Physician Payment Sunshine Act: More marginal thinking

It never ceases to amaze me how we gravitate to the margins in our thinking: margins seem to have a centrifugal force that is nearly impossible to overcome in today's political discourse. Yet the truth almost always lies at the center, the place that does not generate Op-Eds or produce votes.

I have said this before, and I will say it again: industry-physician relationship is not all bad or all good, there is no one within this relationship that is all bad or all evil, and it does not always benefit or always harm patients! The truth, of course, is somewhere in the middle. Contrary to Stossel's thesis, there is plenty to worry about with respect to corruption promoted by the big money exchanging hands between Pharma and doctors. On the other hand, just because there are instances of corruption and its consequences, not all interaction, financial or otherwise, is counterproductive. I am the first to admit that the much-touted innovation in medicine is rare, and we have largely given up its pursuit in favor of predictable markets and returns. Yet without a robust and transparent collaboration between industry and practitioners there is not only little hope of innovation, but any innovation that may stand a chance is likely to be irrelevant.

Yes, I agree with Stossel that the new reporting regulation is overly punitive and will inevitably result in undue administrative burden. But it would be disingenuous of me to disagree with the fundamental idea that there needs to be at least some degree of transparency in the financial dealings between industry and clinicians, if only to avoid the appearance by the docs of serving two masters.

As in everything in life, the devil is in the details. And it is these details that get buried by the gravitational pull of peripheral thinking and discourse. The solution? How about we stop paying attention to these marginal fallacies and start putting our heads together for real to solve these significant problems? How about we start a rational discussion about what is best for the people and not for the corporations or the economy or reputations? The discussion has been subverted by extremism. It is time to give in to the centripetal pull of reason.      

Massachusetts' unwinnable gamble

It is ironic how, just a few days following the startling (?) confirmation by the Robert Wood Johnson Foundation-funded research that an ounce of prevention is indeed worth a pound of cure, the Massachusetts legislature with reckless abandon ushered in yet another mechanism for the erosion of public health: legalized gambling. Really, I have nothing against a little gambling. The issue is that this legislative move does not just open the door to a trickle of small local gambling operations. No, what it does is turn the crank to open a fire hose of "big box" gambling establishments descending upon our state. And it is not just anywhere in the state: it is in the Western part, far removed from the back yards of the legislators who are salivating over the projected licensing and tax revenues.

But I don't want to get into the NIMBY aspect of this misguided bill. I would rather stick to the real issue: selling us out to raise short-term revenue. The move projects 15,000 new jobs (menial with no benefits mostly), $40 million annually in tax income, on top of $85 million licensing fees from each of the three casinos, all this in addition to construction investment and the like. Already the bill allocates $50 million to overhauling healthcare reimbursements in the state. As well, there is a $25 million provision to shore up research into and prevention of problem gambling. And even people who are in staunch opposition to legalizing gambling seem appeased by this provision, which they say makes it the best bill of its kind. But we still have to ask, if prevention is better than cure, why settle for good mitigation strategies when we have the best prevention available to us already: keep casinos out!

Some of you will probably say that I am naive. After all, reason fades when we are talking about such big bucks for the state coffers. Well, just because this kind of a trade-off is something we have come to expect from our politicians does not mean that we should tolerate it. Others will bring up the old free will argument. No, I am not against people exercising their personal decision making, but haven't you read "Nudge?" We are all deeply flawed human beings, and in the face of temptation we fail miserably! And since we know that casinos increase the risk of problem gambling, why not just steer clear of them altogether? This is simply not a winnable gamble.

I hope that some of you are hearing echoes of the food-obesity debate. We deem it an individual rather than a societal problem, and look how well we have done mitigating the obesity epidemic! There is no rocket science here, and it is disingenuous to say that we do not understand the causes of obesity. Human physiology has not changed over a couple of generations, no. What has changed is our constant access to high-calorie cheap concoctions that pass for food; what has changed is our limited access to physical activity; and what has changed is the degree to which we as a society are willing to sign on to corporate and political propaganda designed to get votes and make money at the expense of our health.

So, am I shocked that this casino bill is likely to become law? Not at all. Am I surprised that the public is allowing this to happen in a pathetic perversion of personal freedom? Of course not. Am I going to shut up about what a mistake this is? You bet I am not. And in a decade I will say "I told you so." But I am sure that then, not unlike now, no one will be listening.              

Tipping a sacred cow: QI under the microscope

So much media and journal space has been devoted to financial conflicts of interest, particularly within and related to pharma and device manufacturers, that to write any more about it may be redundant. On this site we have also intermittently addressed COI from other perspectives, such as financial interest of the members of the American College of Radiology in maintaining mammography screening status quo, thinly veiled in its own version of the pernicious "death panel" language. We have also spoken a bit about the non-financial COI. And even though we are so very much aware of COI's potential to lurk around every corner, there are still some surprises.

Take the sacred cow of "quality improvement" in healthcare. Even the name, much like the "pro life" moniker, suggests that it is untouchable in its purity and nobility of purpose. So necessary is it because of the epic magnitude of morbidity and mortality attributed to healthcare itself, that the billions of dollars spent on it seem unquestionably justified. Indeed, much like our public education system, the QI movement garners higher and higher allocations simply due to the sheer face validity of the assumption that more of it is better. And the most fascinating aspect is that, in our current zeal for sensible economic allocation through evidence, QI, much like education, appears immune to scrutiny. This is the very definition of politics driving policy.

I return to the case of ventilator-associated pneumonia, or VAP, as the poster child for this movement. I have already alluded to the fact that definitionally VAP is a slippery slope: its diagnosis varies based not only on the tools used to diagnose it, but also depending on who is doing the diagnosing. Yes, indeed, what one clinician calls VAP another may call absence of VAP. I have also dissected the weak evidence behind some of the strongest purportedly evidence-based recommendations aimed at VAP prevention. But what if VAP itself is the wrong endpoint? What if we are spending untold dollars and other resources on a futile pursuit?

Do you feel yourself bristling yet? If you said "yes", it is a normal response I get from my colleagues and people who read my scholarly papers. Because how can anyone be against QI? Well, I am not against QI. I am simply against sanctifying QI as a sacred cow and thus shielding it from a sensible and rational evaluation.

So, if you are over the initial shock, allow me to explain myself. I am sure you have heard of surrogate endpoints. Here is a definition from Wikipedia:

In clinical trials, a surrogate endpoint (or marker) is a measure of effect of a certain treatment that may correlate with a real clinical endpoint but doesn't necessarily have a guaranteed relationship. The National Institutes of Health (USA) defines surrogate endpoint as "a biomarker intended to substitute for a clinical endpoint".^[1][2]

Surrogate markers are used when the primary endpoint is undesired (e.g., death), or when the number of events is very small, thus making it impractical to conduct a clinical trial to gather a statistically significant number of endpoints. The FDA and other regulatory agencies will often accept evidence from clinical trials that show a direct clinical benefit to surrogate markers. ^[3]

This begs the question of what constitutes a "real" clinical endpoint. Well, in my simplemindedness I think of them as endpoints that matter to the patient or in the long run. So, death, disability, quality of life, functionality, these are the real endpoints. Something that alters one's life or threatens it is a real endpoint. Thus, blood pressure and cholesterol are surrogate endpoints, since they usually, but not always, correlate with the risk of a myocardial infarction or death. But what if such a correlation did not exist? Furthermore, what if a cholesterol level was measured with, say, tea leaves, and therefore was subject to a tremendous variation in detection? Would we then spend hundreds of billions of dollars on trying to alter this factor or would we calmly and rationally walk away and look for something that truly impacts the real outcome of a heart attack or death? I think I am making my point fairly clearly.


Let me explain why I think that VAP is but a surrogate outcome, and, given its diagnostic challenges, not a sensible one in the least. VAP by definition occurs in patients on mechanical ventilation (breathing machine), whose quality of life is fairly badly damaged in the short term. The literature would suggest that not all VAP impacts mortality adversely, but some forms of VAP indeed do, particularly VAP that develops late in the course of illness. So in this VAP does correlate with a real endpoint. Also, there is very little doubt that getting VAP prolongs one's dependence on mechanical ventilation, and increases the duration of the stay in the ICU and hospital overall. So, this can be considered not a very good, albeit real, outcome. An additional point to remember is that VAP engenders the use of additional, usually broad spectrum, antibiotics, putting both the individual and the society at risk for such unwanted consequences as the emergence of highly resistant microorganisms.


So, even though VAP is a surrogate endpoint, it certainly seems to fit the bill for something we would want to prevent. But here is the monkey wrench in this argument: what seem to be great surrogate endpoints do not always end up correlating with clinical reality. The association of VAP with morbidity and mortality has been detected in mostly retrospective observational studies. Trials of VAP prevention rarely, if ever, report any endpoint other than VAP. And, given how elusive VAP diagnosis is, there is plenty of room for gamesmanship so pervasive in the real world to make any data fit our preconceived hypotheses and political needs. 


So, what is my point? My point is that if QI wants to be a science, it needs to be subject to the same rules that all other science is guided by. Since we do not even know how much money we are spending on the ubiquitous QI efforts (likely hundreds of billions), and since we are not sure what they are accomplishing (see my many prior posts on the lack of validity of current claims in VAP prevention), we need to pause and ask ourselves whether the cheering alone justifies such an investment. I hate to say it, but can we really trust those with most to lose, financially and politically, if in reality QI does little more than lather the masses, to be the oracles of truth about the results of these efforts? The cognitive biases alone should disqualify them from being the arbiters of their own success. So, if we do not want to continue to indulge the principle of diminishing returns in QI, we need to take a sober look at what we have invested and what this investment has accomplished. Then and only then can we claim to practice evidence- rather than politics-or dogma-based policy.

Whose perspective?

After a long hiatus filled with travel, work and lack of inspiration to write anything, I have chosen this arguably hottest day of the year to venture forth again. But I will make this brief, as it seems that everything that needs to be thought and said has already been thought and said. Yet who is listening?

Anyhow, to suspend my natural cynicism, I want to talk about perspective. No, not the perspective that makes parallel lines converge in the distance, but the one that gets lost in many of our political, civic, business, and, yes, even scientific discussions. I am talking about my perspective, your perspective, societal perspective, etc. I was inspired to write this because of these tweets by Gary Schwitzer to Kaiser Health News about a story on their web site:

Linking out to the story, I learned that a consulting arm of Disney is teaching hospitals about hospitality. Since there is going to be a financial incentive for these hospitals to deliver good customer service, many are feeling that an investment in this type of training will help them maximize these new reimbursements. Hurrah and ta-da! 

Well, Gary likes to burst these one-sided bubbles, and so he rightfully asked about the costs. What was baffling to me was the response by the KHNews who did not seem to appreciate the importance of reporting the costs or the various perspectives that these costs represent. So, this seemed like a teachable moment, and here is the teaching.

In outcomes research, we are always interested in understanding the perspective for both the costs and the benefits of interventions. In health outcomes these perspectives are broadly represented by the patient, the provider, the hospital, the payer, the employer, the manufacturer, the society, to name a few. These are just some of the examples of the usual stakeholders involved in healthcare decisions. Because our healthcare is such a fragmented disaster, many of these perspectives find themselves at odds with one another. Just think of the patient who wants to get what she perceives as a life-saving treatment that in reality has a 1% chance of helping at a cost of $600,000 per treatment course. From her perspective, since she is insured, this investment is well worth the cost. For a payer, however, this means $600,000 (multiply this by 100 in order to determine the cost to save 1 life) that cannot be spent on something else that can help more people more predictably. And if this payer is the taxpayer, the societal perspective enters the picture, where we have to decide what amount of money is worth spending on possibly saving one life -- is $60 million reasonable? Perhaps. But these are not simple questions, and, as such, do not have simple answers. In addition, all conversations that we hear or engage in have multiple perspectives. This is why a black-and-white approach is so divisive: it generally emphasizes two diametrically opposed perspectives. 

So, next time you hear about death panels or Mickey Mouse teaching hospitals how to maximize their revenue, consider the broader implications from may different perspectives. Chances are you will find yourself agreeing with more than one point of view. And when this happens, you will know that you have learned an important lesson and can now start engaging in more nuanced and thus productive debates, many of which will shape our society's future.

h/t to @garyschwitzer for this KHN story      

Data mining: It's about research efficiency.

I have taken a little break from my reviewing literature series -- work has superseded all other pursuits for a little while. But I did want to do a brief post today, since this JAMA Commentary really intrigued me.

First thing that interested me was the authors. Now, I know who Benjamin Djulbegovic is -- you have to live under a rock as an outcomes researcher not to have heard of him. But who is Mia Djulbegovic? It is an unusual enough surname to make me think that she is somehow related to Benjamin. So, I queried the mighty Google, and it spat out 1,700 hits like nothing. But only one was useful in helping me identify this person, and that was a link to her paper in BMJ from 2010 on prostate cancer screening. On this paper (her only one listed on Medline so far), she is the first author, and her credentials are listed as "student", more specifically in the Department of Urology at the University of Florida College of Medicine in Gainesville, FL. The penultimate author on the paper is none other than Benjamin Djulbegovic, at the University of South Florida in Tampa, FL. So, I am surmising from this circumstantial evidence that Mia is Benjamin's kid who is either a college or a medical student. Why does this matter? Well, there seem to be so few papers in high impact journals that are authored by people without an advanced degree, let alone in the first position, that I am in awe of this young woman, now with two major journals to her name -- BMJ and JAMA. This is evidence that parental mentorship counts for a lot (assuming that I am correct about their relationship). But regardless, kudos to her!

Secondly, the title of the essay really grabbed me: what is the "principle of question propagation", and what does it have to do with comparative effectiveness research (CER) and data mining? Well, basically, the principle of question propagation is something we talk about here a lot: questions beget questions, and the further you go down any rabbit hole, the more detailed and smaller the questions become. This is the beauty and richness of science as well as what I have referred to as "unidirectional skepticism" of science, meaning that a lot of the time, building on existing concepts, we just continue down the same direction in a particular research pursuit. This is why Max Planck was right when he said

A new scientific truth does not triumph by convincing its opponents and making them see the light, but rather because its opponents eventually die, and a new generation grows up that is familiar with it.

So, yes, we build upon previous work, and continue our journey down a single rabbit hole our entire career. Though of course there are countless rabbit holes all being explored at the same time. It is really more of a fractal-like situation than a single linear progression. What is clear, as the authors of the Commentary point out, is that this results in the ever-escalating theoretical complexity of scientific concepts. What does this have to do with anything? This, the authors state, argues for continued use of theory driven hypothesis testing, given that medical knowledge will forever be incomplete. And this brings them to data mining.

Here is where I get a little confused and annoyed. They caution the powers that be from consigning all clinical research to data mining, at the expense of more rigorous studies to pursue hypothesis testing. They argue that mining data that already exist is limiting precisely because it is constrained by the scope of our current knowledge, and that we cannot use these data to generate new associations and new treatment paradigms. They further state that emerging knowledge will require updating these data sets with new data points, and this, according to the authors

...creates a paradox, which is particularly evident when searching for treatment effects insubgroups—one of the purported goals of the IT CER initiative. As new research generates new evidence of the importance for tailoring treatments to a given subpopulation of patients, the existing databases will need to be updated, in turn undermining the original purpose to discover new relationships via existing records.

Come agin? And then they say that "consequently, the data mining approach can never result in credible discoveries that will obviate the need for new data collection". Mmhm, and so? Is this the punch line? Well, OK, they also say that because of all this we will still need to do hypothesis testing research. Is this not self-evident?

I don't know about you, but I have never thought that retrospective data mining would be the only answer to our research needs. Rather, the way to view this type of research is as an opportunistic pursuit of information from massive repositories of existing data. We can look for details that are unavailable in the interventional literature, zoom in on the potentially important bits, and use this information to inform more focused (and therefore pragmatically more realistic) interventional studies.

Don't take me wrong, I am happy that the Djulbegovics published this Commentary. It is really designed more as an appeal to policy makers, who, in their perennial search for one-size-fit-all panaceas, may misinterpret our zeal for data mining as the singular answer to all our questions. No indeed, hypothesis testing will continue. But using these vast repositories of data should make us smarter and more efficient at asking the right questions and designing the appropriate studies to answer them. And then generate further questions. And then answer those. And then... Well, you get the picture.