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Review

Pharmacological modeling and biostatistical analysis of a new drug

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Pages 59-82 | Published online: 29 Apr 2010

References

  • Rosenberger WF, Lachin, JM. Randomization in Clinical Trials: Theory and practice. Philadelpha, PA: John Wiley & Sons, Inc.; 2002.
  • Friedman LM, Furberg CD, DeMets DL. Fundamentals of Clinical Trials. 3rd edition. Chicago, IL: Springer; 1999.
  • Day SJ, Altman DG. Statistics notes: blinding in clinical trials and other studies. BMJ. 2000;321(7259):504.
  • Kolahi J, Bang H, Park J. Towards a proposal for assessment of blinding success in clinical trials: up-to-date review. Community Dent Oral Epidemiol. 2009;37(6):477–484.
  • Bang H, Ni L, Davis CE. Assessment of blinding in clinical trials. Control Clin Trials. 2004;25(2):143–156.
  • Bernstein D, Simmons J. Navigating differences between FDA and EMEA for regulatory compliance during drug development. Bio Pharm Int. 2006;19:16–23.
  • Ono S, Yoshioka C, Asaka O, et al. New drug approval times and clinical evidence in Japan. Contemp Clin Trials. 2005;26(6):660–672.
  • Malinowski HJ, Westelinck A, Sato J, et al. Same drug, different dosing: differences in dosing for drugs approved in the United States, Europe, and Japan. J Clin Pharmacol. 2008;48(8):900–908.
  • Available from: http://www.ich.org/LOB/media/MEDIA486.pdf. Accessed on February 10, 2010.
  • Smith-Tyler J. Informed consent, confidentiality, and subject rights in clinical trials. Proc Am Thorac Soc. 2007;4(2):189–193.
  • Gabrielsson JL. Utilization of physiologically based models in extrapolating pharmacokinetic data among species. Fundam Appl Toxicol. 1991;16(2):230–232.
  • Dixit R, Boelsterli UA. Healthy animals and animal models of human disease(s) in safety assessment of human pharmaceuticals, including therapeutic antibodies. Drug Discov Today. 2007;12(7–8): 336–342.
  • Miller K, Louie A, Baltch AL, et al. Pharmacokinetics of pentoxifylline and its metabolites in healthy mice and in mice infected with Candida albicans. Antimicrob Agents Chemother. 1998;42(9): 2405–2409.
  • Simmons D. The use of animal models in studying genetic disease: Transgenesis and induced mutation. Nature Educ. 2008;1(1).
  • ’t Hart BA, Bajramovic JJ. Non-human primate models of multiple sclerosis. Drug Discovery Today: Disease Models. 2008;5(2):97–104.
  • Coppola BA, Omens JH. Use of larger species such as dog and pig as model systems to study cardiac disease. Drug Discovery Today: Disease Models. 2008;5(3):195–200.
  • Wang L, Martin DR, Baker HJ, et al. Neural progenitor cell transplantation and imaging in a large animal model. Neurosci Res. 2007;59(3):327–340.
  • Scheerlinck JY, Snibson KJ, Bowles VM, et al. Biomedical applications of sheep models: from asthma to vaccines. Trends Biotechnol. 2008;26(5):259–266.
  • Gabrielsson J, Weiner D. Pharmacokinetic and Pharmacodynamic Data Analysis: Concepts and Applications. 4th Edition. Stockholm, Sweden: Swedish Pharmaceutical Press; 2007.
  • Anderson BJ, McKee D, Holford NHG. Size, myths and the clinical pharmacokinetics of analgesia in paediatric patients. Clin Pharmaco-kinet. 1997;33:313–327.
  • Holford NHG. A size standard for pharmacokinetics. Clin Pharmaco-kinet. 1996;30:329–332.
  • West GB, Brown JH, Enquist BJ. A general model for the origin of allometric scaling laws in biology. Science. 1997;276:122–126.
  • The University of Auckland School of Medical Sciences. 2009. MEDSCI 722 workshop timetable. Available from: http://www.fmhs.auckland.ac.nz/sms/pharmacology/holford/teaching/medsci722/timetable.aspx. Accessed on Dec 10, 2009.
  • Bourne D. Biopharmaceutics and pharmacokinetics. 2009. Available from: http://www.boomer.org/cZp4/. Accessed on January 10, 2010.
  • Beal SL. On the Solution to the Michaelis-Menten Equation. J Pharmacokinet Biopharm. 1982;10(1):109–119.
  • Boeckmann AJ, Sheiner LB, Beal SL. NONMEM Users Guide – Part V Introductory Guide. San Francisco, CA: NONMEM Project Group, University of California; 1994.
  • Bonate P. Pharmacokinetic-Pharmacodynamic Modeling and Simulation. 1st edition. Chicago, IL: Springer; 2005.
  • Luo FR, Yang Z, Camuso A, et al. Dasatinib (BMS-354825) pharmacokinetics and pharmacodynamic biomarkers in animal models predict optimal clinical exposure. Clin Cancer Res. 2006;12(23): 7180–7186.
  • Bruno R, Washington CB, Lu JF, et al. Population pharmacokinetics of trastuzumab in patients with HER2+ metastatic breast cancer. Cancer Chemother Pharmacol. 2005;56(4):361–369.
  • Marchetti S, Schellens JH. The impact of FDA and EMEA guidelines on drug development in relation to Phase 0 trials. Br J Cancer. 2007;97(5):577–581.
  • Whitehead J, Zhou Y, Hampson L, et al. A Bayesian approach for dose-escalation in a Phase I clinical trial incorporating pharmacodynamic endpoints. J Biopharm Stat. 2007;17(6):1117–1129.
  • Wang K, Ivanova A. Two-dimensional dose finding in discrete dose space. Biometrics. 2005;61(1):217–222.
  • Normolle D, Lawrence T. Designing dose-escalation trials with late-onset toxicities using the time-to-event continual reassessment method. J Clin Oncol. 2006;24(27):4426–4433.
  • Ivanova A, Wang K. A non-parametric approach to the design and analysis of two-dimensional dose-finding trials. Stat Med. 2004;23(12): 1861–1870.
  • Sp#x0142;awński J, Kuńniar J. Clinical trials: active control vs placebo – what is ethical? Sci Eng Ethics. 2004;10(1):73–79.
  • Terézhalmy GT, Bartizek RD, Biesbrock AR. Plaque-removal efficacy of four types of dental floss. J Periodontol. 2008;79(2): 245–251.
  • Singh D, Brooks J, Hagan G, et al. Superiority of “triple” therapy with salmeterol/fluticasone propionate and tiotropium bromide versus individual components in moderate to severe COPD. Thorax. 2008;63(7):592–598.
  • Hirsch FR, Hansen HH, Hansen M, et al. The superiority of combination chemotherapy including etoposide based on in vivo cell cycle analysis in the treatment of extensive small-cell lung cancer: a randomized trial of 288 consecutive patients. J Clin Oncol. 1987;5(4): 585–591.
  • Snapinn S. Noninferiority trials. Curr Control Trials Cardiovasc Med. 2000;1(1):19–21.
  • Kay R. Statistical Thinking for Non-Statisticians in Drug Regulation. Philadelphia, PA: Wiley-Interscience; 2007.
  • Kieser M, Friede T. Planning and analysis of three-arm non-inferiority trials with binary endpoints. Stat Med. 2006;26(2):253–273.
  • Munk A, Mielke M, Skipka G, et al. Testing noninferiority in threearmed clinical trials based on likelihood ratio statistics. Can J Stat. 2007;35(3):413–431.
  • Blackwelder WC. Current issues in clinical equivalence trials. J Dent Res. 2004;83 Spec No C:C113-C115.
  • Wiens BL, Iglewicz B. Design and analysis of three treatment equivalence trials. Control Clin Trials. 2000;21(2):127–137.
  • Beller EM, Gebski V, Keech AC. Randomisation in clinical trials. Med J Aust. 2002;177(10):565–567.
  • Torgerson DJ, Campbell MK. Use of unequal randomisation to aid the economic efficiency of clinical trials. BMJ. 2000;321(7263):759.
  • Dumville JC, Hahn S, Miles JN, et al. The use of unequal randomisation ratios in clinical trials: a review. Contemp Clin Trials. 2006;27(1): 1–12.
  • Kang M, Ragan BG, Park JH. Issues in outcomes research: an overview of randomization techniques for clinical trials. J Athl Train. 2008;43(2):215–221.
  • McEntegart DJ. Pursuit of balance using stratified and dynamic randomization techniques: an overview. Drug Inf J. 2003;37(3): 293–308.
  • Signorini DF, Leung O, Simes RJ, et al. Dynamic balanced randomization for clinical trials. Stat Med. 1993;12(24):2343–2350.
  • Gebski VJ, Beller EM, Keech AC. Randomised controlled trials: elements of a good study. Med J Aust. 2001;175(5):272–274.
  • Sica GT. Bias in research studies. Radiology. 2006;238(3): 780–789.
  • Chow S, Liu J. Design and Analysis of Clinical Trials: Concepts and methodologies. 2nd Edition. Philadelphia, PA: John Wiley and Sons, Inc.; 2004.
  • Gluud LL. Bias in clinical intervention research. Am J Epidemiol. 2006;163(6):493–501.
  • Thall PF. A review of phase 2–3 clinical trial designs. Lifetime Data Anal. 2008;14(1):37–53.
  • Appel LJ. A primer on the design, conduct, and interpretation of clinical trials. Clin J Am Soc Nephrol. 2006;1(6):1360–1367.
  • Fitzpatrick S. Clinical Trial Design. : The Institute of Clinical Research; 2006.
  • Green S, Liu PY, O’Sullivan J. Factorial design considerations. J Clin Oncol. 2002;20(16):3424–3430.
  • Lathyris DN, Trikalinos TA, Ioannidis JP. Evidence from crossover trials: empirical evaluation and comparison against parallel arm trials. Int J Epidemiol. 2007;36(2):422–430.
  • Quan H, Luo X, Capizzi T. Multiplicity adjustment for multiple endpoints in clinical trials with multiple doses of an active treatment. Stat Med. 2005;24(14):2151–2170.
  • Rosner B. Fundamental of Biostatistics. 5th Edition. Stamford, CT: Duxbury Thomson Learning; 2000.
  • Streiner DL. Missing data and the trouble with LOCF. Evid Based Ment Health. 2008;11(1):3–5.
  • Streiner DL. The case of the missing data: methods of dealing with dropouts and other research vagaries. Can J Psychiatry. 2002;47(1): 68–75.
  • Taylor L, Zhou XH. Multiple imputation methods for treatment noncompliance and nonresponse in randomized clinical trials. Biometrics. 2009;65(1):88–95.
  • Yang X, Li J, Shoptaw S. Imputation-based strategies for clinical trial longitudinal data with nonignorable missing values. Stat Med. 2008;27(15):2826–2849.
  • Kenward MG, Carpenter J. Multiple imputation: current perspectives. Stat Methods Med Res. 2007;16(3):199–218.
  • Shaffer ML, Chinchilli VM. Including multiple imputation in a sensitivity analysis for clinical trials with treatment failures. Contemp Clin Trials. 2007;28(2):130–137.
  • Baselga J, Carbonell X, Castañeda-Soto NJ, et al. Phase II study of efficacy, safety, and pharmacokinetics of trastuzumab monotherapy administered on a 3-weekly schedule. J Clin Oncol. 2005;23(10):2162–2171.
  • Fleming TR. Data monitoring committees and capturing relevant information of high quality. Stat Med. 1993;12(5–6):565–570; discussion 571–573.
  • Grant AM, Altman DG, Babiker AB, et al. Issues in data monitoring and interim analysis of trials. Health Technol Assess. 2005;9(7): 1–238, iii-iv.
  • O’Brien PC, Fleming TR. A multiple testing procedure for clinical trials. Biometrics. 1979;35(3):549–556.
  • Lan KKG, DeMets DL. Discrete sequential boundaries for clinical trials. Biometrika. 1983;70:659–663.
  • DeMets DL, Lan G. The alpha spending function approach to interim data analyses. Cancer Treat Res. 1995;75:1–27.
  • DeMets DL, Lan KK. Interim analysis: the alpha spending function approach. Stat Med. 1994;13(13–14):1341–1352; discussion 1353–1356.
  • Pocock SJ, Hughes MD. Practical problems in interim analyses, with particular regard to estimation. Control Clin Trials. 1989; 10(4 Suppl):209S–221S.
  • Hughes MD, Pocock SJ. Stopping rules and estimation problems in clinical trials. Stat Med. 1988;7(12):1231–1242.
  • Geller NL, Pocock SJ. Interim analyses in randomized clinical trials: ramifications and guidelines for practitioners. Biometrics. 1987;43(1):213–223.
  • Jennison C, Turnbull BW. Group Sequential Trials: Applications to Clinical Trials. Baton Rouge, LA: Chapman and Hall/CRC; 2000.
  • Bretz F, Koenig F, Brannath W, et al. Adaptive designs for confirmatory clinical trials. Stat Med. 2009;28(10):1445–1463.
  • Walker GA. Common Statistical Methods for Clinical Research with SAS Examples. 2nd edition. Cary, NC: SAS Institute Inc.; 2006.
  • Berry DA. Bayesian clinical trials. Nature Rev Drug Discov. 2006;5: 27–36.
  • Biswas S, Liu DD, Lee JJ, Berry DA. Bayesian clinical trials at the University of Texas M.D. Anderson Cancer Center. Clin Trials. 2009;6(3):205–216.
  • Schoenwald RD. Pharmacokinetic Principles of Dosing Adjustments: Understanding the Basics. Baton Rouge, LA: CRC Press LLC.; 2001.
  • Puri PK, Tyrer PJ. Sciences Basic to Psychiatry. Philadelphia, PA: Elsevier Health Sciences; 1998.