One of the things you learned very quickly while growing up in our household was, “Money doesn’t grow on trees, you know!” That was my dad’s way of saying that we were poor. Not grinding-into-the ground, begging-for-food poor, but we started out with much less than our neighbors had. In fact, I was born into a 35-foot travel trailer in my Maw Maw’s back yard in 1961. And within two years, I had had two sisters, too!
Dad and his friends built a 1,400 square-foot house, where, eventually, seven of us lived, sharing a bath and a half and one 30-gallon water heater! I don’t think I had a hot shower until I went to college, frankly. Since Dad didn’t finish college, and Mom was taking care of babies, things were tough for quite a while. That thriftiness served us well throughout life. But, as Dad got older, a few things struck me.
“Sometimes you just get what you pay for, and you need to pay more!” he told me when I was shopping for a used car in my 20s. It was the last thing I expected to hear out of his mouth. But, I realized that he was wiser and he knew that sometimes scrimping to save a few bucks came back to bite you later. I never forgot that one …
Blasting Off Medical Breakthroughs
Medicine, and specifically the pharmaceutical business, is having that sort of you-get-what-you-pay-for revelation today, in a very specific area we’ll call gene therapy.
This is the most high-tech, fascinating field imaginable. Imagine being able to identify, within a sick child, the actual gene that is missing from their body, what effect that missing gene has on them, and then working out a way to put that gene that they weren’t born with IN their body! Amazing!
As I’m studying these new gene therapies and techniques, researchers are teaching me how they do this. It’s essentially sending the body a message with a set of instructions and a package of material. It tells our immune systems how to fix the missing part and what to replace it with. There are a few ways to do this. In some cases, these therapies take a virus and empty out its ability to cause infection, then use it like that container at the bank that goes in the tube with your money. You know the one I mean? Imagine using a virus like that empty case, putting instructions and material in it and using it to carry therapeutic genes into a person’s body for the repair of a missing gene. And then imagine doing that BILLIONS of times in a single person, until that missing gene is placed in their body and doing what it should have been doing all along! Boggles my mind to think about it.
Now that this technique has been nearly perfected and put to use, lots of genetic disorders are on the verge of a clinical solution. We’ve written about one before here. Another gene therapy drug has just come out, and many more are in the pipeline.
The most recent gene therapy drug the U.S. Food & Drug Administration approved is called Hemgenix®. It repairs missing genes that cause a horrible disease called hemophilia B. This condition can keep your blood from clotting, which means your life could be at risk with every little bump and bruise you ever get. The drugs we have today that slow down hemophilia’s symptoms are expensive. And, they have to be used for a patient’s whole lifetime because they only treat symptoms; they don’t improve the condition. These drugs don’t do what Hemgenix can do – get at the missing gene, which is the root cause of this disease.
Launching New Treatments – But Where Do They Land?
Now, you’re thinking, “This sounds too good to be true, what’s the catch?”
The catch, as with most things, is will you get what you pay for? How much?
Most of the diseases that gene therapy drugs treat are rare, affecting very tiny parts of the U.S. population. Hemophilia B, for example, affects 8,000 patients in the U.S. out of 330 million plus citizens. The space-age treatment I’ve described with Hemgenix is the closest thing to a cure people with Hemophilia B have ever seen. One treatment, and people improve dramatically. Because the drug company that developed Hemgenix spent billions of dollars to do it, their only hope of breaking even is — well, you can do that math yourself. The drug company has priced Hemgenix at $3.5 million.
In some ways, that’s a bargain. Today, the drugs hemophilia B patients use to manage symptoms could easily cost $10-$20 million over their lifetimes. Hemgenix has the potential to work in one treatment, reducing, possibly eliminating, future costs to treat symptoms. But, no matter how you slice it, $3.5 million is a lot of money.
These treatments have sparked a lively debate among health insurance carriers like Blue Cross, and among businesses that provide health insurance coverage to their employees. They have a genuine financial risk and concern here. Gene therapy costs are so high, a single employee or covered family member’s treatment can financially wreck a business. These are unpredictable, real considerations that we debate internally all the time.
The Straight Talk is, with 40 more gene therapies in the U.S. Food and Drug Administration’s approval pipeline, we have to be vigilant and innovative in how we cover treatments while keeping costs in line for all members.