7 Références

[1] Little RJ, Lewis RJ. Estimands, Estimators, and Estimates. JAMA 2021 10.1001/jama.2021.2886 [34424281]

[2] Phillips A, Clark T. Estimands in practice: Bridging the gap between study objectives and statistical analysis. Pharm Stat 2021;20:68–76 10.1002/pst.2056

[3] INTERNATIONAL COUNCIL FOR HARMONISATION OF TECHNICAL REQUIREMENTS FOR PHARMACEUTICALS FOR HUMAN USE. ADDENDUM ON ESTIMANDS AND SENSITIVITY ANALYSIS IN CLINICAL TRIALS TO THE GUIDELINE ON STATISTICAL PRINCIPLES FOR CLINICAL TRIALS E9(R1).

[4] Fennell DA, Ewings S, Ottensmeier C, et al. Nivolumab versus placebo in patients with relapsed malignant mesothelioma (CONFIRM): a multicentre, double-blind, randomised, phase 3 trial. The Lancet Oncology 2021;22:1530–40 10.1016/S1470-2045(21)00471-X

[5] Mosenzon O, Blicher TM, Rosenlund S, et al. Efficacy and safety of oral semaglutide in patients with type 2 diabetes and moderate renal impairment (PIONEER 5): a placebo-controlled, randomised, phase 3a trial. Lancet Diabetes Endocrinol 2019;7:515–27 10.1016/S2213-8587(19)30192-5 [31189517]

[6] Pieber TR, Bode B, Mertens A, et al. Efficacy and safety of oral semaglutide with flexible dose adjustment versus sitagliptin in type 2 diabetes (PIONEER 7): a multicentre, open-label, randomised, phase 3a trial. Lancet Diabetes Endocrinol 2019;7:528–39 10.1016/S2213-8587(19)30194-9 [31189520]

[7] Lingvay I, Catarig A-M, Frias JP, et al. Efficacy and safety of once-weekly semaglutide versus daily canagliflozin as add-on to metformin in patients with type 2 diabetes (SUSTAIN 8): a double-blind, phase 3b, randomised controlled trial. Lancet Diabetes Endocrinol 2019;7:834–44 10.1016/S2213-8587(19)30311-0

[8] Rabe KF, Martinez FJ, Ferguson GT, et al. Triple Inhaled Therapy at Two Glucocorticoid Doses in Moderate-to-Very-Severe COPD. New Engl J Med 2020;383:35–48 10.1056/nejmoa1916046

[9] Rosenstock J, Bajaj HS, Janež A, et al. Once-Weekly Insulin for Type 2 Diabetes without Previous Insulin Treatment. New Engl J Med 2020;383:2107–16 10.1056/nejmoa2022474

[10] Heerspink HJL, Parving H-H, Andress DL, et al. Atrasentan and renal events in patients with type 2 diabetes and chronic kidney disease (SONAR): a double-blind, randomised, placebo-controlled trial. The Lancet 2019;393:1937–47 10.1016/S0140-6736(19)30772-X [30995972]

[11] Rosenkranz G. Estimands-new statistical principle or the emperor's new clothes? Pharm Stat 2017;16:4–5 10.1002/pst.1792 [27966259]

[12] Gogtay NJ, Ranganathan P, Aggarwal R. Understanding estimands. Perspect Clin Res 2021;12:106–12 10.4103/picr.picr_384_20 [34012908]