102,930 dimes, or $10,293 a year is how much the anti-narcolepsy medicine costs that I began taking in January. Xyrem, sponsored by Orphan Medical, with a chemical name sodium oxybate or gamma hydroxybutyrate, was approved for prescribing in late 2002 by the U.S. Food and Drug Administration. It had to also be approved by each state’s legislature as well, and Oklahoma’s was one of the very last to do so, in the fall of 2005.
How did a $2,500/year drug end up costing so much? The short answer is that generic street versions have been employed in “date rape.” The longer answer includes other factors, including the fact that sodium oxybate is a naturally occurring substance in the body (in far, far smaller quantities) that needed a means of patent protection to entice a company, any company, into manufacturing the medicine. Sodium oxybate was declared an orphan drug under the 1986 Orphan Drug Act passed during the Reagan administration. The act has been a God-send for the development of many cures or treatments that were otherwise not economically feasible. Further, the U.S. narcoleptic population is relatively small, numbering perhaps between 250,000 to 400,000. Thus the demand was potentially limited. (Despite that the manufacturer’s application as amended only covered the condition of narcolepsy with cataplexy or narcolepsy without cataplexy but also excessive daytime sleepiness, the FDA chose not to prohibit off-label use.)
In approving the distribution of Xyrem, the FDA mandated that the drug would not be available in local pharmacies, but only through one central mail order pharmacy (Express Scripts was designated), with the medicine sent by overnight courier and the patient signing for it in person. The FDA also requires that both patient and doctor complete an education program about the drug.
Behind the limited distribution is the fear, not entirely unreasonable, that local pharmacy distribution posed too great a risk for conversion to illegal use. Under the current program, the physician faxes the scrip to the Xyrem Success Program, which then turns it over to the pharmacy upon proof that the safegaurd regulations have been complied with.
Interestingly, however, the FDA may have passed on one of the best safeguards, and that is to limit the drug to treatment of the condition of narcolepsy, for which it was developed in the first place. Narcolepsy is such a serious, debilitating sleep disorder that those who have it are more likely to not only really need the medicine but to be so appreciative as to faithfully comply with the terms. Indeed, any patient who “loans” their Xyrem to someone else and gets caught can find that the drug’s classification has instantly become “Schedule I, illegal for all purposes.”
It is not that I begrudge others who have serious disorders of excessive daytime sleepiness, or that I oppose for all time trials for things such as chronic fatigue syndrome. Rather, I don’t want anything to risk Xyrem’s future availability. Many law enforcement officials and families of illegal sodium oxybate victims fought hard against Xyrem’s legalization. Most opponents were sincere, although some were unhinged zealots and a few grossly exaggerated the dangers of the drug.
I myself had to wait 12 years beyond the time a neurologist first recommended the medicine for me. So, while I would empathize with others who might have to wait, securing the safe use and distribution of the drug is vital to its continued availability.
I’m not sure yet exactly how much of the medicine is going to be covered by my insurance; I hope a lot, as I’ve so far been paying for it entirely out of my own pocket. I also hope the price drops over time, not just for my sake but because even though there are income-tested assistance programs for the uninsured, it is likely that many will go without a valuable medicine due to its high cost.
So, can you spare 102,930 dimes. Not literally. One way or the other, my expense will be taken care of. I’d like to point out, though, that Xyrem is one drug that even most drug companies would admit would never have been developed if left entirely to the free market system. It really took federal involvement to provide the needed incentives.