The value, qualification, and regulatory use of surrogate end points in drug development

Clinical Pharmacology and Therapeutics

Volumn 86, Issue 1, 2009, Pages 32-43

  Lathia, C D ^a   Amakye, D ^b   Dai, W ^c   Girman, C ^d   Madani, S ^e   Mayne, J ^f   MacCarthy, P ^g   Pertel, P ^h   Seman, L ⁱ   Stoch, A ^d   Tarantino, P ^j   Webster, C ^k   Williams, S ^l   Wagner, J A ^d  

^a Bayer HealthCare Pharmaceuticals Inc   (United States)

^b ASTRAZENECA   (United Kingdom)

^c SANOFI   (France)

^d MERCK RESEARCH LABORATORIES   (United States)

^e NOVARTIS PHARMA AG   (Switzerland)

^f PFIZER INC   (United States)

^g Women's Healthcare   (United States)

^h CUBIST PHARMACEUTICALS INC   (United States)

ⁱ BOEHRINGER INGELHEIM PHARMACEUTICALS INC   (United States)

^j Sepracor   (United States)

^k Biogen Idec   (United States)

^l SomaLogic Inc   (United States)

more..

Author keywords

[No Author keywords available]

Indexed keywords

ACARBOSE; ALENDRONIC ACID; BIOLOGICAL MARKER; CARBOPLATIN; CISPLATIN; DIETHYLSTILBESTROL; DOXAZOSIN; ENCAINIDE; ETIDRONIC ACID; EZETIMIBE; FLECAINIDE; GEFITINIB; GEMCITABINE; GEMFIBROZIL; GLIBENCLAMIDE PLUS METFORMIN; GLIMEPIRIDE; GONADORELIN AGONIST; GOSERELIN; HEMOGLOBIN A1C; IBANDRONIC ACID; LEUPRORELIN; LOW DENSITY LIPOPROTEIN CHOLESTEROL; MORACIZINE; PACLITAXEL; PLACEBO; PRAVASTATIN; RISEDRONIC ACID; SIMVASTATIN; TESTOSTERONE; ZIDOVUDINE;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; ANTINEOPLASTIC ACTIVITY; BLOOD PRESSURE; BONE DENSITY; CANCER INHIBITION; CD4 LYMPHOCYTE COUNT; CLINICAL TRIAL; CORTICOSTEROID INDUCED OSTEOPOROSIS; DISEASE MARKER; DRUG APPROVAL; HEART ARRHYTHMIA; HEART INFARCTION; HEART VENTRICLE EXTRASYSTOLE; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; LUNG NON SMALL CELL CANCER; MEDICAL PRACTICE; NON INSULIN DEPENDENT DIABETES MELLITUS; OSTEOPOROSIS; OUTCOME ASSESSMENT; PRIORITY JOURNAL; PROGNOSIS; PROGRESSION FREE SURVIVAL; PROSTATE CANCER; REVIEW; RISK REDUCTION; STROKE; SUDDEN DEATH; TREATMENT OUTCOME; VERTEBRA FRACTURE; VIRUS LOAD;

ANIMALS; BIOLOGICAL MARKERS; DRUG DISCOVERY; HUMANS; UNITED STATES; UNITED STATES FOOD AND DRUG ADMINISTRATION;

EID: 67650604373     PISSN: 00099236     EISSN: 15326535     Source Type: Journal    
DOI: 10.1038/clpt.2009.69     Document Type: Review

References (51)

1. Lee, J.W. et al. Fit-for-purpose method development and validation for successful biomarker measurement. Pharm. Res. 23, 312-328 (2006).
2. Dybul, M., Connors, M. & Fauci, A. The immunology of human immunodefciency virus infection. In Principles and Practice of Infectious Diseases 6th edn. (eds. Mandell, G.L., Douglas, R.G. & Bennett, J.E.) 1527-1546 (Elsevier, Philadelphia, PA, 2005).
3. Panel on Clinical Practices for Treatment of HIV Infection. Guidelines for the Use of Antiretroviral Agents in HIV-1-Infected Adults and Adolescents. Department of Health and Human Services (DHHS), 2005.
4. Mellors, J.W. et al. Plasma viral load and CD4+ lymphocytes as prognostic markers of HIV-1 infection. Ann. Intern. Med. 126, 946-954 (1997).
5. Hill, A.M., DeMasi, R. & Dawson, D. Meta-analysis of antiretroviral efects on HIV-1 RNA, CD4 cell count and progression to AIDS or death. Antivir. Ther. (Lond.) 3, 139-145 (1998).
6. O'Brien, W.A., Hartigan, P.M., Daar, E.S., Simberkof, M.S. & Hamilton, J.D. Changes in plasma HIV RNA levels and CD4+ lymphocyte counts predict both response to antiretroviral therapy and therapeutic failure. VA Cooperative Study Group on AIDS. Ann. Intern. Med. 126, 939-945 (1997).
7. Greub, G. et al. Intermittent and sustained low-level HIV viral rebound in patients receiving potent antiretroviral therapy. AIDS 16, 1967-1969 (2002).
8. Havlir, D.V. et al. Prevalence and predictive value of intermittent viremia with combination hiv therapy. JAMA 286, 171-179 (2001).
9. US Food and Drug Administration. Center for Drug Evaluation and Research. Guidance for Industry Antiretroviral Drugs Using Plasma HIV RNA Measurements - Clinical Considerations for Accelerated and Traditional Approval. (2002).
10. Palella, F.J. Jr. et al. Declining morbidity and mortality among patients with advanced human immunodefciency virus infection. N. Engl. J. Med. 338, 853-860 (1998).
11. Johnson, J.R., Williams, G. & Pazdur, R. End points and United States Food and Drug Administration approval of oncology drugs. J. Clin. Oncol. 21:1404-1411 (2003).
12. Pignatti, F. et al. Clinical trials for registration in the European Union: the EMEA 5-year experience in oncology. Crit. Rev. Oncol. Hematol. 42, 123-135 (2002).
13. Grundy, S.M. Cholesterol-Lowering Therapy: Evaluation of Clinical Trial Evidence (Marcel Dekker, NY, 2000).
14. Keys, A. Coronary heart disease in seven countries. Circulation 41 (suppl. I), I1-I211 (1970).
15. The multiple risk factor intervention trial (MRFIT). A national study of primary prevention of coronary heart disease. JAMA 235, 825-827 (1976).
16. Kannel, W.B. Contributions of the Framingham study to the conquest of artery disease. Am. J. Cardiol. 62, 1109-1112 (1988).
17. Randomized trial of cholesterol lowering in 4444 patients with coronary heart disease: the Scandinavian Simvastatin Survival Study (4S). Lancet 344, 1383-1389 (1994).
18. Heart Protection Study Collaborative Group. MRC/BHF Heart Protection Study of cholesterol lowering with simvastatin in 20,536 high-risk individuals: a randomised placebo-controlled trial. Lancet 360, 7-22 (2002).
19. Kastelein, J.J. et al. Simvastatin with or without ezetimibe in familial hypercholesteremia. N. Engl. J. Med. 358, 1431-1443 (2008).
20. Brown, B.G. & Taylor, A.J. Does ENHANCE diminish confdence in lowering LDL or in ezetimibe? N. Engl. J. Med. 358, 1504-1507 (2008).
21. The efect of intensive treatment of diabetes on the development and progression of long-term complications in insulin-dependent diabetes mellitus. The Diabetes Control and Complications Trial Research Group. N. Engl. J. Med. 329, 977-986 (1993).
22. Holman, R.R., Paul, S.K., Bethel, M.A., Matthews, D.R. & Neil, H.A. 10-year follow-up of intensive glucose control in type 2 diabetes. N. Engl. J. Med. 359, 1577-1589 (2008).
23. Feinböck, C. et al. Prospective multicentre trial comparing the efcacy of, and compliance with, glimepiride or acarbose treatment in patients with type 2 diabetes not controlled with diet alone. Diabetes Nutr. Metab. 16, 214-221 (2003).
24. Dailey, G.E. Glyburide/metformin tablets: a new therapeutic option for the management of Type 2 diabetes. Expert Opin. Pharmacother. 4, 1417-1430 (2003).
25. The Action to Control Cardiovascular Risk in Diabetes Study Group. Efects of intensive glucose lowering in type 2 diabetes. N. Engl. J. Med. 358, 2545-2559 (2008).
26. The ADVANCE Collaborative Group. Intensive blood glucose control and vascular outcomes in patients with type II diabetes. N. Eng. J. Med. 358, 2560-2572 (2008).
27. Lewington, S., Clarke, R., Qizilbash, N., Peto, R., Collins, R. ; Prospective Studies Collaboration. Age-specifc relevance of usual blood pressure to vascular mortality: a meta-analysis of individual data for one million adults in 61 prospective studies. Lancet 360, 1903-1913 (2002).
28. Turnbull, F.; Blood Pressure Lowering Treatment Trialists' Collaboration. Efects of diferent blood-pressure-lowering regimens on major cardiovascular events: results of prospectively-designed overviews of randomized trials. Lancet 362, 1527-1535 (2003).
29. Hamilton, M., Thompson, E.M. & Wisniewski, T.K. The role of blood-pressure control in preventing complications of hypertension. Lancet 1, 235-238 (1964).
30. Efects of treatment on morbidity in hypertension. Results in patients with diastolic blood pressures averaging 115 through 129 mm Hg. JAMA 202, 1028-1034 (1967).
31. ALLHAT Ofcers and Coordinators for the ALLHAT Collaborative Research Group. The Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial. Major outcomes in high-risk hypertensive patients randomized to angiotensin-converting enzyme inhibitor or calcium channel blocker vs diuretic: the Antihypertensive and Lipid-Lowering Treatment to Prevent Heart Attack Trial (ALLHAT). JAMA 288, 2981-2997 (2002).
32. Huggins, C. & Hodges, C.V. Studies on prostate cancer. I. The efect of castration, of estrogen and of androgen injection on serum phosphatases in metastatic carcinoma of the prostate. Cancer Res. 1, 293-297 (1941).
33. Scher, H.I., Buchanan, G., Gerald, W., Butler, L.M. & Tilley, W.D. Targeting the androgen receptor: improving outcomes for castration-resistant prostate cancer. Endocr. Relat. Cancer 11, 459-476 (2004).
34. Catalona, W.J. Management of cancer of the prostate. N. Engl. J. Med. 331, 996-1004 (1994).
35. Conn, P.M. & Crowley, W.F. Jr. Gonadotropin-releasing hormone and its analogues. N. Engl. J. Med. 324, 93-103 (1991).
36. Leuprolide versus diethylstilbestrol for metastatic prostate cancer. The Leuprolide Study Group. N. Engl. J. Med. 311, 1281-1286 (1984).
37. Beacock, C.J. et al. The treatment of metastatic prostatic cancer with the slow release LH-RH analogue Zoladex ICI 118630. Br. J. Urol. 59, 436-442 (1987).
38. Lesko, L.J. & Atkinson, A.J. Use of biomarkers and surrogate endpoints in drug development and regulatory decision making: criteria, validation, strategies. Annu. Rev. Pharmacol. Toxicol. 41, 347-366 (2001).
39. Ross, P.D., Davis, J.W., Epstein, R.S. & Wasnich, R.D. Pre-existing fractures and bone mass predict vertebral fracture incidence in women. Ann. Intern. Med. 114, 919-923 (1991).
40. Cummings, S.R. et al. Bone density at various sites for prediction of hip fractures. Lancet 341, 72-75 (1993).
41. Black, D.M. et al. Randomized trial of efect of alendronate on risk of fracture in women with existing vertebral fractures. Lancet 348, 1535-1541 (1996).
42. US Food and Drug Administration. Guidelines for Clinical Evaluation of Agents Used in the Treatment and Prevention of Osteoporosis. 1985.
43. US Food and Drug Administration. Guidelines for Preclinical and Clinical Evaluation of Agents in the Prevention or Treatment of Postmenopausal Osteoporosis. US Food and Drug Administration, Rockville, MD, 1994.
44. Riggs, B.L., Seeman, E., Hodgson, S.F., Taves, D.R. & O'Fallon, W.M. Efect of the fuoride/calcium regimen on vertebral fracture occurrence in postmenopausal osteoporosis. Comparison with conventional therapy. N. Engl. J. Med. 306, 446-450 (1982).
45. Kleerekoper, M. et al. A randomized trial of sodium fuoride as a treatment of postmenopausal osteoporosis. Osteoporos. Int. 3, 155-161 (1991).
46. Echt, D.S. et al. Mortality and morbidity in patients receiving encainide, fecainide, or placebo. The Cardiac Arrhythmia Suppression Trial. N. Engl. J. Med. 324, 781-788 (1991).
47. Hulley, S.B. & Grady, D. The WHI estrogen-alone trail - do things look any better? JAMA 291, 1769-1771 (2004).
48. Prentice, R.L. Surrogate endpoints in clinical trials: defnition and operational criteria. Stat. Med. 8, 431-440 (1989).
49. US Food and Drug Administration. Innovation or Stagnation? Challenge and Opportunity on the Critical Path to New Medical Products. (2004).
50. Williams, S.A., Slavin, D.E., Wagner, J.A. & Webster, C.J. A cost efectiveness approach to the qualifcation of biomarkers and surrogate endpoints. Nat. Rev. Drug Discov. 5, 897-902 (2006).
51. Altar, C.A. et al. A prototypical process for creating evidentiary standards for biomarkers and diagnostics. Clin. Pharmacol. Ther. 83, 368-371 (2008).