Regulatory aspects of oncology drug safety evaluation: Past practice, current issues, and the challenge of new drugs

Toxicology and Applied Pharmacology

Volumn 243, Issue 2, 2010, Pages 125-133

  Rosenfeldt, Hans ^a   Kropp, Timothy ^a   Benson, Kimberly ^a   Ricci, M Stacey ^a   McGuinn, W David ^a   Verbois, S Leigh ^a  

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

Author keywords

Cancer; Drug development; Lehman; Preclinical; Regulation

Indexed keywords

ANTINEOPLASTIC AGENT; NEW DRUG;

ANIMAL; DRUG DEVELOPMENT; DRUG EFFECT; DRUG LEGISLATION; FOOD AND DRUG ADMINISTRATION; HUMAN; NEOPLASM; PRACTICE GUIDELINE; REPRODUCTION; REVIEW; SAFETY; UNITED STATES;

ANIMALS; ANTINEOPLASTIC AGENTS; DRUG DISCOVERY; DRUGS, INVESTIGATIONAL; GUIDELINES AS TOPIC; HUMANS; LEGISLATION, DRUG; NEOPLASMS; REPRODUCTION; SAFETY; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 77949374323     PISSN: 0041008X     EISSN: 10960333     Source Type: Journal    
DOI: 10.1016/j.taap.2009.12.020     Document Type: Review

References (48)

1. Ali, R., Ozkalemkas, F., Kimya, Y., Koksal, N., Ozkocaman, V., Gulten, T., Yorulmaz, H., Tunali, A., Imatinib use during pregnancy and breast feeding: a case report and review of the literature. Arch. Gynecol. Obstet. 280 (2009), 169–175.
2. Bedard, P.L., Piccart-Gebhart, M.J., Current paradigms for the use of HER2-targeted therapy in early-stage breast cancer. Clin. Breast Cancer 8:Suppl 4 (2008), S157–S165.
3. Bigwood, E.J., The acceptable daily intake of food additives. CRC Crit. Rev. Toxicol. 2 (1973), 41–93.
4. Brave, M., Goodman, V., Kaminskas, E., Farrell, A., Timmer, W., Pope, S., Harapanhalli, R., Saber, H., Morse, D., Bullock, J., Men, A., Noory, C., Ramchandani, R., Kenna, L., Booth, B., Gobburu, J., Jiang, X., Sridhara, R., Justice, R., Pazdur, R., Sprycel for chronic myeloid leukemia and Philadelphia chromosome-positive acute lymphoblastic leukemia resistant to or intolerant of imatinib mesylate. Clin. Cancer Res. 14 (2008), 352–359.
5. Chu, T.F., Rupnick, M.A., Kerkelä, R., Dallabrida, S.M., Zurakowski, D., Nguyen, L., Woulfe, K., Pravda, E., Cassiola, F, Desai, J., George, S., Morgan, J.A., Harris, D.M., Ismail, N.S., Chen, J.H., Schoen, F.J., Van den Abbeele, A.D., Demetri, G.D., Force, T., Chen, M.H., Cardiotoxicity associated with thyrosine kinase inhibitor sunitinib. Lancet 370 (2007), 2011–2019.
6. Cohen, M.H., Johnson, J.R., Chen, Y.F., Sridhara, R., Pazdur, R., FDA drug approval summary: erlotinib (Tarceva) tablets. Oncologist 10 (2005), 461–466.
7. Cohen, M.H., Williams, G., Johnson, J.R., Duan, J., Gobburu, J., Rahman, A., Benson, K., Leighton, J., Kim, S.K., Wood, R., Rothmann, M., Chen, G., U, K.M., Staten, A.M., Pazdur, R., Approval summary for imatinib mesylate capsules in the treatment of chronic myelogenous leukemia. Clin. Cancer Res. 8 (2002), 935–942.
8. Cohen, M.H., Williams, G.A., Sridhara, R., Chen, G., Pazdur, R., FDA drug approval summary: gefitinib (ZD1839) (Iressa) tablets. Oncologist 8 (2003), 303–306.
9. Dagher, R., Cohen, M., Williams, G., Rothmann, M., Gobburu, J., Robbie, G., Rahman, A., Chen, G., Staten, A., Griebel, D., Pazdur, R., Approval summary: imatinib mesylate in the treatment of metastatic and/or unresectable malignant gastrointestinal stromal tumors. Clin. Cancer Res. 8 (2002), 3034–3038.
10. DeGeorge, J.J., Ahn, C.H., Andrews, P.A., Brower, M.E., Giorgio, D.W., Goheer, M.A., Lee-Ham, D.Y., McGuinn, W.D., Schmidt, W., Sun, C.J., Tripathi, S.C., Regulatory considerations for preclinical development of anticancer drugs. Cancer Chemother. Pharmacol. 41 (1998), 173–185.
11. Environmental Protection Agency. Guidelines and methodology used in preparation of health effects assessment chapters of the consent decree water quality criteria. Federal Register 45 (1980), 79347–79357.
12. European Medicines Agency, 2007. Guideline on Strategies to Identify and Mitigate Risks for First-In-Human Clinical Trials with Investigational Medicinal Products.
13. Food and Drug Administration, 2005. Guidance for Industry: Estimating the Maximum Safe Starting Dose in Initial Clinical Trials for Therapeutics in Adult Healthy Volunteers.
14. Food and Drug Administration. International conference on harmonisation: draft guidance on S9 nonclinical evaluation for anticancer pharmaceuticals; availability. Fed. Regist. 74 (2009), 7445–7446.
15. Food and Drug Administration, 2009b. Oncologic Drug Advisory Committee, July 15, 2009, Morning Session.
16. Food Safety Council. A Proposed Food Safety Evaluation Process. Food Safety Council, 1982, The Nutrition Foundation, Inc., Washington, D.C.
17. Force, T., Krause, D.S., Van Etten, R.A., Molecular mechanisms of cardiotoxicity of tyrosine kinase inhibition. Nat. Rev. Cancer 7 (2007), 332–344.
18. Freireich, E.J., Gehan, E.A., Rall, D.P., Schmidt, L.H., Skipper, H.E., Quantitative comparison of toxicity of anticancer agents in mouse, rat, hamster, dog, monkey, and man. Cancer Chemother. Rep. 50 (1966), 219–244.
19. Green, M.C., Murray, J.L., Hortobagyi, G.N., Monoclonal antibody therapy for solid tumors. Cancer Treat. Rev. 26 (2000), 269–286.
20. Grieshaber, C.K., Marsoni, S., Relation of preclinical toxicology to findings in early clinical trials. Cancer Treat. Rep. 70 (1986), 65–72.
21. Guglin, M., Cutro, R., Mishkin, J.D., Trastuzumab-induced cardiomyopathy. J. Card. Fail. 14 (2008), 437–444.
22. Hazarika, M., Jiang, X., Liu, Q., Lee, S.L., Ramchandani, R., Garnett, C., Orr, M.S., Sridhara, R., Booth, B., Leighton, J.K., Timmer, W., Harapanhalli, R., Dagher, R., Justice, R., Pazdur, R., Tasigna for chronic and accelerated phase Philadelphia chromosome-positive chronic myelogenous leukemia resistant to or intolerant of imatinib. Clin. Cancer Res. 14 (2008), 5325–5331.
23. International Conference on Harmonization, 1997a. Guidance for Industry—Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals; ICH-S6.
24. International Conference on Harmonization, 1997b. Testing for Carcinogenicity of Pharmaceuticals; ICH-S1B.
25. International Conference on Harmonization, 2009. Addendum to ICH-S6: Preclinical Safety Evaluation of Biotechnology-Derived Pharmaceuticals (Draft Consensus Guideline; ICH-S6(R1); Step 2 version; Oct. 29, 2009.
26. Kane, R.C., Farrell, A.T., Saber, H., Tang, S., Williams, G., Jee, J.M., Liang, C., Booth, B., Chidambaram, N., Morse, D., Sridhara, R., Garvey, P., Justice, R., Pazdur, R., Sorafenib for the treatment of advanced renal cell carcinoma. Clin. Cancer Res. 12 (2006), 7271–7278.
27. Kerkelä, R., Grazette, L., Yacobi, R., Iliescu, C., Patten, R., Beahm, C., Walters, B., Shevtsov, S., Pesant, S., Clubb, F.J., Rosenzweig, A., Salomon, R.N., Van Etten, R.A., Alroy, J., Durand, J.B., Force, T., Cardiotoxicity of the cancer therapeutic agent imatinib mesylate. Nat. Med. 12 (2006), 908–916.
28. Klaassen, C.D., Doull, J., Evaluation of safety: toxicological evaluation. Klaassen, C.D., Doull, J., (eds.) Toxicology, 1980, MacMillan, New York.
29. Lehman, A.J., Control of toxicity in foods, drugs and cosmetics. Fed. Proc. 19:Suppl 4 (1960), 13–16.
30. Lehman, A.J., Fitzhugh, O.G., 100-Fold Margin of Safety. Q. Bull. – Assoc. Food Drug Off. U. S. 18 (1954), 33–35.
31. Lehman, A.J., Patterson, W.I., Davidow, B., Hagan, E.C., Woodard, G., Laug, E.P., Frawley, J.P., Fitzhugh, O.G., Bourke, A.R., Draize, J.H., Nelson, A.A., Vos, J.B., Procedures for the appraisal of the toxicity of chemicals in foods, drugs, and cosmetics. Food Drug Cosmet. Law J. 10 (1955), 679–748.
32. Lissauer, H., Zwei fälle von leucaemie. Berl. Klin. Wochenschr. 2 (1865), 403–404.
33. Lowe, M.C., Davis, R.D., The current toxicology protocol of the National Cancer Institute. Helmann, K., Carter, S.K., (eds.) Fundamentals of cancer chemotherapy, 1987, McGraw-Hill, New York, 228.
34. Mahalingam, D., Mita, A., Mita, M.M., Nawrocki, S.T., Giles, F.J., Targeted therapy for advanced non-small cell lung cancers: historical perspective, current practices, and future development. Curr. Probl. Cancer 33 (2009), 73–111.
35. Morris, P.G., King, F., Kennedy, J., Cytotoxic chemotherapy for pregnancy-associated breast cancer: single institution case series. J. Oncol. Pharm. Pract. 15:4 (2009), 241–247.
36. Newell, D.R., Burtles, S.S., Fox, B.W., Jodrell, D.I., Connors, T.A., Evaluation of rodent-only toxicology for early clinical trials with novel cancer therapeutics. Br. J. Cancer 81 (1999), 760–768.
37. 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., Concordance of the toxicity of pharmaceuticals in humans and in animals. Regul. Toxicol. Pharmacol. 32 (2000), 56–67.
38. Pavlovsky, C., Kantarjian, H., Cortes, J.E., First-line therapy for chronic myeloid leukemia: Past, present, and future. Am. J. Hematol. 84 (2009), 287–293.
39. Pinkel, D., The use of body surface area as a criterion of drug dosage in cancer chemotherapy. Cancer Res. 18 (1958), 853–856.
40. Podsypanina, K., Politi, K., Beverly, L.J., Varmus, H.E., Oncogene cooperation in tumor maintenance and tumor recurrence in mouse mammary tumors induced by Myc and mutant Kras. Proc. Natl. Acad. Sci. U. S. A. 105 (2008), 5242–5247.
41. Raimondi, A.R., Vitale-Cross, L., Amornphimoltham, P., Gutkind, J.S., Molinolo, A.A., Rapid development of salivary gland carcinomas upon conditional expression of K-ras driven by the cytokeratin 5 promoter. Am. J. Pathol. 168 (2006), 1654–1665.
42. Rock, E.P., Goodman, V., Jiang, J.X., Mahjoob, K., Verbois, S.L., Morse, D., Dagher, R., Justice, R., Pazdur, R., Food and Drug Administration drug approval summary: sunitinib malate for the treatment of gastrointestinal stromal tumor and advanced renal cell carcinoma. Oncologist 12 (2007), 107–113.
43. Ryan, Q., Ibrahim, A., Cohen, M.H., Johnson, J., Ko, C.W., Sridhara, R., Justice, R., Pazdur, R., FDA drug approval summary: lapatinib in combination with capecitabine for previously treated metastatic breast cancer that overexpresses HER-2. Oncologist 13 (2008), 1114–1119.
44. Vettorazzi, G., Safety factors and their application in the toxicological evaluation. The Evaluation of Toxicological Data for the Protection of Public Health, 1976, Pergamon, Oxford, 207–223.
45. Vettorazzi, G., Handbook of International Regulatory Food Toxicology, Vol. I: Evaluations. 1980, Spectrum, New York.
46. Visco, A.G., Meyer, L.C., Xi, S., Brown, C.G., One disease, two lives. Clin. J. Oncol. Nurs. 13 (2009), 426–432.
47. Vitale-Cross, L., Amornphimoltham, P., Fisher, G., Molinolo, A.A., Gutkind, J.S., Conditional expression of K-ras in an epithelial compartment that includes the stem cells is sufficient to promote squamous cell carcinogenesis. Cancer Res. 64 (2004), 8804–8807.
48. Zambelli, A., Prada, G.A., Fregoni, V., Ponchio, L., Sagrada, P., Pavesi, L., Erlotinib administration for advanced non-small cell lung cancer during the first 2 months of unrecognized pregnancy. Lung Cancer 60 (2008), 455–457.