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Maintaining Optimism in the Face of Reality. Occasional observations on the state of the world, society, business and politics. Usually anchored by facts, always augmented by opinion.


I Could Get Behind This If It Made Them Smarter  | e-mail post

This July, the FDA did a rather interesting thing. They approved Eli Lilly's Humatrope, a Human Growth Hormone (hGH) supplement, for use in treating "short stature" in children who do not suffer from hGH deficiency. Strictly, they approved it for "idiopathic (of unknown origin) short stature." (As a side note "Idiopathic" is rapidly becoming my new favorite word, but is regrettably difficult to use in casual conversation.)
For the details, you can read the FDA release for some specifics.

Two questions: what exactly do I mean, and why on earth do I care?

Some background may help. The FDA has for some time recognized the use of hGH supplements to treat children, reasonably enough, who suffer from an actual hGH deficiency. Because hGH is important to growing bodies, this deficiency usually presents itself by a child being of short stature. Without getting into the definitions, which you can read about in the FDA document, this is the shortest 2.3% of children within their age and sex group. Pediatricians (pediatric endorcrinologists, specifically) have been prescribing Humatrope "off-label" to children who had normal hGH levels, but whose parents apparently felt they were simply too short. (Wow, way to go Mom & Dad, rather than helping develop your child's self-esteem, validate the fear that they are freakishly short!). This sort of off-label use is legal, but insurance companies, not unreasonably, do not cover off-label usage. A full treatment protocol can run up to $200,000. For reference, Eli Lilly sold $93.5 million in Humatrope in their most recent quarter, up 12% from last year.

So, what's my problem with this?

I think categorizing short stature as being an "indication," i.e. a disease, seems somewhat wrong. We are not talking about some type of dwarfism here. We are talking about an adult male who is 5'3" or an adult female who is 4'11". I'm not a real tall guy myself, and I'll admit 5'3" is pretty short, but I have a difficult time thinking of it as an ailment.

Now, if we can agree that this isn't an ailment, per se, there is an ethical question of whether we should be prescribing medications for the purpose of human "enhancement." This is a far bigger debate than I would want to entertain here, and I think that I am in general comfortable with the idea of such uses of medicine. I am troubled in this particular case because it inherently involves adults making decisions about enhancing their children rather than individuals making decisions for themselves. I see this is a clear leading indicator of the genuine market demand for pre-birth genetic enhancement in the future. I'm not talking about the world depicted in Gattaca or anything on that scale, but it does give me pause.

My biggest issue with this new indication, however, is that because it is now an official indication, and has an official diagnosis code (783.43, if you're curious), there will be substantial pressure on insurance companies to cover the treatment of this new indication. Oh, and what does that cost? Lilly claims it runs $10,000 - $25,000 per year of treatment. However, Harvey John Guyda, a pediatric endocrinologist from McGill University, told the panel that each centimeter of growth could cost "40-odd thousand dollars per centimeter" for kids with no hormone deficiency. Now McGill's in Toronto, so maybe he's talking Canadian dollars, but still. Given that even 5 centimeters is only a couple of inches, this is an expensive way to grow a basketball team. And, in all seriousness, average height gains under this treatment are only around 1.5 inches. That's a lot of money for still being pretty short.

You can read the FDA transcript if you'd like. Guyda's testimony is about midway through the transcript.

I am genuinely troubled to think about insurance companies paying for this treatment, for which something on the order of one million or more children apparently would be candidates. This becomes a very expensive entitlement that insurers, and ultimately governments and businesses, will have to pay for, and thus will we all. Think about it this way: even one-tenth of the low-end of the population at the lowest cost estimate by Lilly would total up to a billion per year. A larger patient population at more broadly acknowledged prices quickly surpasses the $10billion mark annually. My concern amplifies when I contemplate that this may be a watershed event in the use of "healthcare" dollars.

And if you think that insurance companies won't be paying for it, I'd be curious about your explanation for Lilly's "Humatrope Access Program". In this program, for qualified patients, Lilly will provide all or part of the treatment for up to twelve months. Of course, "During this time, the Humatrope Reimbursement Center will attempt to find sources of reimbursement for the cost of Humatrope for this patient." Maybe Lilly has a corporate mission to make people taller. I'm betting they are confident that they will be able to wrangle insurers into reimbursing in that time. Also, bear in mind that the treatment duration in the studies for approval of the Idiopathic Short Stature indication was 4.4 years and 6.5 years.

Interestingly, Lilly doesn't seem to make much mention of Humatrope. Their third-quarter results press release didn't mention the FDA approval for Idiopathic Short Stature, although it mentions three others. They also didn't issue a press release about the approval, nor do they say much at all about Humatrope. This is somewhat strange given that it is now generating almost two-thirds as much revenue for them as Prozac does. Maybe they are somewhat uncomfortable with the ethical quandary this approval represents. They arguably have a corporate responsibility to pursue this opportunity, particularly given the apparent demand from the off-label use of the product. At the same time, Lilly seems to be a company that prides itself on an elevated level of ethical behavior, and I imagine they may have misgivings about this.

Of course, if we had this drug 200 years ago, possibly Napoleon would have been a well-adjusted minor noble like his parents and tens of thousands of European lives would have been spared. On the other hand, this would have deprived France if its liberation from Britain and its sole demonstration of military capability or even couilles, as the French call them.
e-mail post | Link Cosmos | [Permalink]  |  | Tuesday, October 28, 2003

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