Cystic fibrosis breakthrough has given patients a chance to live longer

https://www.theatlantic.com/magazine/archive/2024/04/cystic-fibrosis-trikafta-breakthrough-treatment/677471/

by cowboysauce • 2 years ago

422 147 2 years ago

leetrout• 2 years ago

I lost a cousin and a family friend to CF. Both in their 20's. One after a lung transplant added some time.

It is such a horrible disease and the breakthroughs are amazing. So many families will be much more fortunate than mine and I am happy for them!

forgetfreeman• 2 years ago

I had a coworker with CF that started this treatment. Over the course of their first year of treatment, as it became plain to them just how effective it was and what the implications for their long-term survival might be, their behavior changed drastically. Within 18 months of starting treatment they were showing worrying indications of both budding mental health issues and heavy substance abuse. Their marriage, which had been stable for years, was in shambles as well. I can only speculate that their exuberance at being given a stay of execution lead them to "oversteer" into some questionable lifestyle choices.

bane2• 2 years ago

This section seems to speak to your observation (edited for brevity):

Doctors told me they could think of only one other comparable breakthrough in recent memory: the arrival of powerful HIV drugs in the 1990s. Like Trikafta, those drugs were not a cure, but they transformed AIDS from a terminal illness into a manageable chronic one. Young men got up from their deathbed, newly strong and hale. ... This was a remarkable turn of events. But it elicited a complicated mix of emotions, not all of them joyful. Some patients who were no longer dying grew depressed, anxious, and even suicidal at the thought of living. This phenomenon became known as “Lazarus syndrome.”

Death is an end, after all. Life comes with problems...the writer Andrew Sullivan, who is HIV-positive, described life after the advent of the HIV drugs in his essay “When Plagues End”:

   When you have spent several years girding yourself for the possibility of death, it is not so easy to gird yourself instead for the possibility of life. What you expect to greet with the euphoria of victory comes instead like the slow withdrawal of an excuse. And you resist it.
   The intensity with which you had learned to approach each day turns into a banality, a banality that refuses to understand or even appreciate the experience you have just gone through.

For some HIV patients, their reversal of fortune seemed unreal. “He doesn’t trust what’s happening to him,” one doctor said about a patient who had made a dramatic recovery, yet found himself in psychological distress."

gfodor• 2 years ago

I have CF, and my whole life I avoided things like CF communities explicitly because I felt these ties to the disease would lead to a crisis in my life if it was ever something I could stop centering my life around.

Aurornis• 2 years ago

Chronic illness forums are almost universally terrible. I try to navigate the online communities for a family member’s disease to keep up with new developments. A decade ago I found some value in the way they presented news and research and anecdotes.

Now, the forums are overrun by small numbers of constantly online members who feel the need to dominate every conversation. The content has become almost entirely venting and memes, with an unreasonable amount of alternative medicine being pushed as fact. It’s understandable that they’re frustrated, to say the least, but the way their frustration gives way to a communal rage against doctors has weirdly opened doors to alternative medicine peddlers. It’s disgusting to me to see how the alternative medicine pushers have arrived with open arms and comforting smiles for vulnerable communities, which slowly becomes a sales pitch for their products.

I’ve seen everting from people peddling custom diet consulting based on your 23andMe results to invitations to private, paid Telegram channels where they supposedly share their secret cures, to doctors from Eastern Europe who claim to have cured the condition (which has eluded many researchers and pharmaceutical companies) with a custom treatment made from the patients’ own urine. The way these communities set themselves up to rage together at modern medicine opens the door for friendly alternative medicine scams.

It’s depressing.

bdcravens16• 2 years ago

I also have CF, and my diagnosis was different than most: I grew up dirt poor and wasn't diagnosed until I was almost 14. Even after diagnosis, it pretty much became incumbent on me to manage my care. I feel that not being put in a bubble and not being told I was going to die kept CF from shaping my perspective (today I'm 47 and have all the boring problems people my age have. My health is tolerable, sitting at about 50-60% lung capacity)

bdcravens16• 2 years ago

Many have reported severe anxiety due to Trikafta. As I mentioned in another comment, I believe I experienced the same, but I also started Lexapro around the same time for other non-CF reasons, so independent test variables and all that.

qgin• 2 years ago

Imagine if tomorrow, it was announced there had been a discovery that doubled average human life expectancy to 150. There would be happiness, but there would also be chaos. How many people would quit all the things they'd told themselves they were too old to change? The job they were grinding out until retirement. The marriage they'd resigned themselves to? Imagine billions of people getting pulled backwards back across the midlife crisis line, realizing that yes, maybe there IS more to life than "this". The days and weeks after that announcement would be some of the most chaotic the world has ever seen.

dennis_jeeves23• 2 years ago

There would be happiness, but there would also be chaos

Which is good in the short term, correct? Long term people hopefully would be wise to settle for stable relationships, jobs etc...

bdcravens16• 2 years ago

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. It can't undo a life of damage to things like my pancreas, vas deferens, etc, and I still take medicine to digest my food, but overall, it's as close to "normal" as I could hope for at this point.

The only real downside: 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, and went up some 6 inches in the waist. To add insult to injury, it happened during the spring and summer of 2020, when buying clothes was a challenge due to the pandemic.

Speaking of the pandemic, the timing of Trikafta was amazing: it kept the normally full "CF floors" of hospitals empty, opening up those beds for those with COVID and keeping CF patients less exposed.

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.

(copied from the last time Trikafta was mentioned here: https://news.ycombinator.com/item?id=37540731)

COGlory• 2 years ago

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 infection that is going to end one's life will arrive. Is it really possible that Trikafta is causing noticeably worse anxiety?

therein4• 2 years ago

Just an anecdotal experience so doesn't necessarily mean anything but the only person I knew that had CF had absolutely no anxiety from his condition.

If anything he was a very mature guy for his age, having realized he has limited time but of course I wouldn't know the internal struggles he might have kept from us.

mason55• 2 years ago

I would have imagined that most people with CF already had extreme anxiety - wondering when the infection that is going to end one's life will arrive. Is it really possible that Trikafta is causing noticeably worse anxiety?

There's a discussion of this in the article. Essentially, a bunch of people have said that they started experiencing extreme anxiety when they started Trikafta, and that stopping or lowering the dose helped immensely. However, clinicians have said that there's no actual evidence that the Trikafta is causal.

So it seems to be a bit of an open question and I'm sure an emotional one on both sides.

vkou• 2 years ago

People generally get acclimated to the situation they are in.

If you have anxiety over money, for instance, you'd probably still have it, regardless of whether you had $15,000 in your bank account, or $15,000,000.

cbeach3• 2 years ago

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.

bdcravens16• 2 years ago

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.

junon• 2 years ago

Just an anecdote.

My best friend died of CF just a few years ago. He talked candidly about his eventual death, to the extent it was uncomfortable at first (but we ended up being to the level of making jokes about it, which I think was a positive thing for both of us).

He changed his WhatsApp status to a coffin and skull-and-bones emoji right before he went, just as a joke.

He didn't live with a shred of anxiety - least anxious person I've ever met. Dude was down for anything and everything, and could have probably fought off a bear despite being comically short and concerningly skinny. Easily one of the most kind and relaxed souls I've ever met.

matthewtse2• 2 years ago

(I left a previous comment as a similar sufferer of a lifelong genetic disease that was cured by a breakthrough medicine: https://news.ycombinator.com/item?id=37541222)

Funnily enough, although in practical terms my life is infinitely better than it was before the drug in, I would rate my anxiety as higher than it was before.

A lot more options open in the world, a lot more to hope/pine for, a lot more to lose. Back in the days when my expected future was to just hang out in bed, there wasn't much else to do than to just chill and read a book.

therein4• 2 years ago

One of the first friends I made in college was a friend with CF. I didn't know what CF was. He was very mature for our age and generally a very nice, happy, caring person. Got internships, studied with us. Went to bars with us but never drank because he couldn't. He had stories about how Make-a-Wish foundation has been dragging their feet not to give him his wish since his childhood.

He would take his enzymes, do the vibrating jacket treatment. He graduated and got a full time offer from a nice company. As he was relocating, he picked up some infection and passed away in a few weeks. None of us expected it. This was months before I heard of the approval of Trikafta.

bdcravens16• 2 years ago

Never had a problem drinking. (So much so that I had to quit a few years ago)

paulpauper3• 2 years ago

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 people have too much nutrition. Blocking absorption seems easier than dieting, as no willpower required. You shit out the undigested food.

derefr• 2 years ago

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...

bdcravens16• 2 years ago

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.

patmorgan23• 2 years ago

Appetite suppression/regulation seems like a much better route to pursue than intentionally inducing malabsorption.

vrc2• 2 years ago

Orlistat. Look at Alli — very available to buy. A very common side effect is steatorrhea. Oily leakage/poops. But not like, “oops I gotta run to the bathroom” but like, a tiny bear down and oil shoots out your bum. There is a clear warning on it about malabsorption as well of fat soluble vitamins.

pedalpete2• 2 years ago

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?

bdcravens16• 2 years ago

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.

abhisuri97• 2 years ago

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).

jMyles• 2 years ago

Reading your comment, I was certain I remembered it - and I see that you've now added a link to its previous home.

So I just wanted to say: it was an impactful piece of writing, enough so that I remembered it quite clearly after a single read these few months ago.

bdcravens16• 2 years ago

Thank you. My aim isn't to wax poetic or elicit sympathy, but to provide insight for others.

cogman10• 2 years ago

The only real downside: weight gain.

Interesting. I'm an uncle to a family member with CF (and I'm a carrier) and one of their long battles has actually been weight. To the point of doing things like downing olive oil to try and keep up the weight. They've eventually had to take the fairly drastic measure of getting a feeding tube installed. Getting enough calories in just hasn't been possible until the feeding tube.

bdcravens16• 2 years ago

Yeah, it's been quite a shock trying to reverse the first 40+ years of my life where I was told to eat eat eat. Not just for the weight, which is really a vanity thing, but also to counteract the diabetes that most with CF develop, since that can directly impact overall health and the ability to fight off infection.

joney_baloney4• 2 years ago

My younger brother has CF and takes Trikafta and it's absolutely made all the difference in the world, it's wonderful to see. Watching all the years of research and CFF funding finally pay off is really something.

My personal situation is probably pretty unique, in that I have CF as well, but had a double lung tx many years ago. A couple years ago my transplant team (actually it may have been the CF people.. fuzzy memory) asked me if I was interested in getting on Trikafta and after thinking it over for about 30 seconds I told them I'd probably pass. It seemed like it wouldn't have any beneficial effect on my lungs, as they no longer "have CF", and that I assumed it would just make extra weight for my decently healthy but still under par lungs to have to drag around. They didn't disagree.

Sammi2• 2 years ago

Yes but CF affects all liquids in your whole body. You can get diffuse long term damage anywhere and everywhere in your body. Having to eat calorie amounts like someone without CF sounds like a good tradeoff in order to not risk this damage.

abdulhaq• 2 years ago

I'm in a similar situation so it's funny to see someone else here with the same. I had my transplant in 1988. There's also Steve Rasmussen (you can google him) who AFAIK also does not take Trikafta.

sunnybeetroot• 2 years ago

Assuming your anxiety medication is an SSRI or similar, your weight gain could be attributed to this as it’s been observed in people who take these medications.

7thaccount• 2 years ago

I had a close family friend (many decades older than me) lose his young daughter to CF back in the 60s or 70s. He and his family helped run the CF walk each year in their area and I'm sure made some major donations to research regarding it.

Hearing about any kind of advancement is huge. I can't even begin to understand what you've gone through, but am so happy to hear there is a new medicine to help with such a cruel disease and that it's made a world of difference to you. Sometimes it feels like medical science is still in the dark ages and I get saddened that we haven't got flying cars or Dr. Beverly Crusher can't wave some device over me and fix all the ailments I now have. Other days I hear about these kinds of advancements and it makes me smile!

bdcravens16• 2 years ago

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

therein4• 2 years ago

My friend that had CF was eligible. He was asked by the foundation what he wanted when he was 5-6 years old. He said he wanted to go Zorbing in Australia.

They dragged it until he was 18. He always jokingly said "make a wish foundation is waiting, hoping I'll die".

He finally got to go Zorbing when he was 19. Passed away a few years later.

Not surprised the foundation jumped on Trikafta to remove eligibility.

ForHackernews4• 2 years ago

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...

tnias23• 2 years ago

There’s a sign-up process required.

bdcravens16• 2 years ago

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

bane2• 2 years ago

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 particularly lucky, as time went on and I aged, he was probably gone.

My father also recently died from lung cancer, and had a few months where he was effectively drowning in his own lung fluids, requiring doctors to drain his lungs with long needles through his back. That experience also brought me back to that schoolmate, considering what he had had to endure.

Recently, I was reorganizing my personal library and came across my elementary school yearbook and flipping through, saw his picture. It's been decades since he's likely passed on. It gave me pause to contemplate certain priorities in my life and try to cultivate greater compassion.

It's a particularly cruel disease, and this news is wonderful.

jhoechtl• 2 years ago

Those were wonderful words and moved me a lot.

dclowd9901• 2 years ago

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.

speedylight• 2 years ago

Have you tried looking him up? He could still be alive.

dotnet00• 2 years ago

I remember studying about how CF was an agonizing death sentence just 10 years ago in highschool. It's both interesting to learn that even at that time the life expectancy info was outdated, and even cooler to hear this news.

varjag• 2 years ago

It appeared too late to make a difference for my dear friend and the kindest human being I knew. The manifestation of life's injustice drives me mad ngl.

alleycat5000• 2 years ago

Breath From Salt is a great book on CF and it's history in medicine.

https://benbellabooks.com/shop/breath-from-salt/

yalok• 2 years ago

I wonder if this applies to kidneys cystic fibrosis?

I friend of mine passed away a few years ago from it, and it was painful to watch slow degradation in his health over the years.

josefrichter• 2 years ago

Around the time Trikafta became widely available I used to work for a clinical trials company. I remember one guy with CF telling us the full story of his life, how plenty of his friends with CF died in their 20s, and how, after taking the new meds, he felt effects within the first hour. It was like a miracle. If I remember correctly, there’s still some 10% of CF patients for whom it doesn’t work but I hope we will soon eradicate this horrendous disease altogether.

bruce511• 2 years ago

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 last generation to die of it.

I get the insane costs, and risks, of developing these things. I get that the profit motive is what drives there to be any research at all in pharma. I get that the price has to be high for everyone, or insurance companies will balk.

And yet, even knowing all that, there's a sour taste when we -could- (literally) save lives, but, well, money first ya-know...

I don't have an answer to this issue- there are downsides to all proposals I've heard. But this approach seems, well, pretty harsh.

[1] https://www.statnews.com/2023/11/03/trikafta-cystic-fibrosis....

elektor• 2 years ago

"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/...

amplicons4ever• 2 years ago

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

xenospn• 2 years ago

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”).

ploika• 2 years ago

Not necessarily. I don't know about other countries, but I remember there being a campaign here in Ireland (with one of the highest rates of cystic fibrosis in the world) to get Orkambi covered under the Drug Payment Scheme when it was new.

BurningFrog• 2 years ago

Or the country decides to not import the medicine due to the cost.

paulpauper3• 2 years ago

'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.

astrange• 2 years ago

The socialized medicine systems don't have infinite money just like private health systems don't; if they can't negotiate a cheaper price for the medicine they won't purchase it.

(It's not "health insurance". Insurance covers unpredictable outcomes; needing a medicine every month for the rest of your life is not unpredictable.)

refurb• 2 years ago

It’s not. That’s what a lot of people don’t understand.

These drugs from Vertex started being approved in 2014. US insurers pretty much covered them from approval. Even Medicaid (health insurance for low income) started paying for it.

Other countries? The UK just started in 2020.

In Canada? Last I checked only 5 of the 10 provinces paid for it and only if you’re under 18.

The US healthcare is expensive for a lot of reasons, and one of them is new technology gets paid for really quickly.

umanwizard• 2 years ago

No country pays unlimited amounts of public money for brand new drugs.

ForHackernews4• 2 years ago

It is mentioned. Quoting directly from this piece...

Or stopped being covered by insurance? Trikafta’s sticker price is more than $300,000 a year. Insurance typically covers most of that cost—minus what can be significant co-pays and deductibles—and Vertex offers co-pay assistance. But patients’ lives ultimately depend on decisions made by nameless bureaucrats in rooms far away: Insurance plans can suddenly change what they cover, and in 2022, Vertex announced that it would substantially reduce its financial assistance.

Aloha2• 2 years ago

I suspect very strongly at 300k a year, its still cheaper than treating CF via existing means.

nwiswell3• 2 years ago

In many other countries, patents do not apply to personal use, where there is no sale.

Since Trikafta is a blend of 3 small-molecule drugs, is it possibly feasible to publish a do-it-yourself synthesis process?

Or perhaps the community could even come together to set up a lab, where individuals could travel and go through the motions to produce their own?

I recognize this isn't ideal, but it might be legal...

deadbabe• 2 years ago

If you’re going to do all that why bother with legality, just push pills on a black market.

0xB31B1B• 2 years ago

drugs are priced differently country to country and the price has nothing to do with the unit cost to manufacture. The US is the least regulated pharmaceuticals market in the world and drugs here usually cost significantly more than drugs in other locals. For example semaglutide/ozempic costs like 1300/month in the US, but only 100-200/month in European countries. Also, the "1300/month" cost in the US is only paid by people who are paying cash and not covered by insurance, if the drug is covered by the patients insurance than the Pharmacy benefits manager usually pays a fraction of the 1300/month price as it is a "bulk negotiated discount, so they often pay 200-300 USD, the actuall price in these deals is a closely guarded trade secret.

glp1guide• 2 years ago

So note that although Semaglutide/Ozempic cost that amount retail, most are absolutely not paying that price.

https://glp1.guide/content/glp1-pricing-2024-02

Of course, insurers are struggling to not cover the bill, and although it's in the medicaid list it's up to states to choose whether to cover it or not

bdcravens16• 2 years ago

Vertex is very good about providing programs to put it in the hands of those who need it. I have great insurance and a great income, and they even apply deductible assistance to me.

In the online CF communities I'm a part of, I've never heard of anyone who couldn't get access to the medication (aside from the country they're in not approving it) (I'm in the US so my perspective is probably skewed)

cbeach3• 2 years ago

I've seen conversations like this before, regarding the heavy cost that Big Pharma levies on treatments for major diseases. And US companies frequently come up in these discussions.

But the alternative, without the massive cashflows, is a world where the research never happens, and the diseases haunt us forever.

The Big Pharma treatments will eventually become public domain, and may inspire other cheaper treatments prior to that.

The fact that these conversations come up frequently is a symptom of the fact that Big Pharma is frequently doing fantastic things. Curing diseases that have plagued humanity for millenia and killed millions.

We have a very bright future. But the future is not free of charge, as we'll continue to require a lot of medical research, and medical researchers have families to feed, like the rest of us. Medicine costs money - there's no escaping it.

In the UK, we sometimes mistakenly imagine that our socialised healthcare system is "free." It's not. It's one of the most expensive in the world, and employs over 2 million people. The component of tax that I paid to fund the National Health Service was £8919 last year - the largest single expenditure of my taxes, and vastly more than I'd need to spend to get private healthcare.

So while American medical pricing is scary and sometimes ruthless, I think we need to put things in perspective and recognise their leadership in medical research - without which, we'd all be in a worse situation.

kelnos• 2 years ago

What about direct government funding? Isn't that a reasonable alternative? Maybe not a politically-popular one, but I wouldn't be surprised if health care overall would be cheaper under a system where pharma research is done with public money, and then isn't allowed to be patented. Might even in save enough money to avoid much in the way of extra taxes to fund it.

Hell, a lot of medical research is funded (in part) by public dollars, and then the results get locked up behind patents for years, which is gross.

matthewtse2• 2 years ago

I love this reply.

It's a particularly touchy subject because it involves people's health and the ability/inability to pay for it.

But as a sufferer of a debilitating disease with a miracle drug that costs an arm and a leg, I've become a convert to the US pharma-industrial complex. These drugs take billions of dollars and decades to research--that funding/development isn't going to happen without venture capitalists who shoulder that risk for the profit reward. An equal number of pharma venture capitalists lose all their money, researching dead-end drugs.

I'm incredibly blessed to have both a cure invented during my lifetime and the means to pay for it. But even if I lacked the means, I would still infinitely prefer a world where a cure exists that I need to somehow access, as opposed to a hopeless world with no cure.

google234123• 2 years ago

Take away the money and watch the development go away ;)

orzig• 2 years ago

I loved the book The Billion Dollar Molecule (Reviewed by Nature here: https://www.nature.com/articles/nbt0594-521.pdf) about the founding of Vertex Pharma. Really drives home the amount of money, time and luck it takes to make a breakthrough - the author needed to write an entire second book to get to the part where they started making real revenue. The second book isn't as good, but if you like the first enough you might try it anyway.

BenFranklin100• 2 years ago

This article gives short shrift to the role the pharmaceutical industry played in making this life-changing treatment a reality. It barely mentions Vertex. Vertex is the biotech company that made the big bet on cystic fibrosis, a rare disease that most companies wouldn’t touch because of the small market and unknown biology made it too risky. They were the ones that believed in the science and developed multiple CF drugs and got them into the hands of patients. Here’s a 2019 STAT article that gives a fuller account of Vertex’s role:

https://www.statnews.com/2019/10/23/we-conquered-a-disease-h...

jeremiahbuckley• 2 years ago

Thanks for this. Very cool to read these types of stories.

bdcravens16• 2 years ago

Yes, the CFF has partnered with companies like Vertex to take an almost VC-like approach towards developing new drugs (Trikafta being one of many similar drugs they've developed with Vertex)

rakejake• 2 years ago

The book "The Billion Dollar Molecule" is about the founding of Vertex Pharma. Worth a read.

ackbar03• 2 years ago

There's a follow up book as well, The Antidote. I read both to learn more about the industry

ForHackernews4• 2 years ago

This is the future I want to live in: one where radical advances in gene therapy are curing (or nearly curing) horrible illnesses humanity has suffered with for thousands of years.

Scoundreller5• 2 years ago

already a thing: https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9512634/

dennis_jeeves23• 2 years ago

Summarize it in plain layman's language for me please...

kouru225• 2 years ago

When I was a kid, I was told my cousins would die before they turned 30 because of CF. One of them ended up dying when he was 10, but other is now above 30 and seems incredibly healthy.

The amount of development in the treatment of CF over the last 20 or so years is so incredible it’s insane.

clumsysmurf• 2 years ago

I wasn't able to read the article (behind paywall) but this was also in the news recently, related to CF & Zn.

https://newatlas.com/medical/zinc-lung-macrophages-anti-bact...

SEJeff• 2 years ago

The internet archive has your back. Here is TFA: https://archive.is/gD49J

paulpauper3• 2 years ago

If CF like breathing through a coffee straw, as some sources say? I tried it myself, and god I hope not. I lasted a few minutes and was gasping for air.

bdcravens16• 2 years ago

I have CF. My lung capacity is around 55% (many have lower, and around 20-30% is when they tend to start talking lung transplant)

I wouldn't use that analogy. It's more like breathing in a deep swimming pool or a very humid sauna. Slight physical exertion (like stairs or hills) can be like most people doing strenuous exercise. Laziness tends to be a way of life :-)

joney_baloney4• 2 years ago

Once it gets bad, yeah. I was very end stage before my lung transplant and it's an accurate description.

pedalpete2• 2 years ago

https://archive.is/gD49J

dm8• 2 years ago

This is incredible. I wonder if there will be medicine that cures idiopathic pulmonary fibrosis. New class of drugs like Ofev stop worsening of the IPF but doesn't cure it.

optymizer• 2 years ago

This is great news and hopefully with time there will be a definitive cure. I found out about CF when we did genetic testing for family planning. It turned out me and my spouse were carriers of the gene. They said about 25% chance that the embryo would have CF, and since I soon learnt it is a terrible disease I did not want to risk bringing a child in this world with CF.

We did IVF instead and to my surprise, 60% of viable embryos had CF, which I thought was unusually high compared to what I had been told prior to the procedure.

We were very fortunate that we did the testing and had successful IVF pregnancies, but so many other families aren't so lucky. I hope advancements in CF treatment will make it a non-issue for parents in the near future.

ETH_start3• 2 years ago

If I can be allowed a bit of sophistry:

It's a beautiful thing for treatments to be devised for rare conditions, to give people decades more life. It would be more wonderful still if we devised treatments for aging, the universal condition, to grant everyone decades more life.

Hardly anyone deserves to die. The vast majority of people are singularly incredible and worthy of life. They deserve to be equally safe from murder, fatal accidents, rare diseases like cystic fibrosis and the universal disease of aging.

mmustapic2• 2 years ago

Many incurable diseases come with an awful quality of life, like CF. Even cancer, treated or not, is a terrible experience. Just aging, what you call “a disease”, is nothing like that.

ETH_start3• 2 years ago

Aging comes with a terrible quality of life. By the end, the person is severely debilitated.

Osteoporosis can make it so that a fall can lead to major fractures, which leads to a severe reduction in a person's independence. Macular degeneration can make an elderly person blind.

Neurological deterioration can take the form of conditions like Alzheimer's, where a person loses their memory of everyone they love.

The body, as it ages, becomes much more susceptible to other, terrible, diseases as well, like cancer, heart disease, and diabetes, each carrying its own set of debilitating effects that degrade quality of life.

dennis_jeeves23• 2 years ago

It would be more wonderful still if we devised treatments for aging, the universal condition, to grant everyone decades more life.

Well, people are people. Somehow the life of a 90 year old is less worthy that than that of 20 year old. As to how they arrived at the magic number of 90 remains a mystery to the fictitious society where individuals routinely live for 9000 years.

warion• 2 years ago

This sounds bad and can give edge to republicans as generally rightist people have more incidence of CF

Cystic fibrosis breakthrough has given patients a chance to live longer

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