RARE DISEASES – WHO SHOULD PAY? (3)
Hepatitis does not belong in the category of rare diseases. But a doctor who is accidentally pricked by a needle from an infected patient has a 1 in 100 chance of dying. Conversely, the chances of getting sick from a needle infected with HIV is one in a million.
So it turns out that hepatitis C is far more terrifying than HIV, although people are much more scared of HIV.
We gave vaccinations, but if you were unlucky the infection could still creep in. This virus is now inside you and you’ll die from hepatitis, cirrhosis, or liver cancer, which is a very sad and painful fate. But then they came up with all kinds of treatment! There was Interferon, drugs, and injections that had to be administered several times a month. Side effects included loss of appetite, hot flashes, and others, but it did prolong life. Then out of nowhere, just a couple of years ago, one company developed a new medicine. It’s only 14 tablets, and 90% of patients no longer have hepatitis C!
So what does that mean?
The virus completely disappears from the body. The company says this – We put a high price on this unique medicine, but in paying for it society benefits. Cured people will remain useful members of society. They are not relegated to intensive care, and they will not have the usual side effects. They also won’t need a liver transplant, which is an astronomical amount of money. Therefore, when we say that this 2-week course of medicine requires $180,000, we’re not only recouping our own costs for research, testing, and production but also helping society itself.
What does society have to say about that?
Society agrees. Medicare and other insurances pay for it. Moreover, they are urging doctors to more carefully screen patients for hep C.
RARE DISEASES – SO WHAT?
Seems like a happy ending. So what’s the problem?
Suppose a person has hemophilia or Leber’s hereditary optic neuropathy, which is accompanied by a sudden loss of vision in both eyes. Both are very rare. The prevalence of Leber’s in Europe, for example, is 1 in 30,000 to 50,000. And those with hemophilia usually don’t live very long.
Up until recently, they had to receive risky blood transfusions, which were both unpleasant and very expensive. Then researchers developed the clotting factors that hemophiliacs are missing. Injections were given once a month. It cost tens of thousands a year, but at least people weren’t dying.
What about now?
Now there’s a medicine obtained through genetic engineering. You get ONE injection of a modified virus that carries the desired gene. It embeds itself in your system and replicates. As a result, it cures those born with hemophilia. They can still genetically pass the disease on, but themselves are cured and require no further treatment.
But it’s not available yet?
It will soon be approved by the FDA. There are several thousand hemophiliacs in the United States. This is the second medicine of its kind, and the first is for Leber’s. It’s basically the same thing. You get one injection, and a virus carries the necessary genetic information into the patient. So how much should medicine like this cost?
DO RARE DISEASES NOT HAVE A PRICE?
I’m not even going to guess, although it’s clear these medications are priceless.
It’s going for $850,000. But the treatment for hemophilia will be one million. The question is who’s going to pay for it. Genetically engineered drugs, like I described, are coming soon.
So they create these really unique medicines that people need, but then the prices are out-of-reach?
It’s a question for ethics professors, philosophers, and economists. Who pays and how? It could bankrupt any insurance company. The money simply doesn’t exist, so we have to find creative ways to pay for this. The company developing the medication for Leber’s suggested this – they give you the injection and you pay $150 a month for the rest of your life. If the medication doesn’t work, you stop paying.
Back to the eyes. Everything we’ve talked about, when distilled a thousand times, could be related to dry eyes, right?
Of course. If you could get rid of it once and for all, why not pay a hundred bucks a month for a year or so?
I’d do that for sure. Crowdfunding is another creative way to generate money for super-expensive medications. As soon as you have that smart machine that cures dry eyes, I hope we’ll return to this topic.
We installed it a month ago.