The evolution of juvenile animal testing for small and large molecules

Regulatory Toxicology and Pharmacology

Volumn 67, Issue 2, 2013, Pages 125-135

  Baldrick, Paul ^a  

^a Department of Genetic and Molecular Toxicology   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ABATACEPT; ALIPOGENE TIPARVOVEC; APIXABAN; APREPITANT; AZILSARTAN MEDOXOMIL; BELATACEPT; BRIVARACETAM; CARISBAMATE; CASOPITANT; CASPOFUNGIN; CATRIDECACOG; CINACALCET; DALBAVANCIN; DENOSUMAB; EPLIVANSERIN; FOSAPREPITANT; MOXIFLOXACIN; NILOTINIB; PALIPERIDONE; PLERIXAFOR; RETIGABINE; TAPENTADOL; TARANABANT; TELAVANCIN; TELBIVUDINE; TICAGRELOR; UNINDEXED DRUG; USTEKINUMAB; VICRIVIROC; VOCLOSPORIN;

ANIMAL TESTING ALTERNATIVE; ANIMAL TESTING REDUCTION; B LYMPHOCYTE; DEVELOPMENTAL TOXICITY; DRUG SAFETY; HUMAN; IMMUNE SYSTEM; JUVENILE ANIMAL; NONHUMAN; NOTE; POSTMARKETING SURVEILLANCE; POSTNATAL DEVELOPMENT; PRIORITY JOURNAL; REGULATORY MECHANISM; T LYMPHOCYTE; TOXICITY TESTING; TOXICOKINETICS;

ANIMALIA;

JUVENILE ANIMAL TESTING; PAEDIATRIC CLINICAL TRIALS; PAEDIATRIC INVESTIGATION PLAN; PIP; REGULATORY AGENCIES;

ANIMALS; DRUG EVALUATION, PRECLINICAL; MODELS, ANIMAL; RESEARCH DESIGN; TOXICITY TESTS;

EID: 84886591130     PISSN: 02732300     EISSN: 10960295     Source Type: Journal    
DOI: 10.1016/j.yrtph.2013.07.009     Document Type: Note

References (55)

1. Bailey G.P., Marien D. The value of juvenile animal studies "What have we learned from preclinical juvenile toxicity studies? II". Birth Defects Res. (Part B) 2011, 92(4):273-291.
2. Baldrick P. Developing drugs for pediatric use: a role for juvenile animal studies?. Regul. Toxicol. Pharmacol. 2004, 39:381-389.
3. Baldrick P. Juvenile animal testing in drug development - is it useful?. Regul. Toxicol. Pharmacol. 2010, 57:291-299.
4. Baldrick P., Hewings S., Skinner M. Reproduction and juvenile animal toxicology studies in the rat with a new allergy vaccine adjuvanted with monophosphoryl lipid A (MPL®) for the treatment of grass pollen allergy. Reprod. Toxicol. 2011, 32:322-328.
5. Beckman D.A., Feuston M. Landmarks in the development of the female reproductive system. Birth Defects Res. (Part B) 2003, 68(3):137-143.
6. Belatacept EPAR, 2011. European Medicines Agency (EMA): European Public Assessment Report: Belatacept (Nulojix). http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/002098/WC500108357.pdf.
7. Burnett J.E. Dried blood spot sampling: practical considerations and recommendation for use with preclinical studies. Bioanalysis 2011, 3(10):1099-1107.
8. Cappon G.D. Nonclinical support of pediatric drug development in a global context: an industry perspective. Birth Defects Res. (Part B) 2011, 92:269-272.
9. Carleer J., Karres J. Juvenile animal studies and pediatric drug development: a European regulatory perspective. Birth Defects Res. (Part B) 2011, 92:254-260.
10. Cross D.M., Chmielewski G., Lewis E.M., Liu L., Modesitt M.S., Ripp S.L., Sawaryn C.M., Bowman C.J. Non-clinical safety assessment and toxicokinetics of voriconazole and anidulafungin in the juvenile rat: a combination study design in support of a paediatric investigation plan. Regul. Toxicol. Pharmacol. 2012, 63:29-39.
11. Cross D.M., Derzi M., Horsley E., Owen K., Stavros F.L. Evaluation of sitaxentan (Thelin®) toxicity in juvenile rats and regulatory interactions during the development of a European medicines agency pediatric investigation plan. Regul. Toxicol. Pharmacol. 2012, 64:43-50.
12. Dainty T.C., Richmond E.S., Davies I., Blackwell M.P. Dried blood spot bioanalysis: an evaluation of techniques and opportunities for reduction and refinement in mouse and juvenile rat toxicokinetic studies. Int. J. Toxicol. 2012, 31(1):4-13.
13. De Zwart L., Scholten M., Monbaliu J.G., Annaert P.P., Van Houdt J.M., Van den Wyngaert I., De Schaepdrijver L.M., Bailey G.P., Coogan T.P., Coussement W.C., Mannens G.S. The ontogeny of drug metabolizing enzymes and transporters in the rat. Reprod. Toxicol. 2008, 26:220-230.
14. De Zwart L.L., Monbaliu J.G., Annaert P.P. Nonclinical testing procedures - pharmacokinetics. Pediatric Non-Clinical Drug Testing: Principles, Requirements and Practices 2012, Wiley.
15. Duarte D.M., Silva-Lima B. Juvenile animal studies in the development of pediatric medicines: experience from European medicines and pediatric investigation plans. Birth Defects Res. (Part B) 2011, 92:353-358.
16. EMA, 2013a. European Medicines Agency (EMA): Paediatric investigation plans, waivers and modifications. http://www.emea.europa.eu/ema/index.jsp?curl=pages/regulation/document_listing/document_listing_000293.jsp&mid=WC0b01ac0580025b91.
17. EMA, 2013b. European Medicines Agency (EMA): Opinions and decisions on paediatric investigation plans. http://www.ema.europa.eu/ema/index.jsp?curl=pages/medicines/landing/pip_search.jsp.
18. EMEA, 2008: European Medicines Agency (EMEA): Non-clinical Testing in Juvenile Animals on Human Pharmaceuticals for Paediatric Indications. http://www.ema.europa.eu/pdfs/human/swp/16921505en08.pdf.
19. FDA, 2006: United States Food and Drug Administration (FDA): Guidance for Industry: Nonclinical Safety Evaluation of Pediatric Drug Products. http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm079247.pdf.
20. FDA, 2013. Fact Sheet: Pediatric provisions in the Food and Drug Administration (FDA) Safety and Innovation Act (FDASIA). http://www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/FDASIA/ucm311038.htm.
21. Forxiga EPAR, 2012. European Medicines Agency (EMA): European Public Assessment Report: Forxiga (Dapagliflozin). http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Public_assessment_report/human/002322/WC500136024.pdf.
22. Hew K.W., Keller K.A. Postnatal anatomical and functional development of the heart: a species comparison. Birth Defects Res. (Part B) 2003, 68(4):309-320.
23. Holsapple M.P., West L.J., Landreth K.S. Species comparison of anatomical and functional immune system development. Birth Defects Res. (Part B) 2003, 68(4):321-334.
24. ICH, 2009. International Conference on Harmonisation (ICH) Topic M3 (R2): Note for Guidance on Non-Clinical Safety Studies for the Conduct of Human Clinical Trials and Marketing Authorization for Pharmaceuticals. http://www.emea.europa.eu/pdfs/human/ich/028695en.pdf.
25. ICH, 2013. International Conference on Harmonisation (ICH) Topic M3 (R2) - Questions and Answers. http://www.ema.europa.eu/docs/en_GB/document_library/Other/2011/07/WC500109298.pdf.
26. Ilaris EPAR, 2012. European Medicines Agency (EMA): European Public Assessment Report: Ilaris (Canakinumab). http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Assessment_Report_-_Variation/human/001109/WC500140964.pdf.
27. Invokana Product Label (2013). Invokana (Canagliflozin) product label. http://www.accessdata.fda.gov/drugsatfda_docs/label/2013/204042s000lbl.pdf.
28. JMHLW, 2012. Japan Ministry of Health, Labour, and Welfare (JMHLW): Guideline on non-clinical safety studies in juvenile animals for pediatric drug development. http://wwwhourei.mhlw.go.jp/hourei/doc/tsuchi/T121018I0010.pdf.
29. Jonsson O., Villar R.P., Nilsson L.B., Norsten-Höög C., Brogren J., Eriksson M., Königsson K., Samuelsson A. Capillary microsampling of 25 μl blood for the determination of toxicokinetic parameters in regulatory studies in animals. Bioanalysis 2012, 4(6):661-674.
30. Jonsson O., Villar R.P., Nilsson L.B., Eriksson M., Königsson K. Validation of a bioanalytical method using capillary microsampling of 8 μl plasma samples: application to a toxicokinetic study in mice. Bioanalysis 2012, 4(16):1989-1998.
31. Laffan S.B., Posobiec L. Approaches to rat juvenile toxicity studies and case studies: a pharmaceutical perspective. Pediatric Non-Clinical Drug Testing: Principles, Requirements and Practices 2012, Wiley.
32. Leconte I., Bailey G., Davis-Bruno K., Hew K.W., Kim J., Silva Lima B., Liminga U., Moffit J., De Schaepdrijver L., Schmitt G., Tassinari M., Thompson K., Hurtt M. Value of juvenile animal studies. Birth Defects Res. (Part B) 2011, 92:292-303.
33. Lewin G., Hoymann H.G., Fuhst R., Berger-Preiss E., Pohlmann G., Buschmann J. Assessment of pulmonary function and serum substance levels in newborn and juvenile rats. Reprod. Toxicol. 2010, 30:422-428.
34. Lewin G., Muller M., Braun A., Buschmann J. Development of immunological characteristics in newborn rats, PND 2-21. Reprod. Toxicol. 2011, 32:154-155. (Free Communication).
35. Marty M.S., Chapin R.E., Parks L.G., Thorsrud B.A. Development and maturation of the male reproductive system. Birth Defects Res. (Part B) 2003, 68(3):125-136.
36. Morford L.L., Bowman C.J., Blanset D.L., Bøgh I.B., Chellman G.J., Halpern W.G., Weinbauer G.F., Coogan T.P. Preclinical safety evaluations supporting pediatric drug development with biopharmaceuticals: strategy, challenges, current practices. Birth Defects Res. (Part B) 2011, 92:359-380.
37. Nilsson L.B., Ahnoff M., Jonsson O. Capillary microsampling in the regulatory environment: validation and use of bioanalytical capillary microsampling methods. Bioanalysis 2013, 5(6):731-738.
38. Parrott N., Davies B., Hoffmann G., Koerner A., Lave T., Prinssen E., Theogaraj E., Singer T. Development of a physiologically based model for oseltamivir and simulation of pharmacokinetics in neonates and infants. Clin. Pharmacokinet. 2011, 50(9):613-623.
39. Parker G.A., Picut C.A., Scully K.L., Veney D.J. Histologic features of the developing immune system of rats. Toxicol. Sci. 2008, 102(Suppl.):35. (Abstract).
40. Rhodes M., Laffan S., Genell C., Gower J., Maier C., Fukushima T., Nichols G., Bassiri A.E. Assessing a theoretical risk of dolutegravir-induced developmental immunotoxicity in juvenile rats. Toxicol. Sci. 2012, 130(1):70-81.
41. Shimomura K. The value of juvenile animal studies: a Japanese industry perspective. Birth Defects Res. (Part B) 2011, 92:228-266.
42. Silva-Lima B., Due Theilade-Thomsen M., Carleer J., Vidal J.M., Tomasi P., Saint-Raymond A. Juvenile animal studies for the development of paediatric medicines: a description and conclusions from a European medicines agency workshop on juvenile animal testing for nonclinical assessors. Birth Defects Res. (Part B) 2010, 89:467-473.
43. Soellner L., Olejniczak K. The need for juvenile animal studies - a critical review. Regul. Toxicol. Pharmacol. 2013, 65:87-99.
44. Stokes A.H., Moose T.A., Parry S.P., Barfield M., Lovatt C.A., Dopson W.J., Melich D., Overvold C.R., Gade S.D., Spooner N. Determination of drug concentrations using dried blood spots: investigation of blood sampling and collection techniques in Crl:CD(SD) rats. Lab. Anim. 2011, 45(2):109-113.
45. Tassinari M.S., Benson K., Elayan I., Espandiari P., Davis-Bruno K. Juvenile animal studies and pediatric drug development retrospective review: use in regulatory decisions and labeling. Birth Defects Res. (Part B) 2011, 92:261-265.
46. Van Dijck K., De Schaepdrijver L., Fransen J., Starckx S., Vinkern P., Vriens K. Postnatel morphological and functional development of the rat kidney. Reprod. Toxicol. 2009, 28:127. (Abstract).
47. Walthall K., Cappon G.D., Hurtt M.E., Zoetis T. Postnatal development of the gastrointestinal system: a species comparison. Birth Defects Res. (Part B) 2005, 74(2):132-156.
48. Walzer M., Bekersky I., Wanaski S., Collins S., Jortner B., Patterson R., Garman R., Sagar S., Tolbert D. Oral toxicity of vigabatrin in immature rats: characterization of intramyelinic edema. Neurotoxicol 2011, 32:963-974.
49. Watson R.E., Desesso J.M., Hurtt M.E., Cappon G.D. Postnatal growth and morphological development of the brain: a species comparison. Birth Defects Res (Part B) 2006, 77(5):471-484.
50. Weinbauer G.F, Chellman G.J., Rasmussen A.D., Vogelwedde E. Use of primate pediatric model. Pediatric Non-Clinical Drug Testing: Principles, Requirements and Practices 2012, Wiley.
51. Whitney K.M. Testicular histopathology in juvenile rat toxicity studies. Syst. Biol. Reprod. Med. 2012, 58:51-56.
52. Wood S.L., Beyer B.K., Cappon G.D. Species comparison of postnatal CNS development: functional measures. Birth Defects Res. (Part B) 2003, 68(5):391-407.
53. Zoetis T., Hurtt M.E. Species comparison of anatomical and functional renal development. Birth Defects Res. (Part B) 2003, 68(3):111-120.
54. Zoetis T., Hurtt M.E. Species comparison of lung development. Birth Defects Res. (Part B) 2003, 68(3):121-124.
55. Zoetis T., Tassinari M.S., Bagi C., Walthall K., Hurtt M.E. Species comparison of postnatal bone growth and development. Birth Defects Res. (Part B) 2003, 68(3):86-110.