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Health technology assessment

Limitations of traditional health technology assessment methods and implications for the evaluation of novel therapies

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Pages 1635-1642 | Received 12 May 2017, Accepted 20 Jul 2017, Published online: 31 Jul 2017

References

  • Garrison LP, Kamal-Bahl S, Towse A. Toward a broader concept of value: identifying and defining elements for an expanded cost-effectiveness analysis. Value Health 2017;20:213-16
  • Sanders GD, Neumann PJ, Basu A, et al. Recommendations for conduct, methodological practices, and reporting of cost-effectiveness analyses: second panel on cost-effectiveness in health and medicine. JAMA 2016;316:1093-103
  • Neumann PJ, Cohen JT. Measuring the value of prescription drugs. N Engl J Med 2015;373:2595-7
  • Chandra A, Shafrin J, Dhawan R. Utility of cancer value frameworks for patients, payers, and physicians. JAMA 2016;315:2069-70
  • Kotseva K, Wood D, De Bacquer D, et al. EUROASPIRE IV: A European Society of Cardiology survey on the lifestyle, risk factor and therapeutic management of coronary patients from 24 European countries. Eur J Prev Cardiol 2016;23:636-48
  • Aggarwal J, Patel J, Yu J, et al. LDL-C goal achievement after adding or switching to ezetimibe in patients with clinical atherosclerotic cardiovascular disease or probable HeFH. J Manag Care Pharm 2016;222:S86
  • Hartgers M, Besseling J, Lucius B, et al. Clinical characteristics and prevalence of cardiovascular disease risk factors in subjects screened for familial hypercholesterolemia – a cross sectional study in the Netherlands. Atherosclerosis 2016;252:e203
  • Hartgers M, Besseling J, Hovingh G. Attainment of LDL-C treatment target in familial hypercholesterolemia patients: A theoretical model exploring efficacy of current and novel lipid lowering therapies. Atherosclerosis 2016;252:e43-4
  • Lloyd-Jones DM, Morris PB, Ballantyne CM, et al. 2016 ACC Expert consensus decision pathway on the role of non-statin therapies for LDL-cholesterol lowering in the management of atherosclerotic cardiovascular disease risk. A report of the American College of Cardiology Task Force on Clinical Expert Consensus Documents Journal of the American College of Cardiology 2016;68:92-125
  • Singh JA, Saag KG, Bridges SL Jr., et al. 2015 American College of Rheumatology guideline for the treatment of rheumatoid arthritis. Arthritis Rheumatol 2016;68:1-26
  • Garber AJ, Abrahamson MJ, Barzilay JI, et al. Consensus statement by the American Assicoation of Clinical Endocrinologists and Americal College of Endocrinology on the comprehensive Type 2 diabetes management algorithm—2017 executive summary. Endocr Pract 2017;23:207-38
  • American Diabetes Association. 8. Pharmacologic approaches to glycemic treatment. Diabetes Care 2017;40(Supp 1):S64-S74
  • Jansen JP, Incerti D, Mutebi A, et al. Cost-effectiveness of sequenced treatment of rheumatoid arthritis with targeted immune modulators. J Med Econ 2017:1-27
  • Institute for Clinical and Economic Review. Targeted immune modulators for rheumatoid arthritis: effectiveness & value evidence report; 2017. https://icer-review.org/wp-content/uploads/2016/08/NECEPAC_RA_Evidence_Report_Final_040717.pdf. Accessed 26 April 2017
  • Everett BM, Smith RJ, Hiatt WR. Reducing LDL with PCSK9 inhibitors—the clinical benefit of lipid drugs. N Engl J Med 2015;373:1588-91
  • Arrieta A, Page TF, Veledar E, et al. Economic evaluation of PCSK9 inhibitors in reducing cardiovascular risk from health system and private payer perspectives. PLoS One 2017;12:e0169761
  • Jena AB, Blumenthal DM, Stevens W, et al. Value of improved lipid control in patients at high risk for adverse cardiac events. Am J Manag Care 2016;22:e199
  • Gandra SR, Villa G, Fonarow GC, et al. Cost‐effectiveness of LDL‐C lowering with evolocumab in patients with high cardiovascular risk in the United States. Clin Cardiol 2016;39:313-20
  • Kazi DS, Moran AE, Coxson PG, et al. Cost-effectiveness of PCSK9 inhibitor therapy in patients with heterozygous familial hypercholesterolemia or atherosclerotic cardiovascular disease. JAMA 2016;316:743-53
  • Toth PP, Danese M, Villa G, et al. Estimated burden of cardiovascular disease and value-based price range for evolocumab in a high-risk, secondary-prevention population in the US payer context. J Med Econ 2017;20:1-10
  • Institute for Clinical and Economic Review. PCSK9 inhibitors for treatment of high cholesterol: effectiveness, value, and value-based price benchmarks. Boston, MA: ICER; 2015
  • American Diabetes Association, Standards of medical care in diabetes—2016. Diabetes Care 2016;39(Suppl1):S1-S112
  • Huang ES, Liu JY, Moffet HH, et al. Glycemic control, complications, and death in older diabetic patients. Diabetes Care 2011;34:1329-1336
  • Stratton IM, Adler AI, Neil HAW, et al. Association of glycaemia with macrovascular and microvascular complications of type 2 diabetes (UKPDS 35): prospective observational study. BMJ 2000;321:405-12
  • Dilokthornsakul P, Chaiyakunapruk N, Campbell JD. Does the use of efficacy or effectiveness evidence in cost-effectiveness analysis matter? J Asthma 2016;54:1-7
  • Campbell JD, McQueen RB, Briggs A. The “e” in cost-effectiveness analyses. A case study of omalizumab efficacy and effectiveness for cost-effectiveness analysis evidence. Ann Am Thorac Soc 2014;11(Suppl2):S105-S111
  • Weinstein MC, O'Brien B, Hornberger J, et al. Principles of good practice for decision analytic modeling in health-care evaluation: report of the ISPOR Task Force on Good Research Practices—modeling studies. Value Health 2003;6:9-17
  • Rawlins M. De testimonio: on the evidence for decisions about the use of therapeutic interventions. Lancet 2008;372:2152-61
  • Faria R, Hernandez Alava M, Manca A, et al. NICE DSU technical support document 17: the use of observational data to inform estimates of treatment effectiveness for technology appraisal: methods for comparative individual patient data. Sheffield: NICE Decision Support Unit; 2015
  • Doctor JN, Baseman JG, Lober WB, et al. Time-tradeoff utilities for identifying and evaluating a minimum data set for time-critical biosurveillance. Med Decis Making 2008;28:351-8
  • Institute for Clinical and Economic Review. Anabolic therapies for osteoporosis: effectiveness and value, final background and scope. Boston, MA: ICER; 2017. https://icer-review.org/wp-content/uploads/2016/11/ICER_Final_Osteo_Scope_010917.pdf. Accessed 26 April 2017
  • Sarode R, Milling TJ Jr., Refaai MA, et al. Efficacy and safety of a 4-factor prothrombin complex concentrate in patients on vitamin K antagonists presenting with major bleeding: a randomized, plasma-controlled, phase IIIb study. Circulation 2013;128:1234-43
  • Pabinger I, Brenner B, Kalina U, et al. Prothrombin complex concentrate (Beriplex P/N) for emergency anticoagulation reversal: a prospective multinational clinical trial. J Thromb Haemost 2008;6:622-31
  • Preston FE, Laidlaw ST, Sampson B, et al. Rapid reversal of oral anticoagulation with warfarin by a prothrombin complex concentrate (Beriplex): efficacy and safety in 42 patients. Br J Haematol 2002;116:619-24
  • Sarode R. Four-factor prothrombin complex concentrate versus plasma for urgent vitamin K antagonist reversal: new evidence. Clin Lab Med 2014;34:613-21
  • Jones CA, Ducis K, Petrozzino J, et al. Prevention of treatment-related fluid overload reduces estimated effective cost of prothrombin complex concentrate in patients requiring rapid vitamin K antagonist reversal. Expert Rev Pharmacoecon Outcomes Res 2016;16:135-9
  • Winslow BT, Mary KO, Christian MS, et al. Treatment of Alzheimer disease. Am Family Physician 2011;15:1403-12
  • National Institute for Health and Care Excellence. Donepezil, galantamine, rivastigmine and memantine for the treatment of Alzheimer’s disease, in NICE Guidance. London, UK: National Institute for Health and Care Excellence; 2011. p 76
  • Lipton RB, Bigal ME, Diamond M, et al. Migraine prevalence, disease burden, and the need for preventive therapy. Neurology 2007;68:343-9
  • Hepp Z, Bloudek LM, Varon SF. Systematic review of migraine prophylaxis adherence and persistence. J Manag Care Pharm 2014;20:22-33
  • Aurora S, Dodick DW, Turkel CC, et al. OnabotulinumtoxinA for treatment of chronic migraine: results from the double-blind, randomized, placebo-controlled phase of the PREEMPT 1 trial. Cephalalgia 2010;30:793-803
  • Diener H, Dodick DW, Aurora SK, et al. OnabotulinumtoxinA for treatment of chronic migraine: results from the double-blind, randomized, placebo-controlled phase of the PREEMPT 2 trial. Cephalalgia 2010;30:804-14
  • Institute for Clinical and Economic Review. Public comments received on ‘Controvesies in Migraine Management’; 2014. https://icer-review.org/wp-content/uploads/2016/02/Public_comments_081914_part1_v2.pdf. Accessed 26 April 2017
  • Institute for Clinical and Economic Review. Controversies in migraine management: a technology assessment; 2014. https://icer-review.org/wp-content/uploads/2016/01/CTAF_Migraine_Final_Report_081914-2.pdf. Accessed 26 April 2017
  • Li L, Roddam A, Gitlin M, et al. Persistence with osteoporosis medications among postmenopausal women in the UK General Practice Research Database. Menopause 2012;19:33-40
  • Karlsson L, Lundkvist J, Psachoulia E, et al. Persistence with denosumab and persistence with oral bisphosphonates for the treatment of postmenopausal osteoporosis: a retrospective, observational study, and a meta-analysis. Osteoporosis Int 2015;26:2401-11
  • Arden N, Earl S, Fisher DJ, et al. Persistence with teriparatide in patients with osteoporosis: the UK experience. Osteoporosis Int 2006;17:1626-9
  • Chastek B, Cheng L-i, White JC, et al. Persistence with osteoporosis therapies in postmenopausal women in a large US national health plan. Arthritis Rheumatol 2015;67:1625-6
  • Durden E, Pinto L, Lopez-Gonzalez L, et al. Two-year persistence and compliance with osteoporosis therapies among postmenopausal women in a commercially insured population in the United States. Arch Osteoporosis 2017;12:22
  • Heaney R, Watson P. Variability in the measured response of bone to teriparatide. Osteoporosis Int 2011;22:1703-8
  • Facey K, Boivin A, Gracia J, et al. Patients’ perspectives in health technology assessment: a route to robust evidence and fair deliberation. Int J Technol Assess Health Care 2010;26:334-40
  • Angelis A, Lange A, Kanavos P. Using health technology assessment to assess the value of new medicines: results of a systematic review and expert consultation across eight European countries. Eur J Health Econ 2017:1-30
  • Nicod E, Kanavos P. Commonalities and differences in HTA outcomes: a comparative analysis of five countries and implications for coverage decisions. Health Policy 2012;108:167-77
  • Shafrin J, Bruno A, MacEwan JP, et al. Physician and patient preferences for nonvalvular atrial fibrillation therapies. Value Health 2016;19:451-9
  • Osterberg L, Blaschke T. Adherence to medication. N Engl J Med 2005;353:487-97
  • Long Q, Little RJ, Lin X. Causal inference in hybrid intervention trials involving treatment choice. J Am Stat Assoc 2008;103:474-84
  • Howard L, Thornicroft G. Patient preference randomised controlled trials in mental health research. Br J Psychiatry 2006;188:303-4
  • Le QA, Doctor JN, Zoellner LA, et al. Cost-effectiveness of prolonged exposure therapy versus pharmacotherapy and treatment choice in posttraumatic stress disorder (the Optimizing PTSD Treatment Trial): a doubly randomized preference trial. J Clin Psychiatry 2014;75:222-30
  • McCaffery KJ, Turner R, Macaskill P, et al. Determining the impact of informed choice separating treatment effects from the effects of choice and selection in randomized trials. Med Decis Making 2011;31:229-36
  • Clark NM, Janz NK, Dodge JA, et al. The effect of patient choice of intervention on health outcomes. Contemp Clin Trials 2008;29:679-86
  • Janevic MR, Janz NK, Dodge JA, et al. The role of choice in health education intervention trials: a review and case study. Social Sci Med 2003;56:1581-94
  • Dolan P. The measurement of individual utility and social welfare. J Health Econ 1998;17:39-52
  • Abellan-Perpinan JM, Pinto-Prades JL. Health state after treatment: a reason for discrimination? Health Econ 1999;8:701-7
  • Nord E. The trade-off between severity of illness and treatment effect in cost-value analysis of health care. Health Policy 1993;24:227-38
  • Bleichrodt H, Doctor J, Stolk E. A nonparametric elicitation of the equity-efficiency trade-off in cost-utility analysis. J Health Econ 2005;24:655-78
  • Cookson R, Dolan P. Public views on health care rationing: a group discussion study. Health Policy 1999;49:63-74
  • Williams A. Intergenerational equity: an exploration of the ‘fair innings’ argument. Health Econ 1997;6:117-32
  • Stolk EA, Brouwer WB, Busschbach JJ. Rationalising rationing: economic and other considerations in the debate about funding of Viagra. Health Policy 2002;59:53-63
  • Lakdawalla D, Malani A, Reif J. The insurance value of medical innovation. J Public Econ 2017;145:94-102
  • Lakdawalla DN, Chou JW, Linthicum MT, et al. Evaluating expected costs and benefits of granting access to new treatments on the basis of progression-free survival in non–small-cell lung cancer. JAMA Oncol 2015;1:196-202
  • Malani A, Philipson T. Clinical trials, the market for observations, and the cost of medical R&D. The University of Chicago; 2008
  • Moore RA, McQuay HJ. Prevalence of opioid adverse events in chronic non-malignant pain: systematic review of randomised trials of oral opioids. Arthritis Res Ther 2005;7:R1046-51
  • Kalso E, Edwards JE, Moore RA, et al. Opioids in chronic non-cancer pain: systematic review of efficacy and safety. Pain 2004;112:372-80
  • Furlan AD, Sandoval JA, Mailis-Gagnon A, et al. Opioids for chronic noncancer pain: a meta-analysis of effectiveness and side effects. CMAJ 2006;174:1589-94

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