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Bias In Clinical Research Is Inevitable

DrRich has said many times that clinical science is among the least exact of the sciences, and therefore, the results of clinical research are particularly susceptible to “spinning” by various interested parties, in order to yield the kind of results they would prefer to see.

Until recent times in American medicine, the parties who have been most interested in spinning clinical research have been the people who run drug companies and medical device companies (who need clinical research which supports the use of their products), and the medical specialists (who are more likely to be paid for performing medical procedures that are supported by clinical research). In writing about such data-spinning abuses, DrRich has particularly targeted his own Cardiology Guild, but only because he knows and loves cardiologists the best. He suspects that other specialists are doing exactly the same thing.

While DrRich has used reasonably gentle humor (laced, to be sure, with sarcasm and irony) to criticize doctors and their industry collaborators for twisting clinical data to their own ends, others have expressed the same concerns in much more indignant terms, and have threatened to employ professional sanctions, civil and criminal penalties, and everlasting perdition, to curtail such behaviors. Read more »

*This blog post was originally published at The Covert Rationing Blog*

Comparative Effectiveness Research Costs More Than Most Realize

I just finished our first day at the Principle Investigator Meeting for the launch of the Catheter Ablation Versus Anti-arrhythmic Drug Therapy for Atrial Fibrillation (CABANA) trial in Philadelphia today. The trial is a 3000-patient patient trial performed at 140 centers around the world and jointly sponsored by the National Heart, Lung, and Blood Institute (NHLBI), a component of the National Institutes of Health (NIH), and industry (St. Jude Medical and Biosense Webster).

The trial will randomize 3000 previously untreated or incompletely treated patients at high risk of cardiovascular complications in the trial to two arms: 1500 patients to catheter ablation as primary therapy of atrial fibrillation and the other 1500 patients to conventional medical therapy with rate control or rhythm control strategies to determine if catheter ablation is superior to medical therapy at reducing total mortality (the primary endpoint). Secondary endpoints of a composite endpoint of mortality, disabling stroke, serious bleeding, or cardiac arrest will also be studied.

If done properly, this study stands to be a landmark trial for the field of cardiac electrophysiology and has huge ramifications for the treatment of patients with atrial fibrillation. Also, it doesn’t take a lot of rocket science to know that the government will be looking closely at the results of this trial to determine which treatment strategy will receive government funding. Read more »

*This blog post was originally published at Dr. Wes*

Who’s Against Comparative Effectiveness Research?

DrRich’s valued colleague R. W. Donnell, who writes Notes From Dr. RW, has responded to a recent post in which DrRich bravely came out in favor of Comparative Effectiveness Research, even at the cost (DrRich asserted) of alienating the majority of the more conservative-leaning components of his readership.

Dr. RW, noting DrRich’s claim that conservatives have laid out a formal policy of opposition to CER, says:

“OK, stop. Where are these people, conservatives or those of any ilk, who have taken a position against CER? Dr. Rich cites groups who are skeptical and very concerned about the new political agenda for CER, not CER itself.”

Dr. RW is, of course, correct. Research that compares the relative effectiveness of medical procedures or treatments is not only inherently a very good thing, but also is a form of research that has a long and proud history.  Healthcare would be an even more dire activity than it is today without the large body of research that guides physicians in making recommendations to their patients when more than one option is available. So yes, comparative effectiveness research is obviously a valuable and time-honored endeavor, and for anyone (conservatives or anyone else) to come out against it would be akin to coming out against babies, or bunnies. (Though, as one whose effort to grow vegetables has been severely challenged each year by a pride of aggressive rabbits, DrRich, as it happens, is indeed against bunnies.)

So, to reiterate, neither conservatives nor anyone else are really against comparative effectiveness research, just as Dr. RW asserts.

What they are against is Comparative Effectiveness Research. They are against a new government bureaucracy that sets the CER agenda, whose stated goal is to create a more efficient and less expensive healthcare system, and that will have the authority to determine what gets reimbursed and what doesn’t.

Dr. RW has made it plain that he is not confused about the following point, but many are: There is a difference between comparative effectiveness research (whose unambiguous goal is to compare the clinical effectiveness among different treatment options, so as to offer physicians objective guidance in making clinical decisions, and which is as unassailable as babies and bunnies), and Comparative Effectiveness Research (which is to be operated by a new government bureaucracy, whose agenda regarding what kind of effectiveness is actually to be compared is intentionally ambiguous).

The ambiguity of CER (as compared to cer) was made clear recently when Peter Orszag testified on behalf of the administration before the Senate Finance Committee. When queried by skeptical Republicans on the ultimate goal of the proposed CER board, Mr. Orszag was evasive. Specifically, when asked by Senator Kyle (R-Arizona) whether the CER board would be empowered to make decisions on which medical services will be reimbursed, Mr. Orszag finally replied, “Not at this point,”  a reply which did not alleviate the suspicions of the minority party.

To state the ambiguity more plainly, it is clear that while the CER board will mainly be concerned about comparing “cost effectiveness” (which is the only way they can potentially achieve their main goal of reducing healthcare costs), the only kind of effectiveness they are willing to discuss publicly is “clinical effectiveness.”

This studied ambiguity allows proponents of the new government plan to paint opponents of the CER board as being against the “babies and bunnies” form of comparative effectiveness research, and thus reveal those nay-sayers as being beneath contempt, unworthy of anyone’s attention. Meanwhile they will be free to advance their real “cost effectiveness” agenda.

DrRich agrees with conservatives that this kind of deceptive ambiguity is indeed contemptible. But really, it is no more contemptible than the thousands of other forms of covert healthcare rationing we see all around us. (Covert rationing inherently relies on ambiguity – saying we’re doing one thing while all the time we’re doing another.)

Having tried to clarify this distinction between cer and CER, DrRich will now repeat that his prior post was not merely to express support for the “babies and bunnies” variety. As Dr. RW points out, everybody is in favor of that kind of comparative effectiveness research.

Rather – and this is where he further jeopardizes his continued tolerance by his conservative readers – DrRich is offering his support to the other kind of CER, the kind described in the stimulus bill, which (though the administration will not say it publicly) will undoubtedly use comparative effectiveness research to perform cost effectiveness calculations, then coerce physicians, through one form of federal subterfuge and intimidation or another, to employ the least expensive therapies.  The government bureaucrats, just as they are doing today but with less muscle, will shout “quality” while enforcing “cost.”

DrRich supports this kind of CER not because it is a good thing – it decidedly is not. He supports it because here is a form of covert rationing that will at last effect everyone, and will be so blatant that after a time even us Americans will no longer be able to ignore it, try as we might.  DrRich believes that relatively soon, we would notice that here is a cadre of unelected bureaucrats rationing our healthcare – determining which of us lives and dies – through some opaque process, and lying to us about it the whole time. He believes this to be the pathway most likely to get the American people to finally face the fact of healthcare rationing, and to goad them into an open debate on the best and least harmful way to accomplish it.

Go ahead. Call him a cock-eyed optimist.

*This blog post was originally published at The Covert Rationing Blog*

Comparative Clinical Effectiveness Research: How Will It Impact Healthcare?

When I first heard about the new emphasis on comparative clinical effectiveness research (CCER) in Obama’s economic stimulus bill I thought, “Thank goodness! Maybe now science will truly regain its rightful place and we’ll end the CAM, ‘me-too’ drug, and excessive-use-of-technology madness that is wasting so much money in healthcare.” In fact, I was so excited about the new administration’s apparent interest in objective analysis of medical treatment options, that I intended to write a jubilant blog post about it. However, as with most things that seem black and white at first glance, further analysis reduces them to shades of gray.

What Is Comparative Clinical Effectiveness Research?

The new economic stimulus bill, also known as The American Recovery and Reinvestment Act (ARRA) includes 1.1 billion dollars for clinical comparative effectiveness research. Interestingly, CCER is not defined in the bill though AHRQ describes it this way in their glossary:

“A type of health care research that compares the results of one approach for managing a disease to the results of other approaches. Comparative effectiveness usually compares two or more types of treatment, such as different drugs, for the same disease. Comparative effectiveness also can compare types of surgery or other kinds of medical procedures and tests. The results often are summarized in a systematic review.”

Any mention of “comparative cost effectiveness” or value-based language is notably absent.

How Does It Work?

The government’s new CCER initiative will be administered through a Federal Coordinating Council for clinical comparative effectiveness research. The FCC consists of a group of 15 federal employees, half of whom “must be physicians or other experts with clinical expertise.” [Meaning, none have to be physicians.] Some have suggested that the FCC is the first step toward an organization modeled after Britain’s National Institute of Health and Clinical Excellence (NICE). NICE is regularly tasked with helping the NHS to decide which medical treatments should be available to their beneficiaries, and which should not be covered (based on their efficacy and cost).

The budget for the CCER will be divvied up as follows:

400 million – left to the discretion of the Secretary of HHS with 1.5 million to go to the Institute of Medicine for a report regarding where to focus CCER attention initially
400 million – to the office of the director, NIH
300 million – to AHRQ

Here is a quote from the ARRA bill, discussing the mechanics of CCER:

“The funding appropriated in this paragraph shall be used to accelerate the development and dissemination of research assessing the comparative clinical effectiveness of health care treatments and strategies, including through efforts that: (1) conduct, support, or synthesize research that compares the clinical outcomes, effectiveness, and appropriateness of items, services, and procedures that are used to prevent, diagnose, or treat diseases, disorders, and other health conditions and (2) encourage the development and use of clinical registries, clinical data networks, and other forms of electronic health data that can be used to generate or obtain outcomes data: Provided further, That the Secretary shall enter into a contract with the Institute of Medicine, for which no more than $1,500,000 shall be made available from funds provided in this paragraph, to produce and submit a report to the Congress and the Secretary by not later than June 30, 2009 that includes recommendations on the national priorities for comparative clinical effectiveness research to be conducted or supported with the funds provided in this paragraph…”

A Game-Changer For Pharma, Medical Technology, and Biotech

There is no doubt that CCER stands to radically change how industry does business. I anticipate that industry will develop their own internal CCER teams, and begin the process of comparing their new product (to others currently available) as early as phase 3 clinical trials. By and large, that’s probably a good thing – though there are potential unintended consequences that deserve mention.

While it’s appropriate for drug, device, and equipment manufacturers to consider whether or not their new product actually contributes something new/better to our current cadre of treatment options, there will be clear winners and losers in this game. And when companies lose, we lose companies. That’s generally not good for the economy. Manufacturers without diversified product lines are more likely to go out of business – and it will become more difficult for new players to enter the marketplace.

Although comparative clinical effectiveness research is distinct from comparative cost effectiveness research – it is likely that payers will use CCER to build their formularies. This means that even though the government (at this point in time) is not mandating coverage decisions based on CCER, health insurers are going to be using the information liberally to justify coverage preferences and even potential denials of coverage.

There’s also the question of stifling innovation. Blockbuster drugs are rarely discovered in a vacuum. They are the result of incremental steps in understanding the biology of disease, with an ever improving ability to target the offending pathophysiologic process. The first few therapies may offer marginally improved outcomes, but can lead to discoveries that substantially improve their efficacy. If an early drug is found to be only marginally better than the standard of care, an unfavorable comparative effectiveness rating could kill the drug’s sale. Without sales to recoup the R&D losses and reinvestment in the next generation of the drug, development may cease for financial reasons, and the breakthrough drug that could cure patients would never exist.

As Dr. Rich argues in his excellent blog: healthcare rationing is inevitable – but it’s more ethical to do it overtly than covertly. I would also like to suggest that insofar as physicians can be enlisted to translate CCER for patients (rather than being handed down inflexible rules from on high) and help them make the best decision for them – that would be even better. The inflexibility of national decisions about healthcare rationing does make me nervous.

What’s Good For The Geese Isn’t Necessarily Good For The Goose

First of all, most key healthcare stakeholders would like to be able to compare efficacy of one treatment option over another. Informed decision-making is hard to do when head-to-head studies are simply not available for most treatment options.

However, population-based conclusions do not always provide a clear “best choice” for individual patients. Individual genetic differences, allergy profiles, complicated drug regimens, unique constellations of diseases, socioeconomic factors, and psychological issues all influence clinical decision-making.

Dr. Nancy Nielsen recently voiced concern about CCER at the Medicare Policy Summit. She said that the AMA’s position is that CCER is for information purposes, not for coverage decisions. CCER’s goal is to help patients make informed choices, not limit their choices. I’m afraid the horse may have already left the barn on that one – but I agree with Dr. Nielsen’s sentiment. It would be wonderful if CCER could remain in its supportive role for shared physician-patient informed decision-making. My fervent wish is that in doing the right thing by the geese, we don’t kill off the occasional goose. Physicians need the flexibility to make exceptions when necessary for their patients.

Republican Unrest

A certain degree of hysteria related to CCER has recently wafted up through the hallowed halls of government. Are republicans overreacting to the bill? Maybe – though the bill doesn’t include any provisions for using CCER to mandate coverage decisions or ration care, it seems that Pete Stark has made it clear that he’d like the FCC to “direct medicine” which does kind of send a shiver down my physician spine, and provides some insight into what some democrats are hoping to accomplish with CCER – laying the foundation for future government involvement in the diagnosis and treatment of patients.

Also one particular congressional report is proving helpful in “reading the tea leaves” regarding the democrats’ plan for CCER. In describing the comparative effectiveness provision, the report states that items, procedures, and interventions “that are found to be less effective and in some cases, more expensive, will no longer be prescribed.”

While congressional reports are not binding, they do give an indication of intent.

The bottom line is that though CCER is not supposed to be used for “cost effectiveness” decisions – there’s no policy in place to protect that from happening.


Information about the comparative clinical effectiveness of treatment options is critical for the practice of evidence based medicine. Such information supports informed decision-making, and could be the single most important strategy for reducing the use of wasteful or ineffective therapies in healthcare.

On the other hand, CCER will certainly have some negative consequences, both anticipated and unanticipated. When “cost effectiveness” conclusions are drawn from clinical effectiveness data, rationing ensues, patient choices are limited, people lose their jobs, and some companies go out of business. As a recent article in the New England Journal of Medicine points out, “saying no isn’t nice.” I greet this 1.1 billion dollar initiative with muted enthusiasm.

Post Script

In my research for this blog post I came across some interesting quotes. I thought I’d add them here for your consideration:


And before you tell me we need such bills in order to be more scientific, take a minute and ask yourself just how scientific you think the government will be when it applies cost-cutting measures to medicine.  The congress is certainly a hot-bed of evidence-based legislation, isn’t it?

- Edwin Leap, M.D.

When things go wrong, which of course they will, we reach for 2 tools to try to fix them: rules, and incentives. We see this at work in our response to the current financial crisis – but the truth is that neither rules nor incentives are enough to do the job.

When we turn increasingly to rules and incentives, they may make things better in the short run but they create a downward spiral that makes them worse in the long run. Moral skill is chipped away by an over reliance on rules that deprive us of the opportunity to improvise and learn from our improvisations, and moral will is undermined by an incessant appeal to incentives that destroy our desire to do the right thing.

Without intending it, by appealing to rules and incentives we are engaging in a war on wisdom.

Don’t get me wrong, we need rules. Most Jazz musicians need some notes on the page, and we need more rules for the bankers, God knows. But too many rules prevent jazz musicians from improvising and as a result, they lose their gifts – or worse, they stop playing altogether.

We need incentives – people have to make a living. But over-reliance on incentives demoralizes professional activity. It causes people who engage in that activity to lose morale, and it causes the activity itself to lose morality.

- Barry Schwartz, Ph.D. from his lecture at TED

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