A Quantitative approach for making GO/NO-GO decisions in drug development

Drug Information Journal

Volumn 45, Issue 2, 2011, Pages 187-202

  Chuang Stein, Christy ^a   Kirby, Simon ^a   French, Jonathan ^a   Kowalski, Ken ^b   Marshall, Scott ^a   Smith, Mike K ^a   Bycott, Paul ^a   Beltangady, Mohan ^a  

^a PFIZER INC   (United States)

^b ANN ARBOR PHARMACOMETRICS GROUP   (United States)

Author keywords

Diagnostic test; Modelbased drug development; Negative predictive value; Positive predictive value; Prior distribution; Probability of a successful trial

Indexed keywords

AXITINIB; CYCLOOXYGENASE 2 INHIBITOR; GEMCITABINE; NONSTEROID ANTIINFLAMMATORY AGENT; SC 75416; UNCLASSIFIED DRUG;

ARTICLE; CHRONIC PAIN; CLINICAL TRIAL; CLINICAL TRIAL (TOPIC); DECISION MAKING; DIAGNOSTIC TEST; DRUG RESEARCH; HUMAN; HYPOTHESIS; METRIC SYSTEM; OSTEOARTHRITIS; PAIN; PANCREAS CANCER; PREDICTIVE VALUE; PRIORITY JOURNAL; PRODUCT DEVELOPMENT; QUANTITATIVE ANALYSIS; TOOTH PAIN;

EID: 79955708726     PISSN: 00928615     EISSN: None     Source Type: Journal    
DOI: 10.1177/009286151104500213     Document Type: Article

References (46)

1. Masia N. The cost of developing a new drug. In Focus on Intellectual Property Rights. US Department of State; April 23, 2008. http://www.america.gov/st/business-English/2008/April/20080429230904myleen0.5233981.html.
2. Parmar MK, Ungerleider RS, Simon R. Assessing whether to perform a confirmatory randomized clinical trial. J Natl Cancer Inst. 1996;88:1645-1651.
3. Lee SJ, Zelen M. Clinical trials and sample size considerations: another perspective. Stat Sci. 2000;15(2):95-110.
4. O'Hagan A, Stevens JW, Campbell MJ. Assurance in clinical trial design. Pharm Stat. 2005;4:187-201.
5. Chuang-Stein C. Sample size and the probability of a successful trial. Pharm Stat. 2006;5:305-309.
6. Hobbs BP, Carlin BP. Practical Bayesian design and analysis for drug and device clinical trials. J Biopharm Stat. 2008;18:54-80.
7. Kowalski KG, Ewy W, Hutmacher MM, Miller R, Krishnaswami S. Model-based drug development - a new paradigm for efficient drug development. Biopharm Rep. 2007;15(2):2-22.
8. Kowalski KG, French JL, Smith MK, Hutmacher MM. A model-based framework for quantitative decision-making in drug development. Presented at the American Conference on Pharmacometrics, Tucson AZ, March 12, 2008. http://tucson2008.go-acop.org/pdfs/8-Kowalski_FINAL.pdf.
9. Kowalski KG, Olson S, Remmers AE, Hutmacher MM. Modeling and simulation to support dose selection and clinical development of SC-75416, a selective COX-2 inhibitor for the treatment of acute and chronic pain. Clin Pharm Ther. 2008; 83:857-866.
10. Smith MK, French J, Kowalski K, Ewy W. Enhanced quantitative decision making-reducing the likelihood of incorrect decisions. Presented at the PAGE (Population Approach Group in Europe) Predictive Modeling in Drug Development Satellite Meeting, St. Petersburg, Russia, June 23, 2009.
11. Chuang-Stein C, Yang R. A revisit of sample size decision in confirmatory trials. Stat Biopharm Res. 2010;2:239-248.
12. Fleiss JL. Design and Analysis of Clinical Experiments. New York: Wiley; 1999.
13. Browner WS, Newman TB. Are all significant p-values created equal? JAMA. 1987;257:2459-2463.
14. Pater JL, Willan AR. Clinical trials as diagnostic tests. Control Clin Trials. 1984;5:107-113.
15. Wacholder S, Chanock S, Garcia-Closas M, El Ghormli L, Rothman N. Assessing the probability that a positive report is false: an approach for molecular epidemiology studies. J Natl Cancer Inst. 2004;96:434-442.
16. US Department of Health and Human Services, Food and Drug Administration. Innovation or stagnation? Challenge and opportunity on the critical path to new medical products. 2004. http://www.fda.gov/downloads/ScienceResearch/SpecialTopics/CriticalPathInitiative/CriticalPathOpportunitiesReports/ucm113411.pdf.
17. Peck CC, Barr WH, Benet LZ, et al. Opportunities for integration of pharmacokinetics, pharmacodynamics and toxicokinetics in rational drug development. Clin Pharmacol Ther. 1992;51:465-473.
18. Sheiner LB. Learning versus confirming in clinical drug development. Clin Pharmacol Ther. 1997;61:275-291.
19. Grasela TH, Dement CW, Kolerman OG, et al. Pharmacometrics and the transition to modelbased development. Clin Pharm Ther. 2007;82: 137-142.
20. Lalonde RL, Kowalski KG, Hutmacher MM, et al. Model-based drug development. Clin Pharm Ther. 2007;82:21-32.
21. Zhang L, Sinha V, Forgue T, et al. Model-based drug development: the road to quantitative pharmacology. J Pharmacokinet Pharmacodyn. 2006;33: 369-393.
22. Zhang L, Pfister M, Meibohm B. Concepts and challenges in quantitative pharmacology and model-based drug development. AAPS J. 2008; 10:552-559.
23. Ades A, Sutton AJ. Multiple parameter evidence synthesis in epidemiology and medical decisionmaking: current approaches. J R Stat Soc. A. 2006;169:5-35.
24. Mandema JW, Hermann D, Wang W, et al. Modelbased development of gemcabene, a new lipidaltering agent. AAPS J. 2005;7:E513-E522.
25. Ahn JE, French JL. Longitudinal aggregate data model-based meta-analysis with NONMEM: approaches to handling within treatment arm correlation. J Pharmacokinet Pharmacodyn. 2010;37: 179-201.
26. Ito K, Ahadieh S, Corrigan B, French J, Fullerton T, Tensfeldt T, Alzheimer's Disease Working Group. A disease progression meta-analysis model in Alzheimer's disease. Alzheimers Dement. 2010;6:39-53.
27. Smith MK, Marshall S. A Bayesian design and analysis for dose-response using informative prior information. J Biopharm Stat. 2006;16:695-709.
28. Santen G, van Zwet E, Danhof M, Della Pasqual O. From trial and error to trial simulation. Part 1: the importance of model-based drug development for antidepressant drugs. Clin Pharmacol Ther. 2009;86:248-254.
29. Santen G, Horrigan J, Danhof M, Della Pasqual O. From trial and error to trial simulation. Part 2: an appraisal of current beliefs in the design and analysis of clinical trials for antidepressant drugs. Clin Pharmacol Ther. 2009;86:255-261.
30. Sultana SR, Marshall S, Davis J, Littman BH. Experiences with dose finding in patients in early drug development: the use of biomarkers in early decision making in appropriate dose selection-how to optimize clinical drug development. Ernst Schering Foundation Symp Proc. 2007; 59:65-79.
31. de Greef R. Target occupancy biomarkers: schizophrenia. In: Danhof M, Van der Graaf PH, Holford NHG, eds. Measurement and Kinetics of in Vivo Drug Effects: Advances in Simultaneous Pharmacokinetic/Pharmacodynamic Modelling. 6th International Symposium. Leiden/Amsterdam Center for Drug Research; 2010:67-70.
32. Danhof M, Della Pasqua O, Knibbe CAJ, de Lange ECM, Voskuyl RA, Ploeger BA. Quantitative systems pharmacology: what are the targets? In: Danhof M, Van der Graaf PH, Holford NHG, eds. Measurement and Kinetics of in Vivo Drug Effects: Advances in Simultaneous Pharmacokinetic/Pharmacodynamic Modelling. 6th International Symposium. Leiden/Amsterdam Center for Drug Research; 2010:5-12.
33. Van der Graaf PH, Benson N. Bridging systems biology and PKPD: towards novel drugs. In: Danhof M, Van der Graaf PH, Holford NHG, eds. Measurement and Kinetics of in Vivo Drug Effects: Advances in Simultaneous Pharmacokinetic/Pharmacodynamic Modelling. 6th International Symposium. Leiden/Amsterdam Center for Drug Research; 2010:17-22.
34. Chuang-Stein C, Kirby S, Hirsch I, Atkinson G. The role of the minimum clinically important difference and its impact on designing a trial. Pharm Stat. 2010. Published online. DOI 10.1002/pst459.
35. Spano JP, Chodkiewicz C, Maurel J, et al. Efficacy of gemcitabine plus axitinib compared with gemcitabine alone in patients with advanced pancreatic cancer: an open-label randomized phase II study. Lancet. 2008;371:2101-2108.
36. Wang SJ, Hung HMJ, O'Neill RT. Adapting the sample size planning of a phase III trial based on phase II data. Pharm Stat. 2006;5:85-97.
37. Carroll KJ. Back to basics: explaining sample size in outcome trials, are statisticians doing a thorough job? Pharm Stat. 2009;8:333-345.
38. Staquet MJ, Rozencweig M, Von Hoff DD, Mugia FM. The delta and epsilon errors in the assessment of clinical trials. Cancer Treat Rep. 1979;63: 1917-1921.
39. Simon R. Randomized clinical trials and research strategy. Cancer Treat Rep. 1982;66:1083-1087.
40. Simon R. Some practical aspects of the interim monitoring of clinical trials. Stat Med. 1994;13: 1401-1409.
41. Berger J, Sellke T. Testing of a point null hypothesis: the irreconcilability of significance levels and evidence. J Am Stat Assoc. 1987;82:112-122.
42. Liu PY, LeBlanc M, Desai M. False positive rates of randomized phase II designs. Control Clin Trials. 1999;20:343-352.
43. Stallard N, Todd S, Whitehead J. Estimation following selection of the largest of two normal means. J Stat Plan Infer. 2008;138:1629-1638.
44. Bauer P, Koenig F, Brannath W, Posch M. Selection and bias-two hostile brothers. Stat Med. 2010;29(1):1-13.
45. Julious SA, Swank DJ. Moving statistics beyond the individual clinical trial: applying decision science to optimize a clinical development plan. Pharm Stat. 2005;4(1):37-46.
46. Burman CF, Grieve AP, Senn S. Decision analysis in drug development. In: Dmitrienko A, Chuang-Stein C, Agostino R, ed. Pharmaceutical Statistics Using SAS: A Practical Guide. Cary, NC: SAS Institute; 2007:385-428.