Six years ago, after my son Case was diagnosed with Hunter Syndrome, a rare genetic disorder, one of the first things I did was to check the lifetime cap on our health insurance policy.
The medicine FDA-approved to treat the physical effects of Hunter Syndrome, Elaprase or technically, idursulfase, costs around $400,000 per year. I calculated how long it would be until we’d reach our $5 million cap – by my estimate, not including other expenses like the myriad of surgeries, tests, and appointments a child with Hunter Syndrome faces, much less other health care expenses for our family, at about 13 years.
So my brain then said, “Well, he was diagnosed at two years old and the approximate life span of a child with Hunter Syndrome is around 12-15 years old, so maybe even taking into account other expenses, we’ll be covered.”
Is that terrible that I had to consider my child’s “life span” in my calculation of insurance coverage? It sure felt terrible.
But that was the reality before health insurance caps for children were removed by the Affordable Care Act.
And once Case enrolled in a clinical trial for intrathecal idursulfase (a more concentrated and pH modified form of the drug administered into the spinal fluid to stop the brain decline caused by the disease since regular idursulfase doesn’t cross the blood-brain-barrier), we knew that there may be an additional expensive drug to add to Case’s lifesaving treatment cocktail.
So every month, I take delivery of $35,000 of medicine and every Sunday, I infuse around $9,000 worth of that drug into my son. It keeps his legs walking, his airway breathing, his heart pumping, and his liver and spleen from swelling to twice their normal size.
And then every month, he receives what, on a price per milligram basis, constitutes another $5,000 of that molecule. It keeps his brain from deteriorating and his mind from forgetting who I am.
So how do I feel about having a child whose body has already consumed approximately $2 million worth of medication and is projected to receive millions more over the course of his life? How do other such parents feel whose children receive some of the most expensive medicines in the world, like Glybera, Soliris, Naglazyme, Vimizim, and Elaprase?
1. We are thankful. Just ten years ago, these drugs did not exist. Were it not for the Orphan Drug Act’s incentives for developing drugs for rare diseases, our children would still be suffering and dying at very young ages.
2. We feel guilty. We realize the actual and perceived burden that these drugs place on the health care system, both individually and as a whole. Although the cost of one drug in the entire health care system, or even among prescription drugs, is relatively small, we know that it indeed impacts costs, if only the rating of our group health plans. We wish the drugs were less expensive, but we also know that without the incentives and pricing structures, these drugs likely would not exist, see #1 above.
3. We are fearful. We fear not having access to approved drugs (because of governmental or insurance policies), losing access to drugs we see benefiting our kids, or disappearing benefits of the drugs themselves. Because of all the attention on drug pricing, especially in the last few months (thanks a lot Martin Shkreli!), we fear that the Orphan Drug Act will be amended (see Law for rare disease drugs needs revamping, researchers say), removing incentives to develop new drugs to help our kids, or that other legislative changes will be made which will impede orphan drug development. We fear that the drugs in development will not be approved by the FDA, despite the benefits parents clearly see, because we’ve somehow entered a rabbit hole of a system incapable of appropriately measuring those benefits. We fear that no one will ever develop treatments for some of our children’s conditions. We fear that we will not raise enough money to fund the cure ourselves.
We fear hearing songs on the radio and our brain automatically placing them on the shortlist for our child’s funeral. Plain and simple, we fear losing our children to pain, paralysis, and personality vacuums and then ultimately, to the grave.
4. We are hopeful. Despite #3, the smiles and laughter of our children, even if they exist only in our memory or on grainy video, give us hope. We hope that the children diagnosed tomorrow, or 10 years from now, will have a better life. We hope that we’ve made a difference. We know our kids have made a difference, in our lives and in the lives of those around us. We are hopeful for a future where they are free from the disease that steals their body and their mind, but never their joy.
5. We are conflicted. If #3 and #4 listed sequentially didn’t convince you of this already, we are conflicted by diseases that have altered our lives immensely, but in reflection, have made us more thankful, blessed, patient, and reflective human beings. We hate the diseases, but love the children that have emerged from their effect, the siblings that have been refined, and the community that has converged to fight a dragon we never knew existed.
6. We are fighters. In the end, we are fighters. We will fight to remain thankful, resolve our guilt, combat our fears, spread our hope, honor our conflicts, and cure our children.
How are we hoping to cure our children? Visit ProjectAlive.org to learn more.