What Is “Medical Necessity” And What Does It Have To Do With Growth Hormone Treatment?

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.

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