Comparing Apples and Angioplasties  

March 2, 2015

CHW LogoConsumers seeking organic produce have, in the past, had few options.  Many communities had at most one purveyor of such products, and those stores could charge a premium.  (Hence the common nickname “Whole Paycheck” for Whole Foods.)  As more stores, including even Walmart, began to carry organic products, shoppers had choices and prices moderated.  Score one point for free market competition.

Many policy makers have argued that American health care needs a similar dose of market medicine.  More providers will lead to competition for patients, leading to increased quality and decreased costs.  While insurers and business leaders have tended to support such efforts, based on their experience in other industries, is health care different?  Empirical evidence is mixed.  Some studies have shown that in areas with more providers, prices tend to be lower.  Others, however, find higher costs associated with more duplication of services.  There are similarly contradictory findings on quality of care (though the studies that do claim to show higher quality have largely focused on things like wait times for appointments and patient satisfaction; safety and clinical outcomes actually appear to suffer from competition).

With regard to financial outcomes, it is very important to distinguish between price and cost.  Price (or charge), is the amount of money a provider of a product or service demands from the payer.  The cost to the purchaser, however, is a factor not only of the price, but also any negotiated discount, as well as the number of services consumed.  Let’s use the example of a car.  The car has a sticker price, but the cost to me depends on whether I can negotiate a better deal, and on whether I purchase any extras.

Hence the paradox of more competition leading to higher costs. In health care, competition among providers tends to drive down the prices, and it also allows insurers to negotiate better discounts.  However, the total cost can still go up, if people are using more of those services.  And that is where health care is different from groceries.  Consumerism only works if the consumers know the price, but also have information on quality, and can control the amount that is consumed.  In health care, even if prices were transparent, quality data are much more rudimentary.  More importantly, the consumer (patient) has very little real control.  If someone with chest pain is told by their doctor they need a nuclear stress test and a cardiac MRI they are unlikely to argue.  Decisions by physicians drive up to 80% of all spending; it has been said that the most expensive piece of medical equipment is the doctor’s pen.  (Or in the era of the electronic health record, her mouse.)

So while prices can go down, costs can go up.  There is evidence that when the price per unit cost of a health care service drops, providers may simply do more to make up the difference.  Similarly, when there are multiple hospitals in an area with duplication of expensive equipment and other infrastructure, the use of that equipment increases as the hospitals try to bring in enough revenue to pay for the investment.  The Institute of Medicine has estimated that 30% of medical spending – over $1 trillion per year in 2014 – is waste, much of it from medically unnecessary services.

The excess utilization doesn’t even need to be due to overt greed.  Physicians who are under more time pressure may feel a need to get more specialty consultation rather than spending more time themselves evaluating a patient.  Providers with lower levels of expertise – for example, a general emergency physician versus a pediatric emergency physician – may order additional tests or treatments due to a greater degree of uncertainty.  (Being a good pediatric specialist is as much about knowing what not to do as what to do.)

When it comes to groceries, competition can lower both prices and costs.  But when Walmart comes to town and Whole Foods lowers the price of organic rutabagas and tofu, if they get to fill my cart with whatever they want, I’m going to shell out at least as much money.  And as with medical care, more isn’t necessarily better.

 


The Price of Civilization

February 23, 2015

CHW LogoMatch up the number on the left with the fact on the right:

  1. 1400 A.  # of people demonstrated to suffer autism as a result of measles immunization
  2. 2.6 million B.  # of annual deaths from measles in 1950
  3. 0 C.  # of deaths per day from measles in 2014

(Correct answers: 1.C; 2.B, 3.A)

Despite the hysteria spread by several prominent but ill-informed celebrities, fueled at least in part by completely discredited “research,” vaccination remains one of the most important discoveries in the history of medicine, and along with sanitation one of the top advances in public health.  My first year of medical school we saw a movie celebrating the eradication of smallpox, which had been declared by the World Health Organization just a few years earlier in 1979 (the last naturally occurring case was in 1977).  It was moving to see this illness, which was responsible for up to half a billion deaths during the 20th century alone, and the disfigurement of many survivors, wiped from the planet through scientific discovery and international collaboration.  I, of course, have never seen a case of smallpox.  But I have seen plenty of children suffer – and in some cases, die – from diseases that are now vaccine-preventable.  Measles, Hemophilus influenzae, meningococcemia, etc.  As inspiring as the triumph over smallpox was, it was equally disheartening to arrive in Philadelphia for my fellowship on the heels of one of the largest US measles outbreaks in the vaccine era, with over 1000 cases and 9 children dead.  The epidemic was traced to members of two sects who opposed vaccination on religious grounds.

Today, people are at least as likely to reject vaccines (either completely or selectively) for philosophical or personal reasons.  Although every state requires evidence of immunization for school entry, with exemptions for medical contraindications, 48 allow parents to refuse on religious grounds, and 20 (including Wisconsin) allow refusal on personal or philosophical grounds.  This is what is behind the current national measles outbreak.  Ironically, two of the poorest “Bible Belt” states – Mississippi and West Virginia – which do not permit religious exemption, have much higher immunizations rates than affluent communities like Orange County and Santa Barbara, California, where up to 40% or more of children are unimmunized.

Many politicians are framing this as an issue of individual choice.  Those who claim not to be antivaccine support education to convince parents to have their children immunized, but insist that it is a parent’s right to choose.  I’m not convinced by that argument.  We don’t allow parents to choose to abuse their children, and we don’t allow parents to refuse treatment for life-threatening conditions.  I don’t believe they should be allowed to withhold life-saving preventive measures either.  More importantly, though, this isn’t just about parents making decisions for their own children.  These decisions affect other children, too.  Most vaccines are incompletely effective in an individual.  Much of the protective effect is from what is called “herd immunity”: if enough individuals in a population are rendered non-susceptible to a disease, then the disease cannot perpetuate in that population and everyone is spared.  For measles, which is one of the most contagious diseases known, the level of vaccination required to achieve herd immunity is quite high.  Jenny McCarthy choosing not to immunize her kids put mine at risk.  (Well, not literally, since my kids are unlikely to ever be in the same population as hers, but you get the point.)

At some level, infectious diseases like smallpox, tuberculosis, and measles are the product of civilization.  They arise and thrive when people come together in societies.  Oliver Wendell Holmes said “Taxes are what we pay for civilization.”  So are vaccines.  Like taxes, they shouldn’t be optional.


Doc, I Saw This Ad

February 16, 2015

CHW LogoAsk your doctor if MiracleCure™ is right for you!

We’ve all seen the ads for the latest pharmaceutical wonder.  Grimacing people, who presumably suffer from some sort of –itis or –osis, now able to play tennis or cuddle with grandchildren or sit in side-by-side outdoor bathtubs thanks to this amazing product.  There is then a rapid-fire list of potential side effects ranging from flatulence to hair loss to sudden death, followed by the concluding “Ask your doctor…”

How are you or your doctor supposed to know?

You need three basic pieces of information:

How well does it work?

How dangerous is it?

How much does it cost?

Some people who are given a medication will get better, and some won’t.  Similarly, some people will get better without any treatment, or with a different treatment.  If the number who improve with treatment is no bigger than the number who improve without, then the drug is useless and would be unlikely to be approved.  However, clinical trials may show that a drug is better than placebo or an alternative treatment, but the important question is how much better.  This can be summarized in a measure called the “number needed to treat (NNT).”  It quantifies how many people would need to be given a drug in order for one to be expected to benefit.  Let’s say MiracleCure is a new treatment for migraine.  In a clinical trial, 80% of subjects who received MiracleCure had a significant improvement in their headache.  That sounds really good.  But 75% of subjects who received WonderDrug™, a different migraine medication, also improved.  That difference was statistically significant, so it is a legitimate claim that MiracleCure is better than WonderDrug.  How much better?  You could say “a little,” but that is pretty subjective.   We can quantify it. The difference is 80%-75%, or 5%, which is 1 in 20.  Therefore, the NNT is 20, meaning 20 people would need to take MiracleCure instead of WonderDrug in order for one of them to see a benefit.

By itself, the NNT is a step in the right direction, but it’s incomplete to answer the question if it’s right for you.  If MiracleCure were not only better, but also cheaper and safer, than WonderDrug, the answer would be obvious.  But that is rarely the case.  New medications are usually more costly, and sometimes have different side effects.  For now, let’s just stick with the cost issue.  (Risks and side effects can be summarized by a similar number called the number needed to harm, perhaps the topic for a future column.)  Let’s say the newer, more expensive MiracleCure costs $500 per dose, while WonderDrug is $250.  Most people taking either one will get better; we’ve already determined that 20 people would have to take MiracleCure instead of WonderDrug for one of them to benefit.  In other words, 20 people would need to take a drug that costs $250 more, for a total cost difference of 20 times 250, or $5000.  That is the cost needed to treat.

Now, this actually is not quite so straightforward.  Your cost isn’t really $5000.  If you choose MiracleCure over WonderDrug, your cost will be $250 (the difference in price between them).  It’s essentially a $250 bet on a 1 in 20 chance of a benefit.  That can be hard to evaluate in the abstract.  Thinking of it as an overall expected cost of $5000 to achieve the benefit is a more concrete way to put it all together.

Then you can decide.  Is it worth $5000 for that extra shot at making your headache go away?  It may depend on how bad the headaches are, or exactly what symptoms you tend to get.  It may depend on the consequences: if the headache comes on the day before you are scheduled to deliver a major presentation, you may want to pay the extra money for the small additional chance that you’d be able to give the talk, whereas if you have nothing high stakes planned the next day, maybe you’ll take a chance on the slightly less effective drug.

The point is, it’s your decision.  The question shouldn’t be “Ask your doctor if MiracleCure is right for you,” but rather,  “Ask your doctor to help you decide if MiracleCure is right for you.”  And if you are going to make an informed decision, you now know the basic data to request.


Nature v. Nurture

February 10, 2015

CHW LogoThe mention of funding for additional research in “precision medicine” attracted applause from both sides of the aisle in a State of the Union address that was notably thin in bipartisan appeal.  But it was hard for me to bring my hands together over the concept.  When I hear about the promise of the “genetic revolution,” I think of Carly (not her real name).  I took care of Carly, an 8 year old with sickle cell disease, a few years ago when she came to the emergency department with a massive stroke.

In medical school, in 1983, a professor told us with great authority that within 15 years, sickle cell disease would be an historical relic.  Because it was known to be caused by a single mutation coding for a single amino acid in the hemoglobin molecule, it was an obvious target for the emerging field of gene therapy – it would be the simplest genetic disease to cure.  If that professor had been correct, Carly wouldn’t have sickle cell disease, much less a crippling stroke.  I think everyone knows that sickle cell disease is still very much with us.  Carly’s family certainly does.

Now, I know “precision medicine” isn’t about gene therapy.  As described by the White House, the initiative is designed to “provide clinicians with new tools, knowledge, and therapies to select which treatments will work best for which patients.”  The concept is appealing.  Therapies have different effects on people depending on their genetics.  Rather than a “one size fits all” approach, drugs can be targeted to people whose genetics suggest they would be more likely to benefit, and dosages can be adjusted according to genetic differences in metabolism.

But as Mayo clinic anesthesiologist and physiologist Michael Joyner points out in a recent New York Times op-ed, it’s never quite as simple as it seems.  First, most diseases, and certainly the most common ones, turn out to be genetically very complex.  In sickle cell disease, 100% of the risk is associated with a single gene mutation.  For most conditions studied so far – diabetes, heart disease, autism – multiple genes account for at most a few percent of the variability.  Environment and behavior remain far stronger risk factors.  And of course there is an interplay between genes, environment, and behavior that increases the complexity further.

Another “straightforward” example provides further caution.  Metabolism of many drugs is affected by a genetic variants in the cytochrome p450 complex.  The presence of certain alleles alters drug metabolism in predictable ways.  Knowing a patient’s cytochrome p450 alleles should allow a clinician to adjust drug dosages without the need for trial and error – giving more to people known to metabolize more readily, or less to those who are slow metabolizers –  potentially maximizing benefit while avoiding toxicity.  Warfarin (Coumadin®), an anticoagulant commonly prescribed to prevent blood clots in patients with a variety of heart and other conditions, is one of the drugs metabolized in this way.  Moreover, it has a narrow therapeutic index, meaning there is a relatively small zone of appropriate dosages: too little is ineffective, too much is dangerous.    The usual way to find the sweet spot is to start with a given dose and monitor blood tests to check clotting, making frequent adjustments until the desired effect  if achieved.  This can take many weeks, during which time the patient is at risk for either blood clots or excessive bleeding if the dose is too low or too high.  And people can vary in the necessary dosage by a factor of almost 20, in large part due to known variants in the p450 genes.  So when these variants were identified, it was appealing to think that prescribing based on knowing a patient’s p450 genotype would be safer and more effective.  There was a lot of hype about the coming “pharmacogenetic revolution,” and in 2010, the FDA actually changed the labeling on warfarin to recommend that  physicians choose the dose based on p450 genotype.

They appear to have jumped the gun.  A recent study, a meta-analysis of 9 clinical trials comparing genotype-based dosing of warfarin with standard “trial and error,” found no advantage to the “precision” approach in terms of any laboratory or clinical outcomes.

No doubt as our understanding of disease improves, we will be able to target treatments more effectively.  Some things we consider a single condition will turn out to be a cluster of many different conditions, based at least on part on genetic differences, each with a different optimal treatment.  Cancer chemotherapy is already a step in this direction.  But we shouldn’t get overly distracted by this latest shiny object.  The more we learn about the human genome, the more we realize that external factors – environment and behavior – remain the primary determinants of disease and the keys to prevention.  The single biggest advance in the 40+ year “war on cancer” has not come from the lab; it was the reduction in smoking.

Diet, exercise, and other behavioral and environmental factors aren’t very sexy, and they are not the stuff on which academic careers are built, but in the end they are what will make us healthier.  We can’t let the allure of precision medicine threaten support for these mundane but proven strategies.  Gene therapy sounded pretty cool, but it didn’t help Carly.


What is Brain?  

February 2, 2015

CHW LogoOne of the worst episodes of the original Star Trek series was one called “Spock’s Brain.”  I can’t recall all the details (although – nerd alert – I undoubtedly had the entire script committed to memory at one point in my life).  But it involved a technologically backward planet where the males and females lived separate lives: the men (called “morg”) lived on the surface in a sort of late Ice Age existence, while the women (“imorg”) lived in a comfortable underground city, though the whole place apparently ran on autopilot, since both morg and imorg had the intelligence level of 8 year olds. (When asked the whereabouts of Spock’s brain – don’t even ask why – one of the imorg, confused, replied, “Brain and brain! What is brain?”)

Many of the episodes from the 1960s had a hidden – or sometimes quite blatant – moral message, but I could never figure out what it might have been in this episode.  Given how bad it was, maybe there wasn’t one.  But a couple of recent articles shed some light on a possible allegory.

One study examined the ability of randomly created groups of 2-5 members to perform a set of tasks.  Success was only minimally correlated with several hypothesized factors, such as average or maximal IQ, or group cohesion.  The three factors most strongly predictive of success were equal distribution of speaking turns, average social sensitivity of the group, and the proportion of women. (Most of this last effect was mediated by the greater social sensitivity of women, but there was an independent contribution from femaleness itself.)

Other work examines gender differences in decision making.  In low-stress situations, men and women approach decisions similarly, gathering information and weighing risks and benefits, and their average overall risk appetite is the same.  But a series of studies demonstrates that under conditions of stress (physical stress, anxiety), decision-making strategies diverge.  Men tend to take larger risks for larger potential gains, while women tend to go for surer but smaller rewards.  Moreover, when asked to evaluate their strategies, men were less likely to recognize their riskier approaches as actually riskier.

Now, I don’t mean to suggest that men are a bunch of insensitive adrenaline junkies.  And the data don’t say one style is better or worse.  But teams with a diversity of approaches will likely outperform one with a single-minded risk-averse or risk-taking strategy, and this is also borne out by evidence.  A review of 2400 large global companies showed that those with at least one woman on their board outperformed those with all-male boards by 26%.  (You might argue that we also need to see how a large global company with an all-female board would compare.  Sigh.)

Working in pediatrics, and specifically at children’s hospitals, for my entire career, I take this sort of balanced approach to work groups for granted.  But too many organizations are like that planet where the morg live above ground, the imorg below, and both suffer as a result.  Brain seems to require both.


Free Range Kids

January 26, 2015

CHW LogoThis from the Washington Post: The parents of two children, age 10 and 6, were investigated by child protective services after their children walked home alone from a park near their home.  Several years ago,

Since many of you are mandated reporters of suspected abuse and neglect, I may be putting myself at risk by admitting the following:

  • Our kids walked to school since first grade – without their parents
  • When our son was 7 we allowed him to be in the house by himself when we ran around the block
  • At age 10, our older son was given the chore of doing his and his brother’s laundry, which of course meant pouring laundry soap into the machine
  • By age 12 our younger son regularly took the Milwaukee County bus to his guitar lessons
  • All of us regularly walk barefoot in the yard, eat outside, and pick up trash and recycling we find as we walk

Without realizing it, we were in the vanguard of what is becoming known as the “free range kids” movement, a reaction to the notorious “helicopter parenting” trend of the past decade or so: the notion that kids must be protected from all known risks by hovering over them constantly and intervening should any danger such as a pedestrian, a mosquito, or a jellybean dropped on the floor get through the layers of virtual bubble wrap in which those kids are cocooned.

This is, perhaps, a bit harsh.  I don’t mean to suggest we should be cavalier about safety, and I recognize that the fact that I walked to school in first grade, stayed home alone briefly after school, and rode New York City public transit when I was 12, means that everything we did back then was a good idea. (Our kids did not ride in the back of pickup trucks.)  But we tend to overemphasize risks and safety, at the expense of allowing children to experience – and yes, at times fail.  Take stranger phobia; the evidence is that abductions and other crimes involving children are actually less common, though you’d never know it from watching Nancy Grace or other TV “news”.

There is a down side to this sheltering.  Overprotection of children makes them less able to deal with problems when they are adults.  Here’s one example: Boston College has seen a doubling of emergency calls for minor issues like being called a name by a roommate or finding a mouse in the dorm.  So the helicopter continues to hover.  NPR reported a couple of years ago about parents who show up at their children’s job interviews.

I’m all for insisting on kids wearing bicycle helmets.  But at some point you have to take off the training wheels and let them go around the neighborhood on their own.  Just hope someone doesn’t call CPS.


The Age of the Tricorder

January 22, 2015

CHW Logo

“It’s worse than that – he’s dead, Jim,” Dr. McCoy would solemnly intone, after waving a small handheld device over the motionless figure in the red velour shirt.  Of all the gadgets in the Star Trek universe – some of which (warp drive, transporter) remain science fiction, while others are already passé (communicators like 1990s cell phones, 3” square computer disks) – the one I found most fascinating was the tricorder.  Imagine waving something the rough size and shape of a salt shaker over someone and getting a reading of their vital signs, blood chemistries, and even a diagnosis.  If you had one of those, who would even need a doctor; you could hold it in front of your own face and figure out what was wrong, and then ask the computer what you should do about it.

Until recently I assumed the tricorder was more like the transporter than the communicator – unrealistic fantasy rather than established technology.  Perhaps not.  A raft of devices designed to allow people to monitor their own health and self-diagnose is coming to market, some already here.  (Google “medical tricorder” and one of the first hits is the Scanadu Scout™, which measures and records vital signs by scanning your forehead.  For real.)  According to a new book called The Patient Will See you Now from Dr. Eric Topol, a cardiologist and “Professor of Innovative Medicine” at the Scripps Institute, as well as long-time proponent of personalized medicine, the future of medicine lies not with physicians, but with individuals armed with smart phones, miniature electronic sensors, and terabytes of genomic and other data about themselves.

Others, while not disputing the usefulness of some of the new technology, are less certain how soon they will revolutionize health care in the way envisioned by Dr. Topol.  For one thing, data are not information: raw data must be processed to be actionable, and context is important.  While automated language translation programs are certainly getting better, anyone who has used one knows that communicating to a native Mandarin speaker requires more than drawing on a database of Chinese ideograms.  Similarly, humans are more than the sum of a set of physiologic parameters.  The best providers don’t treat numbers, they treat patients.  Besides, despite all the incredible hassles they complain about, patients seem to value seeing their doctor, which may explain why the annual physical remains a popular ritual despite the lack of evidence for its benefit on health outcomes.

What is missing from the patient-as-own-doctor model is the element of empathy, the communication, caring, and connection that characterizes the ideal of the provider-patient relationship.  The tricorder-like gizmos may eventually be able tell you what is wrong with you, but they can’t hold your hand, or give you words of encouragement or sympathy.

As technology advances, the medical profession has an opportunity to increase their value to patients.  We like to blame technology – especially the electronic health record – for interfering with the relationship between patients and providers.  But it can also be liberating.  As rote memorization and manual dexterity, keys to success in medicine in the era before handheld computer devices and robotic surgery, become less important, we can select for and spend more time developing communication and interpersonal skills.  Some innovative training programs have shown promising results.

Sir William Osler said “a physician who treats himself has a fool for a patient.”  Perhaps a patient who treats herself has a fool for a doctor.  But if we can’t figure out and demonstrate how providers can add value in a world of Scanadu™ and Siri, many patients will take their chances without us.


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