothetical Estimands in Clinical Trials: A Unification of Causal Inference and Missing Data Methods,” available at https://arxiv.org/abs/2107.04392.
  • Polverejan, E., and Dragalin, V. (2020), “Aligning Treatment Policy Estimands and Estimators—A Simulation Study in Alzheimer’s Disease,” Statistics in Biopharmaceutical Research, 12, 142–154. DOI: 10.1080/19466315.2019.1689845.
  • Roger, J. (2021), “Reference-based MI via Multivariate Normal RM (the “five macros” and miwithd),” available at https://www.lshtm.ac.uk/research/centres-projects-groups/missing-data/#dia-missing-data.
  • Seaman, S. R., White, I. R., and Leacy, F. P. (2014), “Comment on “Analysis of Longitudinal Trials with Protocol Deviations: A Framework for Relevant, Accessible Assumptions, and Inference via Multiple Imputation,” by Carpenter, Roger, and Kenward,” Journal of Biopharmaceutical Statistics, 24, 1358–1362.
  • Siddiqui, O. (2011), “MMRM versus MI in Dealing with Missing Data—A Comparison based on 25 NDA Data Sets,” Journal of Biopharmaceutical Statistics, 21, 423–436. DOI: 10.1080/10543401003777995.
  • Simuni, T., Siderowf, A., Lasch, S., Coffey, C. S., Caspell-Garcia, C., Jennings, D., Tanner, C. M., Trojanowski, J. Q., Shaw, L. M., Seibyl, J., and Schuff, N. “Longitudinal Change of Clinical and Biological Measures in Early Parkinson’s Disease: Parkinson’s Progression Markers Initiative Cohort,” Movement Disorders, 33, 771–782. DOI: 10.1002/mds.27361.
  • Verschuur, C. V. M., Suwijn, S. R., Boel, J. A., Post, B., Bloem, B. R., van Hilten, J. J., van Laar, T., Tissingh, G., Munts, A. G., Deuschl, G., and Lang, A. E. (2019), “Randomized Delayed-Start Trial of Levodopa in Parkinson’s Disease,” New England Journal of Medicine, 380, 315–324. DOI: 10.1056/NEJMoa1809983.
  • von Hippel, P. T., and Bartlett, J. W. (2021), “Maximum Likelihood Multiple Imputation: Faster Imputations and Consistent Standard Errors Without Posterior Draws,” Statistical Science, 36, 400–420. DOI: 10.1214/20-STS793.
  • Wolbers, M., Noci, A., Delmar, P., Gower-Page, C., Yiu, S., and Bartlett, J. W. (2022), “Standard and Reference-based Conditional Mean Imputation,” Pharmaceutical Statistics. DOI: 10.1002/pst.2234..

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