Modeling hard clinical end-point data in economic analyses

Journal of Medical Economics

Volumn 16, Issue 11, 2013, Pages 1327-1343

  Kansal, Anuraag R ^a   Zheng, Ying ^a   Palencia, Roberto ^b   Ruffolo, Antonio ^b   Hass, Bastian ^b   Sorensen, Sonja V ^a  

^a EVIDERA   (United States)

^b BOEHRINGER INGELHEIM PHARMA GMBH AND CO KG   (Germany)

Author keywords

Cost effectiveness; Hard clinical end point data; Modeling approaches

Indexed keywords

ANTICOAGULANT AGENT;

ARTICLE; BREAST CANCER; CARDIOVASCULAR DISEASE; CEREBROVASCULAR ACCIDENT; CHRONIC OBSTRUCTIVE LUNG DISEASE; CHRONIC RESPIRATORY TRACT DISEASE; CLINICAL TRIAL (TOPIC); COST EFFECTIVENESS ANALYSIS; DECISION TREE; HEALTH ECONOMICS; HEALTH STATUS; HEART FAILURE; HIDDEN MARKOV MODEL; HUMAN; HYBRID; LOWER RESPIRATORY TRACT; LUNG CANCER; MODEL; NEOPLASM; NON INSULIN DEPENDENT DIABETES MELLITUS; PRIMARY PREVENTION; RANDOMIZED CONTROLLED TRIAL (TOPIC); RISK ASSESSMENT; SECONDARY PREVENTION; SIMULATION;

CARDIOVASCULAR DISEASES; COST-BENEFIT ANALYSIS; DIABETES MELLITUS, TYPE 2; HUMANS; MARKOV CHAINS; MODELS, ECONOMIC; NEOPLASMS; PRIMARY PREVENTION; RESEARCH DESIGN;

EID: 84886446981     PISSN: 13696998     EISSN: 1941837X     Source Type: Journal    
DOI: 10.3111/13696998.2013.838960     Document Type: Article

References (75)

1. Morganti A. Randomized clinical trials on surrogate end points: are they useful for evaluating cardiovascular and renal disease protection in hypertension? The case for yes. J Am Soc Nephrol 2006;17(4 Suppl 2):S141-4
2. Yudkin JS, Eggleston EM. 'Hard, ' 'soft' and 'surrogate' endpoints in diabetes. J Epidemiol Community Health 2013;67:295-7
3. D'Agostino Sr RB, Massaro JM. New developments in medical clinical trials. J Dent Res 2004;83(Suppl 1):C18-24
4. US Department of Health and Human Services. Guidance for industry: diabetes mellitus-Evaluating cardiovascular risk in new antidiabetic therapies to treat type 2 diabetes. Silver Spring, MD: US Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), 2008
5. D'Agostino Jr RB. Debate: the slippery slope of surrogate outcomes. Curr Control Trials Cardiovasc Med 2000;1:76-8
6. Hulley S, Grady D, Bush T, et al. Randomized trial of estrogen plus progestin for secondary prevention of coronary heart disease in postmenopausal women. Heart and Estrogen/progestin Replacement Study (HERS) Research Group. JAMA 1998;280:605-13
7. Kochanek KD, Xu JQ, Murphy SL, et al. Deaths: Preliminary data for 2009. National Vital Statistics Reports; Hyattsville, MD: National Center for Health Statistics 2011;59:1-51
8. Beck JR, Pauker SG. The Markov process in medical prognosis. Med Decis Making 1983;3:419-58
9. Caro JJ. Pharmacoeconomic analyses using discrete event simulation. Pharmacoeconomics 2005;23:323-32
10. Caro JJ, Moller J, Getsios D. Discrete event simulation: the preferred technique for health economic evaluations? Value Health 2010;13:1056-60
11. Carson P, Tognoni G, Cohn JN. Effect of Valsartan on hospitalization: results from Val-HeFT. J Card Fail 2003;9:164-71
12. Cohn JN, Tognoni G. A randomized trial of the angiotensin-receptor blocker valsartan in chronic heart failure. N Engl J Med 2001;345:1667-75
13. Cowie MR, Cure S, Bianic F, et al. Cost-effectiveness of highly purified omega-3 polyunsaturated fatty acid ethyl esters in the treatment of chronic heart failure: results of Markov modelling in a UK setting. Eur J Heart Fail 2011;13:681-9
14. de Pouvourville G, Solesse A, Beillat M. Cost-effectiveness analysis of aldosterone blockade with eplerenone in patients with heart failure after acute myocardial infarction in the French context: the EPHESUS study. Arch Cardiovasc Dis 2008;101:515-21
15. Flather MD, Shibata MC, Coats AJ, et al. Randomized trial to determine the effect of nebivolol on mortality and cardiovascular hospital admission in elderly patients with heart failure (SENIORS). Eur Heart J 2005;26:215-25
16. Khush KK, Waters DD, Bittner V, et al. Effect of high-dose atorvastatin on hospitalizations for heart failure: subgroup analysis of the Treating to New Targets (TNT) study. Circulation 2007;115:576-83
17. Pitt B, Remme W, Zannad F, et al. Eplerenone, a selective aldosterone blocker, in patients with left ventricular dysfunction after myocardial infarction. N Engl J Med 2003;348:1309-21
18. Pradelli L, lannazzo S, Zaniolo O. The cost effectiveness and cost utility of valsartan in chronic heart failure therapy in Italy: a probabilistic markov model. Am J Cardiovasc Drugs 2009;9:383-92
19. Rosen VM, Taylor DC, Parekh H, et al. Cost effectiveness of intensive lipid-lowering treatment for patients with congestive heart failure and coronary heart disease in the US. Pharmacoeconomics 2010;28:47-60
20. Tavazzi L, Maggioni AP, Marchioli R, et al. Effect of n-3 polyunsaturated fatty acids in patients with chronic heart failure (the GISSI-HF trial): a randomised, double-blind, placebo-controlled trial. Lancet 2008;372:1223-30
21. Yao G, Freemantle N, Flather M, et al. Long-term cost-effectiveness analysis of nebivolol compared with standard care in elderly patients with heart failure: an individual patient-based simulation model. Pharmacoeconomics 2008;26:879-89
22. Amarenco P, Bogousslavsky J, Callahan 3rd A, et al. High-dose atorvastatin after stroke or transient ischemic attack. N Engl J Med 2006;355:549-59
23. Connolly SJ, Ezekowitz MD, Yusuf S, et al. Dabigatran versus warfarin in patients with atrial fibrillation. N Engl J Med 2009;361:1139-51
24. Freeman JV, Zhu RP, Owens DK, et al. Cost-effectiveness of dabigatran compared with warfarin for stroke prevention in atrial fibrillation. Ann Intern Med 2011;154:1-11
25. Kongnakorn T, Ward A, Roberts CS, et al. Economic evaluation of atorvastatin for prevention of recurrent stroke based on the SPARCL trial. Value Health 2009;12:880-7
26. Pink J, Lane S, Pirmohamed M, et al. Dabigatran etexilate versus warfarin in management of non-valvular atrial fibrillation in UK context: quantitative benefit-harm and economic analyses. BMJ 2011;343:d6333. doi: 10.1136/bmj.d6333
27. Shah SV, Gage BF. Cost-effectiveness of dabigatran for stroke prophylaxis in atrial fibrillation. Circulation 2011;123:2562-70
28. Sorensen SV, Kansal AR, Connolly S, et al. Cost-effectiveness of dabigatran etexilate for the prevention of stroke and systemic embolism in atrial fibrillation: a Canadian payer perspective. Thromb Haemost 2011;105:908-19
29. Anis AH, Sun H, Singh S, et al. A cost-utility analysis of losartan versus atenolol in the treatment of hypertension with left ventricular hypertrophy. Pharmacoeconomics 2006;24:387-400
30. Colhoun HM, Betteridge DJ, Durrington PN, et al. Primary prevention of cardiovascular disease with atorvastatin in type 2 diabetes in the Collaborative Atorvastatin Diabetes Study (CARDS): multicentre randomised placebo-controlled trial. Lancet 2004;364:685-96
31. Dahlof B, Devereux RB, Kjeldsen SE, et al. Cardiovascular morbidity and mortality in the Losartan Intervention For Endpoint reduction in hypertension study (LIFE): a randomised trial against atenolol. Lancet 2002;359:995-1003
32. Lindgren P, Buxton M, Kahan T, et al. The lifetime cost effectiveness of amlodipine-based therapy plus atorvastatin compared with atenolol plus atorvastatin, amlodipine-based therapy alone and atenolol-based therapy alone: results from ASCOT1. Pharmacoeconomics 2009;27:221-30
33. Ohsfeldt RL, Gandhi SK, Smolen LJ, et al. Cost effectiveness of rosuvastatin in patients at risk of cardiovascular disease based on findings from the JUPITER trial. J Med Econ 2010;13:428-37
34. Ramsey SD, Clarke LD, Roberts CS, et al. An economic evaluation of atorvastatin for primary prevention of cardiovascular events in type 2 diabetes. Pharmacoeconomics 2008;26:329-39
35. Ridker PM, Danielson E, Fonseca FA, et al. Rosuvastatin to prevent vascular events in men and women with elevated C-reactive protein. N Engl J Med 2008;359:2195-207
36. Sever PS, Dahlof B, Poulter NR, et al. Prevention of coronary and stroke events with atorvastatin in hypertensive patients who have average or lower-than-average cholesterol concentrations, in the Anglo-Scandinavian Cardiac Outcomes Trial-Lipid Lowering Arm (ASCOT-LLA): a multicentre randomised controlled trial. Lancet 2003;361:1149-58
37. Slejko JF, Page 2nd RL, Sullivan PW. Cost-effectiveness of statin therapy for vascular event prevention in adults with elevated C-reactive protein: implications of JUPITER. Curr Med Res Opin 2010;26:2485-97
38. CAPRIE Steering Committee. A randomised, blinded, trial of clopidogrel versus aspirin in patients at risk of ischaemic events (CAPRIE). Lancet 1996;348:1329-39
39. GISSI-Prevenzione Investigators. Dietary supplementation with n-3 polyunsaturated fatty acids and vitamin E after myocardial infarction: results of the GISSI-Prevenzione trial. Gruppo Italiano per lo Studio della Soprawivenza neH'Infarto miocardico. Lancet 1999;354:447-55
40. 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 2002;360:7-22
41. Berg J, Lindgren P, Spiesser J, et al. Cost-effectiveness of clopidogrel in myocardial infarction with ST-segment elevation: a European model based on the CLARITY and COMMIT trials. Clin Ther 2007;29:1184-202
42. Chen ZM, Jiang LX, Chen YP, et al. Addition of clopidogrel to aspirin in 45, 852 patients with acute myocardial infarction: randomised placebo-controlled trial. Lancet 2005;366:1607-21
43. Crespin DJ, Federspiel JJ, Biddle AK, et al. Ticagrelor versus genotype-driven antiplatelet therapy for secondary prevention after acute coronary syndrome: a cost-effectiveness analysis. Value Health 2011;14:483-91
44. Greenhalgh J, Bagust A, Boland A, et al. Clopidogrel and modified-release dipyridamole for the prevention of occlusive vascular events (review of Technology Appraisal No. 90): a systematic review and economic analysis. Health Technol Assess 2011;15:1-178
45. LaRosa JC, Grundy SM, Waters DD, et al. Intensive lipid lowering with atorvastatin in patients with stable coronary disease. N Engl J Med 2005;352:1425-35
46. Lindgren P, Graff J, Olsson AG, et al. Cost-effectiveness of high-dose atorvastatin compared with regular dose simvastatin. Eur Heart J 2007;28:1448-53
47. Logman JF, Heeg BM, Herlitz J, et al. Costs and consequences of clopidogrel versus aspirin for secondary prevention of ischaemic events in (high-risk) atherosclerotic patients in Sweden: a lifetime model based on the CAPRIE trial and high-risk CAPRIE subpopulations. Appl Health Econ Health Policy 2010;8:251-65
48. Mihaylova B, Briggs A, Armitage J, et al. Lifetime cost effectiveness of simvastatin in a range of risk groups and age groups derived from a randomised trial of 20, 536 people. BMJ 2006;333:1145
49. Pedersen TR, Faergeman O, Kastelein JJ, et al. High-dose atorvastatin vs usual-dose simvastatin for secondary prevention after myocardial infarction: the IDEAL study: a randomized controlled trial. JAMA 2005;294: 2437-45
50. Quilici S, Martin M, McGuire A, et al. A cost-effectiveness analysis of n-3 PUFA (Omacor) treatment in post-Mi patients. Int J Clin Pract 2006;60:922-32
51. Sabatine MS, Cannon CP, Gibson CM, et al. Addition of clopidogrel to aspirin and fibrinolytic therapy for myocardial infarction with ST-segment elevation. N Engl J Med 2005;352:1179-89
52. Sacco RL, Diener HC, Yusuf S, et al. Aspirin and extended-release dipyridamole versus clopidogrel for recurrent stroke. N Engl J Med 2008;359:1238-51
53. Taylor DC, Pandya A, Thompson D, et al. Cost-effectiveness of intensive atorvastatin therapy in secondary cardiovascular prevention in the United Kingdom, Spain, and Germany, based on the Treating to New Targets study. Eur J Health Econ 2009;10:255-65
54. Wallentin L, Becker RC, Budaj A, et al. Ticagrelor versus clopidogrel in patients with acute coronary syndromes. N Engl J Med 2009;361:1045-57
55. Yusuf S, Zhao F, Mehta SR, et al. Effects of clopidogrel in addition to aspirin in patients with acute coronary syndromes without ST-segment elevation. N Engl J Med 2001;345:494-502
56. Asukai Y, Valladares A, Camps C, et al. Cost-effectiveness analysis of peme-trexed versus docetaxel in the second-line treatment of non-small cell lung cancer in Spain: results for the non-squamous histology population. BMC Cancer 2010;10:26
57. Ciuleanu T, Brodowicz T, Zielinski C, et al. Maintenance pemetrexed plus best supportive care versus placebo plus best supportive care for non-small-cell lung cancer: a randomised, double-blind, phase 3 study. Lancet 2009;374:1432-40
58. Giuliani G, Grossi F, de Marinis F, et al. Cost-effectiveness analysis of bev-acizumab versus pemetrexed for advanced non-squamous NSCLC in Italy. Lung Cancer 2010;69(Suppl 1):S11-17
59. Goulart B, Ramsey S. A trial-based assessment of the cost-utility of bevaci-zumab and chemotherapy versus chemotherapy alone for advanced non-small cell lung cancer. Value Health 2011;14:836-45
60. Klein R, Wielage R, Muehlenbein C, et al. Cost-effectiveness of pemetrexed as first-line maintenance therapy for advanced nonsquamous non-small cell lung cancer. J Thorac Oncol 2010;5:1263-72
61. Reck M, von Pawel J, Zatloukal P, et al. Phase III trial of cisplatin plus gemcitabine with either placebo or bevacizumab as first-line therapy for nonsquamous non-small-cell lung cancer: AVAil. J Clin Oncol 2009;27: 1227-34
62. Sandler A, Gray R, Perry MC, et al. Paclitaxel-carboplatin alone or with bevacizumab for non-small-cell lung cancer. N Engl J Med 2006;355: 2542-50
63. Scagliotti GV, Parikh P, von Pawel J, et al. Phase III study comparing cisplatin plus gemcitabine with cisplatin plus pemetrexed in chemotherapy-naive patients with advanced-stage non-small-cell lung cancer. J Clin Oncol 2008;26:3543-51
64. Benedict A, Cameron DA, Corson H, et al. An economic evaluation of docetaxel and paclitaxel regimens in metastatic breast cancer in the UK. Pharmacoeconomics 2009;27:847-59
65. Geyer CE, Forster J, Lindquist D, et al. Lapatinib plus capecitabine for HER2-positive advanced breast cancer. N Engl J Med 2006;355:2733-43
66. Gray R, Bhattacharya S, Bowden C, et al. Independent review of E2100: a phase III trial of bevacizumab plus paclitaxel versus paclitaxel in women with metastatic breast cancer. J Clin Oncol 2009;27:4966-72
67. Jones SE, Erban J, Overmoyer B, et al. Randomized phase III study of docetaxel compared with paclitaxel in metastatic breast cancer. J Clin Oncol 2005;23:5542-51
68. Le QA, Hay JW. Cost-effectiveness analysis of lapatinib in HER-2-positive advanced breast cancer. Cancer 2009;115:489-98
69. Reed SD, Li Y, Anstrom KJ, et al. Cost effectiveness of ixabepilone plus capecitabine for metastatic breast cancer progressing after anthracycline and taxane treatment. J Clin Oncol 2009;27:2185-91
70. Roche. Bevacizumab in combination with taxanes for the treatment of HER2-negative 1st line metastatic breast cancer. Roche Submission to the National Institute for Health and Clinical Excellence. Submitted: 8th March 2010. Single Technology Appraisal (STA). London: National Institute for Health and Clinical Excellence, 2010.
71. Thomas ES, Gomez HL, Li RK, et al. Ixabepilone plus capecitabine for metastatic breast cancer progressing after anthracycline and taxane treatment. J Clin Oncol 2007;25:5210-17
72. Calverley P, Pauwels R, Vestbo J, et al. Combined salmeterol and fluticasone in the treatment of chronic obstructive pulmonary disease: a randomised controlled trial. Lancet 2003;361:449-56
73. Calverley PM, Anderson JA, Celli B, et al. Salmeterol and fluticasone propionate and survival in chronic obstructive pulmonary disease. N Engl J Med 2007;356:775-89
74. Earnshaw SR, Wilson MR, Dalai AA, et al. Cost-effectiveness of fluticasone propionate/salmeterol (500/50 microg) in the treatment of COPD. Respir Med 2009;103:12-21
75. Spencer M, Briggs AH, Grossman RF, et al. Development of an economic model to assess the cost effectiveness of treatment interventions for chronic obstructive pulmonary disease. Pharmacoeconomics 2005;23:619-37