Endodoc

In earlier entries I discussed various aspects of the US health care mess, starting with an overview of the problems (poor access and high costs) and ending with a bit of a detour to discuss rationing health care as a means of controlling costs.  Now it’s time to take a step-by-step approach to solving the problems, starting with access.

What are the reasons for poor access to health care in the US and what is the magnitude of the problem?

First, the problem with access is HUGE; at least 40-50 million people in the US have no health care coverage (by “coverage” I mean health insurance, Medicaid, Medicare, VA Health Care, etc.).  In addition, many millions more (some experts estimate another 40-50 million people) have inadequate coverage.

Access vs. real access

So, part of the problem is that many people do not have adequate access to health care because they do not have health care coverage.  Theoretically, these people can obtain access to health care simply by paying for it “out of pocket.”   In practice, many health care providers will not see people needing health care unless they have some form of insurance (within the medical community, so-called “self-pay patients” are considered synonymous with “no-pay patients”).  If self-pay patients are “lucky enough” to obtain health care services, they are typically charged more (sometimes as much as 2-3 times as much) for the same services than an insured patient; most insurance plans have contracts with health care providers that allow for deep discounts for services.

Even having some form of health care coverage may not guarantee access to care.  Many physicians do not see patients with Medicaid, and a worrisome trend is that a growing number of physicians will not see Medicare patients .  At this point, I do not want to get into all the reasons for these access problems except to say that many physicians find the paperwork load for dealing with Medicaid and Medicare patients is not worth the bother given how low the reimbursement rates are for most services.  So, access doesn’t always mean real access.

Geographic barriers

Another problem with access is a geographic; patients who live in rural areas may be many miles from the closest health care facility and much further from specialty care.  Given the shortcomings of the US transportation system, most people who live far from health care facilities are completely dependent on the automobile to get health care.  What about an elderly person who lives in a rural setting and does not drive a motor vehicle?  That person might have the best health insurance possible but it doesn’t do them much good if they can’t get to the health care facility.

Manpower barriers

Last, but not least, a major problem with access is the shortage of health care professionals, particularly in primary care.  How can physicians afford not to see Medicaid and Medicare patients?  It’s easy.  They are very busy already with customers who pay well.  We need more primary care physicians (family medicine, internal medicine, and pediatrics) and nurse practitioners that specialize in primary care.

Part of the manpower problem is one of geographic  distribution; physicians, nurses, and other health professionals generally want to live near the bright city lights not in rural areas.  In many ways that is good since many more people live in the cities than in the small towns.  But even in large population areas, it’s much easier to get a timely appointment with a cardiologist or a dermatologist than with a primary care doctor.

Access vs. high quality access

Finally, there is a difference between access and high quality access.  Unfortunately, the quality of health care varies greatly in the US.  Some people are lucky enough to have high quality health care plans and easy access to high quality health care providers.  For example wouldn’t you rather see a health care provider who focuses on preventive care and who has access to highly skilled chronic disease management teams (e.g., for patients with diabetes, heart disease, and cancer)?  Some people are not so lucky.  Most reputable health care plans in the US now do include such services (for a good example of what I consider an excellent health care plan, take a look at one of the Kaiser-Permanente HMO plans).

After I posted my last entry (FYI: An Important Newspaper Article About The US Health Care Mess), I got to thinking about the concept of rationing health care- that is, providing health care for some problems and not for others.  It really boils down to money; if one has a health care budget and if the demands cost more than the budget and if one is not willing to allow deficit spending (by either printing more money or borrowing it) some health care demands will not be paid by whomever writes the checks.  Of course, that does not mean a person whose health care service request was denied cannot go out and buy the service on their own, unless such an approach is not permitted.  The idea that someone somewhere will be making life and death decisions about access to health care services makes most people uncomfortable.  But maybe we need to think about rationing in a much broader context?

How much is a human life worth?

Our society already rations many services that put a price tag on how much a human life is worth and seem to  we accept the approach.  For example, private citizens, industry, and the government (federal and state) already determine how much to spend on transportation safety.

In building roads and in setting rules for transportation, governments look at costs and then try to strike a balance between the most safe possible solution and a downright dangerous one.  Having limited access roads, long merge lanes, dedicated lanes for large trucks, etc. makes for safer roads and fewer traffic accidents.  Unfortunately, such roads cost much more to build than roads without such features.

We could build the safest possible roads everywhere except that highway departments have finite budgets and must decide on how best to spend their money.  The decisions are often based on statistical information regarding the likelihood of motor vehicle accidents leading to injuries or deaths given the volume of traffic, speed, etc.  Often historical data are used to help make decisions whether or not to make road improvements, such as adding a traffic light to a certain intersection- only after so many injury motor vehicle accidents at the intersection in question is it “worth” spending  the money.

Individual decisions that balance safety vs. costs

When people shop for cars, they usually have a pretty good idea how much they can afford to spend.  Once they pick out the brand and model they are interested in, they usually then decide which options to purchase, including safety features (thank goodness more and more, vehicle manufacturers are including important safety features as standard equipment, such as multiple air bags and stabilization control) which are known to save lives in the event of an accident.  If money were no object, most people would get the safest vehicle possible.   But costs do matter to people and they routinely make personal safety decisions based on their cost analyses.

Rules that balance safety vs. convenience and personal freedoms

Let me give you  one last set of examples to illustrate the fact that health care rationing is not all that different from  rationing we already do in other areas.  It is well known that using  seat belts save lives, yet the US has moved very slowly to make seat belt use mandatory (and even when mandatory, the consequences for not wearing a seat belt are minimal).  It is also clear that lowering speed limits on roads saves lives, yet we as a society are reluctant to impose major restrictions on individual freedoms.   It’s the same for states that do not have helmet laws for motorcycle riders.  I could go on and on.  I am not saying that the government should or shouldn’t be able to limit individual freedoms if certain rules can save lives but only that we already make many decisions in our society that truly have life and death consequences.

So, in many ways our society does put a price tag on how much a human life is worth.  Of course we do not have a single well accepted formula for calculating the value of a human life and there is no consistency in the approach from issue to issue.  That’s what makes the notion of rationing health care so difficult to come to grips with, yet we must find a way to do that if we are to improve US health care in the end.  I am quite confident that we can control costs and attain universal health care access.  I’ll try to persuade you in subsequent entries.

FYI:  An Important Newspaper Article On the US Health Care Mess

In my last entry, I began a discussion of the US health care system.  I had planned to work my way through the various issues, ending with a plan to control costs.  That is still my plan but I want to let you know about a very interesting and important article published recently in the New York Times (Wednesday, December 3, 2008).  The article is entitled “British Balance Gain Versus Cost Of Latest Drugs,” and was written by Gardiner Harris.

How does information about the British health care system help those of us in the US?

As I discussed earlier, the “big ticket” problems in the US health care system are access and costs.  I submit that it is not possible to assure universal access to high quality health care for all people in the US without controlling costs.  By controlling costs I mean not just slowing the rise in health care costs (greater than the rate of inflation over the past 30 years) but actually cutting current costs by quite a bit- 25-50%.  That’s quite a challenge.

So, the New York Times article discusses the British efforts to control their costs by rationing drugs.  The British government has an agency, the National Institute for Health and Clinical Excellence, known as “NICE,”  which is responsible for determining if a prescribed treatment is affordable; that is, whether or not the British government will pay for the service.  The institute has carried out rather fancy economic analyses and has determined that the British government can afford only about $23,000 to save a citizen’s life for 6 months.  Basically, NICE has calculated what a life is worth.  The agency’s policy has generated considerable controversy.  The newspaper article discussed the case of a British citizen who has kidney cancer with metastases to his lung.  The patient’s physician prescribed a new and very expensive pill which has shown some promise in clinical trials and offers, on average, 6 months longer survival  at a cost of more than $50,000.

Is NICE a nice idea?

The issue of rationing health care is a very complicated one but there is no question that rationing services will be necessary if the US has any hope of controlling health care costs.  I’m not sure “rationing” is really the right term for what the British are doing or what the US will need to do.  Britain unlike the US actually has a health care budget (almost all people in Britain receive medical care paid for by the  government and they have been struggling to control costs.  NICE has been around for about 10 years and its purpose is to decide which services will and will not be covered for patients within the British National Health Service.  Their policies are based on both medical necessity determined on evidence-based medicine principles and costs.

The British Dilemma

The problem the British have is how to best control their health care costs best given that they actually are supposed to operate within the constraints of a health care budget.  Should expensive services be denied even if they are effective in prolonging life or in providing symptom relief?  There are no easy answers to such questions.  One problem, which is addressed in the newspaper article, is how difficult it can be to determine if the cost of the service in question is a reasonable one.  For example, should a $10 pill really cost $10; is the cost of production such that $10 per pill is an appropriate charge?  We know quite a bit about drug pricing in the US.  Typically, when a generic drug is introduced, the price for the drug drops by up to 90% (that’s not a typo- a huge drop in price).  So, how much of the current high and steadily increasing costs for health care services in the US and around the world are more or less price gouging?  Clearly, some of the cost increases are the result of new and better therapies, but no matter what the service, governments and insurers must find ways to get services at reasonable costs (probably a better term would be “appropriate costs”).  Only then will it be possible to determine how much rationing is actually necessary (I still don’t like that term, “rationing”).  What’s interesting is that drug companies and device manufacturers around the world are already anticipating major efforts by payers to assess costs for individual services rather than just accept them as has been the case in the past- the result has been a quiet deflation in costs for many services on a “voluntary” basis.

Back to the US health care system

The New York Times article is well worth reading and if nothing else it makes clear how difficult a task it will be to control health care costs.  I will come back to this issue when I get into the details of how we can achieve universal health coverage and afford it (I promise it won’t be “smoke and mirrors”).

In an earlier entry, I promised that right after the 2008 U.S. presidential election I would address an important subject, the U.S. health care system. Well, I’m a man of my word. My plan is to devote a number of entries to the subject, despite frequent warnings from my webmaster to make every entry a complete piece; there is just too much to discuss to fit into a reasonable length single entry. So the grand plan is to first provide an overview of the subject in this entry. Future entries will focus on the details.

Is health care a right or a privilege?

Before we get into the details,, it is necessary first to address a very basic moral/ethical/legal question: Is quality health care for all a right or is it merely a privilege? So, is access to quality health care just a commodity that one is free to buy on the open market just like a BMW or a ski parka or a condo in Aspen, Colorado? Or, is it a basic right? This is not just an academic question. There is considerable opposition to implementing a universal health care system in the U.S. For some, the opposition is a mixture of philosophy and interpretation of the law- that there is no basis in law (constitutional or otherwise) to spend tax money to assure health care coverage for people who have not “earned” it. It’s sort of survival of the fittest where America is seen as the land of opportunity. If people just work hard enough, they can make a success of themselves by making enough money to buy good health care coverage (and whatever other commodities they want); otherwise, too bad. The other side takes the position that whether or not there is a legal basis for universal health care coverage, the U.S. is a wealthy nation that can afford to do the right thing, i.e., provide universal health care coverage. I have greatly oversimplified the two opposing positions; it’s not just the “good guys against the bad guys.”

I am not going to take sides in this debate. I would point out, however, that many studies have shown that not having high quality access to health care for all, costs both those with and those without health care coverage dearly. For example, did you know that almost 1 of every 3 dollars spent on health care premiums actually goes to cover health care costs for the uninsured. So, as it turns out, unless we as a society are ready to deny all access to the health care system for those who do not have health care coverage (including life-threatening emergencies) or cash “up front,” it is far cheaper in the long run to assure health care coverage for all than to exclude anyone.

For the purposes of this discussion, I will assume that high quality health care coverage is a worthy goal for our society, regardless if one favors this approach on philosophic grounds or merely economic ones.

Is there really a health care crisis in the U.S.?

These days the term “crisis” gets tossed about quite a bit and I think it is way over used. It seems to me that for something to be a “crisis” means that the situation whatever it might involve is such that unless something is done about it in short order, the results are likely to be disastrous. In my opinion, the current U.S. health care system is a mess, and some would say it’s already a disaster. But, does that mean we have a crisis? I would prefer to think about our health care problems as more like a chronic disease that if left untreated (or treated poorly) will just get worse and worse for quite some time until things are so bad that something revolutionary happens- that would be a crisis. I don’t think we should to wait for the revolution (one never knows how a revolution will turn out), but rather, develop a comprehensive effective treatment for this “disease” now.

Despite its problems, doesn’t the U.S. have the best health care system in the world?

Myth: the U.S. has the best health care system in the world. Fact: among the world’s most highly developed countries, the U.S. ranks mostly last in the quality of its health care. Whether it is longevity, infant mortality rates, chronic disease outcomes, or whatever, the U.S. generally falls far short of what many other countries have been able to achieve. The U.S. does have some of the best medical care and research facilities in the world, but that hasn’t resulted in great medical care for many of its people. Some of the reasons for poor performance are related to socioeconomic status but even if one makes allowances for that, the U.S. falls far short- just having lots of money does not assure that a person will get high quality health care in the U.S.

Adding insult to injury: health care costs

So, the quality of U.S. health care is not up to its potential. Yet, on a per capita basis, the U.S. spends far more than any other country in the world on health care, more than $7000 per person per year. In fact, the U.S. spends about twice what the next closest country spends- 2-3 trillion dollars per year.

How come U.S. health care isn’t very good and costs so much?

It is not totally clear exactly why U.S. health care cost so much and isn’t so hot, but most experts point to one fundamental problem- access. Many people in the U.S. do not have easy access to the health care system; even if medical care in the U.S. were uniformly outstanding, if people can’t get the care, it doesn’t matter how state-or-the-art it might be. It is estimated that between 40-50 million people in the U.S. do not have health care coverage (by coverage, I mean commercial insurance or a governmental plan such as Medicare or Medicaid). Another 40-50 million more may have some form of coverage but which is not considered optimal (e.g., no drug prescription plan, very high deductibles and c0-pays, no preventive care coverage). Many experts have concluded that if the U.S. were to have universal coverage of high quality, medical care outcomes in the U.S. would be the best in the world.

But, with medical care costs so high and climbing every day, the question is whether or not it is possible to actually achieve high quality universal coverage in the U.S. without “breaking the bank?” I am confident we can can achieve those twin goals. In upcoming entries I will do my best to provide an easy to read road map showing where the U.S. needs to go (high quality health care access for all, at a price its citizens can afford) and how to get there . As I will emphasize, the U.S. cannot “fix” its health care system without improving high quality access and it cannot improve high quality access without controlling costs. No workable plan can disregard either component.

In my last posting I delved briefly into the politics (nonpartisan, of course) of health care issues in the upcoming presidential election. In that posting (10/10/08) I should have provided some information about the health care proposals put forth by the 2 major candidates, but I wasn’t sure how to do it.

Now I know. To my surprise (and pleasure), the current issue of the New England Journal of Medicine (October 9, 2008) has reasonably detailed statements by Senators Barack Obama and John McCain describing their health care proposals; the editors of the journal had asked the candidates to describe their plans for reforming the U.S. health care system, and voila.

If you want to learn more, the journal sponsored a symposium in which representatives from each of the campaigns participated and which is available as a video at www.nejm.org.

In my opinion the two biggest problems with our health care system (it might be a stretch to call it a “system”) are access and costs. Our collective health care woes cannot be solved without a coordinated effort to attack both problems; I do not believe we can realistically improve access without controlling costs and vice versa. How well do the candidates and their representatives tackle these barriers to a better health care system? I will offer no opinions in this matter until after the election, and you need to sort this out for yourselves anyway. Good luck.

I do promise to get back to health care policy as it affects the health of patients with various endocrine disorders, particularly diabetes mellitus, but only after the election.

This political season I have tried to “sit on my hands” and stay out of the various health care debates until after the presidential election which will finally happen in a few weeks. But, I can’t contain myself. The other day, I read an article in the New York Times entitled “Campaign Myth: Prevention as Cure-All,” written by H. Gilbert Welch, M.D. (NYT, Tuesday, October 7, 2008, page D6). Dr. Welch is a professor of medicine at the Dartmouth Institute for Health Policy and Clinical Practice in Hanover, N.H. His article, which was labeled an “essay,” addressed the question of whether the presidential candidate claims that improving preventative health efforts will save money and improve health care.

What does Dr. Welch think?

Basically, Dr. Welch writes that preventative health these days is mostly overdiagnosis and not worth pursuing. He contends that the issue “boils down to encouraging the well to have themselves tested to be sure they are not sick. And that approach doesn’t save money; it costs money.” Wow, that’s interesting. But is Dr. Welch correct?

A critique of Dr. Welch’s thesis

In my opinion, Dr. Welch is guilty (very guilty) of generalizing to such an extent that his argument is at best misleading, and at worst, very misleading. Let me defend my position with one simple example- screening for diabetes mellitus. First of all, it is important for you to understand that screening IS defined as testing asymptomatic people for the presence of disease. Diabetes mellitus is incredibly common and we SHOULD encourage people to be tested for the disease. Why? We know that many millions of people in the U.S. (to say nothing about the rest of the world) have diabetes and don’t know it. Studies have shown that the average duration of diabetes prior to its discovery is 9 years; patients not infrequently learn that they have had diabetes for years when their eye doctor asks them after a routine eye exam how long they’ve had diabetes? Furthermore, we now know that early detection and proper treatment can prevent all diabetes complications, which account for most of the diabetes care costs. So, at least for diabetes, early detection improves health and saves money, lots of money. If you don’t believe me, ask Kaiser-Permanente why they screen for diabetes and have specialized diabetes management teams for patients enrolled in one of their health care plans.

Maybe I shouldn’t be so hard on Dr. Welch?

I do agree with Dr. Welch that for some disorders, early diagnosis does not improve health outcomes and save money. I also agree that screening for disease needs to be done in a way that few false positives (people who have positive screens but do not really have the disorder) and false negatives (people who really do have the disease but are missed by the screening test) result from the screening. It is also important in making a decision to screen for a disease that an effective treatment for the disease exists. One last thing- it is possible that for some diseases that not making a timely diagnosis saves money by not having to treat the patient with an expensive drug or procedure; but that’s an ethical/moral question that I’m not in a mood to discuss today.

The September 2008 issue of Diabetes Care had a very interesting article that is quite relevant to both diabetes specifically, and to health care delivery in general. The article was entitled “The missed patient with diabetes: how access to health care affects the detection of diabetes” and was written by Xuanping Zhang at colleagues at the Centers for Disease Control (CDC) in Atlanta, Georgia (Diabetes Care 2008;31:1748-53).

Background information

If you’ve read some of my earlier postings, you surely know that the prevalence of diabetes mellitus word-wide has been increasing at an alarming rate over the past 20-30 years; in the U.S. alone it is estimated that 20-25 million people have the disease with 6-7 million of those undetected. On top of those depressing statistics, studies have shown that the average duration of diabetes before it is detected is about 9 years; many people with diabetes first learn they have diabetes when they develop symptoms from some diabetes complication. None of this would really matter much except that we now know that diabetes complications are preventable with early detection and appropriate treatment (check out some of my earlier posts about the Diabetes Control and Complications Trial, or DCCT, The Epidemiology of Diabetes Interventions and Complications Trial, or EDIC, and the United Kingdom Prospective Diabetes Study, or UKPDS).

The study design

The investigators analyzed data from the 1999-2004 National Health and Nutrition Examination Survey or NHANES. I need to explain that one good thing done with your tax dollars (assuming you live in the U.S. and pay your taxes) is that every 8 years or so, the CDC carries out a 6-year studies to assess the health status of people living in the U.S. They have been at this for quite a number of years and over the years, the data gleaned from these studies have been incredibly important and useful. I know quite a bit about the NHANES since my laboratory carried out most of the diabetes-related laboratory analyses (plasma glucose, hemoglobin A1c, serum C-peptide, and serum insulin) between 1987-2004. I’ll vouch for the accuracy of the data.

Zhang and colleagues picked through the NHANES 199-2004 data in an effort to answer the following question: does access to health care affect the detection of diabetes (I guess you’d figure that out from the article title). The question of access to health care is a rather complex subject with a number of components. For their study, Zhang and colleagues focused on health insurance status and utilization of health services (i.e., number of health care visits during the previous 12 months) as their measures of access (some other components to access include availability, organization, and satisfaction).  The investigators analyzed data from approximately 5500 people ages 18-64 years.  They identified 110 people who met criteria for diagnosis of diabetes yet had not been previously diagnosed, 704 who had been previously diagnosed, and 4782 who did not have diabetes.  Thus, 110/5486 people had been “missed.”  What turned out to be so interesting, but not so surprising, was that those people undiagnosed with diabetes had the least favorable profile of access to care of the three groups (missed, previously diagnosed, and no diabetes.  The differences were very large; taking all diabetic people (missed and previously diagnoses), the percentage of people with no health insurance was much greater in the missed group than in the previously diagnosed group.  Continuity of coverage was also important; people with no insurance for > 1 year were much more likely to be in the missed group than in the previously diagnosed group.   Level of education and income were far less important than health care access in determining risk for undetected diabetes.  The investigators concluded that given the data showing undetected diabetes is a serious risk factor for adverse health outcomes, timely detection of diabetes is difficult without access to health care.

So?

These data are just one more piece of evidence that if we want to provide optimal medical care to people with diabetes, we can not do it without providing adequate access (code word for quality health insurance) to appropriate medical care.  It doesn’t matter how skillful the physician and how motivated the patient if the diabetes goes undetected for years and years before it is diagnosed.  Remember, the lion’s share of diabetes health care costs are for the treatment of preventable diabetes complications.  And who actually pays for treating diabetes complications?  The answer is that we all pay. If I had to bet or die, I’d say the situation is very similar for other chronic diseases such as heart disease and cancer, but here in a study of diabetes, Zhang and colleagues have made their case very persuasively.

Recently I received a draft document on growth hormone treatment policies from a leading health insurance company. I was asked to review the document and to offer comments. The document, which was 26 pages long, detailed the company’s proposed changes in the criteria they will use to approve or deny requests by physicians that the insurer pay for use of synthetic human growth hormone. At present, at least 8 pharmaceutical companies manufacture and market human growth hormone (I have no idea how many companies sell bovine and other animal growth hormones- growth hormone is species specific in terms of having biological action). Also, at present, there are at least 10 specific medical diagnoses for which use of growth hormone therapy is FDA-approved.

Why does the insurer want a new set of policies on the use of human growth hormone?

If I had to bet or die, I’d say the insurer has noticed an alarming increase in requests for growth hormone treatment over the past few years and given the costs for growth hormone, which can be up to $100,000 per year per patient, they just did the math and found the results frightening. The proposed new policies are designed to “slow the bleeding.” And who can blame the company for trying to keep costs down- increased costs mean lower profits for them and/or increased costs for insurees? The question I want to address here is whether it is possible for an insurer to maintain quality of service (i.e, pay for services that are needed for the insuree’s health and well-being), yet keep costs from going up and up and up? Here, we’ll focus on human growth hormone, but the question is really a general one that our society will have to come to grips with sooner rather than later.

What is “medical necessity?”

Most people would agree that medical insurers should not “cover” (i.e., pay for) treatments that are not medically necessary. The problem is deciding exactly what is a medically necessary treatment. In the situation under consideration here, the insurer has defined medically necessary as “procedures, treatments, supplies, devices, equipment, facilities or drugs that a medical practitioner, exercising prudent clinical judgment, would provide to a patient for the purpose of preventing, evaluating, diagnosing or treating an illness, injury or disease or its symptoms that are: in accordance with generally accepted standards of medical practice; and clinically appropriate in terms of type, frequency, extent, site and duration and considered effective for the patient’s illness, injury or disease; and not primarily for the convenience of the patient, physician or other health care provider; and not more costly than an alternative service or sequence of services at least as likely to produce equivalent therapeutic or diagnostic results as to the diagnosis or treatment of that patient’s illness, injury or disease.” Furthermore, the insurer defines “generally accepted standards of medical practice” as “standards that are based on credible scientific evidence published in peer-reviewed medical literature generally recognized by the relevant medical community, national physician specialty society recommendations and the views of medical practitioners practicing in relevant clinical areas and any other relevant factors.”

Is treatment with growth hormone medically necessary?

In 1984, biosynthetic growth hormone was FDA-approved for use in children with growth hormone deficiency. The drug was offered by a single pharmaceutical company and it was expensive, but there was no real debate whether the drug was medically necessary in “classic” cases,those patients who were exceedingly short and in whom growth hormone deficiency was well documented. The general principle here was that in treating a patient with growth hormone deficiency, one was correcting a pathological situation with serious consequences if left untreated (even beyond the short stature). That was easy. But next came the hard part- a series of conditions were shown to be “growth hormone responsive” even though patients were not growth hormone deficient. That is, patients with certain clinical disorders associated with short stature who were clearly not growth hormone deficient, showed increased liner growth with growth hormone treatment. Examples include patients with Turner’s syndrome, Prader-Willi syndrome, mild renal disease, intrauterine growth retardation, and adults with growth hormone deficiency. One by one, growth hormone was FDA-approved for these and other disorders. Finally, in 2003, growth hormone was approved for children with “idiopathic short stature,” that is, children who are short but have no specific medical condition to explain their short stature. So, in essence, growth hormone became FDA-approved for children with short stature whatever the reason as long as they are likely not to achieve an adult height in the normal range (nominally defined as 59 inches tall in females and 63 inches tall in males and still have open bone growth centers.

The big question

So, it is not surprising that if a person takes enough growth hormone and for long enough while their bone growth centers are open, they will end up taller. We have known this for many years from patients with pituitary tumors that produce growth hormone (in adults the condition is called acromegaly, and in children it is called pituitary gigantism). So is it medically necessary to treat patients with short stature from whatever diagnosis in order to help them achieve an adult height in the normal range? And, if reaching an adult height of 63 inches is good, why not 73 inches? There is a widespread belief that “taller is better,” and considerable data support that notion in terms of earning power, social acceptance, etc. This is the crux of the problem. Is it medically necessary to make all potentially short adults taller, or is it really a cosmetic therapy, similar to cosmetic rhinoplasty, breast reduction/augmentation, and botox for wrinkles?

There may be moral and ethical questions regarding cosmetic therapies but few argue against the right of individuals to receive these types of therapies, assuming they have been proven to be safe. The question is if an insurer (i.e., all insurees) should be forced to pay for such therapies. It isn’t so easy to come up with answers. For example, is psychological trauma in a teenage girl with a misshapen nose a medical condition that fulfills the definition of medical necessity as described above? Another example is gynecomastia in a teenage boy. Is surgery to correct this abnormality merely cosmetic, even if the situation is psychologically devastating, or is it medically necessary?

Back to growth hormone

I got a bit off track in an effort to explain the problem with using “medical necessity” to determine the appropriateness of growth hormone treatment in some patients with short stature. With respect to growth hormone, the question given the enormous expense of the drug is not whether it is efficacious (i.e., increases height), but rather, whether it is medically necessary. This is really a question of rationing medical care, something that none of us like to talk about but what is surely coming, given the rapidly rising costs of medical care. It is not surprising that insurers are looking first at their most expensive services to determine if some cost savings can be achieved. These are issues that none of us can hide from. In the long run, patients will be better served if medical practitioners and insurers can sit down and discuss how best to manage these complicated situations, given that medical costs are going up and up and someday soon we will need to make painful choices about which medical therapies are clearly medically necessary and which are a luxury.  In my opinion, having an insurer ask pediatric endocrinologists what they think about such issues  with respect to growth hormone prescribing is a step in the right direction.  Of course, one important question that I didn’t discuss at all is why growth hormone costs so much- I’ll leave that question for the economists to debate.

For the past few days I have been struggling with writing an entry about two articles (and 2 accompanying editorials) on diabetes complications published in last week’s New England Journal of Medicine. My struggle has been how to convey my thoughts about these two very important articles in a coherent yet concise manner. I must confess that my webmaster has scolded me on several occasions for rambling on and on in some of my entries. He has told me that most readers have a limited attention span and that “word bites” work better than epics. I respect his opinion about such matters but I don’t agree completely; I would like to think that my readers want to go beyond superficial discussions yet not get bogged down in unimportant details. Anyway, as I thought about these matters, it dawned on me that many readers may not have a clue how to read medical journal articles. By “read” I mean how to analyze whether the authors’ claims are supported by the data presented and whether the study design makes sense.

I am not suggesting that all my readers should be expert readers of medical research papers, but rather, that one should understand how it can be that firm conclusions reached in medical studies based on analysis of the data are often completely wrong (we’re not talking about data that has been falsified).

An example of good data yet incorrect conclusions

Let me give you an example . In 1973, the New England Journal of Medicine published a study that showed increased levels of hemoglobin A1c (HbA1c) in the blood of people with diabetes; HbA1c is a minor hemoglobin component that represents about 5% of the total hemoglobin (I assume you already know that hemoglobin is the blood protein that carries oxygen around). In people with diabetes, the level of HbA1c was found to be about 10%, confirming the results of a small study carried out a few years earlier. The investigators were not able to demonstrate any relationship between the level of HbA1c and anything they could measure- blood glucose levels, diabetes complications, etc. They concluded that HbA1c was possibly a genetic marker for diabetes. Of course, many years later we all know that HbA1c is a very valuable measure of average blood glucose and a risk factor for diabetes complications. So here we have reliable data on hemoglobin A1c but faulty conclusions because the assessments for blood glucose and diabetes complications were not sensitive enough to uncover what HbA1c was really all about.

Analyzing the structure of medical journal scientific articles

Of course, the first thing one sees in looking at a medical report is the title and a list of the authors including each author’s place of work, typically a university, scientific institute, or company. Next there is the “summary,” which is, as the name implies, a brief discussion of the study goals, methods, results, and conclusions. The summary is useful for scanning medical journal articles to identify those that might be worthwhile to read. A word of caution- one shouldn’t assume that the summary conclusion has any validity.

Next, there is the “introduction,” which ideally provides background information about the study subject and the specific study goals. Basically, this section provides the rationale for the study and what the investigators were hoping to learn.

Next comes the “methods” section which describes in some detail how the study was designed and what statistical methods were used to analyze the data. Thus, a study comparing the effects of 2 different antibiotics on strep throat would provide detailed information on the study population, including how the subjects were selected (e.g, age range), how strep throat was diagnosed , and what things were assessed to carry out the comparison (e.g., number of days of fever after starting the antibiotic). In many studies, the methods section contains information that is crucial in determining if the study conclusions are valid given the study results. For example, if the study population included only people over age 40 years, it would be risky to generalize the conclusions to people of all ages.

The next section is “results.” Here, the authors present the data, unadorned by opinion. The data might include how many patients were compliant with their treatment regimen, what side-effects were found, etc. Well-written studies present the results in a very organized way and take advantage of tables and figures if they make it easier for the reader to digest the material. This section includes the results of any statistical analyses carried out (i.e, were there statistically significant differences in outcomes comparing the 2 antibiotics and how significant were the differences, if any?).

The last section is the “discussion.” This is where the authors get to interpret the study results and convey their opinions regarding the significance (meaning the importance of the results as opposed to the statistical aspects) of the results. For example, they may conclude based on the results that antibiotic B was far superior to antibiotic A and with fewer side-effects, and should become the standard of care for strep throat. In this section, authors are supposed to address what the limitations of the study might have been. For example, if the study showed no statistically significant differences between the 2 antibiotics, maybe there might have been differences if the antibiotics doses had been different?

It is the job of the reader to determine if the conclusions expressed in the discussion section accurately reflect the data in the results section. Also, the reader needs to consider if statistically significant differences found in a study are also clinically significant. For example, it might be that antibiotic B decreased the duration of fever on average from 4 days to 3.8 days. The different might be statistically significant but not clinically important (particularly if antibiotic B had more side effects or cost 3 times as much as antibiotic A).

Summary

Now are you are ready to tackle the medical journals with authority? I hope I conveyed just how complicated it can be to evaluate medical claims, even for highly sophisticated folk. I think the most important thing is to determine if the study conclusions are appropriate given the results. You may be surprised to find that often study conclusions are either much more speculative than the data warrant or that serious limitations of the study results have not been addressed.

Every medical student has heard the old adage (which, by the way, I can’t find in any anthology of famous quotations): “When you hear hoof-beats, think of horses, not zebras. ” What this means is that common medical disorders are more frequently encountered than rare ones. For example, if a patient comes in with a “runny nose,” a physician should think of an upper respiratory illness (i.e, a common cold) before considering the possibility of a cerebrospinal fluid leak. Unfortunately, many medical educators often spend much more time focusing on those zebras than the horses during medical school and residency training.

Our medical school has an excellent “problem-based learning approach” in which medical students take case history examinations at the end of each “learning block.” A recent examination was about a patient with gastrointestinal symptoms who turned out to have a very rare disorder (Whipple’s disease, if you are interested). I was a grader for the examination and enjoyed  one student’s discussion of his differential diagnosis in which he wrote something to the effect that “the diagnosis must be Whipple’s disease since it’s so rare and the type of diagnosis that might turn up on a problem-solving examination.” How right the student was. Even if he becomes a gastroenterologist, it is unlikely that he will ever encounter a case of Whipple’s disease.

How to tell a horse from a zebra

I’m not saying that it is always so easy to make diagnoses and that we should not consider uncommon disorders as part of differential diagnoses. Rather, that physicians should, if it’s not an immediate life or death situation, first focus on the most likely reasons for the patient’s signs and symptoms than on all of the possibilities, no matter how remote the likelihood. Such a “staged” approach can save both an incredible amount of money and patient discomfort, or even risk of complications from this or that diagnostic procedure or empiric therapy. More and more I am finding young physicians “throwing the kitchen sink at” a set of patient signs and symptoms rather than first thinking things through and maybe looking to see if there are any horses in the pasture.

In my next entry, we’ll come back to this question of common versus rare disorders as it relates to the diagnosis of congenital hypothyroidism.