Buphenyl, or sodium phenylbutyrate, the drug that keeps Jacob alive, is an orphan drug. This means that it is a drug developed for a disorder that affects fewer than 200,000 people in the United States. Normally, pharmaceutical companies would not both to fund research on these drugs, as they would not be profitable because of the small market. Thankfully, the 1983 Orphan Drug Act created incentives for pharmaceutical companies to develop and market these drugs, so that today we have something to help Jacob clean up the excess waste nitrogen in his system.
However, knowing that the drug exists is one thing. Getting your hands on it is quite another. I’m pretty sure I could get a month’s worth of Walter White’s blue meth with less effort. Forget CVS or Walgreens… you need a compounding pharmacy. After spending a few days calling around, I finally found a compounding pharmacy in St. Petersburg who would make the drug for me. Here was my conversation with the pharmacist. We’ll call her Rachel.
Rachel: “Okay, we can make the sodium phenylbutyrate oral suspension for you. But it will be very expensive. Are you sure you want it?”
Me: “My son needs it to live, so, yes, I’m sure. How much is it?”
Rachel: “$258 for a one month supply”
Me: “Okay, do you take AmEx?”
Really, what did she expect me to say? Thanks, but I don’t want to pay that much for the medication that keeps my son from suffering severe brain damage? Of course, Aetna did not cover it, despite several appeals and 24 pages of documentation from his metabolic geneticist. So, I was very happy to learn that Jacob qualified for Children’s Medical Services, a division of Medicaid. I called the pharmacist back to have her process the medication under his new insurance. “I’m sorry,” she said, “but we don’t contract with Medicaid.”
So, today I have spent the better part of the afternoon searching for (1) a compounding pharmacy (2) within a two-hour drive (3) that will make Jacob’s two medications and (4) contracts with Medicaid.
When I find it, I’m pretty sure it will be in between the fountain of eternal youth and a unicorn stable.