While the concept is not new, "comparative effectiveness" is starting to be a term that is more widely known due to its curious inclusion in President Obama's $789 billion spending package. The fact that liberal health care issues somehow found their way into a supposed "stimulus" package is strange enough; but the arguments being made to defend the supposed innocence of "comparative effectiveness" are even stranger.

As far as I can tell, there is no use for comparative effectiveness other than as a first step towards the rationing of pharmaceutical products and health care services. And, somehow, that first step was funded to the tune of $1 billion in the spending package.

What Is Comparative Effectiveness?

Comparative effectiveness essentially means the same thing as "cost-benefit."

A more mundane (and somewhat less rationing-sounding) definition is:

Comparative effectiveness quite simply means comparing two or more treatments for a given condition... Comparative effectiveness evaluations may focus only on the relative medical benefits and risks of each option, or they may also weigh both the costs and the benefits of those options. In some cases, a given treatment may prove to be more effective clinically or more cost-effective for a broad range of patients, but frequently a key issue is determining which specific types of patients would benefit most from it...

Comparative effectiveness is increasingly being viewed as a viable way to help drive down spiraling health care costs while continuing to provide quality care.

Generally speaking the concept sounds innocent enough: Who would be opposed to using cost-effective solutions to continue to provide quality health care? However, the concern that many people have is in the precedent that is established and where it leads.

First, commenting on the quoted definition above, it should be noted that this really isn't about comparing the effectiveness of different courses of treatment. Comparing effectiveness of different options is--and has always been--a standard practice in medicine that has been utilized by researchers, doctors, and patients since, well, since pretty much forever. You get sick and the doctor tells you, "You can take these pills and there's a 40% chance of success with these side effects... or you can have surgery and there's an 80% chance of success with these other side effects." And you then make a decision based on the factors that are important to you. Those factors might include the probability of success, the potential implications of failure, chances of recurrence, quality of life, and, of course, cost. As different courses of treatment are chosen by patients and doctors, more information becomes available to researchers automatically so that they are better able to evaluate the degree of success that can be expected in different cases.

Regardless of how some may want to spin it, the only thing that is different about "comparative effectiveness" is that the analysis really boils down to one of cost-benefit.

"Information on which to compare the results from drugs with the same purpose is often not available. For example, both Lucentis [ranibizumab, Genentech] and Avastin [bevacizumab, Genentech] are promising new drugs for treatment of macular degeneration, but head-to-head information on the relative outcomes is not available--and one costs about 20 times the amount of the other..."

Stuart Altman, Ph.D., a prominent health economist from Brandeis University, told the conference that over the past seven years, healthcare cost increases have been steeper than at any time in our history. "We are not slowing down.... It is very serious." He said the employment-based, private-based health insurance system is running into serious problems as premiums have gone up over 90% in recent years.

Altman said clearly the nation needs comparative effectiveness research, "so that we can get the kind of care that is appropriate. Appropriate not only in the clinical sense.... It's also a cost issue."

It's really about the cost more than the effectiveness.

If one course of treatment costs $100 and there's a 20% chance of success and there's another option that costs $600 for a 60% chance of success, the comparative effectiveness is that the second option gives you three times the effectiveness but at six times the cost. From a cost-benefit analysis, the first option is a comparatively better value.

Statistically and economically speaking, society would be better off today using the first option. If you have 100 people that require some kind of treatment and the first option was used, 20 people would experience success at a total societal cost of $10,000. If the second option were used, only 16 people could be treated for $10,000, of which a total of 9 would experience success. Given that information, the macroeconomic and social argument can be made that the first option should be used. $10,000 will cure 20 people using the first option instead of only 9 that would be cured with the second option--even though the second option is more effective.

The problem is that this isn't a typical cost-benefit analysis. Health, quality of life, and even life itself hangs in the balance. The economy overall benefits from the first option being used since twice as many people can be cured for the same cost. But that is little comfort to the individual that is faced with a 20% chance of success given the society-approved treatment but a 60% chance if the second option is available.

The individual is almost always interested in effectiveness. Society (and government) is interested in cost-effectiveness.

Update 5/22/2009: Two months after writing this article, a court case seems to have supported the opinion that state bureaucrats do have the right to ration health care for which the state is paying. This could very well be a glimpse of things to come under comparative effectiveness and Obama's health care plan.



Who should have control over your medical care: your family doctor, or a bureaucrat you've never met whose sole job is to look out for the government's financial bottom line?

A ruling earlier this month by a panel of the 11th U.S. Circuit Court of Appeals went a long way toward answering that question, as federal judges ruled in favor of three states that had filed suit to have final medical decision-making authority transferred from doctors to state bureaucrats....

The thrust of the states' argument in Moore was summed up in a brief written by the attorneys representing the state of Florida. 'Treating physicians,' they wrote, 'cannot be trusted with this sort of decision. When left to their own devices, they advocate for their patients' - something state governments resent due to its interference in the execution of their cost-effectiveness analyses - 'and deem all manner of unproven, dangerous, ineffective, cosmetic, unnecessary, bizarre and controversial treatments as ‘medically necessary.''

While bureaucrats 'will consider doctors' determinations,' said attorney Robert Highsmith in oral arguments on March 24, the 'final arbiter' of medical decisions is and should be 'the state.' The panel of the 11th Circuit agreed.



So the main point of the court case was that states were arguing that doctors, left to their own devices, will advocate for their patients. And that's not in the interest of the finances of the state.

I hope this clearly demonstrates why state participation in the health care system is do dangerous.

What Obama's Spending Package Includes

The reference to comparative effectiveness can be found on pages 156 through 160 of the House/Senate compromise legislation (13MB PDF ).

It should be noted that Obama's spending package does not prohibit patients from obtaining any specific treatment regardless of its comparative effectiveness. And the compromise legislation that came out of the negotiations between the Senate and the House apparently modified the text to state that the funds were for "research that compares the clinical outcomes, effectiveness, and appropriateness" of different products and services (page 157, line 17).

The significance of the word "clinical" is that it presumably limits the research to compare the effectiveness of treatments without regards to cost. Of course, this would seem to make the entire $1.1 billion expenditure rather wasteful since researchers and doctors already have a pretty darned good idea of the effectiveness of each treatment option; and where that information is lacking, the information will become more complete as time goes on as a logical consequence of applying the treatment to real patients. No government funding is necessary for that.

The Slippery Slope of Health Rationing

Notwithstanding the seemingly harmless, if not wasteful, funding of clinical comparative effectiveness, the debate has to center around the ultimate purpose of any such research. While this initial legislation--buried in a stimulus package--only provides for clinical comparative effectiveness, subsequent legislation addressing health care could very easily widen the mandate of the research and use these funds for the new expanded purpose.

Ultimately the question has to be asked: What is the usefulness of comparative effectiveness if not to judge the cost-effectiveness of different treatments? And what is the benefit of knowing the cost-effectiveness of any given treatment?

Update 5/4/2009: Two months after publishing this article I found the following quote in which Obama effectively chooses to dance around the subject of providing health care to the terminally ill.



[President Obama said]: "I would have paid out of pocket for that hip replacement, just because she's my grandmother. Whether, sort of in the aggregate, society making those decisions to give my grandmother, or everybody else's aging grandparents or parents, a hip replacement when they're terminally ill is a sustainable model is a very difficult question."...

Advocates on the other side of the debate reject the label. 'I think Obama was trying to invoke the notion of tradeoffs more than rationing,' said Len Nichols, who directs the health care program at the New America Foundation, a Washington research organization. 'Curative care for his grandmother was futile. Rationing is when efficacious care is denied to save money, perhaps to provide basic care to another, but nevertheless consciously denied.'



While the latter quote doesn't necessarily speak for the administration, it might be one of the first attempts to redefine what rationing is. According to this quote, denying someone health care services if they're dying anyway isn't rationing. I would disagree. But the fact is that just two months after I wrote my original article we are having discussion about what it means to ration health care.

Further:



'There is going to have to be a conversation that is guided by doctors, scientists, ethicists,' Mr. Obama said. 'And then there is going to have to be a very difficult democratic conversation that takes place. It is very difficult to imagine the country making those decisions just through normal political channels. And that's part of why you have to have some independent group that can give you guidance. It's not determinative, but I think has to be able to give you some guidance.'



So here President Obama seems to be saying that the question of what procedures are appropriate can't be decided by politicians so it's going to have to be decided by an "independent group." It would seem likely that that "independent group" that's going to be making health rationing decisions has been funded to the tune of $1.1 billion in the stimulus bill.

Common sense would suggest that patients will, in virtually all cases where all else is equal, prefer the most effective treatment available to them regardless of the comparative effectiveness. If a treatment costs $600 and provides a 60% chance of success, I'm not going to be interested in what an alternative with 20% success costs. It won't matter to me whether the government or the doctor considers the other treatment more cost-effective. As a patient I'm just looking at 60% vs. 20%. All else being equal, that's the end of my comparison. And if I'm the one paying for the treatment, it should be of no interest to the government or the doctor how much it costs.

The problem, of course, is that it's not usually the individual that is paying for the treatment. It's usually an insurance company or the government. And both the insurance companies and the government are looking for ways to minimize costs and get the most bang for their buck. As an insurance company or government entity, wouldn't you rather cure 20 people than 9 people? Or cure 9 people for half the cost?

Indeed, Andrew Holtz of the ECRI Institute says:

There are many examples of research results not being applied by clinicians or not accepted by consumers, as well as political backlash against federal agencies that recommended unpopular changes in clinical practice... Nevertheless, the need to manage rising costs currently drives important comparative effectiveness efforts...

Throughout the conference, the discussion often touched on two key dialectics: whether cost-effectiveness should be an integral component of comparative effectiveness analyses and whether greater attention to comparative effectiveness would impair innovations.

Those that promote comparative effectiveness research recognize the political challenges they're up against. Apparently they envision "federal agencies" recommending "unpopular changes in clinical practice." They admit that "rising costs" is driving the effort and they ask--at least publicly--whether or not cost-effectiveness should be a part of their analysis... They also recognize the threat that comparative effectiveness poses to medical innovation.

The Threat to Medical Innovation

The threat that comparative effectiveness poses to medical innovation is real.

It is common knowledge that cutting edge technologies (medical or otherwise) are often relatively expensive compared to other alternatives. However, as the technology is improved, the price comes down and becomes more accessible to more people. Eventually what was once considered unreasonable from a cost-benefit standpoint becomes the most effective option and also the most cost-effective solution.

However, these advances in technology cannot and will not occur unless the technology is allowed to "run its course" through the high-cost/low-availability phase all the way to the final low-cost/high-availability phase.

A forced focus on "comparative effectiveness" will always tend to favor cheap, existing options to the possible detriment of the development of new technologies that, while initially more expensive, may eventually become cheaper and more effective.

Comparative effectiveness could have the undesired side-effect of deterring medical innovation.

The Threat to the Individual

As mentioned above, Obama's spending plan does not prohibit anyone from obtaining any treatment that they and their doctor deem appropriate.

However, given that the logical progression of comparative effectiveness is one of cost-benefit, consider the logical consequence of a government-sanctioned entity declaring the relative comparative effectiveness of different treatment options. Even if the government doesn't require anyone to use its conclusions, it's not unlikely that insurance companies could look towards those findings to make decisions on what treatments are covered under their policies and which aren't. The findings of comparative effectiveness become an official stamp of government approval and insurance companies, interested in minimizing their costs, could easily add clauses to their coverage that indicate that they will only cover treatments that the government indicates meet some level of "comparative effectiveness." In fact, that precise possibility was mentioned in an article at the New York Times about the comparative research funding in the stimulus bill:

Congress did not say exactly how the findings should be used. Private insurers can use the data in deciding whether to cover new drugs and medical procedures, but it is unclear how Medicare will use the information.

Even if that does not happen initially, it seems likely that the government would quickly limit hospitals and practitioners that receive Medicare/Medicaid funding to those services that meet the government's "comparative effectiveness" standards. This would become a de facto standard. Even if private insurers did not adopt those same standards, the sheer volume of government-funded health care would put general pressure on providers to avoid those treatments that the government didn't deem to be comparatively effective.

This would have the dual negative impact of reducing patient choice and deterring potential medical innovation. Government's allegedly good intentions to reduce health care costs ultimately could result in lower quality care for the patient today and fewer options in the future.

It's About the Money

It really seems that it comes down to a cost-benefit analysis. And as demonstrated earlier in this article, a legitimate case can be made at the macro level for using a treatment that is 20% successful rather than one is 60% successful if the latter is more than three times as expensive. But that argument is based on the assumption that an individual should be forced to sacrifice his health for society's economic interests.

It's also based on the short-sighted view that ignores the fact that treatments that cost a lot today may become very economical tomorrow. In the example above, what if that 60% effective treatment that costs $600 today would end up costing $250 in five years if it were allowed to persist in the market? At that point, the new treatment would be able to cure more people than the previously cheaper treatment. However, if comparative effectiveness decides that its currently-high cost doesn't justify its use, it may simply disappear before it has a chance to come down in price. Or, perhaps, the medical research that would lead to the product might never take place since the company would know the option wouldn't be competitive in the beginning and would die a quick commercial death.

The reason the government and insurance companies are interested in comparative effectiveness is to reduce cost. While cost is certainly important to patients, it's even more important to insurance companies whose profitability depends on minimizing costs; and to the government whose long-term solvency depends on the same thing. In fact, that's the only reason government is involved in this issue--so much government money and the future solvency of the federal government depends on being able to control its control health care costs.

Considering that comparative effectiveness research doesn't provide any clear improvements to individual patient care, will arguably reduce future medical innovations, and limits the options of patients, is this really something the taxpayers want to be funding?

The only way to avoid the type of health care rationing that this will seemingly lead to is to reduce--not increase--government's involvement in health care.