Cost-Effectiveness in a Flat World — Can ICDs Help the United States Get Rhythm?
http://www.100md.com
《新英格兰医药杂志》
The remarkable reduction of 50 percent or more in the age-adjusted mortality rate from coronary heart disease over the past four decades in the United States1 has been driven by three parallel phenomena: an understanding of the risk factors that influence the likelihood and presentation of coronary events, the use of randomized trials to create an indisputable evidence base on which to make decisions, and the flexibility to change systems of care to incorporate new knowledge into practice. Despite the failure of some physicians to adopt proven interventions2 and substantial disparities in the U.S. health care system,3 the reduction in the rate of death from coronary causes can be directly linked to the adoption of proven healthier lifestyles and the application of evidence-based interventions.4 Even more remarkably, the overwhelming majority of these interventions also meet typical criteria for acceptable cost-effectiveness in the types of patients for whom they are beneficial.5
One of the remaining areas of uncertainty is the use of implantable cardioverter–defibrillators (ICDs). In this issue of the Journal, Sanders et al.6 demonstrate that the lifetime cost per quality-adjusted year of life gained by the implantation of an ICD ranged from $34,000 to $70,200 among patients whose expected relative benefit was 23 percent or greater, thereby approximating the usually accepted threshold of about $35,000 to $50,000, on the basis of the presumed cost of Medicare's end-stage renal disease program.
This experienced group's methods generally followed guidelines,7 ranging from the absence of industry sponsorship to the analytic details. Nevertheless, they may have overestimated the benefits of ICDs by assuming that the rate of death from noncardiac causes after the implantation of an ICD would be the same as age- and sex-specific rates of death from noncardiac causes in the overall U.S. population, since recipients of ICDs are more likely to have smoked and have other atherosclerotic diseases. In addition, patients whose lives are saved by ICDs may otherwise be sicker than the survivors of myocardial infarction whose data Sanders et al. used for their estimation of costs. The authors performed numerous sensitivity analyses, in which prespecified assumptions varied within reasonable ranges, but they did not perform an uncertainty analysis, in which all estimates are varied simultaneously within a reasonable range, the analysis is run 1000 or more times, and confidence intervals are created around every estimate.8
Their baseline analysis also assumed that the receipt of an ICD would not change a patient's quality of life, although such an event could decrease the quality of life of a patient who is bothered by implanted hardware or increase it by increasing a patient's self-assuredness. Even small changes in a patient's quality of life can drive cost-effectiveness: for example, the favorable cost-effectiveness of dual-chamber as compared with single-chamber pacing for sinus-node dysfunction is critically dependent on small improvements in the quality of life.9
Nevertheless, the fundamental message of Sanders and colleagues is that ICDs probably have an acceptable cost-effectiveness ratio when used in populations in which a significant device-related reduction in mortality exists. No therapy should be assumed to have a single, simple value for cost-effectiveness, since its cost-effectiveness can vary substantially depending on the type of patient being considered.6,10
The methods available to perform a cost-effectiveness analysis like that reported by Sanders et al. are sophisticated and reasonably reliable. Given such methods, it is remarkable that Medicare, the single largest payer of medical expenditures in the United States, does not consider costs in any of its reimbursement decisions and is not permitted to negotiate for lower drug prices. In this issue of the Journal, Neumann et al.11 provide a variety of reasons, overwhelmingly political, for the failure of the United States to join other countries in which governmental payment has been the stimulus, rather than the inhibitor, of cost-effective decisions. Detractors point to the imprecision of cost-effectiveness methods, especially the potential for unscrupulous or unintentionally biased investigators to tilt their findings. However, methods have improved and become more standardized, and leading journals now expect researchers to meet this higher standard. Perhaps the greatest misunderstanding is the assumption that reliance on cost-effectiveness analysis will reduce medical care expenditures. The reality is that cost-effectiveness analysis is a way to optimize value, either within a predetermined budget or at a threshold of predefined value. Its wider use will probably identify interventions that are worthwhile but underused,2,6,10,12 simultaneously encouraging the extension of worthwhile health coverage to a broader segment of the population while discouraging collective payment for interventions that society may nevertheless want to allow more affluent persons, with a different threshold for value, to purchase privately.
Another issue is the lack of agreement on a threshold of cost-effectiveness. However, overreliance on a single threshold would trigger the natural incentive in a capitalistic society to price every intervention at that threshold: why charge less when the system will pay more? The future of medical expenditures is further complicated by the fact that thresholds may change over time with inflation and increasing economic productivity. For example, one estimate of the cost of treatment for end-stage renal disease now exceeds $120,000 per quality-adjusted year of life (Chertow G: personal communication), a threshold that might justify the use of medical interventions that heretofore have been considered financially unattractive. By comparison, the United States currently spends about 15 percent of its $38,000 per capita annual gross domestic product on health care — about $6,000 per year of life per person.
In cost-effectiveness analysis, the therapy itself is always a key cost. For example, it is projected that the otherwise expensive benefit of the use of continuous clopidogrel therapy for all patients with coronary disease would be reasonably cost-effective if the price were reduced by 75 percent or so.13 If the cost of ICDs were reduced by about two thirds, the cost-effectiveness ratio would be close to the threshold of $50,000 per quality-adjusted year of life, even when its benefit is as small as a relative reduction in the risk of death of 20 to 25 percent.6
Ignoring cost-effectiveness ignores value and diminishes pressure on costs. The continued increase in U.S. health care expenditures, now over 15 percent of the gross domestic product, is a direct reflection of higher prices,14 owing to the relatively tame price competition in health care as compared with the fierce competition (think Wal-Mart) in the rest of the economy. It also reflects the tendency to extend effective medical interventions to populations in which their comparatively marginal effectiveness yields less desirable or even frankly unbearable cost-effectiveness ratios.
Recently, a British man, frustrated by the delays in the national dental care system, sought private care — not in the United Kingdom, where the price of his care was ￡2,000, but rather in Poland, where the price was ￡280 including airfare — and the outcome was excellent.15 In The World Is Flat,16 Friedman emphasizes that the availability of electronic communication and international travel has leveled the playing field, so that products and services can be provided wherever labor and other costs are lowest. Outsourcing to the developing world, originally confined to unskilled manufacturing jobs, now includes customer-assistance call centers and sophisticated software development in countries in which highly educated persons work for lower wages in efficient systems. The international supply chain is driven by value: price and quality.
Meanwhile, medicine has lagged badly in the adoption of electronic and other mechanisms to standardize production and delivery and to improve quality.17 Adjusted for inflation, automobiles are no more expensive than they were 10 years ago even though they are of higher quality, and computers are cheaper yet better. It is now easier to qualify for a home-equity loan for hundreds of thousands of dollars than to obtain preauthorization for a prescription for less than $100.
In a flat world, medicine remains one of the last protected parts of our economy, not because of tariffs but because of political pressures and the unusual reality that the user rarely pays the price. However, if a British man can go to Poland for dental care, does it follow that Americans will soon get their prescriptions from Canada or go to India for bypass surgery? Will American hospitals contract with cardiologists and radiologists from across the globe to read routine echocardiograms and radiographs?
Medical practice has been slow to change and has too often ignored cost and cost-effectiveness, but the changes afoot in the fields of technology and communication may be irresistible. Maybe we will finally not only permit but also actually mandate the agency that pays so much of U.S. health care expenses to negotiate for lower prices and consider costs in its reimbursement policies, so that the increased costs of indicated care, such as implanting additional worthwhile ICDs,6,12 will be partially offset by lower prices and the avoidance of inappropriately costly care.
Source Information
From the Department of Medicine, University of California, San Francisco, San Francisco.
References
Morbidity & mortality: 2004 chart book on cardiovascular, lung, and blood diseases. Rockville, Md.: National Heart, Lung, and Blood Institute, May 2004.
Ma J, Sehgal NL, Ayanian JZ, Stafford RS. National trends in statin use by coronary heart disease risk category. PLoS Med 2005;2:e123-e123.
Lillis-Blanton M, Maddox TM, Rushing O, Menash GA. Disparities in cardiac care: rising to the challenge of Healthy People 2010. J Am Coll Cardiol 2004;44:503-508.
Hunink MGM, Goldman L, Tosteson ANA, et al. The recent decline in mortality from coronary heart disease, 1980-1990: the effect of secular trends in risk factors and treatment. JAMA 1997;277:535-542.
Hlatky MA, Mark DB. Economics and cardiovascular disease. In: Zipes DP, Libby P, Bonow RO, Braunwald E, eds. Braunwald's heart disease: a textbook of cardiovascular medicine. 7th ed. Vol. 1. Philadelphia: Elsevier Saunders, 2005:20-6.
Sanders GD, Hlatky MA, Owens DK. Cost-effectiveness of implantable cardioverter-defibrillators. N Engl J Med 2005;353:1471-1480.
Gold MR, Siegel JE, Russell LB, Weinstein MC, eds. Cost-effectiveness in health and medicine. New York: Oxford University Press, 1996.
Briggs AH. Handling uncertainty in cost-effectiveness models. Pharmacoeconomics 2000;17:479-500.
Rinfret S, Cohen DJ, Lamas GA, et al. Cost-effectiveness of dual-chamber pacing as compared with ventricular pacing for sinus node dysfunction. Circulation 2005;111:165-172.
Goldman L, Weinstein MC, Goldman PA, Williams LW. Cost-effectiveness of HMG-CoA reductase inhibition for primary and secondary prevention of coronary heart disease. JAMA 1991;265:1145-1151.
Neumann PJ, Rosen AB, Weinstein MC. Medicare and cost-effectiveness analysis. N Engl J Med 2005;353:1516-1522.
Voigt A, Ezzeddine R, Barrington W, et al. Utilization of implantable cardioverter-defibrillators in survivors of cardiac arrest in the United States from 1996 to 2001. J Am Coll Cardiol 2004;44:855-858.
Gaspoz J-M, Coxson PG, Goldman PA, et al. Cost-effectiveness of aspirin, clopidogrel, or both for secondary prevention of coronary heart disease. N Engl J Med 2002;346:1800-1806.
Anderson GF, Reinhardt UE, Hussey PS, Petrosyan V. It's the prices, stupid: why the United States is so different from other countries. Health Aff (Millwood) 2003;22:89-105.
Burnley man flies to Poland to see dentist. Daily Times. August 23, 2005. (Accessed September 15, 2005, at http://www.dailytimes.com.pk/default.asp?page=story_25-1-2005_pg9_5.)
Friedman TL. The world is flat: a brief history of the twenty-first century. New York: Farrar, Straus & Giroux, 2005.
Reid PP, Compton WD, Grossman JH, Fanjiang G, eds. Building a better delivery system: a new engineering/health care partnership. Washington, D.C.: National Academies Press, 2005. (Also available at http://www.nap.edu/openbook/030909643X/html/R1.html.)(Lee Goldman, M.D.)
One of the remaining areas of uncertainty is the use of implantable cardioverter–defibrillators (ICDs). In this issue of the Journal, Sanders et al.6 demonstrate that the lifetime cost per quality-adjusted year of life gained by the implantation of an ICD ranged from $34,000 to $70,200 among patients whose expected relative benefit was 23 percent or greater, thereby approximating the usually accepted threshold of about $35,000 to $50,000, on the basis of the presumed cost of Medicare's end-stage renal disease program.
This experienced group's methods generally followed guidelines,7 ranging from the absence of industry sponsorship to the analytic details. Nevertheless, they may have overestimated the benefits of ICDs by assuming that the rate of death from noncardiac causes after the implantation of an ICD would be the same as age- and sex-specific rates of death from noncardiac causes in the overall U.S. population, since recipients of ICDs are more likely to have smoked and have other atherosclerotic diseases. In addition, patients whose lives are saved by ICDs may otherwise be sicker than the survivors of myocardial infarction whose data Sanders et al. used for their estimation of costs. The authors performed numerous sensitivity analyses, in which prespecified assumptions varied within reasonable ranges, but they did not perform an uncertainty analysis, in which all estimates are varied simultaneously within a reasonable range, the analysis is run 1000 or more times, and confidence intervals are created around every estimate.8
Their baseline analysis also assumed that the receipt of an ICD would not change a patient's quality of life, although such an event could decrease the quality of life of a patient who is bothered by implanted hardware or increase it by increasing a patient's self-assuredness. Even small changes in a patient's quality of life can drive cost-effectiveness: for example, the favorable cost-effectiveness of dual-chamber as compared with single-chamber pacing for sinus-node dysfunction is critically dependent on small improvements in the quality of life.9
Nevertheless, the fundamental message of Sanders and colleagues is that ICDs probably have an acceptable cost-effectiveness ratio when used in populations in which a significant device-related reduction in mortality exists. No therapy should be assumed to have a single, simple value for cost-effectiveness, since its cost-effectiveness can vary substantially depending on the type of patient being considered.6,10
The methods available to perform a cost-effectiveness analysis like that reported by Sanders et al. are sophisticated and reasonably reliable. Given such methods, it is remarkable that Medicare, the single largest payer of medical expenditures in the United States, does not consider costs in any of its reimbursement decisions and is not permitted to negotiate for lower drug prices. In this issue of the Journal, Neumann et al.11 provide a variety of reasons, overwhelmingly political, for the failure of the United States to join other countries in which governmental payment has been the stimulus, rather than the inhibitor, of cost-effective decisions. Detractors point to the imprecision of cost-effectiveness methods, especially the potential for unscrupulous or unintentionally biased investigators to tilt their findings. However, methods have improved and become more standardized, and leading journals now expect researchers to meet this higher standard. Perhaps the greatest misunderstanding is the assumption that reliance on cost-effectiveness analysis will reduce medical care expenditures. The reality is that cost-effectiveness analysis is a way to optimize value, either within a predetermined budget or at a threshold of predefined value. Its wider use will probably identify interventions that are worthwhile but underused,2,6,10,12 simultaneously encouraging the extension of worthwhile health coverage to a broader segment of the population while discouraging collective payment for interventions that society may nevertheless want to allow more affluent persons, with a different threshold for value, to purchase privately.
Another issue is the lack of agreement on a threshold of cost-effectiveness. However, overreliance on a single threshold would trigger the natural incentive in a capitalistic society to price every intervention at that threshold: why charge less when the system will pay more? The future of medical expenditures is further complicated by the fact that thresholds may change over time with inflation and increasing economic productivity. For example, one estimate of the cost of treatment for end-stage renal disease now exceeds $120,000 per quality-adjusted year of life (Chertow G: personal communication), a threshold that might justify the use of medical interventions that heretofore have been considered financially unattractive. By comparison, the United States currently spends about 15 percent of its $38,000 per capita annual gross domestic product on health care — about $6,000 per year of life per person.
In cost-effectiveness analysis, the therapy itself is always a key cost. For example, it is projected that the otherwise expensive benefit of the use of continuous clopidogrel therapy for all patients with coronary disease would be reasonably cost-effective if the price were reduced by 75 percent or so.13 If the cost of ICDs were reduced by about two thirds, the cost-effectiveness ratio would be close to the threshold of $50,000 per quality-adjusted year of life, even when its benefit is as small as a relative reduction in the risk of death of 20 to 25 percent.6
Ignoring cost-effectiveness ignores value and diminishes pressure on costs. The continued increase in U.S. health care expenditures, now over 15 percent of the gross domestic product, is a direct reflection of higher prices,14 owing to the relatively tame price competition in health care as compared with the fierce competition (think Wal-Mart) in the rest of the economy. It also reflects the tendency to extend effective medical interventions to populations in which their comparatively marginal effectiveness yields less desirable or even frankly unbearable cost-effectiveness ratios.
Recently, a British man, frustrated by the delays in the national dental care system, sought private care — not in the United Kingdom, where the price of his care was ￡2,000, but rather in Poland, where the price was ￡280 including airfare — and the outcome was excellent.15 In The World Is Flat,16 Friedman emphasizes that the availability of electronic communication and international travel has leveled the playing field, so that products and services can be provided wherever labor and other costs are lowest. Outsourcing to the developing world, originally confined to unskilled manufacturing jobs, now includes customer-assistance call centers and sophisticated software development in countries in which highly educated persons work for lower wages in efficient systems. The international supply chain is driven by value: price and quality.
Meanwhile, medicine has lagged badly in the adoption of electronic and other mechanisms to standardize production and delivery and to improve quality.17 Adjusted for inflation, automobiles are no more expensive than they were 10 years ago even though they are of higher quality, and computers are cheaper yet better. It is now easier to qualify for a home-equity loan for hundreds of thousands of dollars than to obtain preauthorization for a prescription for less than $100.
In a flat world, medicine remains one of the last protected parts of our economy, not because of tariffs but because of political pressures and the unusual reality that the user rarely pays the price. However, if a British man can go to Poland for dental care, does it follow that Americans will soon get their prescriptions from Canada or go to India for bypass surgery? Will American hospitals contract with cardiologists and radiologists from across the globe to read routine echocardiograms and radiographs?
Medical practice has been slow to change and has too often ignored cost and cost-effectiveness, but the changes afoot in the fields of technology and communication may be irresistible. Maybe we will finally not only permit but also actually mandate the agency that pays so much of U.S. health care expenses to negotiate for lower prices and consider costs in its reimbursement policies, so that the increased costs of indicated care, such as implanting additional worthwhile ICDs,6,12 will be partially offset by lower prices and the avoidance of inappropriately costly care.
Source Information
From the Department of Medicine, University of California, San Francisco, San Francisco.
References
Morbidity & mortality: 2004 chart book on cardiovascular, lung, and blood diseases. Rockville, Md.: National Heart, Lung, and Blood Institute, May 2004.
Ma J, Sehgal NL, Ayanian JZ, Stafford RS. National trends in statin use by coronary heart disease risk category. PLoS Med 2005;2:e123-e123.
Lillis-Blanton M, Maddox TM, Rushing O, Menash GA. Disparities in cardiac care: rising to the challenge of Healthy People 2010. J Am Coll Cardiol 2004;44:503-508.
Hunink MGM, Goldman L, Tosteson ANA, et al. The recent decline in mortality from coronary heart disease, 1980-1990: the effect of secular trends in risk factors and treatment. JAMA 1997;277:535-542.
Hlatky MA, Mark DB. Economics and cardiovascular disease. In: Zipes DP, Libby P, Bonow RO, Braunwald E, eds. Braunwald's heart disease: a textbook of cardiovascular medicine. 7th ed. Vol. 1. Philadelphia: Elsevier Saunders, 2005:20-6.
Sanders GD, Hlatky MA, Owens DK. Cost-effectiveness of implantable cardioverter-defibrillators. N Engl J Med 2005;353:1471-1480.
Gold MR, Siegel JE, Russell LB, Weinstein MC, eds. Cost-effectiveness in health and medicine. New York: Oxford University Press, 1996.
Briggs AH. Handling uncertainty in cost-effectiveness models. Pharmacoeconomics 2000;17:479-500.
Rinfret S, Cohen DJ, Lamas GA, et al. Cost-effectiveness of dual-chamber pacing as compared with ventricular pacing for sinus node dysfunction. Circulation 2005;111:165-172.
Goldman L, Weinstein MC, Goldman PA, Williams LW. Cost-effectiveness of HMG-CoA reductase inhibition for primary and secondary prevention of coronary heart disease. JAMA 1991;265:1145-1151.
Neumann PJ, Rosen AB, Weinstein MC. Medicare and cost-effectiveness analysis. N Engl J Med 2005;353:1516-1522.
Voigt A, Ezzeddine R, Barrington W, et al. Utilization of implantable cardioverter-defibrillators in survivors of cardiac arrest in the United States from 1996 to 2001. J Am Coll Cardiol 2004;44:855-858.
Gaspoz J-M, Coxson PG, Goldman PA, et al. Cost-effectiveness of aspirin, clopidogrel, or both for secondary prevention of coronary heart disease. N Engl J Med 2002;346:1800-1806.
Anderson GF, Reinhardt UE, Hussey PS, Petrosyan V. It's the prices, stupid: why the United States is so different from other countries. Health Aff (Millwood) 2003;22:89-105.
Burnley man flies to Poland to see dentist. Daily Times. August 23, 2005. (Accessed September 15, 2005, at http://www.dailytimes.com.pk/default.asp?page=story_25-1-2005_pg9_5.)
Friedman TL. The world is flat: a brief history of the twenty-first century. New York: Farrar, Straus & Giroux, 2005.
Reid PP, Compton WD, Grossman JH, Fanjiang G, eds. Building a better delivery system: a new engineering/health care partnership. Washington, D.C.: National Academies Press, 2005. (Also available at http://www.nap.edu/openbook/030909643X/html/R1.html.)(Lee Goldman, M.D.)