Are We Mature Enough to Make Use of Comparative Effectiveness Research?

Thanks to White House budget director Peter Orszag, a Dartmouth Atlas aficionado, $1.1 billion found its way into the stimulus piñata for “comparative effectiveness” research. Terrific, but – to paraphrase Jack Nicholson – can we handle the truth?

In other words, are we mature enough to use comparative effectiveness data to make tough decisions about what we will and won’t pay for? I worry that we’re not.

First, a bit of background. Our health care system, despite easily being the world’s most expensive, produces (by all objective measures) relatively poor quality care. Work begun 3 decades ago by Dartmouth’s Jack Wennberg and augmented more recently by Elliott Fisher has made a point sound-bitey enough for even legislators to understand: cost and quality vary markedly from region to region, variations that cannot be explained by clinical evidence and do not appear to be related to health care outcomes. In other words, plotting a 2×2 table with costs on one axis and quality on the other, we see a state-by-state Buckshot-o-Gram.

Three key conclusions flow from this “variations research”:

  • Lots of what we do in health care is costly and ineffective
  • We must somehow goose the system to move all providers and patients into the high quality, low cost quadrant on that 2×2 table; and
  • Better evidence about what works would help with such goose-ing.

Since nothing can happen without the research, the new funding for comparative effectiveness is welcome and helpful. But will it be sufficient to move the needle?

Here’s where things get dicey. A chief medical officer I know was once discussing unnecessary procedures in his health care system. In a rare moment of unvarnished truth telling, one of his procedural specialists told him, “I make my living off unnecessary procedures.” Even if we stick to the correct side of the ethical fault line, doctors and companies inevitably believe in their technologies and products, making it tricky to get them to willingly lay down their arms. Robert Pear described the political challenges surrounding effectiveness research in last week’s New York Times:

[the legislation has become] a lightening rod for pharmaceutical and medical-device lobbyists, who fear the findings will be used by insurers or the government to deny coverage for more expensive procedures and, thus, to ration care. In addition, Republican lawmakers and conservative commentators complained that the legislation would allow the federal government to intrude in a person’s health care by enforcing clinical guidelines and treatment protocols.

At this moment, Medicare’s rules – yes, the same Medicare that’s slated to go broke in a decade or so – forbid it to consider cost in its coverage decisions. Rather, its mandate is to cover treatments that are “reasonable and necessary.” So if Medicare comes to believe that a new chemotherapy will offer patients an extra week of life at a cost of $100,000 per patient, it is pretty much obligated to cover it. This is insane, obviously, but such are the rules.

And, if anybody tries to put the Kybosh on the Chemo, you can count on boatloads of oncologists, patient advocates, and pharma companies to descend on Washington like teenagers with Obama inaugural tickets, hammering the authorities to “be humane” and “take the decisions out of the hands of government bureaucrats and MBAs” and “put them in the hands of doctors, where they belong.” (This is precisely what happened in at Medicare’s hearings regarding cardiac CT, a technology that Medicare decided to cover despite a striking dearth of evidence of effectiveness). And TV news magazines will be right there, telling the compelling and tragic story of the kindly grandma who will never see her grandchildren’s bar mitzvahs because of Medicare’s heartlessness.

As Stalin said, “a single death is a tragedy, a million deaths a statistic.” Such is the problem with trying to make rational, evidence-based tradeoffs (that lead some people to not get the care they want) in a media-saturated open society.

But we can’t give up. We need to get a handle on healthcare costs, and it’s far better to do it by jettisoning non-evidence-based, wasteful care than by getting rid of the good stuff.

Luckily, we’ve waited long enough that we have some models to learn from – and some cautionary tales. Let’s begin by talking NICE. Literally.

A decade ago, Britain’s National Health Service launched NICE, the National Institute for Health and Clinical Excellence. In a recent NEJM article entitled “Saying No Isn’t NICE,” Robert Steinbrook reviewed the “travails” of NICE:

Since 2002, National Health Service organizations…have been required to pay for medicines and treatments recommended in NICE “technology appraisals.” The NHS usually does not provide medicines or treatments that are not recommended by NICE… NICE can be viewed as either a heartless rationing agency or an intrepid and impartial messenger for the need to set priorities in health care…

As we look to NICE for a roadmap, it is worth remembering the differing dynamics of a closed, tax-funded system such as the NHS, and the pluralistic, chaotic hodgepodge that is American health care. NICE’s physician-chair told Steinbrook that the Institute had to

be fair to all the patients in the National Health Service… If we spend a lot of money on a few patients, we have less money to spend on everyone else. We are not trying to be unkind or cruel. We are trying to look after everybody.

NICE, with its 270-member staff and $50M budget, not only reviews whether treatments work, but explicitly analyzes cost-effectiveness (leading some drug manufacturers to cut their prices to achieve better C-E ratios and chances of NICE approval). Although the cost-effectiveness cutoff is a bit fluid, NICE generally does not recommend treatments whose cost per quality-adjusted-life-year is more than about $40,000. According to American health care mythology, our cutoff is $50,000, but in reality it is hard to find examples of practices that have been withheld based on cost-effectiveness considerations.

Even in relatively non-litigious Britain, about one-third of NICE’s decisions are appealed, and several have generated impassioned pleas by patients and advocates for re-consideration. But most decisions have held up. Steinbrook praises NICE for helping to focus global attention on cost-effectiveness, but notes that

It remains to be seen… how many other countries will follow its lead. After all, saying no takes courage – and inevitably provokes outrage.

To me, NICE’s experience shows that rationing based on cost-effectiveness can be done, but we can count on it being about ten times harder in the United States (with our fragmented healthcare system, our sensationalist media, our hypertrophied legal system, and our tradition of individual benefit trumping the Good of the Commons) than it has been in the UK.

A second cautionary note: In November, the Times ran an article describing the sad case of the ALLHAT trial, the 2002 JAMA hypertension study that found that diuretics, costing pennies a day, worked better than 3 other classes of drugs (ACE inhibitors, calcium channel blockers, and alpha blockers) that cost up to 20 times more. The study, which took nearly a decade and cost over $100 million, was largely ignored – six years after its publication, the number of hypertensive patients on diuretics has bumped by an underwhelming 5% (from 35 to 40%).

Why the wimpy response to ALLHAT’s results? Partly resistance to change, partly new drugs that came out as the study was being conducted, and partly pharmaceutical company lobbying. As Medicare’s former CMO Sean Tunis said, “there’s a lot of magical thinking that [the application of comparative effectiveness studies] will all be science and won’t be politics.”

And if that isn’t depressing enough for anyone favoring science and rationality, here’s one last cautionary tale:

In the mid-1990s, the buzzword for encoding evidence-based practice was “practice guidelines,” and an agency called the Agency for Health Care and Policy Research (AHCPR) set out to create such guidelines using clinical evidence. Sound familiar? One of the first procedures AHCPR addressed was surgical management of back pain, bringing together a panel of national experts (led by Seattle’s Rick Deyo) to review the literature and recommend evidence-based practice standards.

You can guess the rest. The AHCPR panel found virtually no evidence supporting thousands of back surgeries each year, and recommended against them. Orthopedic surgeons worried that the guidelines were the first step to blocking insurance coverage for one of their favorite pastimes. As described by Jerome Groopman in a 2002 New Yorker article,

…almost as soon as the panel convened, it came under attack. Contending that the deliberations were not an open process and that the panelists were biased against surgery, a group of spine surgeons, led by Dr. Neil Kahanovitz, an orthopedist who was then a board member of the North American Spine Society, lobbied Congress to cut off AHCPR’s funding. Deyo recently told me [Groopman] that the line taken by the opponents of the panel was “ ‘These guys are anti-surgery, they’re anti-fusion.’ But we really had no axe to grind,” he went on. “Our aim was to critically examine the evidence and outcomes of common medical practices.”

Congress, led by then-House Speaker Newt Gingrich and in a nasty, budget-slashing mood, “zeroed-out” AHCPR’s funding. Though the Agency survived (a fraction of its budget was restored by the Senate), it did the only thing it could – ending the guideline program, re-branding itself as being about producing evidence but not recommending practice, and even changing its name to the Agency for Healthcare Research and Quality (AHRQ), a masterful series of moves by the late John Eisenberg credited with saving the agency from bureaucratic purgatory. As much as we like to blame the politicos, the drug and device companies, and the MBAs, the AHCPR fiasco demonstrated that physicians are every bit as capable of self-interested venality as any other group.

So, is it worth wasting our time and money on comparative effectiveness research? I’m hoping that this is a new day – the coming implosion of the healthcare system is now well recognized, as is the quality chasm. We simply must find ways to drive the system to produce the highest quality, safest care at the lowest cost, and we need to drag the self-interested laggards along, kicking and screaming if need be. Comparative effectiveness research is the scientific scaffolding for this revolution, so bring it on.

But let’s not be naïve about it – one person’s “cost-ineffective” procedure may be a provider’s mortgage payment, a manufacturer’s stock-levitator, and a patient’s last hope for survival.

So my hope is that we have the brains to produce the right kinds of data, and the maturity to act on it, humanely but responsibly.

Robert Wachter, MD, is widely regarded as a leading figure in the modern patient safety movement. Together with Dr. Lee Goldman, he coined the term “hospitalist” in an influential 1996 essay in The New England Journal of Medicine. His most recent book, Understanding Patient Safety, (McGraw-Hill, 2008) examines the factors that have contributed to what is often described as “an epidemic” facing American hospitals. His posts appear semi-regularly on THCB and on his own blog “Wachter’s World.”

39 replies »

  1. Doing Expired Hydrocodone Mixing Hydrocodone Promethazine [url=http://geodon.onlinecanadianpharmacyusa.com/ ]buy geodon online without prescription[/url]. What Does Hydrocodone Doo To You Hydrocodone 35 92 How Do You Use Hydrocodone . Hydrocodone Ortho Mcneil Hydrocodone Pain Tablets . Medications With Hydrocodone Withdrawal Symptoms Buy Hydrocodone Rx Skelaxin [url=http://hydrocodone.onlinecanadianpharmacyusa.com/ ]order hydrocodone online[/url] Interaction Tramadol Hydrocodone Hydrocodone No Rx Needed Vicodin Lortab Xanax On Line Hydrocodone Vicodin Buy Xanax Hydrocodone

  2. Well, I hate to be radical, but I think one possible solution is to institute DRG’s for physicians for inpatient care, with or without the additional step of bundling physician and hospital DRG payments and letting them fight it out over who gets what. I cite the latter with reluctance, because I think hospital administrators would hold the whip hand the way hospitals are currently organized, but one must at least consider it.
    What other way can one force physicians and hospitals to pay attention to comparative effectiveness research? As I commented to Dr. E. Novak recently, there is already research to show that stents and back surgery do NOT benefit large groups of patients, but these procedures continue to be performed for economic reasons only. There has to be a way to make that stop. This is not rationing, it’s regulation, in the same sense that the now-devastating Wall Street securities should have been regulated – are we going to learn our lesson, or aren’t we?
    i agree
    altın çilek

  3. Bob & Dan–
    Bob–Thanks for a very good post. I’m afraid it will be more difficult in the U.S. than in the U.K. for all the reasons you suggest. But, as Obama keeps telling us, time to grow up.
    Dan- Take a look at who the Obama adminisatration has appointed to the panel that will oversee comparative reserach.
    The 16-member panel is composed mainly of physicians–people like Jim Weinstein (of Dartmouth) and Christine
    Cassell (head of the American Board of Internal Medicine.) The only insurers represented: George Isham of Health Partners, the head of research at Kaiser and the chief medical officer of Independence Blue Cross.
    Two of the three are provider organizations as well as insurers and all are non-profits (unless I’m mistaken about Blue Cross.)
    Merrill Goozner reported on this on GoozNews, but as others have mentione, it seems that the software here no longer allows copying and pasting a URL.

  4. I agree with many of your points about profit from unnecessary procedures, but there is a deeper story to comparative effectiveness (CE).
    I was one of the leaders in the movement to block Medicare’s attempt to eliminate reimbursement for Cardiac CT. Our action was not based upon a disregard for EBM, but instead, the methodology that was used to justify the coverage decision. This methodology and back story holds many lessons for CE:
    -The proposed Medicare decision was for coverage of 2 indications that were not appropriate for Cardiac CT. The medical basis for coverage (via clinical trials) of these indications were based upon a misuse of the Framingham risk score. Taken together, Medicare’s proposed decision indicated that they did not understand the decision they were making.
    – Medicare disregarded the input of the governing medical societies and overlooked 75% of the published peer-reviewed scientific studies on Cardiac CT (n=40). Instead, it based its decision on the report of the BCBS TEC committee, a non-peer reviewed report that is sponsored by a for-profit health insurer (and one of the few that do not cover Cardiac CT).
    – The former CMO of Medicare was actively lobbying Medicare to eliminate Cardiac CT reimbursement, for them to require clinical trials, and for his special interest group to control of the resulting clinical trials. His group, CMTP, is sponsored by Blue Cross, Aetna and has strong ties to AHIP. Moreover, a staff member of CMTP was place on a Medicare decision making panel that weighed in on Cardiac CT’s coverage, while standing to profit from the trial.
    While Comparative Effectiveness is a worthy goal, the lessons learned from our experience in this realm is that CE must be approached cautiously. We have reviewed many CE study designs for Cardiac CT (submitted by groups or physicians sponsored by private payers) that are designed so that the “comparative” endpoints do not give the new technologies a fair trial. New technologies, such as imaging and genetic testing, are a serious threat to private payer profits (especially if they involve early detection for disease).
    The possible corruption of CE to be used in reimbursement decision is primarily sponsored by private payers to control medical decision making via reimbursement. Clearly, a system where for-profit health insurers are calling the shots is going to create a streamlined reimbursement system that advantages those companies’ obligations to build profits for their shareholders. One can only imagine the negative impact this would have on the future of genetic testing, molecular imaging for cancer, and personalized and preventive medicine.

  5. We don’t have a national institute to determine which automobile or house or organic pork chop is most effective. We rely on individual choice. Health insurers should be free to state “we cover any treatment that costs up to $50,000/QALY as determined by (insert name of non-profit institute)” or whatever other QALY and allow individuals to decide the value. See Michael Cannon’s nice briefing paper from CATO Institute on how the government prevents the private sector from producing CER.

  6. A great post. I live and work in England and have been watching the development of NICE and the evidence based healthcare movement here. The going is not easy, and many of the challenges you foresee in the US remain extremely contentious here, despite 10 years of overt “rationing” by NICE. We have not avoided serious and ongoing dissent, despite the differences between our healthcare system and yours (universal health insurance, less fragmentation, less litigation and an electorate more used to – but not particularly accepting of – “greater good” arguments). Given the challenges here, in an apparently more receptive culture and system, the challenges in the US may well be even greater than you describe, and it’s good to rehearse and be rea. But that is not a reason to turn away from the task.
    Evidence about comparative- and cost-effectiveness is not sufficient to make healthcare better and more efficient. But it is necessary. Healthcare will continue to be influenced by lobbyists and campaign funders, and change will always be opposed by those with vested interests in the status quo. Arguably it should be – in a democracy, policy judgements are governed by robust debate among those with different perspectives, beliefs and values.
    But in the absence of comparative effectiveness data such debate becomes a pitched battle between emotive and entrenched positions, to which measured discussion and rational negotiation fall casualty. We have to know how many lives or limbs drug A saves compared with surgery B and at what price we buy those benefits. Otherwise how do we ever have a meaningful discussion about whether it’s worth it? How do we ever begin to argue for better value for money?
    I’ve come across people who argue that the “is it worth it?” debate is one we need never have. Such people – few and far between these days – have their heads in the sand. Healthcare costs are already beginning to hold our personal and national finances hostage. Soon – like it or not – either I or my insurer is going to have to ask “is it worth it?” That’s a meaningless question without knowing what “it” is.
    Finally, another observation from the British experience. We too have people who choose to see the evidence-based movement as inspired by left wing ideology. I suspect that this is a fall-back position when more logical arguments fail. Personally, I do not see it in an ideological light – purely a pragmatic one. However, for those who are stuck on the notion, it’s worth pointing out that informed consumer choice and a functioning free market requires the very thing that evidence-based healthcare seeks to achieve – the exposure of the hitherto opaque relationship between cost and clinical quality.

  7. Excellent post. Nuanced, and understands all the issues at play, including here in the UK.

  8. Because the only case which you discuss is one in which supposedly greedy doctors perform ineffective surgery for profit, one might be left with the impression that the principal problem in healthcare is restraining rapacious doctors.
    It is well known in certain segments of the medical community that back surgery, and cardiac angioplasty are largely ineffective. It is also well known that regulators with government sponsorship have a limited grasp of statistics and science, and an uncanny tendency to target effective procedures as often as stupid ones. Don’t be surprised if you don’t like the result once a soviet style Supreme Extra-ordinary Medical Committee makes enforcable decisions about what heathcare is on your treatment menu.
    Remember that an early target of those who would use government to eliminate medical progress: The CT scan. The assault on the Cat scanner was nearly successful. When you torture the data enough, a CT scanner can seem like a silly thing to use. Why not practice like they did at the dawn of time? Reason is seven eighths of treason, and intelligence is what the other side uses, so the argument goes. The self styled experts – often unhappy doctors who could not abide the job they were trained in — talked themselves into the proposition that the CT scan was not useful. You can’t really blame them. Like Dr. Wolfe, these regulatory doctors had not seen a patient in decades and did not know from experience that a long drawn out physical exam did not provide anywhere near the accurate data that a rapid CT scan does. Imagine what the world would be like without that advance.
    How about bendictin? breast implants? The list goes on. These outlawed perfectly safe medical treatments were outlawed by so called public interest doctors, using statistics that would not let you pass a high school statistics course.
    It is the diversity in medical care that allows us to even know what is good and what is not. Soviet style top down management will not have the result we all would like to see.
    On another matter,
    It is impossible to have evidence based medicine without comprehensive tort reform, which curiously is never mentioned in this blog. Trial lawyers, that bankroll the movement do not want evidence based malpractice. And they tend to get what they want. You can’t have evidence based treatment, and bad outcome=new jet for the trial lawyer based malpractice law.
    Until these issues are addressed, universal healthcare is doomed.

  9. Well, I hate to be radical, but I think one possible solution is to institute DRG’s for physicians for inpatient care, with or without the additional step of bundling physician and hospital DRG payments and letting them fight it out over who gets what. I cite the latter with reluctance, because I think hospital administrators would hold the whip hand the way hospitals are currently organized, but one must at least consider it.
    What other way can one force physicians and hospitals to pay attention to comparative effectiveness research? As I commented to Dr. E. Novak recently, there is already research to show that stents and back surgery do NOT benefit large groups of patients, but these procedures continue to be performed for economic reasons only. There has to be a way to make that stop. This is not rationing, it’s regulation, in the same sense that the now-devastating Wall Street securities should have been regulated – are we going to learn our lesson, or aren’t we?

  10. Over one billion dollars of the economic stimulus package will be used to study which procedures, drugs, and devices are the most effective to treat disease and carry the least risk to the patient We already have scientific evidence that many of our most expensive tests and treatments are unneccessary, risky and very expensive, adding over $700 billion to our healthcare costs. One of the leading culprits is coronary care; $60 billion is spent on unnecessary angioplasties and coronary artery bypass surgeries. In additions, thousands of women are having unnecessary Cesarean sections (that cost 50% more than natural delivery) simply because it is more “convenient” and thousands more are having back surgery that in 90% of cases works no better than physical therapy. There are also countless examples of unnecessary use of expensive drugs and medical devices-all based on scientific evidence.
    One problem we have is that doctors and hospitals don’t pay attention to the scientific evidence because doing so would cost them $$$$. Surgery makes $$$$$, prescribing pills makes $. If having more scientific evidence is so important we would be applying what we know to the practice of medicine. However, Medicare and private insurance companies continue to reimburse providers by the number of procedures they perform, not by whether the procedure was necessary and improved the health of the patient. Until we reform the payment structure, for example, pay by performance rather than procedure, we will continue to have treatments that are of little value and very expensive (but very lucrative for doctors). I do agree that much of medicine is based on hunches rather than evidence and more needs to be done, but let’s first spend the money on reforming healthcare reimbursement so what we do know about evidence-based medical care is applied in the medical community.
    The public are also responsible for the increase in healthcare costs. The public need to understand that more care does not equal better care-just because insurance pays for it doesn’t mean it’s better or should be done. We cannot and should not perform everything that is technologically possible on every patient without considering the scientific evidence supporting it. This is what is meant by rationing of care-using the scientific evidence supporting treatment options.

  11. Well, Barry, I think what your’re getting at is that Emmanuel is proposing differentiated co-pays and tiering based on significant differences in clinical efficacy. Fine and dandy by me. And to be fair, this is what’s happening right now in most pharma formularies. It just hasn’t extended so well to diagnostics and other procedures.
    My point is that “consumer oriented” strategies can’t help at all because only a very few patients will understand the Comparative Efficacy Research well enough to know whether to pay the higher co-pay. Last time we tried this with the “good HMOs” in the 1980s, docs and patients basically got this sort of management outlawed. Some sort of NICE-ish organization must exist along with legislation that says “the only thing you Mr. Insurer are on the hook for is the canonically effective course of treatment” or the medical lobby and patient advocacy groups will scuttle it again.
    Emmanuel, Enthoven, Fuchs, et.al. have better ideas. I hope Kathleen and other people interested enough to read THCB will read books and journal articles too. But they should be looking for a “better solution” not “the solution”.

  12. Tom,
    It’s interesting that you referred to a discussion of Dr. Emanuel’s plan. In my comment, when I cited the example of paying for standard radiation or seed implantation but not the far more expensive proton beam therapy in prostate cancer cases, it was originally brought to my attention by Dr. Emanuel in a speech to the California Healthcare Foundation describing his plan. His point was that the standard therapy is “good enough” and anyone who wants proton beam therapy for five times the price should pay the difference out of pocket. The translation for most of us who couldn’t afford to do that is: you can’t have it because it’s not cost-effective. He also talked about examples of different drugs in the same therapeutic class that differ significantly in price but not efficacy, among other things.
    In his book, “Healthcare Guaranteed,” the portion about funding health insurance with a VAT was the weakest part, I think. The fact is that the broadest based VAT taxes in Europe only apply to about 40% of GDP or so. To generate anywhere near the revenue he says we need, we would have to tax virtually everything from food bought in supermarkets to healthcare to college tuition to government purchases. John Oberlander, a professor at UNC Chapel Hill, in a recent Health Affairs article estimated that if we were to enact a VAT, it could raise 0.4% of GDP for each percentage point of tax which is consistent with the European experience. Thus, the 10% tax proposed by Emanuel would raise 4% of GDP, not nearly enough to do the job he wants to do.

  13. Kathleen asks:
    > Are differentiated co-pays or tiering the solution?
    > If you have a better idea, speak up! We’re listening.
    No — patients are in a very poor position to understand the value they’re getting for their “extra” money.
    Look here for a better idea. There are variations on it of course. We can argue about whether a VAT is a good way to go.

  14. Excellent post and comments. Thank you.
    Barry Carol suggests that what we need is a prescription that doctors are ready to recommend and Americans are ready to swallow. Are differentiated co-pays or tiering the solution? If you have a better idea, speak up! We’re listening.

  15. The pressing need for addressing the evidence and changing our systems of care has been addressed in a comprehensive and compelling way in the Institute of Medicine (IOM) document “Crossing the Quality Chasm.” Not much happened as a result. In his excellent article, “The Silence”, Michael Millenson called us all to task for our continued refusal to take quality improvement actions, undermining the moral foundation of medical professionalism. Michael said that “The IOM quality committee, to retain its moral authority, should forgo issuing more reports and instead lead an emergency corrective-action campaign comparable to Flexner’s crusade against charlatan medical schools.” We are being too delicate here. Flexner’s job is not complete. There will always be a battle to protect the public interest against the special interest. Wikepedia says “the person called a charlatan is being accused of resorting to quackery, pseudoscience, or some knowingly employed bogus means of impressing people in order to swindle his victims by selling them worthless nostrums or similar goods or services that will not deliver on the promises made for them.” It speaks for itself.

  16. What rbar said. From my anecdotal experience, that’s a lot more expensive problem than this generic class of drugs vs. that generic class of drugs.
    A second cautionary note: In November, the Times ran an article describing the sad case of the ALLHAT trial, the 2002 JAMA hypertension study that found that diuretics, costing pennies a day, worked better than 3 other classes of drugs (ACE inhibitors, calcium channel blockers, and alpha blockers) that cost up to 20 times more. The study, which took nearly a decade and cost over $100 million, was largely ignored – six years after its publication, the number of hypertensive patients on diuretics has bumped by an underwhelming 5% (from 35 to 40%).
    A nice illustration of the CER problem. A few weeks after the ALLHAT trial was published, the equally high quality ANBP2 trial was published with the opposite conclusion. Where the ALLHAT trial showed that the ACE-I and the diuretic had identical results in all but a few subgroups, the ANBP2 trial gave the edge to the ACE-I. Based on the synthesis of those two and other trials, the JNC-VII gave the nod to ACE-Is for diabetics and diuretics for non-diabetics (the minority of hypertensives). Who will be the arbiter of multiple trials? When the ALLHAT trial was started only branded ACE-Is existed, but now there are generics available and currently the cost of chlorthalidone is $120/year vs. lisinopril at $128/yr (“insignificant difference” would be more accurate than “costing up to 20 times more”). What’s the cut off for differences? It sounds petty, but it’s really frustrating now when a patient is limited to a formulary medication that they don’t tolerate as well as an equally priced non-formulary medication.

  17. I find the Scott Gottleib/Sally Pipes argument about CER being used to “ration” health care to be totally disingenuous. All health care systems ration care now, and always will as long as finite supply of resources is exceeded by infinite demand. In 1963 Enoch Powell pointed out “there is virtually no upper limit to the amount of health care an individual can consume.” Little has changed since then.
    We have been rationing care in the US for many years in perhaps the most inequitable possible way–on ability to pay. The political right is often more well to do and is therefore largely exempt from this restriction. Its members resent the potential loss of their right to waste their money in any way they see fit. The political left has its own foibles regarding health care, but its adherents at least understand that most people do see access to basic health care as a right–a view that is remarkably consistent across most of the developed world.
    Perhaps the best way to square this circle is to encourage CER to go forward and to use the results to establish coverage and co-payment levels for the majority of Americans. Care that does not pass an evidence-based review will be subject to a 100% co-insurance. The dwindling numbers of the very rich will still be permitted to do whatever they please with their own money, and the suppliers of unproven products will be permitted to use whatever marketing tools they wish to reach out this small but potentially very lucrative subset. Who knows, we might wind up using residents of the Hamptons guinea pigs to prove the value of a new technology for the rest of us.

  18. Excellent post.
    I would add the following:
    1) Agree with commenter BK: “Culturally, we are not ready for health care rationing” – I see that myself practicing in Germany (where there actually is a relatively high degree of technical-procedural medicine and volume based reimbursement, but not coming close to US standards). The question is whether the public can be educated. Unfortunately, most media products solidify the unhealthy culture, and “mystery diagnosis” even deepens the problem. The questions is whether an educational campaign could help – but chances are slim since physicians are not (yet) unified here (for financial and/or cultural reasons).
    2) Diagnostic overkill, something not really discussed by Dr. Wachter who focusses on treatment. The very widespread US american mentality is: “If there is any chance that (noninvasisve) test X could help me, I want it (right now), even if the yield of this test may approach zero in my situation” (that is culture as mentioned above.) The doctor thinks: my patient wants test X, and even though the yield is very low, enough colleagues do it … if the outcome is bad and he/she is unhappy, I can be sued (maybe not succesfully so, but I still have to defend myself) and may have to prove that I met the standard of “reasonable care”. What the heck, I am not paying for it, neither is the patient … let’s just order the MRI/US/CT/lab test, even though it has a less than small chance of changing anything.
    I am not sure whether this has ever been studied formally, but I am confident that the US is a worldwide leader in the ratio of studies showing relevant findings to normal/unrevealing studies, by a wide margin. And I don’t think that this diagnostic overkill is beneficial at all – but the public does not know that any noninvasive study bears the risk of subsequent invasive studies and misdiagnosis resulting in unnecessary treatment.

  19. Other than the sacred cow of Medicare what control mechanism is there to implement CE? As long as we cling to this for profit system there will be no way to say no.

  20. It makes the most sense to implement cost/quality controls on procedures that not only lower costs but are more effective. One argument not mentioned above is the rationalization of inducing more procedure based protocol for improving the quality of care. This would reduce longer term cost on thing such as prenatal care and preventing secondary complications. Rationalizing more preventive care through the use of EMR’s meta-analysis could provide the key in reducing the cost of healthcare across the board. Giving insurance agencies the data to back more preventative and less everything else.

  21. Dr. Wachter,
    The concept of comparative effectiveness is perhaps not new. Publicly funded healthcare systems use it extensively under different names. Mr. Carol has made some very thoughtful comments but I respectfully disagree with him regarding his assessment and simply suggesting NICE type review process. No process or system is going to be ideal with the great degree of variance which exists (as mentioned by Mr. Andrews). I think that every country has to deal with this enormous challenge in a customized way based on its economics, social fabric and politics. 20% of GDP expenditure on healthcare by 2018 or 2020 is not going to be sustainable and it may drown the whole economy by pushing many countries into a vicious cycle of poor cost effectiveness.

  22. Simply put, current American voters who enjoy traditional health insurance coverage through their employers simply aren’t going to approve of CMS using CE studies to approve care – yet. We just aren’t there and there isn’t enough political capital for politicians from voters to overcome the inevitable attempts by several key stakeholders in HC to prevent this and the inevitable backlash that would initially set in.
    I would imagine it the implementation of strict CE for healthcare plays out two ways that are largely dependent upon the overall economy. If the U6 numbers get bad enough though in the US for college-educated workers in the next year or so (say general unemployment north of 10% by this time next year), then I think you have a fighting chance that this gets enacted sooner than later including the chance for more radical health care reform along the lines of a single-payer system.
    However, it is more likely that we have a period of much higher general unemployment/underemployment but that it is not severe enough among college-educated workers to give Democrats the political capital they need to enact more radical healthcare reform including enacting CE as a part of FDA approval and CMS payment.
    As for the pharma/biotech/med device firms, I do think though that the more savvy pharma and medical device executives see the proverbial handwriting on the wall. They know they have been found “wanting” and that the days of the US healthcare market being their principal cash cow are gradually waning. It is not necessarily because the American market is really tapped in terms of penetration (say like the US auto market or segments of the food industry) but more so in terms of raw pricing power.
    The US will remain a key market just due to the raw size of the overall US economy and number of people but it they realize they are going to have to diversify much more globally. A bunch of pharma and med devices are starting to take some initial steps to that accord. I just think they hope though that they have several more years where the prices in the US market remain quite high and can pad their gradual transition to a more global revenue stream.

  23. Re: Wennberg, et al., what is even more disturbing than the variance between regions is the variance in each hospital from day-to-day. Comparative effectiveness is nice to consider as holy grail, but it will not make any difference until hospitals and physicians and other clinical personnel eliminate the variance that currently exists.

  24. I personally would prefer to see the U.S. copy the NICE approach and simply refuse to cover and pay for services, tests, procedures, drugs, etc. that cannot pass a reasonable QALY metric or other cost-effectiveness test. As Bob and others note, this will be a tough sell to put it mildly. We might, however, be able to diffuse the rationing argument by using differentiated co-pays or tiering instead. Such differences would have to be high enough to get the attention of both doctors and patients. When there is a more cost-effective alternative to a high cost approach, we might agree to pay what the cost-effective approach would have cost. For example, if I have prostate cancer and decide that I would prefer proton beam therapy over standard radiation or seed implantation because the side effects are fewer and milder but it costs five times as much, CMS or private insurers might agree to pay for standard radiation or seed implantation and tell me that I need to cover the difference of $50-$60K myself. Obviously, most people aren’t wealthy enough to cover such a high cost, but I don’t think that should be society’s concern because the proton beam therapy is wildly expensive relative to the established alternatives.
    At the other extreme, if a Federal Health Board decides not to cover PSA tests because they’re not cost-effective, most people could afford to self-pay if they really want it. Or, if Prilosec is just as good as Nexium but far cheaper to treat acid reflux, patients should pay the difference between the two drugs if they want the more expensive Nexium. Encouragingly, I read recently that after a comment period, CMS is likely to stop paying for virtual colonoscopies because they don’t do as good a job of finding small polyps and, if there are polyps present, a standard colonoscopy is required to remove them anyway.
    We are in very difficult economic times not seen since the Great Depression of the 1930’s. If there was ever a time to embrace sensible strategies that might start to bend the medical cost growth curve down toward a level that we can afford and sustain, now is that time. Everyone recognizes that healthcare costs are unsustainable even before the housing bust and crisis in the financial system. For doctors and hospitals that resist comparative effectiveness research and payment policy reform, I have the following question regarding the unsustainability of healthcare costs: What’s your answer and, more importantly, what’s your contribution to solving the problem? Insurers, trial lawyers, drug and device manufacturers and individual patients / consumers need to answer the same question as well, but doctors and hospitals are the key stakeholder groups that need to get on board.

  25. And, it isn’t always just money that drives the malfeasance of specialist physicians trade groups. (The Supreme Court ruled that they are “trade groups”, not “professional societies”, BTW; you can look it up.) Specialty trade group also advocate for their members’ convenience and comfort, not just their incomes.
    A signature example of this is the Infectious Disease Society of America (IDSA) publishing egregious guidelines for the treatment of Lyme disease essentially saying to just treat the easy cases. In the mid-eighties the IDSA realized Lyme borreliosis was a nasty infection with no easy diagnosis or treatment and it certainly did not fit the IDSA model of two weeks of antibiotics producing a cure for everything, making the infectious disease specialist look like a hero in the process. It was easier to toss Lyme borreliosis over the wall to the neurologists and rheumatologists who could call it MS, lupus, fibromyalgia, or whatever and get years of office visits and outrageously over-priced insurance-covered drugs paid for, bumping up the whole medical economy.
    The Attorney General of Connecticut tried to stop the insanity of the phony Lyme disease guidelines, and the IDSA just goosed their PR people and lobbyists into action. (Look at what JAMA is saying about guidelines this month. Look at the compromise the IDSA designed.) Physicians crush societal good for the interests of physicians yet again.
    Wachter is right. Physicians need to take responsibility for the U.S. health care mess, but they probably are not going to fix anything soon. Just as with the zombie banks, the government is going to have to step in to take care of this. Doctors can ignore effectiveness research and basic reality, but the rest of us don’t have to put up with this.

  26. It will be interesting to see if the “individual” perspective on rational rationing changes by the end of this recession, as the ranks of the uninsured sweeps well into the middle class. Rationing is OK when society (we) do it to the poor and disenfranchised.
    Perhaps starting comparative effectiveness guidelines with the FEHP and especially Congress and senior administration officials rather than Medicare or the VA might improve its chances of surviving and maturing. If it is good enough for elected and appoint officials then it will be good enough for the rest of us. Let’s see if our leaders really lead and walk their talk.
    Lynn Bailey
    Healthcare Economist
    Columbia, SC

  27. Thanks- Excellent historical perspective
    Reality is we CANNOT afford NOT to go down this path with rigor and speed.
    It will be a painfull economic re-adjustment for all who profit excessively from not having done so sooner.So they killed the proverbial goose that was laying those golden eggs.(Old story of what greed can do)
    Dr. Rick Lippin

  28. This is an absolutely superb post that deserves wide readership. Bob is dead on right that there is an almost touching technocratic belief that it is the absence of scientific knowledge, rather than inappropriate incentives sustained by fundamental flaws in our political system, which result in much health spending with no demostrable social benefit.
    Our political system, particularly Congress, has shown since the advent of Medicare a chronic inability to separate strong from weak claims in health services. While this weakness is a compelling argument for a Federal Reserve like entity to manage the process, the politics will not disappear at the appearance of this deux ex machina solution.
    Sadly, Tom Daschle was uniquely positioned to make the argument to his Congressional colleagues for restraining and rechannelling this eminently abusable political power. No governor or health policy wonk, not matter how widely respected, and no President, regardless of how powerful, can effectively address this systemic political weakness. Bowing to pressure on Daschle’s appointment was, sadly, a damaging political error.
    If Congressional leadership cannot be persuaded to stay its own hand, all the comparative effectiveness research in the world will not make much difference in how we actually spend our trillions on healthcare. It will just be too easy to succumb to the astroturf campaigns and the sob stories. . .

  29. Cost effectiveness analysis is about tradeoffs, denying certain care to certain patients so that more cost effective care can be provided to others. This is a rational approach at a societal level, but is not the way that we think about healthcare, which is delivered on an individual level and in which doctors are supposed to do anything possible to help a patient. Alain Enthoven said “The core fiction of American health care is that insurance should cover every useful medical procedure no matter how small the benefit or prohibitive the cost.” Culturally, we are not ready for health care rationing. I believe it is going to take being on a major precipice of healthcare collapse for us to make the difficult shift required to make a comparative effectiveness system work. Until then, this body will be rendered impotent by a lack of enforceability of whatever guidance it issues.

  30. “NICE, with its 270-member staff and $50M budget, not only reviews wether treatments work, but explicitly analyzes cost-effectiveness.”
    Dr. Scott Gottlieb reminded us in a June 24, 2008 WSJ op-ed piece he penned, “Medicare doesn’t have a single oncologist on staff, yet since the year 2000 the program issued 165 restrictions and directives on the use of cancer drugs and diagnostic tools. Medicare has only about 20 doctors and 40 total clinicans working in its coverage office, and fewer than a dozen in the office that sets doctors’ reimbursement rates.”
    However, in regards to cancer drugs, Medicare has radically expanded its authorization for use of cancer drugs by putting off-label decision making in the hands of compendia writers in the private sector, many of whom are on the payrolls of the companies that make the drugs?

  31. One persons high cost of health care is another persons (spine surgeons) 3rd home