| This is kind of a common issue with genetic disorders. When I was active on cystic fibrosis lists, sometimes articles were posted that said stuff like "It costs an average of $100k annually to treat CF" and parents would discuss it and go "That sounds low to me" and some older patient would chime in and say "Last year, my drug prescriptions alone were $100k. That's not counting the cost of doctor's visits and hospitalizations." (Estimates in articles vary wildly. I searched briefly and just trying to find something that makes sense for like two minutes just makes me tired. So if you want to jump up and dispute that with some googled up article, I don't feel like arguing with you about it. I've already told you actual people living with the condition read those articles and go "Where on earth are they getting these ridiculous low ball figures???") So then they come out with some very expensive drug that costs like a quarter million or more annually and it only treats around five percent or so of patients with CF. The price on Wikipedia [1] is currently listed at over $300k annually. [1] https://en.wikipedia.org/wiki/Ivacaftor And then who can afford that? If you have been sick your whole life, you probably don't have a lot of savings or a lot of ability to come up with big bucks, etc. You are probably deeply in debt and not making much money. I don't know the answer. Conventional medicine doesn't really work for people with genetic disorders. It tends to be crazy expensive and also merely mitigates things somewhat. The standard expectation is that you will suffer a bit less and maybe live a bit longer, but you won't ever really be well. So such people understandably want a cure. That's the holy grail for folks coping with genetic disorders. I think there are potentially other avenues for some portion of people with genetic disorders. But I don't currently have the words for that and I get tired of being ganged up on by boatloads of internet strangers who want to inform me that I'm imagining things and my entire life experience is a hallucination and I don't actually know nothing about genetic anything. So let's just leave it at "I have this opinion and maybe it provides a way out of this trap and maybe it doesn't." Because the current approach of inventing incredibly expensive drugs that might provide a cure is essentially failing as a method. And all other approaches are so awful it's why such things get labeled "dread diseases" -- because what it does to your life is so terrible that if a doctor were not prescribing it, it would be in violation of the Geneva Convention. It's just not humane. |