Trial Termination and Drug Misclassification in Sequential Adaptive Clinical Trials
Published Online:20 Sep 2018https://doi.org/10.1287/serv.2018.0217
References
- (2009) I-Spy 2: An adaptive breast cancer trial design in the setting of neoadjuvant chemotherapy. Clin. Pharmacol. Therapeut. 86(1):97–100.Crossref, Google Scholar
- (2008) Early stopping of randomized clinical trials for overt efficacy is problematic. J. Clin. Epidemiol. 61(3):241–246.Crossref, Google Scholar
- , Group S-S (2010) Stopping randomized trials early for benefit and estimation of treatment effects: Systematic review and meta-regression analysis. JAMA 303(12):1180–1187.Crossref, Google Scholar
- (1990) Application of the triangular test to phase II cancer clinical trials. Statist. Med. 9(8):907–917.Crossref, Google Scholar
- (2011a) Adaptive clinical trials in oncology. Nature Rev. Clin. Oncol. 9(4):199–207.Crossref, Google Scholar
- (2011b) Adaptive clinical trials: The promise and the caution. J. Clin. Oncol. 29(6):606–609.Crossref, Google Scholar
- (2000) The odds ratio. BMJ 320(7247):1468.Crossref, Google Scholar
- (2008) Adaptive design methods in clinical trials—A review. Orphanet J. Rare Diseases 3(11):169–190.Google Scholar
- (2011) Adaptive Design Methods in Clinical Trials (CRC Press, Boca Raton, FL).Crossref, Google Scholar
- (2008) Design and Analysis of Clinical Trials: Concepts and Methodologies (John Wiley & Sons, Hoboken, NJ).Google Scholar
- (2012) Handbook of Statistics in Clinical Oncology (CRC Press, Boca Raton, FL).Google Scholar
- (2009) Pharmaceutical R&D: The road to positive returns. Nature Rev. Drug Discovery 8(8):609–610.Crossref, Google Scholar
- (2007) Adaptive learning and risk taking. Psych. Rev. 114(1):177–187.Crossref, Google Scholar
- (2011) Seeking positive experiences can produce illusory correlations. Cognition 119(3):313–324.Crossref, Google Scholar
- (2001) Adaptation as information restriction: The hot stove effect. Organ. Sci. 12(5):523–538.Link, Google Scholar
- (2016) Innovation in the pharmaceutical industry: New estimates of R&D costs. J. Health Econom. 47:20–33.Crossref, Google Scholar
- (2014) Innovation and Marketing in the Pharmaceutical Industry: Emerging Practices, Research, and Policies(Springer, New York).Crossref, Google Scholar
- (2010) Bias and trials stopped early for benefit. JAMA 304(2):158.Crossref, Google Scholar
- (2011) How small system dynamics models can help the public policy process. System Dynam. Rev. 27(1):22–44.Google Scholar
- (2003) A randomized trial of letrozole in postmenopausal women after five years of tamoxifen therapy for early-stage breast cancer. N. Engl. J. Med. 349(19):1793–1802.Crossref, Google Scholar
- (2012) Futility stopping in clinical trials. Statist. Its Interface 5(4):415–423.Crossref, Google Scholar
- (1991) Organizational learning: The contributing processes and the literatures. Organ. Sci. 2(1):88–115.Link, Google Scholar
- (2003) Planning and monitoring of placebo‐controlled survival trials: Comparison of the triangular test with usual interim analyses methods. Br. J. Clin. Pharmacol. 55(3):299–306.Crossref, Google Scholar
- (2012) Stopping clinical trials early for futility: Retrospective analysis of several randomised clinical studies. Br. J. Cancer 107(6):910–917.Crossref, Google Scholar
- (1987) Design and analysis of group sequential tests based on the type i error spending rate function. Biometrika 74(1):149–154.Crossref, Google Scholar
- (2010) Bias and trials stopped early for benefit. JAMA 304(2):157–158.Crossref, Google Scholar
- (1983) Discrete sequential boundaries for clinical trials. Biometrika 70(3):659–663.Crossref, Google Scholar
- (2012) Magnified effects of changes in NIH research funding levels. Service Sci. 4(4):382–395.Link, Google Scholar
- (2017) Statistical controversies in clinical research: Futility analyses in oncology–lessons on potential pitfalls from a randomised controlled trial. Ann. Oncol. 28(7):1419–1426.Crossref, Google Scholar
- (1982) On choosing the number of interim analyses in clinical trials. Statist. Med. 1(1):25–36.Crossref, Google Scholar
- . (2005) Randomized trials stopped early for benefit: A systematic review. JAMA 294(17):2203–2209.Crossref, Google Scholar
- (1979) A multiple testing procedure for clinical trials. Biometrics 35(3):549–556.Crossref, Google Scholar
- (2009) The future of drug development: Advancing clinical trial design. Nature Rev. Drug Discovery 8(12):949–957.Crossref, Google Scholar
- (2012) Multilevel simulations of health delivery systems: A prospective tool for policy, strategy, planning, and management. Service Sci. 4(3):253–268.Link, Google Scholar
- (2013) A mathematical model for maximizing the value of phase 3 drug development portfolios incorporating budget constraints and risk. Statist. Medicine 32(10):1763–1777.Crossref, Google Scholar
- (2010) How to improve R&D productivity: The pharmaceutical industry’s grand challenge. Nature Rev. Drug Discovery 9(3):203–214.Crossref, Google Scholar
- Pharmaceutical Research and Manufacturers of America (2018) 2017 PhRMA Annual Membership Survey. Accessed March 2, 2018, http://phrma-docs.phrma.org/files/dmfile/PhRMA_membership-survey_2017.pdf.Google Scholar
- (1977) Group sequential methods in the design and analysis of clinical trials. Biometrika 64(2):191–199.Crossref, Google Scholar
- (1982) Interim analyses for randomized clinical trials: The group sequential approach. Biometrics 38(1):153–162.Crossref, Google Scholar
- (2005) When (not) to stop a clinical trial for benefit. JAMA 294(17):2228–2230.Crossref, Google Scholar
- (1999) Trials stopped early: Too good to be true? Lancet 353(9157):943–944.Crossref, Google Scholar
- (2005) The data monitoring experience in the candesartan in heart failure assessment of reduction in mortality and morbidity (charm) program. Amer. Heart J. 149(5):939–943.Crossref, Google Scholar
- (2005) Using group‐sequential analysis to achieve the optimal sample size. Internat. J. Obstetrics Gynaecology 112(5):529–533.Crossref, Google Scholar
- (2013) Meta‐analysis of clinical trials with early stopping: An investigation of potential bias. Statist. Med. 32(28):4859–4874.Crossref, Google Scholar
- (2000) Comparison of four sequential methods allowing for early stopping of comparative clinical trials. Clin. Sci. 98(5):569–578.Crossref, Google Scholar
- (1979) Corrected diffusion approximations in certain random walk problems. Advances Appl. Probab. 11(4):701–719.Crossref, Google Scholar
- (2010) Explaining odds ratios. J. Canadian Acad. Child Adolescent Psychiatry 19(3):227–229.Google Scholar
- (2010) Chemoradiotherapy for locally advanced head and neck cancer: 10-year follow-up of the UK head and neck (Ukhan1) trial. Lancet Oncol. 11(1):66–74.Crossref, Google Scholar
- (2001) Interim analyses and sequential designs in phase III studies. Br. J. Clin. Pharmacol. 51(5):394–399.Crossref, Google Scholar
- UKCCCR Anal Cancer Trial Working Party (1996) Epidermoid anal cancer: Results from the UKCCCR randomised trial of radiotherapy alone versus radiotherapy, 5-fluorouracil, and mitomycin. Lancet 348(9034):1049–1054.Crossref, Google Scholar
- US Food and Drug Administration (2010) Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics (Food and Drug Administration, Washington, DC).Google Scholar
- US Food and Drug Administration (2011) Oncologic Drugs Advisory Committee (Odac) meeting report. Accessed May 28, 2015, http://www.fda.gov/downloads/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/OncologicDrugsAdvisoryCommittee/UCM245644.pdf.Google Scholar
- US Food and Drug Administration (2014) Fast track, accelerated approval, and priority review. Accessed May 28, 2015, http://www.fda.gov/ForPatients/Approvals/Fast/ucm405447.htm.Google Scholar
- (2003) Early stopping in clinical trials and epidemiologic studies for “futility”: Conditional power vs. sequential analysis. J. Clin. Epidemiol. 56(7):610–617.Crossref, Google Scholar
- (1997) The Design and Analysis of Sequential Clinical Trials (John Wiley & Sons, Chichester, UK).Crossref, Google Scholar
- (2002) Sequential methods in clinical trials. Sequential Anal. 21(4):285–308.Crossref, Google Scholar
- (2004) Stopping clinical trials by design. Nature Rev. Drug Discovery 3(11):973–977.Crossref, Google Scholar
- (2004) The double triangular test in practice. Pharmaceutical Statist. 3(1):39–49.Crossref, Google Scholar
- (1993) Behind closed doors: The data monitoring board in randomized clinical trials. Statist. Med. 12(5–6):419–424.Crossref, Google Scholar
- (2012) When to stop a clinical trial early for benefit: Lessons learned and future approaches. Circulation: Heart Failure 5(2):294–302.Crossref, Google Scholar

