Today is my sister’s 17th birthday. She can’t eat her favorite ice cream cake anymore, but my mother ordered a pink and purple ice cream cake anyway. Batten disease does its best to steal everything, but we grin and bear it and do our best to have normal experiences, because that’s what Taylor would do.
Unfortunately, Taylor is spending her 17th birthday in the hospital. After a rough night, she ended up being taken to the hospital by medic late this morning. The doctors can’t figure out exactly what’s wrong. But it’s likely a recent forced change to one of her medications is the main culprit.
Taylor needs this drug to remain stable, but the FDA hasn’t approved the compounding agent for it. In short, that means my family’s insurance won’t approve it, and it’s an incredibly expensive drug. And because Taylor doesn’t have this particular drug, her already diminished quality of life is taking a nosedive. Today – on what should have been all about celebrating my sister’s life – the problem came to a head. The worst part is that even if the doctors here at the hospital “fix” the issue, Taylor may not regain what she’s lost as a result of this bout. When it comes to Batten disease, that’s the ugliest part of all.
One thing that makes me so angry about watching my sister suffer is that we could help take a promising gene therapy treatment to clinic for the small price of a few million dollars. I want to shake every person who says, “But it’s so rare…” as an excuse for why we don’t do more to fight disorders like Batten disease. “Rare” is a sorry excuse for letting children suffer. The fact is, most of these people think it’s just not worth the money to save so few people.
If that’s true, how do we justify the millions of dollars it costs for hospital trips by medic and hospital admissions and expensive medications and medical equipment? My sister has a $17,000 hospital bed, a $4,000 shower chair and a $4,000 wheelchair that doesn’t even work all that well. She has a surgically implanted device to control seizures that cost almost $60,000 – not including the surgery itself. Her health care in 2014 alone cost about half a million dollars.
I love my sister, so watching her suffer cuts me to the core. But she’s one person. Take her story and think about the fact that one in 10 Americans suffers from a rare disease. Consider the fact that 95 percent of rare diseases do not have a single FDA approved treatment. That’s a lot of exorbitantly priced drugs and wheelchairs and hospital visits. Someone has to pay for all of that. Wouldn’t we rather FIX these diseases? On top of the fact that it’s the right thing to do, it’s just good business sense.
How much pain and suffering will it take for us to stop and say, “Enough is enough?”