Medicine as the trolley problem

Are you familiar with the trolley problem? It is an ethics dilemma first formulated by the great Philippa Foot as a part of a series of such dilemmas. Her formulation goes roughly like this. Imagine there is a tram hurtling down a track. If it keeps going straight, it will hit and kill 5 people who are working on that track. The conductor is able to throw a switch and divert the train to another part of the track, where 1 single worker will be killed by the trolley. The question is what should the conductor do? Most people when asked respond that yes, he should throw the switch and sacrifice 1 life to save 5. After all, the net benefit is n=4.

There are literally thousands of alternative formulations of this problem, but one of them from the philosopher Judith Jarvis Thomson merits special consideration. The problem starts out similarly, with 5 lives on a track in potential peril. The vantage point and the solution are quite different, though. Now there is a bridge over the rail track, and a very large man is looking at the tracks from the bridge. One way to stop the train is to throw a heavy object in its path, like this large man, for example. You are on the bridge standing behind the man. Would you be justified in pushing him off the bridge in front of the tram to meet his death in order to spare the 5 workers down the tracks? Most people when faced with this formulation say an emphatic "no." This is somehow puzzling, since the net benefit is the same, n=4, as in the original Foot formulation.

Philosophy professors have puzzled over this difference for decades, and there are several potential explanations for why we respond differently to the two scenarios. One explanation has to do with the proximity of the operator (conductor in the first case and the person doing the pushing in the second) to the sacrificial lamb -- in the first case one is enough removed from the action of killing by merely redirecting the tram, whereas in the second the action is, well, more active, and the operator is actually pushing an innocent person to his death.

Though in some ways the scenarios seem to bear no practical distinction from one another, we see the morals and ethics of each differently. This difference in the view point is instructive to the field of medicine, where it has implications to how policy relates to the individual patient encounter. Here is what I mean.

Suppose you are a policy maker, and you recommend that every woman at age 40 start to receive an annual screening mammogram to reduce deaths from breast cancer. At the population level, if we screen 1,000 women for about 30 years, we will save approximately 8 of them from a breast cancer death. (Yes, it's 8, not 80, and not 800). At the same time, among these 1,000 women, there will be over 2,000 false alarms, and over 150 of these will result in an unnecessary biopsy. Some of these biopsies will incur further complications, though currently we  do not seem to have the data to quantify this risk. But what if even one of these biopsies were to lead to death of or another dire lasting complication in a woman who turned out not to have cancer? And by the way the accounting is not all that different when applying the new USPSTF mammography screening recommendations. Well, then we have the trolley problem, don't we? We are potentially sacrificing 1 individual to save 8. And who does the sacrificing is where the variations of the trolley problem come in.

Payers levy financial penalties on primary care physicians when they fail to comply with screening recommendations in their patient panels. The payer certainly sees this issue as the original formulation of the problem: Why not throw this financial switch to achieve net life savings? But for a clinician who deals with the individual patient this may be akin to pushing her over the bridge toward a potentially fatal event. Because we don't have a crystal ball, we cannot say which woman will die or incur a terrible complication. But the same population data that tell us about benefits must also give us pause when reflecting on the risks. Add the ubiquitous uncertainty (and lack of data) into this equation, and the implications are even more shocking. So, while making policy recommendations based on population data is sensible, policing uniform application of these recommendations to individual patients is fraught: of course, clinicians and patients need to be cautious about making individual decisions even when in population data benefits outweigh risks.

On the surface risk-benefit equations for many interventions may appear favorable, leading to blanket policy recommendations to employ them on everyone who qualifies. In the office, the clinician, caught in a tug of war between mountains of new literature and the ever-shrinking appointment times, is hard-pressed to take the time to consider these recommendations in the context of the individual patient. And furthermore, financial incentives from payers act as a short-hand justification, a "nudge," for doing as recommended rather than for giving it thought. So, who must look out for the patient's interest? The patient, that's who. Who understands the patient's attitude toward the risks and the benefits? The patient, that's who. Who now has to be responsible for making the ultimate informed decision about which track to stand on? The patient, that's who.

For me the trolley problem gives clarity to the reservations that I walk around with every day. I have done a lot of soul searching about why it is that, even if the benefits seem to outweigh the risks, I am still more often than not skeptical about whether a particular intervention is right for me. And since every intervention in medicine has a real risk, though mostly quite low, of going terribly awry, my skepticism is justified. This is my approach to evaluating these risks and benefits, based on my values and my understanding of the data as it is today.

What's the answer to this ethical conundrum in medicine? I cannot see that policy makers will stop throwing the switch in the near future, and so as a society we will be forced to accept the tram's collateral damage. And while this may make sense in an area such as vaccination, where thousands of lives can be saved by sacrificing a very few by throwing the switch, in most everyday less clear-cut medical decisions the answer is less clear-cut. Will doctors rebel against being forced to throw some patients on the tracks in order to save some marginally larger number of others? I don't think that they have the time or the energy or the incentive to do this, since the framing of the switch-throwing is through the rhetoric of "evidence." Right or wrong, doctors are shackled by the stigma of ignorance that comes with not following evidence-based guidelines, and this may act to perpetuate blind compliance. This leaves the patients, for some of whom the right thing will be just to get themselves off the tracks altogether, far away from the hurtling trolley until its brakes are fixed.                        

If you like Healthcare, etc., please consider a donation (button in the right margin) to support development of this content. But just to be clear, it is not tax-deductible, as we do not have a non-profit status. Thank you for your support!

Healthfinder.gov: Education or indoctrination?

Ever heard of healthfinder.gov? It's a web site from the US Department of health and Human Services

...where you will find information and tools to help you and those you care about stay healthy.

Sounds like a laudable goal, right? Great! Now, help me! Here is the "help" that I found when I went to the page called "Colorectal Cancer Screening: Questions for the doctor":

What do I ask the doctor?

It helps to have questions for the doctor written down ahead of time. Print out these questions and take them to your next appointment. You may want to ask a family member or close friend to come with you to take notes.

So far so good. But here is the list that follows:

• What puts me at risk for colorectal cancer?

• When do I need to start getting tested?

• How often do I need to get tested?

• What screening test do you recommend? Why?

• What’s involved in screening? How do I prepare?

• Are there any dangers or side effects involved?

• How long will it take to get the results?

• What can I do to reduce my risk of colorectal cancer?

Note the wording: "When do I need to start getting tested?" "How often do I need to get tested?" And these "needs" come well before the "why?" In fact, the "why" never really comes. The oblique "why" about which test is recommended is too little too late. The real "why" is why, or even whether, I need to get tested in the first place. I am happy to see a question on the dangers of screening, but again it leaves plenty of room for the clinician to minimize and patronize.

The list of questions is built upon one (erroneous) assumption: Everyone is bound to perceive the risk-benefit equation of colorectal cancer screening the same way. We know this is false, and each person needs to make an individual decision based in what we know today and according to the values he/she places on the outcomes. The way the questions are written, they simply reinforce the bullying attitude of the screening bias, making those who swim against this tide feel irrational and unreasonable. But may I point out that some of us spoke out against universal mammography screening even before it became the main-stream recommendation? So perhaps there are good reasons to be more cautious with screening for everything, even colon cancer.

Science evolves, our knowledge evolves. What we think we know today will be modified tomorrow. I take a strong exception to this dogmatic and one-sided formulation of how to have a discussion about testing whose risk and benefit profile may not (and should not) elicit the same unbridled enthusiasm from everyone. So please, healthfinder.gov, rethink your "helpful" questions so as to educate, rather than indoctrinate.

Hat tip to @DCPatient for pointing me to this page  

If you like Healthcare, etc., please consider a donation (button in the right margin) to support development of this content. But just to be clear, it is not tax-deductible, as we do not have a non-profit status. Thank you for your support!

Fast science: No time for uncertainty

Reading Barbara Ehrenreich's "Bright-Sided" has been liberating in that is has given me permission to let my pessimistic nature out of the closet. Well, it's not exactly that I am pessimistic, but certainly I am not given over to brightness and cheer all the time. My poison is worry. Yes, I am a worrier, in case you had not noticed. So, imagine how satisfying it is for me to find new things to worry about. As if climate change were not enough, lately I started to worry about science.

No, my anxiety about how we do clinical science overall is not new; this blog is overrun with it. However, the new branch of that anxiety relates to something I have termed "fast science." Like fast food it fills us up, but the calories are at best empty and at worst detrimental. What I mean is that science is a process more than it is a result, and this process cannot and should not be microwaved. Don't believe me? Let me give you a couple of instances where slow science may be the answer to our woes.

1. Lies and damned lies
Remember this story in the Atlantic that rattled us with its incendiary message? Researcher John Ioannidis has been making headlines with his assertion that most, if not all, of what we know in medicine is in doubt, given how we do and publish research. And how we do and publish research has everything to do with the speed of "progress." Academic careers are made with positive results, to sell news the media demand positive results, and to respond to this demand academic journals prefer only to publish positive results (this last phenomenon is referred to as "publication bias," and is something Ben Goldacre rails against at length). A further manifestation of this fast science is that "no replicators need apply." I am, of course, referring to an extension of the publications bias, whereby journals are not interested in publishing even a positive study that replicates a previous finding -- this is simply not sexy. Thus, results have to be quick and positive to grab a share of our attention and sell academic prestige, journals and news.

2. Science output to drive business profits
In his book Supercapitalism, Robert Reich describes the growing demand by investors over the last several decades to squeeze ever-growing profits. It is clear that this chase after short-term profits has resulted in job loss in the US through outsourcing, the widening of the economic gap, and even the crash of the world economy following the collapse of the mortgage-backed securities house of cards. Much of the profit can be counted on to come through scientific innovations which may or may not improve our quality of life.
In medicine, where scientific progress is applied to our fragile being, being reasonably sure of our findings seems pretty important. Yet speed is once again the order of the day. I will grant you that speed is of importance in such diseases as advanced cancer, for example, where we may and should accept a level of uncertainty that we would ordinarily run away from in other circumstances. But doesn't it make sense to be much more cautious before broadly accepting an intervention that happens before one gets sick, one that is meant to diagnose either early disease or a precursor to one? Should we not demand slower science before we allow anyone to medicalize such normal events in life as menopause and aging? Should this caution also not apply to screening for diseases that may or may not impact us in the long term, yet the chase could hurt us substantially in the immediate future?
But this is not the way to stimulate the economy or to make a profit. The half-life of a medical device, for example, is less than 1 year. After that a new "improved" version of the device is expected, whether it does or does not improve outcomes. For decades we were told to get screening mammography after the age of 40, only to find out now that the risks of this may well outweigh its benefits for many. The American Lung Association has just endorsed CT screening for lung cancer among current or former heavy smokers, yet the jury on its risk-benefit-uncertainty equation should still be in the thick of deliberations.

3. Science denialism
We hear a lot about how people are turning away from science. The state of Tennessee is about to descend back into the dark ages when superstitions instead of scientific theories dominated the classroom. A strong and largely anti-scientific lobby wants to bury any mention of human-driven climate change; fortunately, it looks like they are not succeeding. The anti-vaccination groups are getting more instead of less vocal following repeated debunking of any link between vaccination and autism. Science denialism is so rampant that there was even a need for a conference on how to address it. What gives?
While blaming everything on fast science alone may be reductionist, fast science in the setting of our growing societal innumeracy is a recipe for disaster, as we are seeing unfold. Our schools have failed spectacularly in their duty to educate kids about the process of science, while at the same time arming them with the "single-right-answer-to-every-question" attitude toward knowledge. This pernicious combination, along with the publication and reporting of sexy science at the expense of the more thorough analytic and introspective approach, seals the impression that the roller coaster of scientific knowledge represents not the very essence of how science should be done, but that science (and scientists) has failed.
Is slow science the answer to this fiasco? Only in part, I am afraid. Without altering fundamentally how we teach science at all levels, it would not be the cure, even if it were possible to execute. No, I am afraid that without teaching what science is, it is not even possible to get it to slow down.

Let me reiterate: the pace of scientific discovery is slow. This does not mean that we need to hide every step of it from view until we get the results that we deem worthy of sharing. On the contrary, I agree with those who think that sharing at the more interim steps can only improve what we do. Yet the innumeracy, fame and fortune are forces that put such free sharing in peril by misrepresenting it as the final answer to everything. And when the answer is changed, which is not only expected, but indeed desired in scientific pursuits, the public opinion punishes science.

Let me end with a quote I read on one of my favorite web sites, Brain Pickings, in a review of the book boldly entitled Ignorance: How It Drives Science:

Are we too enthralled with the answers these days? Are we afraid of questions, especially those that linger too long? We seem to have come to a phase in civilization marked by a voracious appetite for knowledge, in which the growth of information is exponential and, perhaps more important, its availability easier and faster than ever.

[...]There are a lot of facts to be known in order to be a professional anything — lawyer, doctor, engineer, accountant, teacher. But with science there is one important difference. The facts serve mainly to access the ignorance… Scientists don’t concentrate on what they know, which is considerable but minuscule, but rather on what they don’t know…. Science traffics in ignorance, cultivates it, and is driven by it. Mucking about in the unknown is an adventure; doing it for a living is something most scientists consider a privilege. 

So, let's celebrate uncertainty. Let's take time to question, answer and question again. Slow down, take a deep breath, cook a slow meal and think.

If you like Healthcare, etc., please consider a donation (button in the right margin) to support development of this content. But just to be clear, it is not tax-deductible, as we do not have a non-profit status. Thank you for your support!

Five ways to tame the risk-benefit-uncertainty troika

There was a story on NPR this morning that sent me in a radical direction. It discussed the increase in use of brachytherapy for localized breast cancer. The idea is that this is a concentrated dose delivered much more locally and rapidly (over 5 days) than the conventional external beam radiation (over 6 weeks). The issue is that it has not been tested rigorously in a randomized controlled trial yet, and some oncologists are concerned about its outcomes as they compare to the conventional approach. One of the concerns stems from an increase in the rates of subsequent mastectomies, which are double in brachytherapy relative to conventional. At the same time, there are clear benefits, not the least of which is the period of exposure and the hassle associated with daily trips for radiation for 6 weeks.

Several points popped up in my head in response to the story:
1. When discussing this predominantly women's disease, the expert voices mostly heard from were male (5 of the 6 doctors quoted). Does this matter? Not sure.
2. The priorities addressed by the experts were the traditional outcomes -- survival, recurrence, metastasis. The priorities described by the patient were about her time and convenience today.
3. The concern about the procedure stems from the observed doubling of the need for mastectomy within 5 years among patients treated with brachytherapy, an event "rare no matter what what kind of radiation women got."

So what's my point? Do I think that more rigorous testing is not indicated? Not at all; we need a more rigorous evaluation of the technique. No, what I am wondering is at what point should a procedure like this (or any intervention, for that matter) become available to patients as an option to be considered? Should its availability be determined in a dark room by bespectacled men around a conference table, or should it be put on the menu of choices, along with its risks, benefits and uncertainties, as soon as it looks safe enough, whatever that looks like?

The larger question this raises is what is the degree of uncertainty that we are willing to accept around interventions that become available, be it a drug or a procedure or a device? How do we incorporate patients' priorities for outcomes that are important to them into these decisions? Remember ACT-UP and how they moved the FDA to make more rapid decisions about treatments for HIV/AIDS? Have we swung too far in the opposite direction today, whereby we want a virtual guarantee of safety before a technology is approved?

I offer these 5 potential question to help with making these decisions:
1. How severe/deadly is the disease in question?
2. What is (are) the known potential benefit(s) of the intervention?
3. What is (are) the known potential risk(s) of the intervention?
4. What uncertainties bracket this risk-benefit equation?
5. How does the patient feel about the extent of this risk-benefit-uncertainty balance in the context of her condition?

I think that the first four are the questions that the FDA struggles with every day. They are the gatekeepers for the availability of new technologies, and, therefore, for the relevance of the fifth question. What I am wondering is whether it is not better to start bringing a lot more of the public perspective to the discussion much earlier, so that the patient can have the option of evaluating more choices sooner. I know I may be treading on thin ice here, but I am ignoring any market forces or special interests for the moment. The question I am asking is "In the best of all possible worlds, where no one is trying to sell you anything, when is the best time to give the patient an opportunity to accept or reject an intervention, given the risk-benefit-uncertainty profile?"

Bottom line: There are no guarantees. Just because something is available on the market does not mean that it is completely safe or completely effective. Most importantly, it does not mean that we come even close to being certain about these attributes. As a corollary, just because there are uncertainties about the risks and benefits of an intervention, does it mean that it should not be available as an option for a patient? My guess is that there is a balance of this troika of properties that may be optimal on average, but I am also guessing that that this average balance will miss a substantial volume of outliers. Just as some people thrive on the thrill of bungee jumping while others clamp down just at the mere thought of it, so some patients may surprise us with their position on this risk-benefit-uncertainty continuum.

I apologize if my argument is not clear -- I am definitely thinking about this stuff actively. The one thing I am absolutely sure of is this: Unless the public and clinicians are educated about how to have these conversations, we will always have to rely on and, consequently, blame someone else for making decisions for us.

If you like Healthcare, etc., please consider a donation (button in the right margin) to support development of this content. But just to be clear, it is not tax-deductible, as we do not have a non-profit status. 


Thank you for your support!

Tinkering with health is not a laboratory job

Do you love Brussels sprouts? I do. And broccoli, chard and kale, too. Why do I ask?

Well, last week my friend Kent Bottles did a blog post on what the future of medicine may look like according to two of our prominent medical futurists, David Agus and Eric Topol. It left me scratching my head, so I went to find Agus on the interwebz, and came upon his 2011 TEDMED talk, which can be found here. The Brussels sprouts were just the beginning of nearly 20 minutes of bewilderment. I will get to the meat (ahem) of it momentarily, but I just could not get past his assertion at around 3:48, where he states that people in their 90s do not take up healthcare resources -- no mechanical ventilation, no weeks in the ICU -- and that they "die with dignity from whatever process ails them at that point." Really? In what country?

Here is the reality -- you can consult the Dartmouth Atlas for a lot of this info, but many other sources exist as well. There are approximately 2.5 million deaths in the US annually. Fully 1/3 of them occurs in the hospital, more that 1/2 of which involve ICU care. And incidentally, not to get all cost-conscious or anything, but 80% of all of the associated costs were due to ICU care. But wait, you say, this is not necessarily people in their 90s, right? OK, let's take it down a layer.

Among the 1/3 of all the annual deaths that occur in the hospital, nearly 3/4 are among the Medicare population, or those who are 65 years old or older. Furthermore, according to none other than the Dartmouth Atlas, up to 1/4 of all Medicare enrollees spend 1 week in the ICU in the last 6 months of life. OK, then. So, where are the data that old age is associated with low medical costs? Not here, that's for sure.

After this dubious beginning, Dr. Agus states the undeniable: humans are complex systems, and we need to think of them as such. Additionally, he advocates skepticism because much of what is done in medicine is not based on "true" data. OK, I can certainly go along with that. Then he goes astray. Here is how.

At around 10:30 he gets into technological solutions. You may be surprised that I do not fundamentally disagree with technology as the answer to disease. No, I disagree that technology is the answer to health -- this is where Brussels sprouts come in. At about 11:00 he starts to talk about aspirin and all the fantastic health benefits that are associated with it -- here is a screen shot of his slide:

And he suggests that aspirin should be mandatory, and that society should not have to pay for these diseases that develop due to what? Aspirin deficiency? Now, as veteran readers of my blog, do you see something funny about this slide? Is there something missing? Yes, you are right, where are the references for these statements? No, I did not cut off the bottom -- there are no studies referenced. One other critical piece of information is missing: whenever data on benefit are presented, data on risks must also be presented. Where are they? So, yes, be very skeptical. One more picky point: he brings up Michael Dell at 11:30 or so, telling the story of how his employees who smoked had to pay higher insurance premiums. By extension, Dr. Agus contends that we should charge higher premiums to employees who do not comply with aspirin. Unfortunately to get into the full controversy about the role of aspirin in these conditions is way beyond the scope of this post. But do let me give you a taste of what a balanced discussion of aspirin as a prevention for heart disease looks like -- here the Mayo Clinic web site is exemplary in providing a well-informed approach. Around 12:00 Agus builds the same argument for statins. And then he knocks down vitamins and supplements and suggests that people who take them should be penalized with higher premiums. OK, in my humble opinion this branch of inquiry has always been a fool's errand in a society that is fairly well fed, but higher premiums? Come on. I will not belabor this. And finally he sprinkles his comments with a few words on the microbiome. 

So what does Dr. Agus seem to say overall? My impression is that he thinks that we should tinker with maintaining our health by looking to manufactured drugs, such as aspirin and statins, as well as whatever we learn from the microbiome (more drugs?). What is missing here is the discussion of the risks vs. the benefits of such tinkering in healthy people. What is also missing is data to back up some of his fundamental assertions (see above). 

So, final words? Technology is not the enemy. If used correctly it can help us understand and cure disease. Tinkering with the healthy human is the job of evolution, not the laboratory. The potential "unintended consequences" of such tinkering are too colossal to ignore.  

          

    

H1N1 vaccine and the burden of proof

I am following with great interest all of the reports, both scientific and popular, on the reluctance to subject oneself to the H1N1 vaccine. Yesterday's WaPo reported that

A nationally representative poll of 1,042 adults released Friday by the Harvard School of Public Health found that only 40 percent were sure they would receive the vaccine and that about half were certain their children would. Recent research by the University of Michigan and by Consumer Reports yielded similar results.

Similarly, a CBS story reported that

Outside New York 's capitol building, health care workers - shouting "Give me liberty!" - vowed to fight an unprecedented order from state health officials: a requirement for every health care worker to get seasonal and H1N1 flu shots or face the possibility of being fired.

By some strange coincidence, the CBS story reports that, in any given year, the rate of voluntary vaccination against seasonal flu among healthcare workers is 40%. This begs the question: What is it about flu vaccination that engenders skepticism in over one-half of the population, whether healthcare professionals or lay people? And further, upon whom does the burden of proof in this argument fall? That is, does the government, who is mandating universal vaccination among NY healthcare providers, need to prove safety and efficacy to the satisfaction of the people or do the protesters need to prove to the government that their concerns are legitimate?

Let's take safety and efficacy. The mere fact that the vaccine has been approved by the FDA (and other regulatory bodies around the globe) should give us some comfort about its profile. Of course, we are all too aware of the recent debacles of removal of medications from the market due to safety signals, Vioxx being the most memorable of all. This drug, having undergone the FDA's scrutiny, was approved and then pulled because of an unacceptably high rate of cardiac events detected in treated patients in post-marketing studies. This has unquestionably colored the public's attitude toward drugs, even those receiving the FDA's blessing.

But is there something special about vaccines? From where I stand, vaccines are subject to an exaggerated suspicion on the part of the public, more so than other therapies. Well, my guess is that because most of the time the population for these injections consists of young and healthy individuals, the risk-benefit balance has to be much more weighted to the benefit side. What I mean is that the more seriously ill the person is, the more risk we are willing to accept for a small incremental benefit. Take cancer chemotherapy: when the alternative is certain death without a drug, the often severe side effects of that drug are acceptable given that it improves one's survival odds. Not so among the young and healthy. It would be absurd to accept the same risk profile from a vaccine that we would in a chemotherapy drug. OK, so there is the risk-benefit profile.

One important question that goes mostly unaddressed in safety discussions is that of time. That is, risk-benefit over what period of time? The flu vaccines, whether seasonal or H1N1, are developed annually and over a fairly contracted timeline. Can we possibly fathom the potential long-term risks of these vaccines in someone who is 20 years old today? Our drug approval process gives us a false comfort in that it only examines drug profiles in the short term. However, the human body is a complex network, and an exposure today may not make itself known until 20 or 30 years from now -- look at cigarettes and cancer. Further complicating this issue are several factors. First, we do not have a great system in the US for tracking adverse events, be it from drugs or from vaccines. The systems are voluntary, and for this reason incomplete and fraught with bias. Second, the task of connecting the potential exposure to an outcome separated by 20 or 30 years is a highly complex cognitive exercise. This task is made even more challenging if the exposure rate to the agent in question, here the vaccine, is very high. Under these circumstances, it may be difficult or impossible to connect the outcome to the exposure causally.

So, where does the burden of proof lie? The fact that even healthcare professionals have a hard time understanding this balance speaks to the complexity of the issue. As for the H1N1 mandatory vaccination of the healthcare workers, the added potential risk to the society may make the explicit risk-benefit balance easier to derive, given the immediacy of the outcomes. Nonetheless, I go back to my previous assertion that there is a dire need for better individual risk-benefit communication -- decision scientists and behavioral economists need to be recruited in droves to help with these dilemmas; we have the technology! In the long term, however, nothing will replace an educated consumer who is empowered to participate in this nuanced exercise.