March 27, 2015

CHW LogoI’m a bit of a Cold War nerd.  I loved the novels of John le Carre, and can still recite the list of leaders of the Soviet Union from the pictures that hung on the walls of my 10th grade social studies classroom.  In some ways, the world seemed simple.  There were two big alliances: NATO and the Warsaw Pact.  The world could be divided into East and West.  While these blocs came to represent competing ideologies and politico-economic systems, the original purpose was to assure mutual defense through shared, coordinated military effort. Over time, it came to seem that not only could the world be divided into two camps, but that it had to be.  You were either East or West.  Yet we forget that the majority of nations in the world, especially in Africa but even in Europe, the epicenter of the Cold War, were part of the non-aligned movement.

Health care in 21st century Wisconsin is coming to resemble mid-20th century Europe.  Two large state-wide alliances of health systems are emerging – Integrated Health Network (IHN) and abouthealth.  The former includes, among others, Froedtert, MCW, Wheaton, Ministry, Dean, and HSHS, while the latter counts Aurora, ProHealth, UW Health, ThedaCare, and Gundersen Lutheran among its members.  Such accountable care organizations (ACOs) are an approach to managing health for populations.  Alliances of providers (including physicians, hospitals, and others) jointly take responsibility for both the costs and outcomes of care.  The specifics can vary widely, but some of the common principles include:

  • The providers take on some or all of the financial risk for providing care. Rather than payment for volume, there is payment for value.  Better quality and lower cost are rewarded; poor quality or high cost put the provider at risk.
  • Value is driven by coordination of care, emphasis on prevention, and use of shared data to drive informed decisions by both clinicians and administrators

While this sounds appealing in theory, it is difficult in practice.  Studies of ACOs to date show that achieving the desired combination of improved outcomes and lower cost is challenging at best, with many performing below expectations.  Scale seems to matter, as does expertise.  And most of the experience to date is with adult populations (especially Medicare).

As we work to figure this out in Wisconsin, Children’s is poised to be one of those non-aligned nations, working with both alliances to advance the health of children in our state.  With our scale and expertise, not only in specialty and primary care, but also with care management and coordination (as in Children’s Community Health Plan, our Medicaid HMO), we are uniquely capable of taking on the task of managing the health of the population of the children in the state.  We like to say kids are not just small adults.  This is as true when it comes to managing their health as when managing their illnesses.

Which is why you didn’t see our name listed in either of the two statewide alliances listed above. We do, and hope to continue to, work with both.  There are only about 1.5 million kids in Wisconsin, spread out among many different health systems.  If we are forced to pick one side or another, it is likely that neither of them will have sufficient scale and expertise to do the best job of managing the population of children, in sickness and in health.  I hope the health care equivalent of the Berlin Wall doesn’t get in the way of our vision of the kids in Wisconsin being the healthiest in the nation.

Say That in English, Please

March 16, 2015

CHW LogoObserving on rounds one morning, I could see the patient’s mother’s brow furrow and her eyes narrow as the resident presented.  “…Respiratory-wise, she’s tachypneic but sats are good, so wean the O2.  FEN-GI-wise, we can try something PO, then decrease the D5….”  After the presentation the attending physician, sensing the mother’s confusion, said gently, “That’s great.  Now let’s just summarize the plan for mom so she can understand what to expect.”

One of the domains of quality health care is patient-centeredness.  Including families on rounds and accounting for their preferences in decision-making are important steps toward that.  But it is all for naught without effective communication.  Patients and families differ in their health literacy, the degree to which individuals can have the capacity to obtain, process, and understand basic health information and services needed to make appropriate health decisions.  It depends on education, prior experience with medical care, culture, and a host of other factors.  But health care providers often make it more difficult by the language they use.

Language is meant to convey information and achieve mutual understanding.  In medicine and many technical fields, precision is critical, so words should be chosen to maximize it.  While medical jargon, typically derived from Latin and Greek roots, is much maligned, a good deal of it is intended to increase precision.  Sometimes the word “skin” suffices, but if it is important to differentiate the layer involved, we might use the terms “dermis” or “epidermis.”  Blister is a lay term that describes a fluid-filled lesion on the skin but it gives no sense of size; the more jargon-y terms “vesicle” and “bulla” describe blisters that are small (<5 mm) or larger.  So using those terms actually adds to meaning.

Too often, though, it simply confuses.  “Tachypneic” means “breathing fast.”  They are interchangeable.  Using the term “tachypneic” doesn’t add to the precision or convey any extra information.  Almost everyone understands “breathing fast”; few people who are not medical professionals, regardless of their educational attainment, know the word “tachypneic.”  So if the words are interchangeable, why not use the simpler, more widely understood term when talking with families?

Some of many examples of non-value added medical terms include:

Erythema (redness)

Chalazion (stye)

Urticaria (hives)

Eructation (burping)

Sometimes a term doesn’t necessarily add precision, but it is more efficient.  One of my favorite (albeit obscure) examples is “photoptarmosis”: sneezing due to bright light.  A whole concept in one word!  But in each of the above examples, the medical term is actually longer than the lay one.  No greater clarity, and more effort.

Abbreviations sometimes fall into this category.  We love our TLAs*.  They can be handy when writing long hand.  It is certainly easier to write or say “T&A” instead of “tonsillectomy and adenoidectomy” (or even “removal of the tonsils and adenoids).  And in the correct context, one is unlikely to confuse “T&A” the medical procedure for other meanings of “T&A.”  But abbreviations can be confusing.  Is BMT “bilateral myringotomy tubes” or “bone marrow transplant”?  Therefore, they should be used cautiously.  The advantage when typing or dictating is probably not worth the confusion.  And there may not even be any savings when speaking.  “PO” is a term that means “by mouth.”  Two letters vs. seven.  But both are two syllables.  So when talking to a parent, why not say “give something by mouth,” instead of “give something PO”?  When you factor in the extra explanation required, it’s clearly not worth it.

It’s great we have interpreters for patients and families whose primary language is not English.  We shouldn’t need them for everyone.

*Three-letter abbreviations

Safe or Out?

March 9, 2015

CHW LogoTo commemorate National Patient Safety Week, I am reprising this column from a year ago.

The only apparent sign of spring around here is the fact that the baseball pre-season has begun.  Now, I’m not a member of the “baseball as a metaphor for life” school of thought, but it does seem that baseball is in some ways a lot like medical care.  Both involve a large team of people with specialized jobs who spend most of their time in what I would term “anticipatory inaction” (i.e., standing around and waiting for something to happen).  And when they do go into action, the goal is to get someone to home.  (One could also add the unexplained ridiculous prices – a $9 Miller Lite is even less defensible than a $25 Tylenol.)

They also share the concept of being safe, but here they diverge.  In baseball, “safe” is a result – one branch of a dichotomous outcome, the opposite of “out.”  But in healthcare, “safe” is much more complex.  It does involve good outcomes: one measure of safety is the absence of harm caused by the care provided, such as wound infections or pressure ulcers.  However, while such outcomes are an important measure of safety, and are necessary, they are not sufficient.  No wound infections can result from safety or from luck.  Safety is as much about the structures and processes that create a safe environment.  A car without airbags or seatbelts is unsafe even if you drive it many miles without getting injured.

I was thinking about this recently as we at Children’s increase our commitment to providing the best and safest care.  We participate in something called Solutions for Patient Safety, a collaborative of almost 80 children’s hospitals around the country.  The hospitals share data and best practices to try to eliminate patient harm.  When I have seen the data, on most measures we perform better than most, so it would seem that we are a “safe” hospital.  But even if we have a rate of 0, that by itself does not mean we are safe.  I used to have this argument with my son: I’d catch him riding his bike without a helmet, and tell him that wasn’t safe; he would argue that he hadn’t gotten hurt.  To which I would respond, “Yet.”

So while I feel good about our record, we can’t get complacent.  That’s why we are embarking on an effort to increase education among all of the staff and providers on what it takes to be a high reliability organization – the kind of place where not only do harmful events not happen, but they can’t happen.  It means having systems in place that make it easy to do the right thing and hard to do the wrong one.  Redundancy, double checks, alerts, standardized approaches, checklists, etc.

It also means having an environment and culture of safety.  This is an environment where people continually seek to improve by asking questions, raising issues, and intervening when there is a problem.  There are many elements critical to a functioning culture of safety, one of which is what we call “just culture.”  This means treating errors, when they do occur, as a system flaw, not a character flaw.  If I order the wrong medication, or forget to give someone a tetanus shot, it doesn’t mean I’m a bad person.  In a just culture, people are more willing to acknowledge errors, and more willing to point them out.

Creating a just culture and an environment of safety are challenging in practice.  For one thing, it requires overcoming the rather natural tendency to blame on the one hand, and to be defensive on the other.  It’s one thing for an organization to say “we’re not blaming you,” but only after an extended period of acting that way will people feel confident in the attitude shift.  In hospitals in particular, there is a traditional hierarchy that must be overcome.  It doesn’t matter how many posters we put up asserting “You have our permission to speak up,” it is never going to be easy for a nurse six months out of school to tell the chief of surgery that he or she is about to use the wrong instrument.  And it’s even harder if that physician makes it difficult by their response.

In recent years, physician performance has been defined around six core competencies.  Among these are medical knowledge and patient care, which are somewhat easy to define and measure.  Another key one, professionalism, has been shown to be a key element in creating the kind of atmosphere that promotes safety.  Much has been written about disruptive behavior –up to 5% of providers are estimated to be regularly disruptive.  But professionalism, and unprofessional behavior,  is much harder to measure.  It can range from an attitude of dismissiveness to frank verbal or physical abuse.  To paraphrase Justice Potter Stewart, it may be that you know it when you see it.  But the more objectively we can define it, the easier it is to identify when there are problems, to create actionable solutions, and monitor progress.  It allows for fair processes to hold people accountable and ultimately improve, which is the goal.  As difficult as it is to live out a “just culture” when it comes to things like medication errors, it’s that much harder when the “error” is a behavior.  It’s one reason we have moved away from the older language of “problem physicians” to “disruptive behavior.”

Not long ago, I was approached by my medical director and patient care manager with a concern about how I might be making the emergency department unsafe.  They told me that a nurse had indicated, in a survey, that “Dr. Gorelick is difficult to work with.  He is hard to approach with questions and dismissive of the nurses.”  I was absolutely floored.  I don’t consider myself to be a difficult person, and I pride myself on my respect for nurses and indeed all the members of the care team.  (My mother is a nurse, after al!)  But while I heard “Dr. Gorelick is a difficult person who disrespects nurses,” what was actually said was quite different.  It was about my actions, and a specific set of actions at that.  It caused me to reflect, and I realized it was true.  As I’ve written about before, I find it increasingly difficult for whatever reason (I’m sure it’s not aging…)  to get back on task when I’m interrupted.  So if I’m entering an order or writing a note and someone stops me to ask me a question, I do sometimes get snappy.  I worry that it will at least set me back in the task I was doing, and may even increase the risk of an ordering error.  In the parlance of our mood elevator, I go to the lower floors of judgmental and irritated.  But by being hard to approach, I was creating my own safety hazard.  The next time a child needed immediate attention, nurses might hesitate to seek me out, putting that child at risk.

As Warren Buffett said, “It takes 20 years to build a reputation, and five minutes to ruin one.”  My reputation was also at risk, and I needed to fix it.  The first step was acknowledging the problem, and owning it.  The problem was not the nurses’ inability to prioritize, it was my reaction to being interrupted.  Rather than being irritable, I needed to be curious – why does this person want my attention now?  If it turns out to be something that could wait, let’s have a respectful discussion about the risks of unnecessary interruptions, which could lead to a constructive solution (e.g., some way to let me know I’m needed soon but not immediately).

This sounds easier than it is.  I’m sure I still seem less than approachable at times in the emergency department.  As much as I’ve tried to objectify this and focus on my behaviors, it still feels like a questioning of my character.  And no doubt people’s perceptions are colored by my prior actions: even a slight hesitation in responding could be perceived as being “difficult” again.  But if I want to restore my reputation – and more importantly, contribute to the kind of environment that allows us to provide the best and safest care – I have to be accountable for my role.  Professional behavior is like an RBI, allowing our patient to get home safe.  Sometimes we have to attend batting practice.

EPILOGUE:  A recent article demonstrated a link between unsafe attitudes and negative safety outcomes: http://www.ncbi.nlm.nih.gov/pubmed/24874115

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.


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