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Cystic fibrosis breakthrough has given patients a chance to live longer

theatlantic.com

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Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#51

Post by bdcravens »

ForHackernews wrote: post #28
bdcravens wrote: post #5

Interesting note about Trikafta and other advancements in cystic fibrosis: the Make a Wish Foundation announced that children with CF now no longer automatically qualify for their program due to the advancements in care. https://wish.org/cf-update

This is also mentioned in the article: > Recently, Make-A-Wish announced that children with CF would no longer automatically be eligible for the program, because “life-changing advances” had radically improved the outlook for them. I know this is a long piece, but half the comments in this thread seem like people didn't even bother to read it before commenting...

Fair enough. As someone who takes Trikafta, I skimmed the article since it wasn't news to me :-)

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#52

Post by amplicons4ever »

elektor wrote: post #24
bruce511 wrote: post #21

Trikafta is a real breakthrough for CF patients. What's not mentioned is the cost - about $300 000 per person per year. [1] In 2037 the patent will run out, and the generic price will likely be 90% less. Fortunately, for Americans with good-enough health insurance, it's covered, so ... yay? For those without insurance, or in other countries where $300k is basically unaffordable, well bummer for you, you'll be the las…

"In 2037 the patent will run out, and the generic price will likely be 90% less." Unfortunately, that's not guaranteed to bring a price drop. Humira is a good example of that; it recently went generic but much of the savings went to higher rebates to pharmacy benefits managers. Some reading for anyone curious: https://www.reuters.com/business/healthcare-pharmaceuticals/...

You're talking apples and oranges...

Humira is a monoclonal antibody (a biologic drug), not a small molecule. Biologics require cell culture systems and a completely different manufacturing process than small molecules, and are very complex drugs. The "generics" (called biosimilars) aren't significantly cheaper because they're simply very expensive to manufacture at scale. Humira or its "generics/biosimilars" will never be cheap. It's physically impossible with today's bio processing technology.

Trikafta is a mix of three small molecules, which are manufactured in large chemical batches and are the more traditional class of drugs. Many even have total synthesis pathways known which means you can basically make them by the train car scale for cheap.

When the patent on Trikafta runs out, it'll be very cheap.

I'll note that vanishingly few patients ever pay the full list price in the US--if you have insurance, the copay is small.

Your insurance pays (as they should, that's why you pay them premiums!). The company even pays the co-pay in most cases so the actual cost to patients in many cases is $0.

If you truly need it and don't have insurance, the company provides it for basically free with a patient assistance program, it's there on their website.

Kudos to the scientists who invented this, I don't feel bad for the insurance companies really, they charge their premiums and it's their responsibility to the policyholders to pay the applicable fees for care.

*edit: typo

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#53

Post by cbeach »

COGlory wrote: post #6
bdcravens wrote: post #4

I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory infections that just wouldn't go away. I was mentally preparing myself for inevitable decline and eventual death. Trikafta changed my situation dramatically. I've had no hospitalizations, and most the classic CF symptoms are either gone or extremely diminished.…

Sorry for the potentially insensitive question, but I really am wondering about this: >One side effect I should mention: many report extreme anxiety. However, I was starting an anxiety medication for the first time (something I should have done 20 years ago, but alas ...) and so those effects were muted or hidden to me. I would have imagined that most people with CF already had extreme anxiety - wondering when the in…

On the subject of correlation/causation, I once took a drug called Accutane to cure my teenage acne. Listed side effects included: "Mental health issues depression, psychosis, aggressive behaviour and suicide ideation and attempts"

Accutane was the best medicine I ever took. It resolved my acne (permanently) in a matter of weeks. I felt elated afterwards and never experienced mental side effects.

However, prior to the drug, I'd experienced several of the listed side effects, due to the acne itself and its devasting effect on my self-esteem.

For many teenagers suffering from serious acne, they'd also have mental side effects due to the skin disorder, meaning there would be a strong correlation between Accutane use and people suffering mental episodes. And if the wonder drug didn't cure the acne, as promised, I can imagine that failure might push some acne sufferers over the edge.

So, Accutane itself could be harmless, but the circumstances of its use might suggest causal link to mental side effects which are not causal at all.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#54

Post by bdcravens »

pedalpete wrote: post #50
bdcravens wrote: post #4

I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory infections that just wouldn't go away. I was mentally preparing myself for inevitable decline and eventual death. Trikafta changed my situation dramatically. I've had no hospitalizations, and most the classic CF symptoms are either gone or extremely diminished.…

Can you elaborate on the weight gain? I'm curious why that is a downside of the Trikafta? You say with CF the pancreas is blocked, so why did the weight come as a side-effect of the drug, and not with the disease initially?

Some of the blockage of the pancreas is due to scarring, some of it to thick mucus. The scarring can't be undone, but with a reduction of mucus blockage, food may be digested better. That said, I think that there's other causes, given how fast most who start Trikafta gain weight.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#55

Post by abhisuri97 »

pedalpete wrote: post #50
bdcravens wrote: post #4

I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory infections that just wouldn't go away. I was mentally preparing myself for inevitable decline and eventual death. Trikafta changed my situation dramatically. I've had no hospitalizations, and most the classic CF symptoms are either gone or extremely diminished.…

Can you elaborate on the weight gain? I'm curious why that is a downside of the Trikafta? You say with CF the pancreas is blocked, so why did the weight come as a side-effect of the drug, and not with the disease initially?

Med student here. My guess is the following: So with CF your pancreas can’t secrete many of the enzymes that are necessary to actually digest food. You can take medications to help with that digestion, but regardless, you aren’t actually getting all the calories in your food with CF because it isn’t making its way into your body (notably patients with CF have steatorrhea which is fat in their stool because they can’t absorb a lot of fat from their foods like a non-CF patient). The med helps the pancreas recover some of its ability to secrete digestive enzymes and so patients can now eat and get more from their food. The issue is that there needs to be a recalibration in terms of how much patients are eating. Previously a 2000 calorie diet may not have gotten them so far because they didn’t absorb much of it, but now they’re absorbing a lot more of it. Plus increased work of breathing with CF expends more calories compared to a non-CF patient (and patients on this drug).

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#56

Post by derefr »

paulpauper wrote: post #44
bdcravens wrote: post #4

I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory infections that just wouldn't go away. I was mentally preparing myself for inevitable decline and eventual death. Trikafta changed my situation dramatically. I've had no hospitalizations, and most the classic CF symptoms are either gone or extremely diminished.…

weight gain. With CF the pancreas is blocked, so you lack in digestive enzymes, and it's a struggle to maintain a healthy enough weight to battle respiratory infections. After Trikafta, I gained some 30 pounds, and have a big belly on my small frame This seems like a no-brainer way to treat obesity, by restricting or blocking such enzymes. Obviously the downside is diarrhea and other malabsorption symptoms, but obese…

They sort of tried this in the '90s — not in the form of suppressing the required enzymes for nutrient absorption, but rather in the form of diet foods that contained inherently non-absorbable nutrients. The downsides were basically the same — the non-absorbed nutrients had to come out. People didn't generally respond well to these products. See e.g. https://web.archive.org/web/20060113084223/http://www.zug.co...

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#57

Post by dclowd9901 »

bane wrote: post #7

Oh wow. I remember a kid in elementary school with CF. I didn't understand at the time what he and his family was going through. I remember his personality and intelligence, his skill on the soccer team. He was unusually driven for an 8-9 year old. As I got older and into my 20s, I thought about him a lot -- it was understood that most CF patients don't make it out of their early 20s and I knew even if he were partic…

My brother passed about a decade ago from complications from a double lung transplant, which he needed because of CF.

It seems like something every day reminds me of him. I’m really glad there’s hope for people with it. It’s a particularly shitty disease and he was the nicest most thoughtful person I’ve ever known. I miss him dearly.

Bittersweet. Glad there’s progress, wish it were earlier.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#58

Post by bdcravens »

COGlory wrote: post #6
bdcravens wrote: post #4

I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory infections that just wouldn't go away. I was mentally preparing myself for inevitable decline and eventual death. Trikafta changed my situation dramatically. I've had no hospitalizations, and most the classic CF symptoms are either gone or extremely diminished.…

Sorry for the potentially insensitive question, but I really am wondering about this: >One side effect I should mention: many report extreme anxiety. However, I was starting an anxiety medication for the first time (something I should have done 20 years ago, but alas ...) and so those effects were muted or hidden to me. I would have imagined that most people with CF already had extreme anxiety - wondering when the in…

Most of us, especially adults, are pretty much settled in with the state of our condition - we've had it our entire lives. Anecdotal, but many CF patients (including in online communities I'm a part of) have reported a lot of increased anxiety.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#59

Post by bdcravens »

paulpauper wrote: post #44
bdcravens wrote: post #4

I have CF, and I take Trikafta. Before Trikafta, I usually had a 5+ day stay in the hospital every year, and sometimes I have would very stubborn respiratory infections that just wouldn't go away. I was mentally preparing myself for inevitable decline and eventual death. Trikafta changed my situation dramatically. I've had no hospitalizations, and most the classic CF symptoms are either gone or extremely diminished.…

weight gain. With CF the pancreas is blocked, so you lack in digestive enzymes, and it's a struggle to maintain a healthy enough weight to battle respiratory infections. After Trikafta, I gained some 30 pounds, and have a big belly on my small frame This seems like a no-brainer way to treat obesity, by restricting or blocking such enzymes. Obviously the downside is diarrhea and other malabsorption symptoms, but obese…

I've played with my enzyme dosage, and there are a lot of unpleasant side effects as you've mentioned. Overall, the best approach has been what everyone else has to do: watch carbs and sugar, reasonable exercise, etc.

Re: Cystic fibrosis breakthrough has given patients a chance to live longer

#60

Post by paulpauper »

xenospn wrote: post #25
bruce511 wrote: post #21

Trikafta is a real breakthrough for CF patients. What's not mentioned is the cost - about $300 000 per person per year. [1] In 2037 the patent will run out, and the generic price will likely be 90% less. Fortunately, for Americans with good-enough health insurance, it's covered, so ... yay? For those without insurance, or in other countries where $300k is basically unaffordable, well bummer for you, you'll be the las…

In other countries this will be covered by the social safety net (that doesn’t exist in the US, hence the need for “good-enough health insurance”).

'other countries' would not even developed the drug...Vertex, a US pharma company, developed Trikafta. No one is paying the 300k out of pocket. The high price incentivizes research. Expensive drugs seems like a tradeoff for more innovation.
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