In the end what matters most? A review of clinical endpoints in advanced breast cancer

Oncologist

Volumn 16, Issue 1, 2011, Pages 25-35

  Verma, Sunil ^a   McLeod, Deanna ^b   Batist, Gerald ^c   Robidoux, André ^d   Martins, Ilídio R S ^b   Mackey, John R ^e  

^a UNIVERSITY OF TORONTO   (Canada)

^b Kaleidoscope Strategic   (Canada)

^c MCGILL UNIVERSITY   (Canada)

^d CENTRE HOSPITALIER DE L UNIVERSITÉ DE MONTRÉAL   (Canada)

^e UNIVERSITY OF ALBERTA   (Canada)

Author keywords

Breast cancer; Clinical trial design; FDA; Metastatic; Regulatory approval; Surrogate endpoint

Indexed keywords

ANTHRACYCLINE DERIVATIVE; BEVACIZUMAB; CAPECITABINE; CYCLOPHOSPHAMIDE; DOCETAXEL; DOXORUBICIN; EPIDERMAL GROWTH FACTOR RECEPTOR 2; EPIRUBICIN; FLUOROURACIL; GEMCITABINE; IXABEPILONE; LAPATINIB; PACLITAXEL; TAXANE DERIVATIVE; TRASTUZUMAB;

BREAST CANCER; CANCER CHEMOTHERAPY; CANCER HORMONE THERAPY; CANCER SURVIVAL; DRUG APPROVAL; DRUG EFFICACY; FOOD AND DRUG ADMINISTRATION; HUMAN; METHODOLOGY; MOLECULARLY TARGETED THERAPY; OVERALL SURVIVAL; PHASE 3 CLINICAL TRIAL (TOPIC); PRIORITY JOURNAL; PROGRESSION FREE SURVIVAL; RANDOMIZED CONTROLLED TRIAL (TOPIC); REVIEW; STANDARD; SURVIVAL RATE; UNITED STATES;

BREAST NEOPLASMS; CLINICAL TRIALS AS TOPIC; DISEASE-FREE SURVIVAL; ENDPOINT DETERMINATION; FEMALE; HUMANS; NEOPLASM METASTASIS;

EID: 79952602636     PISSN: 10837159     EISSN: 1549490X     Source Type: Journal    
DOI: 10.1634/theoncologist.2010-0278     Document Type: Review

References (41)

1. U.S. Department of Health and Human Services, U.S. Food and Drug Administration. Guidance for Industry: Clinical Trial Endpoints for the Approval of Cancer Drugs and Biologics. Available at http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm071590.pdf, accessed April 20, 2010.
2. Pazdur R. Endpoints for assessing drug activity in clinical trials. The Oncologist 2008;13(suppl 2):19-21.
3. Oncologic Drugs Advisory Committee. Transcript From Meeting on December 5, 2007. CDER Meeting Documents 2007. Available at http://www.fda.gov/OHRMS/DOCKETS/AC/cder07.htm#OncologicDrugs,accessed April 20, 2010.
4. Hackshaw A, Knight A, Barrett-Lee P et al. Surrogate markers and survival in women receiving first-line combination anthracycline chemotherapy for advanced breast cancer. Br J Cancer 2005;93:1215-1221.
5. Miksad RA, Zietemann V, Gothe R et al. Progression-free survival as a surrogate endpoint in advanced breast cancer. Int J Technol Assess Health Care 2008;24:371-383.
6. Sherrill B, Amonkar M, Wu Y et al. Relationship between effects on timeto- disease progression and overall survival in studies of metastatic breast cancer. Br J Cancer 2008;99:1572-1578.
7. Burzykowski T, Buyse M, Piccart-Gebhart MJ et al. Evaluation of tumor response, disease control, progression-free survival, and time to progression as potential surrogate end points in metastatic breast cancer. J Clin Oncol 2008;26:1987-1992.
8. Saad ED, Katz A. Progression-free survival and time to progression as primary end points in advanced breast cancer: Often used, sometimes loosely defined. Ann Oncol 2009;20:460-464.
9. Saad ED, Katz A, Hoff PM et al. Progression-free survival as surrogate and as true end point: Insights from the breast and colorectal cancer literature. Ann Oncol 2010;21:7-12.
10. Sheik-Yousouf A, Gandhi S, Dukhovny S et al. A comparison of physician and patient perceptions of clinically important endpoints in the treatment of metastatic breast cancer (MBC). Eur J Cancer Suppl 2010;8:1.
11. Sledge GW. The measure of our success. Clin Breast Cancer 2007;7:1.
12. Chakravarty A, Sridhara R. Use of progression-free survival as a surrogate marker in oncology trials: Some regulatory issues. Stat Methods Med Res 2008;17:515-518.
13. Freidlin B, Korn EL, Hunsberger S et al. Proposal for the use of progression-free survival in unblinded randomized trials. J Clin Oncol 2007;25: 2122-2126.
14. Johnson JR, Williams G, Pazdur R. End points and United States Food and Drug Administration approval of oncology drugs. J Clin Oncol 2003;21: 1404-1411.
15. Dagher R, Johnson J, Williams G et al. Accelerated approval of oncology products: A decade of experience. J Natl Cancer Inst 2004;96:1500-1509.
16. Sridhara R, Johnson JR, Justice R et al. Review of oncology and hematology drug product approvals at the US Food and Drug Administration between July 2005 and December 2007. J Natl Cancer Inst 2010;102:230-243.
17. U.S. Food and Drug Administration: Department of Health and Human Services. 21 Code of Federal Regulations, Part 314.200. 2009 (last revision).
18. Richey EA, Lyons EA, Nebeker JR et al. Accelerated approval of cancer drugs: Improved access to therapeutic breakthroughs or early release of unsafe and ineffective drugs? J Clin Oncol 2009;27:4398-4405.
19. Clinton B, Gore A. Reinventing the Regulation of Cancer Drugs, National Performance Review 1996. Available at http://www.fda.gov/ohrms/dockets/ac/05/briefing/2005-4191B1_01_03-Reinvent-Cancer-Drugs.pdf, accessed April 20, 2010.
20. U.S. Senate and House of Representatives. Food and Drug Administration Modernization Act of 1997. Available at http://www.fda.gov/downloads/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/FDAMA/FullTextofFDAMAlaw/UCM089145.pdf, accessed April 20, 2010.
21. Roberts TG Jr, Chabner BA. Beyond fast track for drug approvals. N Engl J Med 2004;351:501-505.
22. Oncologic Drugs Advisory Committee. Transcript From Meeting on June 7, 1999. CDER Meeting Documents 1999. Available at http://www.fda.gov/ohrms/dockets/ac/cder99t.htm#Oncologic%20Drugs%20Advisory%20Committee, accessed April 20, 2010.
23. Oncologic Drugs Advisory Committee. Transcript From Meeting on November 8, 2005. CDER Meeting Documents 2005. Available at http://www.fda.gov/ohrms/dockets/ac/cder05.html#OncologicDrugs, accessed April 20, 2010.
24. Takeda A, Loveman E, Harris P et al. Time to full publication of studies of anti-cancer medicines for breast cancer and the potential for publication bias: A short systematic review. Health Technol Assess 2008;12:3, 9-10, 1-46.
25. Creagan ET, Green SJ, Ahmann DL et al. A phase III clinical trial comparing the combination cyclophosphamide, adriamycin, cisplatin with cyclophosphamide, 5-fluorouracil, prednisone in patients with advanced breast cancer. J Clin Oncol 1984;2:1260-1265.
26. Pannuti F, Iafelice G, Martoni A et al. A new six-drug antiblastic regimen (R 14) at low doses (micropolychemotherapy) compared to CMF in the treatment of metastatic breast cancer: Phase III study. Chemioterapia 1984; 3:216-219.
27. Albain KS, Nag SM, Calderillo-Ruiz G et al. Gemcitabine plus paclitaxel versus paclitaxel monotherapy in patients with metastatic breast cancer and prior anthracycline treatment. J Clin Oncol 2008;26:3950-3957.
28. U.S. Food and Drug Administration: Center for Drug Evaluation and Research. Drugs@FDA: FDA Approved Drug Products. October 27, 2009. Available at http://www.accessdata.fda.gov/scripts/cder/drugsatfda/, accessed October 27, 2009.
29. Cortazar P, Johnson JR, Justice R et al. Metastatic breast cancer (MBC): FDA approval overview. J Clin Oncol 2008;26(15 suppl):1013.
30. Saad ED, Katz A, Buyse M. Overall survival and post-progression survival in advanced breast cancer: A review of recent randomized clinical trials. J Clin Oncol 2010;28:1958-1962.
31. Wilcken N, Dear R. Chemotherapy in metastatic breast cancer: A summary of all randomised trials reported 2000-2007. Eur J Cancer 2008;44:2218-2225.
32. Broglio KR, Berry DA. Detecting an overall survival benefit that is derived from progression-free survival. J Natl Cancer Inst 2009;101:1642-1649.
33. Greenberg PA, Hortobagyi GN, Smith TL et al. Long-term follow-up of patients with complete remission following combination chemotherapy for metastatic breast cancer. J Clin Oncol 1996;14:2197-2205.
34. Arveux P, Grosclaude P, Reyrat E et al. Breast cancer survival in France: A relative survival analysis based on 68,449 cases treated in the 20 French comprehensive cancer centers between 1980 and 1999. Proc Am Soc Clin Oncol 2003;22:Abstract 3437.
35. Giordano SH, Buzdar AU, Smith TL et al. Is breast cancer survival improving? Cancer 2004;100:44-52.
36. Chia SK, Speers CH, D'yachkova Y et al. The impact of new chemotherapeutic and hormone agents on survival in a population-based cohort of women with metastatic breast cancer. Cancer 2007;110:973-979.
37. Oncologic Drugs Advisory Committee. Transcript From Meeting on July 20, 2010. CDER Meeting Documents 2010. Available at http://www.fda.gov/AdvisoryCommittees/CommitteesMeetingMaterials/Drugs/OncologicDrugsAdvisoryCommittee/ucm195226.htm, accessed 19 August, 2010.
38. Bokemeyer C, Bondarenko I, Makhson A et al. Fluorouracil, leucovorin, and oxaliplatin with and without cetuximab in the first-line treatment of metastatic colorectal cancer. J Clin Oncol 2009;27:663-671.
39. Tol J, Koopman M, Cats A et al. Chemotherapy, bevacizumab, and cetuximab in metastatic colorectal cancer. N Engl J Med 2009;360:563-572.
40. Van Cutsem E, Köhne CH, Hitre E et al. Cetuximab and chemotherapy as initial treatment for metastatic colorectal cancer. N Engl J Med 2009;360: 1408-1417.
41. Amado RG, Wolf M, Peeters M et al. Wild-type KRAS is required for panitumumab efficacy in patients with metastatic colorectal cancer. J Clin Oncol 2008;26:1626-1634.