Scientific and Regulatory Policy Committee: Recommended ("Best") Practices for Determining, Communicating, and Using Adverse Effect Data from Nonclinical Studies

Toxicologic Pathology

Volumn 44, Issue 2, 2016, Pages 147-162

  Kerlin, Roy ^a   Bolon, Brad ^b   Burkhardt, John ^c   Francke, Sabine ^d   Greaves, Peter ^e   Meador, Vince ^f   Popp, James ^g  

^a PFIZER INC   (United States)

^b GEMpath Inc   (United States)

^c ABBVIE INC   (United States)

^d CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

^e LEICESTER ROYAL INFIRMARY   (United Kingdom)

^f 206 Carnegie Center   (United States)

^g Stratoxon   (United States)

Author keywords

adversity; NOAEL; nonclinical; risk assessment; toxicity study

Indexed keywords

ENVIRONMENTAL CHEMICAL; XENOBIOTIC AGENT;

HUMAN; METABOLISM; NO-OBSERVED-ADVERSE-EFFECT LEVEL; NONHUMAN; PATHOLOGY; POLICY; PRIORITY JOURNAL; PRODUCT DEVELOPMENT; REVIEW; RISK FACTOR; TOXICITY; ANIMAL; LEGISLATION AND JURISPRUDENCE; METHODOLOGY; PROCEDURES; RISK ASSESSMENT; STANDARDS; TOXICITY TESTING;

ANIMALS; HUMANS; NO-OBSERVED-ADVERSE-EFFECT LEVEL; RESEARCH DESIGN; RISK ASSESSMENT; TOXICITY TESTS;

EID: 84960457410     PISSN: 01926233     EISSN: 15331601     Source Type: Journal    
DOI: 10.1177/0192623315623265     Document Type: Review

References (42)

1. Amacher D. E., Schomaker J., Burkhardt J. E., (1998). The relationship among microsomal enzymes induction, liver weights, and histological change in rat toxicology studies. Food Chem Toxicol 36, 831-39.
2. Ammerschlaeger M., Beigel J., Klein K. U., Mueller S. O., (2004). Characterization of the species-specificity of peroxisome proliferators in rat and human hepatocytes. Toxicol Sci 78, 229-40.
3. Bassett L., Troncy E., Pouliot M., Paquette D., Ascah A., Authier S., (2014). Telemetry video-electroencephalography (EEG) in rats, dogs and non-human primates: Methods in follow-up safety pharmacology seizure liability assessments. J Pharmacol Toxicol Methods 70, 230-240.
4. Bokkers B. G., Slob W., (2005). A comparison of ratio distributions based on the NOAEL and the benchmark approach for subchronic-to-chronic extrapolation. Toxicol Sci 85, 1033-40.
5. Bokkers B. G., Slob W., (2007). Deriving a data-based interspecies assessment factor using the NOAEL and the benchmark dose approach. Crit Rev Toxicol 37, 355-73.
6. Bowen R., (1996). Physiology of Vomiting. Accessed May, 2015. http://arbl.cvmbs.colostate.edu/hbooks/pathphys/digestion/stomach/vomiting.html.
7. Calabrese E. J., Baldwin L. A., (1994). Improved method for selection of the NOAEL. Regul Toxicol Pharmacol 19, 48-50.
8. Choi S.-Y., Hwang J.-S., Kim I.-H., Hwang D.-Y., Kang H.-G., (2011). Basic data on the hematology, serum biochemistry, urology, and organ weights of beagle dogs. Lab Anim Res 27, 283-91.
9. CLP Regulation. (2008). Regulation (EC) No. 1272/2008 of the European Parliament and of the Council of 16 December 2008 on classification, labelling and packaging of substances and mixtures, amending and repealing Directives 67/548/EEC and 1999/45/EC, and amending Regulation (EC) No 1907/2006. section 3.7.2.3.2. and 3.7.2.5.5.
10. Dekkers S., Telman J., Rennen M. A., Appel M. J., de Heer C., (2006). Within-animal variation as an indication of the minimal magnitude of the critical effect size for continuous toxicological parameters applicable in the benchmark dose approach. Risk Anal 26, 867-80.
11. Dorato M. A., Engelhardt J. A., (2005). The no-observed-adverse-effect-level in drug safety evaluations: Use, issues, and definition(s). Regul Toxicol Pharmacol 42, 265-74.
12. Elwood C., Devauchelle P., Elliott J., Freiche V., German A. J., Gualtieri M., Hall E., den Hertog E., Neiger R., Peeters D., Roura X., Savary-Bataille K., (2010). Emesis in dogs: A review. J Small Anim Pract 51, 4-22.
13. FDCA (Food Drug and Cosmetic Act and amendments). (1938).
14. Fletcher A. P., (1978). Drug safety tests and subsequent clinical experience. J R Soc Med 71, 693-696.
15. Fortner J. H., Milisen W. B., Lundeen G. R., Jakowski A. B., Marsh P. M., (1993). Tapetal effect of an azalide antibiotic following oral administration in beagle dogs. Fundam Appl Toxicol 21, 164-73.
16. Freireich E. J., Gehen E. A., Rall D. P., Schmidt L. H., Skipper H. E., (1966). Quantitative comparison of toxicity of anticancer agents in mouse, rat, hamster, dog, monkey, and man. Cancer Chemother Reports 50, 219-44.
17. Greaves P., Williams A., Eve M., (2004). First dose of potential new medicines to humans: How animals help. Nat Rev Drug Discov 3, 226-36.
18. Hall A. P., Elcombe C. R., Foster J. R., Harada T., Kaufmann W., Knippel A., Küttler K., Malarkey D. E., Maronpot R. R., Nishikawa A., Nolte T., Schulte A., Strauss V., York M. J., (2012). Liver hypertrophy: A review of adaptive (adverse and non-adverse) changes-Conclusions from the 3rd International ESTP Expert Workshop. Toxicol Pathol 40, 971-94.
19. Hard G. C., Rodgers I. S., Baetcke K. P., Richards W. L., McGaughy R. E., Valcovic L. R., (1993). Hazard evaluation of chemicals that cause accumulation of alpha 2u-globulin, hyaline droplet nephropathy, and tubule neoplasia in the kidneys of male rats. Environ Health Perspect 99, 313-49.
20. Holsapple M. P., Wallace K. B., (2008). Dose response considerations in risk assessment-An overview of recent ILSI activities. Toxicol Lett 180, 85-92.
21. Hosford D. A., Lai E. H., Riley J. H., Xu C. F., Danoff T. M., Roses A. D., (2004). Pharmacogenetics to predict drug-related adverse events. Toxicol Pathol 32, 9-12.
22. IPCS. (2004). International Programme on Chemical Safety (IPCS) harmoninzation project: Risk assessment terminology. Part 1: IPCS/OECD key generic terms used in chemical hazard/Risk assessment part 2: IPCS glossary of key exposure assessment terminology. World Health Organization.
23. IRIS (Integrated Risk Information System). (2007). U.S. Environmental Protection Agency. Integrated Risk Information System (IRIS): Glossary of IRIS terms. www.epa.gov/iris.
24. Jorkasky D. K., (1998). What does the clinician want to know from the toxicologist? Toxicol Lett 102-103, 539-43.
25. Karbe E., Williams G., Lewis R., Kimber I., Foster P., (2002). Distinguishing between adverse and non-adverse effects. Exp Toxic Pathol 54, 51-55.
26. Keller D. A., Juberg D. R., Catlin N., Farland W. H., Hess F. G., Wolf D. C., Doerrer N. G., (2012). Identification and characterization of adverse effects in 21st century toxicology. Toxicol Sci 126, 291-97.
27. Kimber I., Dearman R. J., (2002). Immune responses: Adverse versus non-adverse effects. Toxicol Pathol 30, 54-58.
28. Klaassen C., Eaton D., (1991). Principles of Toxicology (Amdu M., Doull J., Klaassen C., eds.), pp. 12-49. Casarett and Doull's, New York.
29. Krewski D., Acosta D. Jr, Andersen M., Anderson H., Bailar J. C., Boekelheide K., Brent R., Charnley G., Cheung V. G., Green S., Kelsey K. T., Kerkvliet N. I., Li A. A., McCray L., Meyer O., Patterson R. D., Pennie W., Scala R. A., Solomon G. M., Stephens M., Yager J., Zeise L., and Staff of Committee on Toxicity Testing and Assessment of Environmental Agents. (2010). Toxicity testing in the 21st century: A vision and a strategy. J Toxicol Environ Health Part B: Crit Rev 13, 51-138.
30. Lewis R. W., Billington R., Debryune E., Gamer A., Lang B., Carpanini F., (2002). Recognition of adverse and nonadverse effects in toxicity studies. Toxicol Pathol 30, 66-74.
31. Mattia A., (2011). Statutory and regulatory framework for FDA's review of food additives, GRAS substances, and food contact substances. Presentation at the Pew Charitable Trust's Pre-Workshop Webinar, March 29. http://www.pewtrusts.org/∼/media/legacy/uploadedfiles/wwwpewtrustsorg/reports/food-additives/fdasupplementalmaterialspdf.pdf.
32. Muller P. Y., Milton M. N., (2012). The determination and interpretation of the therapeutic index in drug development. Nat Rev Drug Discov 11, 751-61.
33. Olson H., Betton G., Robinson D., Thomas K., Monro A., Kolaja G., Lilly P., Sanders J., Sipes G., Bracken W., Dorato M., Van Deun K., Smith P., Berger B., Heller A., (2000). Concordance of the toxicity of pharmaceuticals in humans and in animals. Reg Toxicol Pharmacol 32, 56-67.
34. Owens A., (1962). Predicting anticancer drug effects in man from laboratory animal studies. J Chron Dis 15, 223-28.
35. Renwick A. G., Barlow S. M., Hertz-Picciotto I., Boobis A. R., Dybing E., Edler L., Eisenbrand G., Greig J. B., Kleiner J., Lambe J., Müller D. J., Smith M. R., Tritscher A., Tuijtelaars S., van den Brandt P. A., Walker R., and Kroes R., (2003). Risk characterisation of chemicals in food and diet. Food Chem Toxicol 41, 1211-71.
36. Schein P. S., Davis R. D., Carter S., Newman J., Schein D. R., Rall D. P., (1970). The evaluation of anticancer drugs in dogs and monkeys for the prediction of qualitative toxicities in man. Clin Pharmacol Ther 11, 3-40.
37. Sergeant A., (2002). Ecological risk assessment: History and fundamentals. In Human and Ecological Risk Assessement: Theory and Practice (Paustenbach D., ed.), pp. 369-442. John Wiley and Sons, New York.
38. Tvarijonaviciute A., Tecles F., Ceron J. J., (2010). Relationship between serum butyrylcholinesterase and obesity in dogs: A preliminary report. Vet J 186, 197-200.
39. U.S. EPA (Environmental Protection Agency). (2012). Benchmark dose (BMD) methodology. Accessed July, 2015. http://www.epa.gov/ncea/bmds/bmds-training/methodology/intro.htm#Noael.
40. U.S. FDA-CDER (U.S. Food and Drug Administration, Center for Drug Evaluation and Research). (2005). Guidance for industry: Estimating the maximum safe starting dose in initial clinical trials for therapeutics in adult healthy volunteers. Accessed July, 2015. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm078932.pdf.
41. U.S. FDA-CFSAN (U.S. Food and Drug Administration, Center for Food Safety and Applied Nutrition). (2007). Guidance for industry and other stakeholders toxicological principles for the safety assessment of food ingredients. Redbook 2000 (revision of 2007). Accessed July, 2015. http://www.fda.gov/Food/GuidanceRegulation/GuidanceDocumentsRegulatoryInformation/IngredientsAdditivesGRASPackaging/ucm2006826.htm.
42. Willson C. J., Chandra S. A., Kimbrough C. L., Jordan H. L., (2012). Effect of estrous cycle phase on clinical pathology values in beagle dogs. Vet Clin Pathol 41, 71-76.