References
- Fleury MJ, Djouini A, Huynh C, Tremblay J, Ferland F, Menard JM, Belleville G. Remission from substance use disorders: a systematic review and meta-analysis. Drug Alcohol Depend. 2016;168:293–306.
- McLellan AT, Lewis DC, O’Brien CP, Kleber HD. Drug dependence, a chronic medical illness: implications for treatment, insurance, and outcomes evaluation. JAMA. 2000;284:1689–95. doi:https://doi.org/10.1001/jama.284.13.1689.
- Kelly JF, Greene MC, Bergman BG, White WL, Hoeppner BB. How many recovery attempts does it take to successfully resolve an alcohol or drug problem? Estimates and correlates from a national study of recovering U.S. adults. Alcohol Clin Exp Res. 2019;43:1533–44. doi:https://doi.org/10.1111/acer.14067.
- Lappan SN, Brown AW, Hendricks PS. Dropout rates of in-person psychosocial substance use disorder treatments: a systematic review and meta-analysis. Addiction. 2020;115:201–17. doi:https://doi.org/10.1111/add.14793.
- Dutra L, Stathopoulou G, Basden SL, Leyro TM, Powers MB, Otto MW. A meta-analytic review of psychosocial interventions for substance use disorders. Am J Psychiatry. 2008;165:179–87. doi:https://doi.org/10.1176/appi.ajp.2007.06111851.
- Mattson ME, Del Boca FK, Carroll KM, Cooney NL, DiClemente CC, Donovan D, Kadden RM, McRee B, Rice C, Rycharik RG, et al. Compliance with treatment and follow-up protocols in project MATCH: predictors and relationship to outcome. Alcohol Clin Exp Res. 1998;22:1328–39.
- National Research Council. The prevention and treatment of missing data in clinical trials. Washington, DC: The National Academies Press; 2010.
- Witkiewitz K, Falk DE, Kranzler HR, Litten RZ, Hallgren KA, O’Malley SS, Anton RF, in conjunction with the Alcohol Clinical Trials Initiative (ACTIVE) Workgroup. Methods to analyze treatment effects in the presence of missing data for a continuous heavy drinking outcome measure when participants drop out from treatment in alcohol clinical trials. Alcohol Clin Exp Res. 2014;38:2826–34.
- International Council for Harmonisation. Statistical principles for clinical trials E9. 1998. [accessed 2021 Jun 18]. https://database.ich.org/sites/default/files/E9_Guideline.pdf.
- Hernan MA, Robins JM. Per-protocol analyses of pragmatic trials. N Engl J Med. 2017;377:1391–98. doi:https://doi.org/10.1056/NEJMsm1605385.
- Murray EJ, Caniglia EC, Swanson SA, Hernandez-Diaz S, Hernan MA. Patients and investigators prefer measures of absolute risk in subgroups for pragmatic randomized trials. J Clin Epidemiol. 2018;103:10–21. doi:https://doi.org/10.1016/j.jclinepi.2018.06.009.
- Rudolph JE, Naimi AI, Westreich DJ, Kennedy EH, Schisterman EF. Defining and identifying per-protocol effects in randomized trials. Epidemiology. 2020;31:692–94. doi:https://doi.org/10.1097/EDE.0000000000001234.
- Gravel J, Opatrny L, Shapiro S. The intention-to-treat approach in randomized controlled trials: are authors saying what they do and doing what they say? Clin Trials. 2007;4:350–56. doi:https://doi.org/10.1177/1740774507081223.
- International Council for Harmonisation. Efficacy Guidelines. [ accessed 2020 Dec 10]. https://www.ich.org/page/efficacy-guidelines.
- International Council for Harmonisation. Addendum on estimands and sensitivity analysis in clinical trials to the guideline on statistical principles for clinical trials E9(R1). 2019. [accessed 2020 Aug 17]. https://database.ich.org/sites/default/files/E9-R1_Step4_Guideline_2019_1203.pdf.
- Ritz C, Ronn B. Estimands: improving inference in randomized controlled trials in clinical nutrition in the presence of missing values. Eur J Clin Nutr. 2018;72:1291–95. doi:https://doi.org/10.1038/s41430-018-0207-x.
- Callegari F, Akacha M, Quarg P, Pandhi S, von Raison F, Zuber E. Estimands in a chronic pain trial: challenges and opportunities. Stat Biopharm Res. 2020;12:39–44. doi:https://doi.org/10.1080/19466315.2019.1629997.
- Degtyarev E, Zhang Y, Sen K, Lebwohl D, Akacha M, Hampson LV, Bornkamp B, Maniero A, Bretz F, Zuber E. Estimands and the patient journey: addressing the right question in oncology clinical trials. JCO Precis Oncol. 2019;3. doi:https://doi.org/10.1200/PO.18.00381
- Fiero MH, Pe M, Weinstock C, King-Kallimanis BL, Komo S, Klepin HD, Gray SW, Bottomley A, Kluetz PG, Sridhara R. Demystifying the estimand framework: a case study using patient-reported outcomes in oncology. Lancet Oncol. 2020;21:e488–e94.
- Lawrance R, Degtyarev E, Griffiths P, Trask P, Lau H, D’Alessio D, Griebsch I, Wallenstein G, Cocks K, Rufibach K. What is an estimand & how does it relate to quantifying the effect of treatment on patient-reported quality of life outcomes in clinical trials? J Patient Rep Outcomes. 2020;4:68.
- Aroda VR, Saugstrup T, Buse JB, Donsmark M, Zacho J, Davies MJ. Incorporating and interpreting regulatory guidance on estimands in diabetes clinical trials: the PIONEER 1 randomized clinical trial as an example. Diabetes Obes Metab. 2019;21:2203–10. doi:https://doi.org/10.1111/dom.13804.
- Catz SL, Jack LM, McClure JB, Javitz HS, Deprey M, Zbikowski SM, McAfee T, Richards J, Swan GE. Adherence to varenicline in the COMPASS smoking cessation intervention trial. Nicotine Tob Res. 2011;13:361–68.
- Gray KM, Sonne SC, McClure EA, Ghitza UE, Matthews AG, McRae-Clark AL, Carroll KM, Potter JS, Wiest K, Mooney LJ, et al. A randomized placebo-controlled trial of N-acetylcysteine for cannabis use disorder in adults. Drug Alcohol Depend. 2017;177:249–57.
- McClure EA, Sonne SC, Winhusen T, Carroll KM, Ghitza UE, McRae-Clark AL, Matthews AG, Sharma G, Van Veldhuisen P, Vandrey RG, et al. Achieving cannabis cessation — evaluating n-acetylcysteine treatment (ACCENT): design and implementation of a multi-site, randomized controlled study in the national institute on drug abuse clinical trials network. Contemp Clin Trials. 2014;39:211–23.
- Heishman SJ, Singleton EG, Liguori A. Marijuana craving questionnaire: development and initial validation of a self-report instrument. Addiction. 2001;96:1023–34. doi:https://doi.org/10.1046/j.1360-0443.2001.967102312.x.
- Heishman SJ, Evans RJ, Singleton EG, Levin KH, Copersino ML, Gorelick DA. Reliability and validity of a short form of the Marijuana craving questionnaire. Drug Alcohol Depend. 2009;102:35–40. doi:https://doi.org/10.1016/j.drugalcdep.2008.12.010.
- Foster KT, Li N, McClure EA, Sonne SC, Gray KM. Gender differences in internalizing symptoms and suicide risk among men and women seeking treatment for cannabis use disorder from late adolescence to middle adulthood. J Subst Abuse Treat. 2016;66:16–22. doi:https://doi.org/10.1016/j.jsat.2016.01.012.
- van Boven JF, Vemer P. Higher adherence during reimbursement of pharmacological smoking cessation treatments. Nicotine Tob Res. 2016;18:56–63.
- Browning KK, Wewers ME, Ferketich AK, Diaz P, Koletar SL, Reynolds NR. Adherence to tobacco dependence treatment among HIV-infected smokers. AIDS Behav. 2016;20:608–21. doi:https://doi.org/10.1007/s10461-015-1059-1.
- Leischow SJ, Muramoto ML, Matthews E, Floden LL, Grana RA. Adolescent smoking cessation with bupropion: the role of adherence. Nicotine Tob Res. 2016;18:1202–05. doi:https://doi.org/10.1093/ntr/ntv179.
- Johnson BA, Rosenthal N, Capece JA, Wiegand F, Mao L, Beyers K, McKay A, Ait-Daoud N, Anton RF, Ciraulo DA, et al. Topiramate for treating alcohol dependence: a randomized controlled trial. JAMA. 2007;298:1641–51.
- Winhusen TM, Kropp F, Lindblad R, Douaihy A, Haynes L, Hodgkins C, Chartier K, Kampman KM, Sharma G, Lewis DF, et al. Multisite, randomized, double-blind, placebo-controlled pilot clinical trial to evaluate the efficacy of buspirone as a relapse-prevention treatment for cocaine dependence. J Clin Psychiatry. 2014;75:757–64.
- Trivedi MH, Greer TL, Rethorst CD, Carmody T, Grannemann BD, Walker R, Warden D, Shores-Wilson K, Stoutenberg M, Oden N, et al. Randomized controlled trial comparing exercise to health education for stimulant use disorder: results from the CTN-0037 STimulant reduction intervention using dosed exercise (STRIDE) study. J Clin Psychiatry. 2017;78:1075–82.
- Hedeker D, Mermelstein RJ, Demirtas H. Analysis of binary outcomes with missing data: missing = smoking, last observation carried forward, and a little multiple imputation. Addiction. 2007;102:1564–73. doi:https://doi.org/10.1111/j.1360-0443.2007.01946.x.
- Hallgren KA, Witkiewitz K. Missing data in alcohol clinical trials: a comparison of methods. Alcohol Clin Exp Res. 2013;37:2152–60. doi:https://doi.org/10.1111/acer.12205.
- West R, Hajek P, Stead L, Stapleton J. Outcome criteria in smoking cessation trials: proposal for a common standard. Addiction. 2005;100:299–303. doi:https://doi.org/10.1111/j.1360-0443.2004.00995.x.
- Mallinckrodt C, Molenberghs G, Lipkovich I, Ratitch B. Estimands, estimators and sensitivity analyses in clinical trials. Boca Raton, FL: CRC Press;; 2020.