Practical Anticipation of Human Efficacious Doses and Pharmacokinetics Using Preclinical In Vitro and in Vivo Data

Predictive ADMET: Integrated Approaches in Drug Discovery and Development

Volumn 9781118299920, Issue , 2014, Pages 319-352

  Heimbach, Tycho ^a   Gollen, Rakesh ^b   He, Handan ^a  

^a NOVARTIS INSTITUTES FOR BIOMEDICAL RESEARCH   (United States)

^b LONG ISLAND UNIVERSITY   (United States)

Author keywords

Anticipation of human dose (AHD); Human efficacious doses; in vitro data; in vivo data; Pharmacodynamic (PD) properties; Pharmacokinetic (PK) properties; Physiologically based pharmacokinetic (PBPK) absorption model

Indexed keywords

BIOCHEMISTRY; PHYSIOLOGICAL MODELS; PHYSIOLOGY; UNCERTAINTY ANALYSIS;

ABSORPTION MODEL; ANTICIPATION OF HUMAN DOSE; HUMAN EFFICACIOUS DOSE; IN VITRO DATA; IN VIVO DATA; IN-VITRO; IN-VIVO; PHARMACODYNAMIC PROPERTY; PHARMACOKINETIC PROPERTIES; PHYSIOLOGICALLY BASED PHARMACOKINETIC ABSORPTION MODEL; PROPERTY;

PHARMACOKINETICS;

EID: 84926400433     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1002/9781118783344.ch15     Document Type: Chapter

References (81)

1. Heimbach, T., Lakshminarayana Suresh, B., Hu, W., He, H. (2009). Practical anticipation of human efficacious doses and pharmacokinetics using in vitro and preclinical in vivo data. The AAPS Journal, 11(3), 602-614.
2. Lowe, P.J., Hijazi, Y., Luttringer, O., Yin, H., Sarangapani, R., Howard, D. (2007). On the anticipation of the human dose in first-in-man trials from preclinical and prior clinical information in early drug development. Xenobiotica, 37(10/11), 1331-1354.
3. Thomas, V.H., Bhattachar, S., Hitchingham, L., Zocharski, P., Naath, M., Surendran, N., Stoner, C.L., El-Kattan, A. (2006). The road map to oral bioavailability: An industrial perspective. Expert Opinion Drug Metabolism and Toxicology, 2(4), 591-608.
4. Miller, R., Ewy,W., Corrigan, B.W., Ouellet, D., Hermann, D.,Kowalski, K.G., Lockwood, P., Koup, J.R., Donevan, S., El-Kattan, A., Li, C.S.W.,Werth, J.L., Feltner, D.E., Lalonde, R.L. (2005). How modeling and simulation have enhanced decision making in new drug development. Journal of Pharmacokinetics Pharmacodynamics, 32(2), 185-197.
5. Huang, C., Zheng, M., Yang, Z., Rodrigues, A.D., Marathe, P. (2008). Projection of exposure and efficacious dose prior to first-in-human studies: How successful have we been?. Pharmaceutical Research, 25(4), 713-726.
6. Vuppugalla, R., Marathe, P., He, H., Jones, R.D., Yates, J.W., Jones, H.M., Gibson, C.R., Chien, J.Y., Ring, B.J., Adkison, K.K., Ku, M.S., Fischer, V., Dutta, S., Sinha, V.K., Bjornsson, T., Lave, T., Poulin, P. (2011). PhRMA CPCDC initiative on predictive models of human pharmacokinetics, part 4: Prediction of plasma concentration-time profiles in human from in vivo preclinical data by using the Wajima approach. Journal of Pharmaceutical Science, 100(10), 4111-4126.
7. Ring, B.J., Chien, J.Y., Adkison, K.K., Jones, H.M., Rowland, M., Jones, R.D., Yates, J.W., Ku, M.S., Gibson, C.R., He, H., Vuppugalla, R., Marathe, P., Fischer, V., Dutta, S., Sinha, V.K., Bjornsson, T., Lave, T., Poulin, P. (2011). PhRMA CPCDC initiative on predictive models of human pharmacokinetics, part 3: Comparative assessement of prediction methods of human clearance. Journal of Pharmaceutical Science, 100(10), 4090-4110.
8. Amidon, G.L., Lennernaes, H., Shah, V.P., Crison, J.R. (1995). A theoretical basis for a biopharmaceutic drug classification: The correlation of in vitro drug product dissolution and in vivo bioavailability. Pharmaceutical Research, 12(3), 413-420.
9. Custodio, J.M., Wu, C.-Y., Benet, L.Z. (2008). Predicting drug disposition, absorption/ elimination/transporter interplay and the role of food on drug absorption. Advanced Drug Delivery Reviews, 60(6), 717-733.
10. Li, S., He, H., Parthiban, L.J., Yin, H., Serajuddin, A.T.M. (2005). IVIVC considerations in the development of immediate-release oral dosage form. Journal of Pharmaceutical Science, 94(7), 1396-1417.
11. Lipinski, C.A. (2000). Drug-like properties and the causes of poor solubility and poor permeability. Journal of Pharmacology and Toxicology Methods, 44(1), 235-249.
12. Kasim, N.A., Whitehouse, M., Ramachandran, C., Bermejo, M., Lennernaes, H., Hussain, A.S., Junginger, H.E., Stavchansky, S.A., Midha, K.K., Shah, V.P., Amidon, G.L. (2004). Molecular properties of WHO essential drugs and provisional biopharmaceutical classification. Molecular Pharmaceutics, 1(1), 85-96.
13. Gabrielsson, J.D.H., Gillberg, P.G., Bredberg, U., Benthem, B., Duker, G. (2009). Early integration of pharmacokinetic and dynamic reasoning is essential for optimal development of lead compounds: strategic considerations. Drug Discovery Today, 14(7-8), 358-372.
14. FDA Guidance for Industry - Estimating the Safe Starting Dose in Clinical Trials for Therapeutics in Adult Healthy Volunteers Center for Drug Evaluation and Research (CDER). 2005, pp. 1-30.
15. Reigner, B.G., Blesch, K.S. (2002). Estimating the starting dose for entry into humans:Principles and practice. European Journal of Clinical Pharmacology, 57(12), 835-845.
16. Sharma, V., McNeill, J.H. (2009). To scale or not to scale: The principles of dose extrapolation. British Journal of Pharmacology, 157(6), 907-921.
17. Mahmood, I., Green, M.D., Fisher, J.E. (2003). Selection of the first-time dose in humans:Comparison of different approaches based on interspecies scaling of clearance. Journal of Clinical Pharmacology, 43(7), 692-697.
18. EMEA. Guidline on requirements for First-in-Man clinical trials for potential high-risk medicinal products, Committee for Medicinal Products for Human Use. London, UK, 2007, pp. 1-11.
19. Colburn,W.A. (2000). Optimizing the use of biomarkers, surrogate endpoints, and clinical endpoints for more efficient drug development. Journal of Clinical Pharmacology, 40(12), 1419-1427.
20. Reigner, B.G., Williams, P.E., Patel, I.H., Steimer, J.L., Peck, C.,Van Brummelen, P. (1997). An evaluation of the integration of pharmacokinetic and pharmacodynamic principles in clinical drug development. Experience within Hoffmann La Roche. Clinical Pharmacokinetics, 33(2), 142-152.
21. Iavarone, L., Hoke, J.F., Bottacini, M., Barnaby, R., Preston, G.C. (1999). First time in human for GV196771: Interspecies scaling applied on dose selection. Journal of Clinical Pharmacology, 39(6), 560-566.
22. Muller, P.Y., Brennan, F.R. (2009). Safety assessment and dose selection for first-in-human clinical trials with immunomodulatory monoclonal antibodies. Clinical Pharmacology & Therapeutics, 85(3), 247-258.
23. Zou, P.,Yu,Y., Zheng, N.,Yang,Y., Paholak, H.J.,Yu, L.X., Sun, D. (2012). Applications of human pharmacokinetic prediction in first-in-human dose estimation. The AAPS Journal, 14(2), 262-281.
24. Agoram, B.M. (2009). Use of pharmacokinetic/ pharmacodynamic modelling for starting dose selection in first-in-human trials of high-risk biologics. British Journal of Clinical Pharmacology, 67(2), 153-160.
25. Tannergren, C., Bergendal, A., Lennernas, H., Abrahamsson, B. (2009). Toward an increased understanding of the barriers to colonic drug absorption in humans: Implications for early controlled release candidate assessment. Molecular Pharmaceutics, 6(1), 60-73.
26. FDA. S6 Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals,Center for Drug Evaluation and Research (CDER). Rockville, MD, 1997, pp. 1-14.
27. FDA. Guidance on Nonclinical Safety Studies for the Conduct of Human Clinical Trials and Marketing Authorization for Pharmaceuticals M3(R2), Food Drug Administration. Silver Spring, MD, 2008, pp. 1-14.
28. Chaikin, P., Rhodes, G.R., Bruno, R., Rohatagi, S., Natarajan, C. (2000). Pharmacokinetics/ pharmacodynamics in drug development: An industrial perspective. Journal of Clinical Pharmacology, 40(12), 1428-1438.
29. De Buck, S.S., Sinha, V.K., Fenu, L.A., Nijsen, M.J., Mackie, C.E., Gilissen, R.A.H.J. (2007). Prediction of human pharmacokinetics using physiologically based modeling:A retrospective analysis of 26 clinically tested drugs. Drug Metabolism & Disposition, 35(10), 1766-1780.
30. Fura, A., Vyas,V., Humphreys,W., Chimalokonda, A., Rodrigues, D. (2008). Prediction of human oral pharmacokinetics using nonclinical data: Examples involving four proprietary compounds. Biopharmaceutics & Drug Disposition, 29(8), 455-468.
31. Sinha, V.K., De Buck, S.S., Fenu, L.A., Smit, J.W., Nijsen, M., Gilissen, A.H.J., Van Peer, A., Lavrijsen, K., Mackie, C.E. (2008). Predicting oral clearance in humans: How close can we get with allometry?. Clinical Pharmacokinetics, 47(1), 35-45.
32. Wajima, T., Yano, Y., Fukumura, K., Oguma, T. (2004). Prediction of human pharmacokinetic profile in animal scale up based on normalizing time course profiles. Journal of Pharmaceutical Science, 93(7), 1890-1900.
33. Mahmood, I. (2006). Prediction of human drug clearance from animal data: Application of the rule of exponents and 'fu corrected intercept method' (FCIM). Journal of Pharmaceutical Science, 95(8), 1810-1821.
34. Mahmood, I., Yuan, R. (1999). A comparative study of allometric scaling with plasma concentrations predicted by species-invariant time methods. Biopharmaceutics & Drug Disposition, 20(3), 137-144.
35. Tang, H., Mayersohn, M. (2005). A novel model for prediction of human drug clearance by allometric scaling. Drug Metabolism & Disposition, 33(9), 1297-1303.
36. Mahmood, I. Interspecies pharmacokinetic scaling: Allometric principles and applications, Pine House Publishers. Rockville, MD, 2005.
37. Obach, R.S., Baxter, J.G., Liston, T.E., Silber, B.M., Jones, B.C., Macintyre, F., Rance, D.J., Wastall, P. (1997). The prediction of human pharmacokinetic parameters from preclinical and in vitro metabolism data. The Journal of Pharmacology and Experimental Therapeutics, 283(1), 46-58.
38. Obach, R.S., Lombardo, F.,Waters, N.J. (2008). Trend analysis of a database of intravenous pharmacokinetic parameters in humans for 670 drug compounds. Drug Metabolism & Disposition, 36(7), 1385-1405.
39. Hosea, N., Collard,W.T., Cole, S., Maurer, T.S., Fang, R.X., Jones, H., Kakar, S.M., Nakai, Y., Smith, B.J.,Webster, R., Beaumont, K. (2009). Prediction of human pharmacokinetics from preclinical information: Comparative accuracy of quantitative prediction approaches. Journal of Clinical Pharmacology, 49(5), 513-533.
40. Oie, S., Tozer, T.N. (1979). Effect of altered plasma protein binding on apparent volume of distribution. Journal of Pharmaceutical Science, 68(9), 1203-1205.
41. Jones, R.D., Jones, H.M., Rowland, M., Gibson, C.R., Yates, J.W., Chien, J.Y., Ring, B.J., Adkison, K.K., Ku, M.S., He, H., Vuppugalla, R., Marathe, P., Fischer, V., Dutta, S., Sinha, V.K., Bjornsson, T., Lave, T., Poulin, P. (2011). PhRMA CPCDC initiative on predictive models of human pharmacokinetics, part 2: Comparative assessment of prediction methods of human volume of distribution. Journal of Pharmaceutical Science, 100(10), 4074-4089.
42. Stoner, C.L., Cleton, A., Johnson, K., Oh, D.-M., Hallak, H., Brodfuehrer, J., Surendran, N., Han, H.-K. (2004). Integrated oral bioavailability projection using in vitro screening data as a selection tool in drug discovery. International Journal of Pharmaceutics, 269(1), 241-249.
43. Jones, H.M., Parrott, N., Jorga, K., Lave, T. (2006). A novel strategy for physiologically based predictions of human pharmacokinetics. Clinical Pharmacokinetics, 45(5), 511-542.
44. Luttringer, O., Theil, F.-P., Poulin, P., Schmitt-Hoffmann, A.H., Guentert, T.W., Lave, T. (2003). Physiologically based pharmacokinetic (PBPK) modeling of disposition of epiroprim in humans. Journal of Pharmaceutical Sciences, 92(10), 1990-2007.
45. Parrott, N., Jones, H., Paquereau, N., Lave, T. (2005). Application of full physiological models for pharmaceutical drug candidate selection and extrapolation of pharmacokinetics to man. Basics & Clinical Pharmacology & Toxicology, 96(3), 193-199.
46. Mahmood, I., Balian, J.D. (1996). Interspecies scaling: Predicting pharmacokinetic parameters of antiepileptic drugs in humans from animals with special emphasis on clearance. Journal of Pharmaceutical Sciences, 85(4), 411-414.
47. Mahmood, I. (2002). Prediction of clearance in humans from in vitro human liver microsomes and allometric scaling. A comparative study of the two approaches. Drug Metabolism & Drug Interaction, 19(1), 49-64.
48. Tang, H., Hussain, A., Leal, M., Mayersohn, M., Fluhler, E. (2007). Interspecies prediction of human drug clearance based on scaling data from one or two animal species. Drug Metabolism & Disposition, 35(10), 1886-1893.
49. Wajima,T., Fukumura, K.,Yano,Y., Oguma,T. (2002). Prediction of human clearance from animal data and molecular structural parameters using multivariate regression analysis. Journal of Pharmaceutical Science, 91(12), 2489-2499.
50. Houston, J.B., Galetin, A. (2003). Progress towards prediction of human pharmacokinetic parameters from in vitro technologies. Drug Metabolism Reviews, 35(4), 393-415.
51. Houston, J.B., Carlile, D.J. (1997). Prediction of hepatic clearance from microsomes, hepatocytes, and liver slices. Drug Metabolism Reviews, 29(4), 891-922.
52. Bonate, P.L., Howard, D.R., Pharmacokinetics in Drug Development, AAPS. Arlington, VA, 2004, p. 451.
53. Boxenbaum, H. (1984). Interspecies pharmacokinetic scaling and the evolutionarycomparative paradigm. Drug Metabolism Reviews, 15(5-6), 1071-1121.
54. Sawada, Y., Hanano, M., Sugiyama, Y., Harashima, H., Iga, T. (1984). Prediction of the volumes of distribution of basic drugs in humans based on data from animals. Journal of Pharmacokinetics and Biopharmaceutics, 12(6), 587-596.
55. Lin, T., Heimbach, T., Gollen, R., He, H. Mass Balance Studies in Animals and Humans, Wiley. New York, 2012, pp. 1-39.
56. Bittner, B., Mountfield, R.J. (2002). Intravenous administration of poorly soluble new drug entities in early drug discovery: The potential impact of formulation on pharmacokinetic parameters. Current Opinion in Drug Discovery & Development, 5(1), 59-71.
57. Dannenfelser, R.-M., He, H., Joshi, Y., Bateman, S., Serajuddin, A.T.M. (2004). Development of clinical dosage forms for a poorlywater soluble drug I: Application of polyethylene glycol-polysorbate 80 solid dispersion carrier system. Journal of Pharmaceutical Sciences, 93(5), 1165-1175.
58. Kesisoglou, F.,Wu,Y. (2008). Understanding the effect of API properties on bioavailability through absorption modeling. The AAPS Journal, 10(4), 516-525.
59. Tang, H., Mayersohn, M. (2005). A mathematical description of the functionality of correction factors used in allometry for predicting human drug clearance. Drug Metabolism & Disposition, 33(9), 1294-1296.
60. Rowland, M., Peck,C., Tucker, G. (2011). Physiologically-based pharmacokinetics in drug development and regulatory science. Annual Review of Pharmacology and Toxicology, 51, 45-73.
61. Jamei, M., Marciniak, S., Feng, K., Barnett, A., Tucker, G., Rostami-Hodjegan, A. (2009). The Simcyp population-based ADME simulator. Expert Opinion on Drug Metabolism & Toxicology, 5(2), 211-223.
62. Agoram, B., Woltosz, W.S., Bolger, M.B. (2001). Predicting the impact of physiological and biochemical processes on oral drug bioavailability. Advanced Drug Delivery Reviews, 50(1), S41-S67.
63. Jamei, M., Turner, D., Yang, J., Neuhoff, S., Polak, S., Rostami-Hodjegan, A., Tucker, G. (2009). Population-based mechanistic prediction of oral drug absorption. The AAPS Journal, 11(2), 225-237.
64. Yu, L.X., Lipka, E., Crison, J.R., Amidon, G.L. (1996). Transport approaches to the biopharmaceutical design of oral drug delivery systems: Prediction of intestinal absorption. Advanced Drug Delivery Reviews, 19(3), 359-376.
65. Gabrielsson, J., Weiner, D., Pharmacokinetic and Pharmacodynamic Data Analysis:Concepts and Applications, Swedish Pharmaceutical Press. Sweden, 2007, pp. 1039-1042.
66. Lukacova, V., Parrott, N.J., Lavé, T., Fraczkiewicz, G., Bolger, M.B.,Woltosz, W.S., Role of Fraction Unbound in Plasma in Calculations of Tissue, Plasma Partition Coefficients. Atlanta, GA, 2008.
67. Rodgers, T., Leahy, D., Rowland, M. (2007). Physiologically based pharmacokinetic modeling: Predicting the tissue distribution of moderate-to-strong bases. Journal of Pharmaceutical Science, 96(11), 3151-3152.
68. Rodgers, T., Rowland, M. (2007). Physiologically-based pharmacokinetic modeling 2:Predicting the tissue distribution of acids, very weak bases, neutrals and zwitterions. Journal of Pharmaceutical Science, 96(11), 3153-3154.
69. Meibohm, B., Derendorf, H. (1997). Basic concepts of pharmacokinetic/ pharmacodynamic (PK/PD) modelling. International Journal of Clinical Pharmacology & Therapeutics, 35(10), 401-413.
70. Davies, B., Morris, T. (1993). Physiological parameters in laboratory animals and humans. Pharmaceutical Research, 10(7), 1093-1095.
71. FDA. Guidance for Industry - Food - Effect Bioavailability and Fed Bioequivalence Studies, Center for Drug Evaluation and Research (CDER). Rockville, MD, 2002, pp. 1-12.
72. Tang, H., Hussain, A., Leal, M., Mayersohn, M., Fluhler, E. (2007). Interspecies prediction of human drug clearance based on scaling data from one or two animal species. Drug Metabolism and Disposition, 35(10), 1886-1893.
73. Tang, H., Mayersohn, M. (2005). A novel model for prediction of human drug clearance by allometric scaling. Drug Metabolism and Disposition, 33(9), 1297-1303.
74. Poulin, P., Theil, F.P. (2000). A priori prediction of tissue: Plasma partition coefficients of drugs to facilitate the use of physiologically-based pharmacokinetic models in drug discovery. Journal of Pharmaceutical Science, 89(1), 16-35.
75. Dressman, J., Reppas, C. (2007). Drug solubility: How to measure it, how to improve it. Advance Drug Delivery Reviews, 59(7), 531-532.
76. Selick, H.E., Beresford, A.P., Tarbit, M.H. (2002). The emerging importance of predictive ADME simulation in drug discovery. Drug Discovery Today, 7(2), 109-116.
77. Poulin, P., Jones, H.M., Jones, R.D., Yates, J.W., Gibson, C.R., Chien, J.Y., Ring, B.J., Adkison, K.K., He, H., Vuppugalla, R., Marathe, P., Fischer, V., Dutta, S., Sinha, V.K., Bjornsson, T., Lave, T., Ku, M.S. (2011). PhRMA CPCDC initiative on predictive models of human pharmacokinetics, part 1: Goals, properties of the PhRMA dataset, and comparison with literature datasets. Journal of Pharmaceutical Science, 100(10), 4050-4073.
78. Poulin, P., Jones, R.D., Jones, H.M., Gibson, C.R., Rowland, M., Chien, J.Y., Ring, B.J., Adkison, K.K., Ku, M.S., He, H., Vuppugalla, R., Marathe, P., Fischer, V., Dutta, S., Sinha, V.K., Bjornsson, T., Lave, T., Yates, J.W. (2011). PHRMA CPCDC initiative on predictive models of human pharmacokinetics, part 5: Prediction of plasma concentrationtime profiles in human by using the physiologically-based pharmacokinetic modeling approach. Journal of Pharmaceutical Science, 100(10), 4127-4157.
79. Ito, K., Houston, J.B. (2005). Prediction of human drug clearance from in vitro and preclinical data using physiologically based and empirical approaches. Pharmaceutical Research, 22(1), 103-112.
80. Lave, T., Coassolo, P., Reigner, B. (1999). Prediction of hepatic metabolic clearance based on interspecies allometric scaling techniques and in vitro-in vivo correlations. Clinical Pharmacokinetics, 36(3), 211-231.
81. Lopworth, B.J. (1996). Pharmacokinetics of inhaled drugs. British Journal of Clinical Pharmacology, 42(6), 697-705.