The American Recovery and Reinvestment Act of 2009 includes $1.1 billion for comparative effectiveness research – research that compares medical treatments and procedures to determine which ones are most effective.
Critics cry that comparative effectiveness is a code name for rationing or will inevitably lead to it. A Wall Street Journal editorial said “the comparative effectiveness outfit will start to ration care to control costs.” George Will declared that comparative effectiveness research “would dramatically advance government control – and rationing – of health care.” In print and online many other outlets have carried similar stories.
To support their “slippery slope” argument, the critics point to Britain’s National Institute for Health and Clinical Excellence (NICE), which uses comparative effectiveness and cost-effectiveness analysis to advise the British National Health Service on what should be provided through the service. But the critics are wrong: wrong about the sequence and wrong about the role of NICE. In fact, the British system is a case in point that demonstrates the difference between rationing and comparative effectiveness research.
Created in 1947, the British National Health Service (NHS) rationed care very effectively for more than 50 years before NICE was established, simply by setting a budget for the service each year and sticking to it. Because its budget is well controlled, the NHS has always recognized that it cannot do everything for everybody. In fact, health spending in Britain has grown faster since NICE was created than in any previous decade, the result of a deliberate policy by the Blair government.
What NICE does is help ensure that the money available to the NHS is spent effectively and fairly. NICE attempts to get the most health benefit possible from the NHS budget. One of its main goals is to counter disparities in access to care.
When NICE was created in 1999, decades of research had demonstrated persistent inequities in access to the best in medical care. Dr. Gillian Leng, a member of NICE’s Board and Deputy Chief Executive and Chief Operating Officer for NHS Evidence, described the situation this way: “While one provider was getting good results by using a state-of-the-art procedure in treating some diagnosis, the provider next door might be using an outdated, ineffective approach with less satisfactory results. It was important to develop some way to standardize treatment approaches at the highest levels among NHS providers in order to try to ensure uniformly good care. We had to eliminate what was sometimes referred to as the ‘health care lottery.’”
The health care lottery is an apt description of how care is distributed in the United States today, where geographical and socioeconomic inequities in access are even more acute than they were 10 years ago. The critics ignore the fact that care is already rationed here depending on whether patients have insurance, what type they have, whether they live in underserved areas, and whether it is profitable to serve them, as David Leonhardt pointed out recently in The New York Times.
Moreover, the criteria used for coverage decisions by American health plans, part D insurance carriers, and even Medicare and Medicaid are not transparent to anyone. Cost plays a role, but the public has no idea to what extent benefit and safety have been sacrificed to save money because costs, and even the prices paid for services, are never discussed.
Critics have focused on the fact that when NICE recommends against a treatment it invokes cost-effectiveness. But they have missed two more important facts.
First, as Anthony Culyer points out here, when NICE says yes, the NHS is obligated to provide that care to everyone in the country. Second, NICE is accountable to the public. Consumer groups, manufacturers, physician groups, and other stakeholders know exactly what evidence, assumptions, and other considerations led to a decision. Over time, this has led to a robust system of public comment, reconsideration and appeal as well as explicit consideration of equity and other values in decision-making.
Recently, for example, after issuing a preliminary finding that it was effective but not cost-effective, NICE approved lenalidomide for patients with multiple myeloma who had failed two or more other treatments. Critics of NICE point to the preliminary decision as “rationing,” but fail to mention how the final decision benefited patients as well as taxpayers.
For patients, NICE’s careful analysis helped define the subgroup most likely to benefit. The analysis showed that for many – those who have failed one previous treatment – it is uncertain whether lenalidomide is as effective as treatments that are already available.
NICE also pointed out that, within the fixed NHS health care budget, broader approval of the drug would have meant taking away access to other services that provide more health benefits for the money. The manufacturer, which conducted the cost-effectiveness analysis in the first place, acknowledged this point and agreed to a unique arrangement under which, for each patient, the NHS pays for the first two years of treatment and the manufacturer pays to continue treatment past that time.
In the U.S., where there is no fixed budget for health care, comparative effectiveness information cannot influence how much money goes to health care, but it can inform decisions by individual patients and physicians, affecting which tests and treatments people choose and which ones they do not. Not surprisingly, then, a December 2007 Congressional Budget Office report showed that comparative effectiveness will not cut spending by much.
Compared with NICE, the U.S. comparative effectiveness effort focuses less on cost and more on generating new evidence. At present, because of the lack of comparative studies, medical decisions are made in a cloud of uncertainty, a situation in which no one knows which tests and treatments are demonstrably better and safer than the alternatives.
For drugs and devices, for example, manufacturers conduct more research to meet the Food and Drug Administration’s regulations and less that answers patients’ and health professionals’ questions about what works best and is safest, and for whom. Few studies compare different kinds of treatments, such as drug versus nondrug alternatives, and fewer still seek to distinguish patients who have the greatest benefit and lowest risk of side effects from other patients who benefit less.
As Hal Sox, editor of the Annals of Internal Medicine, recently said in his keynote address to the American College of Physicians 2009 Internal Medicine meetings: “the public isn’t getting its money’s worth from our system of industry-sponsored clinical research. The public pays the costs of drug trials through higher drug prices but gets research that doesn’t tell us everything we need to know to make good decisions. We get more for our money with the NIH-sponsored trials that we support with our taxes. However, the NIH funds far fewer trials than industry.”
In fact, in the U.S. the attack on comparative effectiveness research is not really about rationing. It is about fear of the free flow of independent information about benefits, harms, and costs. Without rationing at all, publicly funded comparative effectiveness research could improve health care in the U.S. by producing and publicizing independent, comprehensive research about what works best for patients.
Mark Helfand, M.D., is editor-in-chief of the journal Medical Decision Making, a staff physician at the Portland V.A. Medical Center, and a professor of medicine at Oregon Health & Sciences University, where he directs the Oregon Evidence-based Practice Center. helfand@ohsu.edu, 503–494-4277. Louise Russell, PhD, is an economist and research professor in the Institute for Health and a professor in the Department of Economics at Rutgers University. She is an associate editor of the journal. lrussell@ifh.rutgers.edu, 732–932-6507.
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Comparative Effectiveness and Rationing: Lessons from NICE
American attempts at comparative effectiveness research have often prompted charges of rationing. However, a free flow of information about benefits, harms, and costs through publicly funded comparative effectiveness research could improve health care in the U.S. Examining how Britain’s NICE uses this research could help us understand its potential benefits.
The American Recovery and Reinvestment Act of 2009 includes $1.1 billion for comparative effectiveness research – research that compares medical treatments and procedures to determine which ones are most effective.
Critics cry that comparative effectiveness is a code name for rationing or will inevitably lead to it. A Wall Street Journal editorial said “the comparative effectiveness outfit will start to ration care to control costs.” George Will declared that comparative effectiveness research “would dramatically advance government control – and rationing – of health care.” In print and online many other outlets have carried similar stories.
To support their “slippery slope” argument, the critics point to Britain’s National Institute for Health and Clinical Excellence (NICE), which uses comparative effectiveness and cost-effectiveness analysis to advise the British National Health Service on what should be provided through the service. But the critics are wrong: wrong about the sequence and wrong about the role of NICE. In fact, the British system is a case in point that demonstrates the difference between rationing and comparative effectiveness research.
Created in 1947, the British National Health Service (NHS) rationed care very effectively for more than 50 years before NICE was established, simply by setting a budget for the service each year and sticking to it. Because its budget is well controlled, the NHS has always recognized that it cannot do everything for everybody. In fact, health spending in Britain has grown faster since NICE was created than in any previous decade, the result of a deliberate policy by the Blair government.
What NICE does is help ensure that the money available to the NHS is spent effectively and fairly. NICE attempts to get the most health benefit possible from the NHS budget. One of its main goals is to counter disparities in access to care.
When NICE was created in 1999, decades of research had demonstrated persistent inequities in access to the best in medical care. Dr. Gillian Leng, a member of NICE’s Board and Deputy Chief Executive and Chief Operating Officer for NHS Evidence, described the situation this way: “While one provider was getting good results by using a state-of-the-art procedure in treating some diagnosis, the provider next door might be using an outdated, ineffective approach with less satisfactory results. It was important to develop some way to standardize treatment approaches at the highest levels among NHS providers in order to try to ensure uniformly good care. We had to eliminate what was sometimes referred to as the ‘health care lottery.’”
The health care lottery is an apt description of how care is distributed in the United States today, where geographical and socioeconomic inequities in access are even more acute than they were 10 years ago. The critics ignore the fact that care is already rationed here depending on whether patients have insurance, what type they have, whether they live in underserved areas, and whether it is profitable to serve them, as David Leonhardt pointed out recently in The New York Times.
Moreover, the criteria used for coverage decisions by American health plans, part D insurance carriers, and even Medicare and Medicaid are not transparent to anyone. Cost plays a role, but the public has no idea to what extent benefit and safety have been sacrificed to save money because costs, and even the prices paid for services, are never discussed.
Critics have focused on the fact that when NICE recommends against a treatment it invokes cost-effectiveness. But they have missed two more important facts.
First, as Anthony Culyer points out here, when NICE says yes, the NHS is obligated to provide that care to everyone in the country. Second, NICE is accountable to the public. Consumer groups, manufacturers, physician groups, and other stakeholders know exactly what evidence, assumptions, and other considerations led to a decision. Over time, this has led to a robust system of public comment, reconsideration and appeal as well as explicit consideration of equity and other values in decision-making.
Recently, for example, after issuing a preliminary finding that it was effective but not cost-effective, NICE approved lenalidomide for patients with multiple myeloma who had failed two or more other treatments. Critics of NICE point to the preliminary decision as “rationing,” but fail to mention how the final decision benefited patients as well as taxpayers.
For patients, NICE’s careful analysis helped define the subgroup most likely to benefit. The analysis showed that for many – those who have failed one previous treatment – it is uncertain whether lenalidomide is as effective as treatments that are already available.
NICE also pointed out that, within the fixed NHS health care budget, broader approval of the drug would have meant taking away access to other services that provide more health benefits for the money. The manufacturer, which conducted the cost-effectiveness analysis in the first place, acknowledged this point and agreed to a unique arrangement under which, for each patient, the NHS pays for the first two years of treatment and the manufacturer pays to continue treatment past that time.
In the U.S., where there is no fixed budget for health care, comparative effectiveness information cannot influence how much money goes to health care, but it can inform decisions by individual patients and physicians, affecting which tests and treatments people choose and which ones they do not. Not surprisingly, then, a December 2007 Congressional Budget Office report showed that comparative effectiveness will not cut spending by much.
Compared with NICE, the U.S. comparative effectiveness effort focuses less on cost and more on generating new evidence. At present, because of the lack of comparative studies, medical decisions are made in a cloud of uncertainty, a situation in which no one knows which tests and treatments are demonstrably better and safer than the alternatives.
For drugs and devices, for example, manufacturers conduct more research to meet the Food and Drug Administration’s regulations and less that answers patients’ and health professionals’ questions about what works best and is safest, and for whom. Few studies compare different kinds of treatments, such as drug versus nondrug alternatives, and fewer still seek to distinguish patients who have the greatest benefit and lowest risk of side effects from other patients who benefit less.
As Hal Sox, editor of the Annals of Internal Medicine, recently said in his keynote address to the American College of Physicians 2009 Internal Medicine meetings: “the public isn’t getting its money’s worth from our system of industry-sponsored clinical research. The public pays the costs of drug trials through higher drug prices but gets research that doesn’t tell us everything we need to know to make good decisions. We get more for our money with the NIH-sponsored trials that we support with our taxes. However, the NIH funds far fewer trials than industry.”
In fact, in the U.S. the attack on comparative effectiveness research is not really about rationing. It is about fear of the free flow of independent information about benefits, harms, and costs. Without rationing at all, publicly funded comparative effectiveness research could improve health care in the U.S. by producing and publicizing independent, comprehensive research about what works best for patients.
Mark Helfand, M.D., is editor-in-chief of the journal Medical Decision Making, a staff physician at the Portland V.A. Medical Center, and a professor of medicine at Oregon Health & Sciences University, where he directs the Oregon Evidence-based Practice Center. helfand@ohsu.edu, 503–494-4277. Louise Russell, PhD, is an economist and research professor in the Institute for Health and a professor in the Department of Economics at Rutgers University. She is an associate editor of the journal. lrussell@ifh.rutgers.edu, 732–932-6507.