Fundamentals of population pharmacokinetic modelling: Validation methods

Clinical Pharmacokinetics

Volumn 51, Issue 9, 2012, Pages 573-590

  Sherwin, Catherine M T ^a   Kiang, Tony K L ^b   Spigarelli, Michael G ^a   Ensom, Mary H H ^b,c  

^a UNIVERSITY OF UTAH SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^c CHILDREN S AND WOMEN S HEALTH CENTRE OF BRITISH COLUMBIA   (Canada)

Author keywords

pharmacokinetic modelling; pharmacokinetics; population pharmacokinetics

Indexed keywords

COMPUTER PROGRAM; DIAGNOSTIC TEST; EVALUATION RESEARCH; HUMAN; KINETICS; POPULATION PHARMACOKINETIC MODELLING; POPULATION RESEARCH; PRACTICE GUIDELINE; PRIORITY JOURNAL; REVIEW; VALIDATION STUDY;

HUMANS; MODELS, BIOLOGICAL; PHARMACOKINETICS; REPRODUCIBILITY OF RESULTS; RESEARCH DESIGN;

EID: 84864755266     PISSN: 03125963     EISSN: 11791926     Source Type: Journal    
DOI: 10.2165/11633940-000000000-00000     Document Type: Review

References (69)

1. Ette EI, Williams PJ. Pharmacometrics: the science of quantitative pharmacology. Hoboken (NJ): John Wiley, 2007
2. FDA. Guidance for industry: population pharmacokinetics. US Department of Health and Human Services; Food and Drug Administration; Centre for Drug Evaluation and Research & Centre for Biologics Evaluation and Research, 1999 Feb; CP 1 online. Available from URL: http://www.fda.gov/downloads/Drugs/ GuidanceComplianceRegulatoryInformation/Guidances/UCM072137.pdf?utm-campaign= Google2&utm-source=fdaSearch&utm-medium=website&utm-term=Guidance for industry population pharmaco kinetics&utm-content=1 Accessed 2011 Sep 1
3. EMA. Guideline on reporting the results of population pharmacokinetic analyses. European Medicines Agency 21 June 2007 (Doc. Ref. CHMP/EWP/185990/06) online. Available from URL: http://www.ema.europa.eu/docs/en-GB/document- library/Scientific-guideline/2009/09/WC500003067.pdf Accessed 2011 Sep 2
4. Wade JR, Edholm M, Salmonson T. A guide for reporting the results of population pharmacokinetic analyses: a Swedish perspective. AAPS J 2005; 7: 45
5. Hennig S, Wainwright CE, Bell SC, et al. Population pharmacokinetics of itraconazole and its active metabolite hydroxy-itraconazole in paediatric cystic fibrosis and bone marrow transplant patients. Clin Pharmacokinet 2006; 45: 1099-114 (Pubitemid 44631389)
6. Falck P, Midtvedt K, Vân Lê TT, et al. A population pharmacokinetic model of ciclosporin applicable for assisting dose management of kidney transplant recipients. Clin Pharmacokinet 2009; 48: 615-23
7. Perez-Ruixo JJ, Zannikos P, Hirankarn S, et al. Population pharmacokinetic meta-analysis of trabectedin (ET-743, Yondelis) in cancer patients. Clin Pharmacokinet 2007; 46: 867-84 (Pubitemid 47443311)
8. Musuamba FT, Rousseau A, Bosmans J-L, et al. Limited sampling models and Bayesian estimation for mycophenolic acid area under the curve prediction in stable renal transplant patients co-medicated with ciclosporin or sirolimus. Clin Pharmacokinet 2009; 48: 745-58
9. Lahu G, Hü nnemeyer A, Diletti E, et al. Population pharmacokinetic modelling of roflumilast and roflumilast N-oxide by total phosphodiesterase-4 inhibitory activity and development of a population pharmacodynamic-adverse event model. Clin Pharmacokinet 2010; 49: 589-606
10. Kerbusch T, de Kraker J, Mathô t RAA, et al. Population pharmacokinetics of ifosfamide and its dechloroethylated and hydroxylated metabolites in children with malignant disease: a sparse sampling approach. Clin Pharmacokinet 2001; 40: 615-25 (Pubitemid 32758583)
11. Djebli N, Rousseau A, Hoizey G, et al. Sirolimus population pharmacokinetic/pharmacogenetic analysis and Bayesian modelling in kidney transplant recipients. Clin Pharmacokinet 2006; 45: 1135-48 (Pubitemid 44631392)
12. Sam WJ, Tham LS, Holmes MJ, et al. Population pharmacokinetics of tacrolimus in whole blood and plasma in Asian liver transplant patients. Clin Pharmacokinet 2006; 45: 59-75 (Pubitemid 43145015)
13. Lehr T, Staab A, Tillmann C, et al. A quantitative enterohepatic circulation model: development and evaluation with tesofensine and meloxicam. Clin Pharmacokinet 2009; 48: 529-42
14. Nielsen EI, SandströmM, Honoré PH, et al. Developmental pharmacokinetics of gentamicin in preterm and term neonates: population modelling of a prospective study. Clin Pharmacokinet 2009; 48: 253-63
15. Pilla Reddy V, Kozielska M, Johnson M, et al. Structural models describing placebo treatment effects in schizophrenia and other neuropsychiatric disorders. Clin Pharmacokinet 2011; 50: 429-50
16. Krekels EH, DeJongh J, van Lingen RA, et al. Predictive performance of a recently developed population pharmacokinetic model for morphine and its metabolites in new datasets of (preterm) neonates, infants and children. Clin Pharmacokinet 2011; 50: 51-63
17. Han K, Bies R, Johnson H, et al. Population pharmacokinetic evaluation with external validation and bayesian estimator of voriconazole in liver transplant recipients. Clin Pharmacokinet 2011; 50: 201-14
18. Beal SL. Validation of a population model. NONMEMusers group (NMusers), 1994 Feb 1 online. Available from URL: http://www.cognigencorp.com/nonmem/nmo/ topic006.html Accessed 2011 Sep 2
19. International Programme on Chemical Safety (IPCS). Characterization and application of physiologically based pharmacokineticmodels in risk assessment Geneva: WHO, 2010 online. Available from URL: http://www.inchem. org/documents/harmproj/harmproj/harmproj9.pdf Accessed 2012May 28
20. Barton HA, Chiu WA, Woodrow Setzer R, et al. Characterizing uncertainty and variability in physiologically based pharmacokinetic models: state of the science and needs for research and implementation. Toxicol Sci 2007; 99: 395-402 (Pubitemid 351753057)
21. Babuska I. Verification and validation in computational engineering and science: basic concepts. Comput Methods Appl Mech Eng 2004; 193: 4057-66 (Pubitemid 39142612)
22. Sun H, Fadiran EO, Jones CD, et al. Population pharmacokinetics: a regulatory perspective. Clin Pharmacokinet 1999; 37: 41-58
23. Mesnil F, Mentre F, Dubruc C, et al. Population pharmacokinetic analysis of mizolastine and validation from sparse data on patients using the nonparametric maximum likelihood method. J Pharmacokinet Biopharm 1998; 26: 133-61 (Pubitemid 28496596)
24. Williams PJ, Ette EI. The role of population pharmacokinetics in drug development in light of the Food and Drug Administration's 'Guidance for Industry: population pharmacokinetics'. Clin Pharmacokinet 2000; 39: 385-95 (Pubitemid 32099467)
25. Ette EI, Ludden TM. Population pharmacokinetic modeling: the importance of informative graphics. Pharm Res 1995; 12: 1845-55 (Pubitemid 26031769)
26. Box GEP, Draper NR. Empirical model-building and response surfaces. New York: John Wiley & Sons, 1987
27. Karlsson MO, Savic RM. Diagnosing model diagnostics. Clin Pharmacol Ther 2007; 82: 17-20 (Pubitemid 46944205)
28. Ette EI. Stability and performance of a population pharmacokinetic model. J Clin Pharmacol 1997; 37: 486-95 (Pubitemid 27265649)
29. Vilicich M. Validation Strategy for NONMEM. NONMEM users group (NMusers) 2008 Oct 17 online. Available from URL: http://www.cognigencorp. com/nonmem/current/2008-October/1214.html Accessed 2011 Sep 2
30. Brendel K, Dartois C, Comets E, et al. Are population pharmacokinetic and/or pharmacodynamic models adequately evaluated? A survey of the literature from 2002 to 2004. Clin Pharmacokinet 2007; 46: 221-34 (Pubitemid 46425648)
31. Frame B, Koup J, Miller R, et al. Population pharmacokinetics of clinafloxacin in healthy volunteers and patients with infections: experience with heterogeneous pharmacokinetic data. Clin Pharmacokinet 2001; 40: 307-15 (Pubitemid 32458156)
32. Feillet F, Clarke L, Meli C, et al. Pharmacokinetics of sapropterin in patients with phenylketonuria. Clin Pharmacokinet 2008; 47: 817-25
33. Sugiyama E, Kaniwa N, Kim S-R, et al. Population pharmacokinetics of gemcitabine and its metabolite in Japanese cancer patients: impact of genetic polymorphisms. Clin Pharmacokinet 2010; 49: 549-58
34. Parke J, Holford NH, Charles BG. A procedure for generating bootstrap samples for the validation of nonlinear mixed-effects population models. Comput Methods Programs Biomed 1999; 59: 19-29 (Pubitemid 29125558)
35. Benkali K, Prémaud A, Picard N, et al. Tacrolimus population pharmacokinetic-pharmacogenetic analysis and Bayesian estimation in renal transplant recipients. Clin Pharmacokinet 2009; 48: 805-16
36. Moltó J, Barbanoj MJ, Miranda C, et al. Simultaneous population pharmacokinetic model for lopinavir and ritonavir in HIV-infected adults. Clin Pharmacokinet 2008; 47: 681-92
37. Saint-Marcoux F, Royer B, Debord J, et al. Pharmacokinetic modelling and development of Bayesian estimators for therapeutic drug monitoring of mycophenolate mofetil in reduced-intensity haematopoietic stem cell transplantation. Clin Pharmacokinet 2009; 48: 667-75
38. Efron B. The jackknife, the bootstrap, and other resampling plans. Philadelphia (PA): Society for Industrial and Applied Mathematics, 1982
39. Zahr N, Amoura Z, Debord J, et al. Pharmacokinetic study of mycophenolate mofetil in patients with systemic lupus erythematosus and design of Bayesian estimator using limited sampling strategies.Clin Pharmacokinet 2008; 47: 277-84 (Pubitemid 351367437)
40. Coulter CV, Isbister GK, Duffull SB. The pharmacokinetics of methanol in the presence of ethanol: a case study. Clin Pharmacokinet 2011; 50: 245-51
41. Stockis A, Toublanc N, Sargentini-Maier ML, et al. Retrospective population pharmacokinetic analysis of levetiracetam in children and adolescents with epilepsy: dosing recommendations. Clin Pharmacokinet 2008; 47: 333-41 (Pubitemid 351508119)
42. Wilde S, Jetter A, Rietbrock S, et al. Population pharmacokinetics of the BEACOPP polychemotherapy regimen in Hodgkin's lymphoma and its effect on myelotoxicity. Clin Pharmacokinet 2007; 46: 319-33 (Pubitemid 46507010)
43. Ette EI, Williams PJ, Kim YH, et al. Model appropriateness and population pharmacokinetic modeling. J Clin Pharmacol 2003; 43: 610-23 (Pubitemid 36613120)
44. Mentre F, Ebelin M. Validation of population pharmacokinetic/ pharmacodynamic analyses: review of proposed approaches. In: Balant L, Aarons L, editors. The population approach: measuring and managing variability in response, concentration and dose. Brussels: Commission of the European Communities, 1997: 147-60
45. Wahlby U, Jonsson EN, Karlsson MO.Assessment of actual significance levels for covariate effects inNONMEM. J Pharmacokinet Pharmacodyn 2001; 28: 231-52 (Pubitemid 32643366)
46. Montgomery D, Peck E. Introduction to linear regression analysis. New York: Wiley, 1982
47. Bachman W. Model diagnostics. Nmusers; 2003 online. Available from URL: http://www.cognigencorp.com/nonmem/nm/99may012003.html Accessed 2006 Jan 12
48. Yamaoka K, Nakagawa T, Uno T. Application of Akaike's information criterion (AIC) in the evaluation of linear pharmacokinetic equations. J Pharmacokinet Biopharm 1978; 6: 165-75 (Pubitemid 8384279)
49. Ludden TM, Beal SL, Sheiner LB. Comparison of the Akaike information criterion, the Schwarz criterion and the F test as guides to model selection. J Pharmacokinet Biopharm 1994; 22: 431-45
50. Sheiner LB, Beal SL. Some suggestions for measuring predictive performance. J Pharmacokinet Biopharm 1981; 9: 503-12
51. Hooker AC, Staatz CE, KarlssonMO. Conditional weighted residuals (CWRES): a model diagnostic for the FOCE method. Pharm Res 2007; 24: 2187-97 (Pubitemid 350060706)
52. Pérez-Ruixo JJ, Krzyzanski W, Bouman-Thio E, et al. Pharmacokinetics and pharmacodynamics of the erythropoietin Mimetibody-construct CNTO 528 in healthy subjects. Clin Pharmacokinet 2009; 48: 601-13
53. Yano Y, Beal SL, Sheiner LB. Evaluating pharmacokinetic/pharmacodynamic models using the posterior predictive check. J Pharmacokinet Pharmacodyn 2001; 28: 171-92
54. Mentre F, Escolano S. Prediction discrepancies for the evaluation of nonlinear mixed-effects models. J Pharmacokinet Pharmacodyn 2006; 33: 345-67 (Pubitemid 44020385)
55. Wiczling P, Lowe P, Pigeolet E, et al. Population pharmacokinetic modelling of filgrastim in healthy adults following intravenous and subcutaneous administrations. Clin Pharmacokinet 2009; 48: 817-26
56. Mueck W, Frey R. Population pharmacokinetics and pharmacodynamics of cinaciguat, a soluble guanylate cyclase activator, in patients with acute decompensated heart failure. Clin Pharmacokinet 2010; 49: 119-29
57. Lindauer A, Siepmann T, Oertel R, et al. Pharmacokinetic/pharmacodynamic modelling of venlafaxine: pupillary light reflex as a test system for noradrenergic effects. Clin Pharmacokinet 2008; 47: 721-31
58. Retlich S, Duval V, Ring A, et al. Pharmacokinetics and pharmacodynamics of single rising intravenous doses (0.5 mg-10 mg) and determination of absolute bioavailability of the dipeptidyl peptidase-4 inhibitor linagliptin (BI 1356) in healthy male subjects. Clin Pharmacokinet 2010; 49: 829-40
59. Samtani MN, Vermeulen A, Stuyckens K. Population pharmacokinetics of intramuscular paliperidone palmitate in patients with schizophrenia: a novel once-monthly, long-acting formulation of an atypical antipsychotic. Clin Pharmacokinet 2009; 48: 585-600
60. Mukonzo JK, Nanzigu S, Rekic D, et al. HIV/AIDS patients display lower relative bioavailability of efavirenz than healthy subjects. Clin Pharmacokinet 2011; 50: 531-40
61. Jauregizar N, de la Fuente L, Lucero ML, et al. Pharmacokinetic- pharmacodynamic modelling of the antihistaminic (H1) effect of bilastine. Clin Pharmacokinet 2009; 48: 543-54
62. Mueck W, Lensing AWA, Agnelli G, et al. Rivaroxaban: population pharmacokinetic analyses in patients treated for acute deep-vein thrombosis and exposure simulations in patients with atrial fibrillation treated for stroke prevention. Clin Pharmacokinet 2011; 50: 675-86
63. Wang DD, Zhang S. Standardized visual predictive check-how and when to used it in model evaluation abstract no. 1501. PAGE. Abstracts of the 18th AnnualMeeting of the PopulationApproach Group inEurope; 2009 Jun 23-26; St Petersburg online. Available from URL: http://www.page-meeting. org/?abstract=1501 Accessed 2012 May 28
64. Karlsson MO, Holford NH. A tutorial on visual predictive checks abstract no. 1434. PAGE. Abstracts of the 17th Annual Meeting of the Population Approach Group in Europe; 2008 Jun 18-20; Marseille online. Available from URL: http://www.page-meeting.org/?abstract=1434 Accessed 2012 May 28
65. Bergstrand M, Hooker AC, Wallin JE, et al. Prediction-corrected visual predictive checks for diagnosing nonlinear mixed-effects models. AAPS J 2011; 13: 143-51
66. Brendel K, Comets E, Laffont C, et al. Metrics for external model evaluation with an application to the population pharmacokinetics of gliclazide. Pharm Res 2006; 23: 2036-49
67. Brendel K, Comets E, Laffont C, et al. Evaluation of different tests based on observations for external model evaluation of population analyses. J Pharmacokinet Pharmacodyn 2010; 37: 49-65
68. Mandema JW, Verotta D, Sheiner LB. Building population pharmacokinetic-pharmacodynamic models: I. Models for covariate effects. J Pharmacokinet Biopharm 1992; 20: 511-28 (Pubitemid 23034413)
69. Sheiner LB. Analysis of pharmacokinetic data using parametric models: III. Hypothesis tests and confidence intervals. J Pharmacokinet Biopharm 1986; 14: 539-55 (Pubitemid 17201338)