Pretty cool what it can do (sadly it doesn't cure it though), but with that price tag, it's pretty expensive for a person to pay to live...IF they can even afford it.
I roleplay with a friend who's nephew has cystic fibrosis. The kid is very cute and his illness distresses me. Its got to be vastly worse for his family. I hope this will wind up working for him.
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If you're going to whine and b~#!# about cost or availability, please go start another thread to do it in. This is intended to be a happy thread where we can celebrate an awesome milestone in battling a terrible disease which affects several friends of mine - all of whom have expressed nothing but happiness about this news.
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For the low, low price of a quarter of a million dollars a year.
Agreed. It's expensive.
Available-but-expensive, however, is a significant step along the road from unavailable to available-and-cheap. A lot of people would argue it's the hardest step. Let's not let the perfect be the enemy of the good, or in this case the enemy of the not merely good but effin' AWESOME!
So, Go Vertex!
At least they are making progress. Yes, it is expensive. Having said that, the cost SHOULD go down and even if it doesn't, this gives us clues as to what direction to start looking for other treatments.
Edit - Besides, ALL research is expensive. That is what the initial cost tends to go to. In addition to that, often the manufacturing process is also difficult and expensive.
Costs will come down at worst when the patent runs out.
And the principles behind the new treatment will hopefully be some sort of breakthrough, which will lead to other treatments.
Yes, research is expensive. However, often the initial breakthroughs are made by academics at universities. Drug companies get involved later, getting approvals et al, purchase the rights and get most of the profits.
But as Aberzombie insists, this is great news and hopefully this will help his sick friends.
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Two of my fraternity brothers have this disease. The older one is Steven, our first Chapter President. He's 51 years old and counting. He's one of the most inspiring people in my life. He's been in an out the hospital all his life, watched other, younger people be taken, and even lost his own brother. He never gives up.
The other is a younger man whom I've not had the privilege to meet yet (and who's name escapes me). Next year, at the chapter's 30th anniversary, I will shake his hand.
The wife of another brother also has CF. They were college sweethearts a bit younger than me, so I know them both. She is another strong one. She's had a double lung transplant.
There are others I could name - most of whom I met only briefly, in the course of my fraternity's philanthropic work. Several of them are no longer with us.
Aberzombie- Is that a University fraternity?
I have 2 degrees and we don't have fraternities in Australia. And from the media you would think US fraternities never involved anything except getting blind drunk and behaving like an animal. It's good to hear this isn't true, or at least only part of it. If that is the fraternity you are talking about.
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It's Delta Tau Delta International Fraternity. Greek organizations are far more than the media and other craven jacknuts make them out to be. Most of them do a tremendous amount of charity work. My own also has an excellent record for academics. And many have some great leadership programs. Then there's the sheer pleasure of being part of a brotherhood that stretches back over 150 years.
Are their problems? Yes. Terrible ones sometimes. And those are things we (old and young, active and alumni) constantly work together to solve. It's not perfect, but it's a damn hell of a lot better than is typically portrayed.
Are they all zombies?
Barely remember any of the fraternities on my campus doing anything other than throwing the occcasional party or going to bars. If they did any charity work, I didn't see it.
Good to know about this med. Here's hoping the company behind it is a good one. Cystic fibrosis is a monstrous disease.
Well, the media cleche's are part of the truth too. Sadly.
Your fraternity was formed by Greek immigrants? Just a guess.
Well, the media cleche's are part of the truth too. Sadly.
Your fraternity was formed by Greek immigrants? Just a guess.
American college fraternities are generally named after Greek letters. Little to do with actual Greece.
This is an international one though - I'd be interested to find out about how it was founded and became international.
This is an international one though - I'd be interested to find out about how it was founded and became international.
For the low, low price of a quarter of a million dollars a year.
Good news if you're one of the people the drug works for and if your insurance will cover it. A cruel joke if you're a candidate and can't afford it.
The cost will come down. Antibiotics were an absurdly expensive top secret government project when they came out.
Edit - Besides, ALL research is expensive. That is what the initial cost tends to go to. In addition to that, often the manufacturing process is also difficult and expensive.
I don't know about this current drug, but I have some figures on the previous drug that Vertex developed for CF. They received $75 million from the Cystic Fibrosis Foundation, a charity, to fund their research. This doesn't include money from other charitable sources, or the federal government. In return the CFF got a cut of sales, the exact amount isn't available, but it's estimated in the single digit to low teens as a % of sales. They sold those rights to the profits for $150 million, so they could put that money back into research.
The CFF mentioned their concern over costs, but they couldn't press too hard on it. For one, it would eat up a portion of their return on investment. The more the drug costs, the more they get in royalties.
Vertex also uses the CFF as a marketing firm. The fastest way to have your new drug adopted is for new guidelines for treating the disease to be released and recommend your drug. The CFF helps put together these guidelines, with in-house experts and specialists who have received funding from them at other institutions.
In 2013, the company released positive data on Kalydeco (their older CF drug) and their up and coming CF drug. Their stock went from $52.87 to $85.60. Ian Smith, the chief financial officer of the company had bought his stock options at the $29-39 range, roughly 750,000 shares. He exercised his option that day and pocketed roughly $60 million. And that doesn't include the $4.8 million that the company paid him in 2013, or the $15 million it paid him in 2014. The top 5 executives combined took home $90.84 million in compensation in 2014.
Kalydeco, and now orkambi, cost about $300,000 a year. Prior to these drugs, CF patients paid about $30,000 a year for all their drugs.
Developing these drugs does take money. At the same time though, drug companies and their executives are pocketing huge sums of money. Their claims of "woe is me" sound pretty hollow when they're profiting so handsomely.
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Edit - Besides, ALL research is expensive. That is what the initial cost tends to go to. In addition to that, often the manufacturing process is also difficult and expensive.I don't know about this current drug, but I have some figures on the previous drug that Vertex developed for CF. They received $75 million from the Cystic Fibrosis Foundation, a charity, to fund their research. This doesn't include money from other charitable sources, or the federal government. In return the CFF got a cut of sales, the exact amount isn't available, but it's estimated in the single digit to low teens as a % of sales. They sold those rights to the profits for $150 million, so they could put that money back into research.
The CFF mentioned their concern over costs, but they couldn't press too hard on it. For one, it would eat up a portion of their return on investment. The more the drug costs, the more they get in royalties.
Vertex also uses the CFF as a marketing firm. The fastest way to have your new drug adopted is for new guidelines for treating the disease to be released and recommend your drug. The CFF helps put together these guidelines, with in-house experts and specialists who have received funding from them at other institutions.
In 2013, the company released positive data on Kalydeco (their older CF drug) and their up and coming CF drug. Their stock went from $52.87 to $85.60. Ian Smith, the chief financial officer of the company had bought his stock options at the $29-39 range, roughly 750,000 shares. He exercised his option that day and pocketed roughly $60 million. And that doesn't include the $4.8 million that the company paid him in 2013, or the $15 million it paid him in 2014. The top 5 executives combined took home $90.84 million in compensation in 2014.
Kalydeco, and now orkambi, cost about $300,000 a year. Prior to these drugs, CF patients paid about $30,000 a year for all their drugs.
Developing these drugs does take money. At the same time...
You need to keep in mind just how mingboggingly expensive clinical trials are, especially when you consider the rate at which drugs -don't- make it through the trials to market and you end up taking all of those expenses for a loss. Plus the years of preclinical development work with candidate compounds even before you can consider years of clinical trials. You're not just having to charge on drugs to recoup your direct investment on that drug, but also on all of the other potential drugs that never made it to market.
Then once you manage to get a minute fraction of drugs to market, assuming that they compete against other drugs out there for the same target, you only have the duration window of the patent to recoup costs and make a profit to drive future drug development.
I know it's easy to blame high drug costs on the boogieman of evil corporate big pharma yearning for profits over people *insert scare quotes*, but it's a naive view at best. I've been in preclinical cell therapy and drug development for a decade.
...Edit - Besides, ALL research is expensive. That is what the initial cost tends to go to. In addition to that, often the manufacturing process is also difficult and expensive.I don't know about this current drug, but I have some figures on the previous drug that Vertex developed for CF. They received $75 million from the Cystic Fibrosis Foundation, a charity, to fund their research. This doesn't include money from other charitable sources, or the federal government. In return the CFF got a cut of sales, the exact amount isn't available, but it's estimated in the single digit to low teens as a % of sales. They sold those rights to the profits for $150 million, so they could put that money back into research.
The CFF mentioned their concern over costs, but they couldn't press too hard on it. For one, it would eat up a portion of their return on investment. The more the drug costs, the more they get in royalties.
Vertex also uses the CFF as a marketing firm. The fastest way to have your new drug adopted is for new guidelines for treating the disease to be released and recommend your drug. The CFF helps put together these guidelines, with in-house experts and specialists who have received funding from them at other institutions.
In 2013, the company released positive data on Kalydeco (their older CF drug) and their up and coming CF drug. Their stock went from $52.87 to $85.60. Ian Smith, the chief financial officer of the company had bought his stock options at the $29-39 range, roughly 750,000 shares. He exercised his option that day and pocketed roughly $60 million. And that doesn't include the $4.8 million that the company paid him in 2013, or the $15 million it paid him in 2014. The top 5 executives combined took home $90.84 million in compensation in 2014.
Kalydeco, and now orkambi, cost about $300,000 a year. Prior to these drugs, CF patients paid about $30,000 a year for all their drugs.
Developing these drugs does take
I wouldn't say it's naive, it depends greatly on what companies we are talking about and the time period we are referring to. The 90s was a particularly awful time.
...Edit - Besides, ALL research is expensive. That is what the initial cost tends to go to. In addition to that, often the manufacturing process is also difficult and expensive.I don't know about this current drug, but I have some figures on the previous drug that Vertex developed for CF. They received $75 million from the Cystic Fibrosis Foundation, a charity, to fund their research. This doesn't include money from other charitable sources, or the federal government. In return the CFF got a cut of sales, the exact amount isn't available, but it's estimated in the single digit to low teens as a % of sales. They sold those rights to the profits for $150 million, so they could put that money back into research.
The CFF mentioned their concern over costs, but they couldn't press too hard on it. For one, it would eat up a portion of their return on investment. The more the drug costs, the more they get in royalties.
Vertex also uses the CFF as a marketing firm. The fastest way to have your new drug adopted is for new guidelines for treating the disease to be released and recommend your drug. The CFF helps put together these guidelines, with in-house experts and specialists who have received funding from them at other institutions.
In 2013, the company released positive data on Kalydeco (their older CF drug) and their up and coming CF drug. Their stock went from $52.87 to $85.60. Ian Smith, the chief financial officer of the company had bought his stock options at the $29-39 range, roughly 750,000 shares. He exercised his option that day and pocketed roughly $60 million. And that doesn't include the $4.8 million that the company paid him in 2013, or the $15 million it paid him in 2014. The top 5 executives combined took home $90.84 million in compensation in 2014.
Kalydeco, and now orkambi, cost about $300,000 a year. Prior to these drugs, CF patients paid about $30,000 a year for all their drugs.
Developing these drugs does take
I don't think you understand my point.
I'm not claiming that research or development is cheap. I'm saying that the consumer cost of this drug is out of line with those research and development costs. I know that's a subtle difference, but it's an important one.
I'm not saying the company can't make a profit at all. My point is that the profit margin should be smaller.
Drug patents last 20 years. Vertex has spent $5 billion on R&D since 1989. Kalydeco (their older CF drug) makes roughly $450 million in sales per year. Now, I highly doubt that the drug costs more than 10% of it's sale price in manufacturing, but lets make it more costly to produce than most drugs cost to the consumer and have it account for roughly 33%, or $100,000 to manufacture one year's supply for one person.
That's still a yearly profit of $200,000 per patient. There are about 2000 patients in the US for this drug (Kalydeco), so $200,000,000 in profit per year. A drug patent lasts 20 years, so that comes to $4,000,000,000 profit over the life time of the patent, so from 1 drug, they nearly recoup ALL of their R&D expenses from the entire lifetime of the company.
Of course, the drug approved last week costs slightly less ($275,000 to the patient), but it will also have 20 times as many patients. Let's say they only make $150,000 per patient, per year. Over the next 20 years they are looking at roughly $240 billion in profit.
This drug doesn't cure CF. It prevents the disease from causing damage and so must be taken for the patients entire life time.
Of course my numbers, other than the number of patients and how much the company is charging, are taken out of thing air. But manufacturing costs for drugs are rarely the prohibitive factor. If I had to hazard a more realistic manufacturing cost, it would probably be in the 1-3% range of what they're charging, and even that could be generously high.
I'm okay with drug companies making a profit. I don't think they should bend patients over a barrel to do so though and lets be honest, that's what these prices are doing.
We know companies continue to sell their products in countries with price controls, because they still make money. Yes, they make less money, but they still make money.
And don't forget, their R&D budgets include donations from charities and funding from the government. Vertex has received $150 million from the CFF for these 2 drugs. Now that's a lot less than their $5 billion total, but it's not an insignificant amount. That's 3% of their funding from just one source.
CFF recently sold off the rest of their shares of the royalties of Kalydeco for $3.3 billion, which accounted for approximately 10% of the profits, give or take a few % points. Vertex is still sitting on the other ~90% which has an market estimated value of $30 billion, much higher than what my example figures put it at.
Does Vertex NEED $30 billion in profits from one drug, when their ENTIRE research and development spending has been $5 billion since 1989? Could we agree that they'd probably be okay at say $10 or $15 billion in profit?
Also, I don't like the fact that the charity is involved in the profit engine of the company. Maybe they aren't abusing it, maybe all their actions have been pure and good. I just think as a system though, it's too opportune for someone to abuse it.
I'd rather see charities devoting their funding leverage to negotiating lower prices for the people suffering from the disease instead of having a vested interest in a higher price.
Hey, I've got another great idea. If you want to discuss the potential evils of pharmaceutical companies, start another thread.
Is your goal purely to have a thread with 50 consecutive posts repeating "that's great"? That's not really a discussion.
The cost issue is real and deserves to be mentioned.
In fact, it's a real issue that is affecting people who have cystic fibrosis. Arkansas is a cash strapped state, so in an attempt to save money they tried to rewrite their rules for eligibility of treatments. This effectively denied the earlier version (FDA approved) to several people.
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Drug patents last 20 years. Vertex has spent $5 billion on R&D since 1989. Kalydeco (their older CF drug) makes roughly $450 million in sales per year. Now, I highly doubt that the drug costs more than 10% of it's sale price in manufacturing, but lets make it more costly to produce than most drugs cost to the consumer and have it account for roughly 33%, or $100,000 to manufacture one year's supply for one person.
That's still a yearly profit of $200,000 per patient. There are about 2000 patients in the US for this drug (Kalydeco), so $200,000,000 in profit per year. A drug patent lasts 20 years, so that comes to $4,000,000,000 profit over the life time of the patent, so from 1 drug, they nearly recoup ALL of their R&D expenses from the entire lifetime of the company.
Yeah, that sounds about right.
About 8% of drugs make it the approval stage. A substantial fraction still fail even at the phase III clinical trial stage, so it's not like all the expenses and risk are front-loaded. This means that roughly one drug in 12 will actually make the company any money at all, and each successful drug needs to make back at least 12x its development costs in order just to break even.
Vertex has six drugs in the pipeline right now. Using your numbers, doing some rough math and assuming that's the steady state, Vertex has about 200 drug-years of R&D, for a total development cost of $25M per drug-year. The average time-to-market is 11-14 years, so we're looking at roughly $300M development costs for the drug.
12 times $300M is $3600M or about $4B. That's roughly what Vertex needs to make on this drug -- on any drug -- to make it a financial success.
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Hey, I've got another great idea. If you want to discuss the potential evils of pharmaceutical companies, start another thread.Is your goal purely to have a thread with 50 consecutive posts repeating "that's great"? That's not really a discussion.
Well, there's your first mistake, cupcake - thinking I actually want a discussion instead of just popping up a thread that celebrates something good for some friends of mine. "That's great!" is an awesome response. "That's f*+*ing awesome" is an even better response.
Watch, I'll show you how easy it is.....
Except that the number is going to be much higher, since my assumed manufacturing cost is $100,000 per year per patient. If we assume a much more reasonable cost of $5,000 to $10,000 (which I bet is still very high) per year, that adds another $2 billion to the profit of the drug.
It also ignores the fact that a second drug was developed off this one. Those numbers were for Kalydeco. The one approved this week is Orkambi, which has a similar cost, but 20-30 times as many patients, though the price is slightly lower.
Orkambi isn't really a standalone drug. It's kalydeco combined with lumacaftor. The turn around time from kalydeco to orkambi was less than 3 years.
Recently a 12% share in both drugs was sold for $3.3 billion. That means that that company (Royalty Pharma, a company that exists solely to buy the intellectual property rights of drugs) thinks the two drugs will make at least $28 billion in profit.
Lastly, I can't find a breakdown of Vertex's R&D budget. I know they've received $100 million from the CFF (with $50 million more pledged), but I have no idea how much they've gotten from other charities or federal or state governments. How much work they've used from universities. Right now we know that CFF accounts for 3% of their R&D budget so far.
That was easy.
Yikes Irontruth. What do you do for a living?
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That is awesome. I have a friend who will be very happy with the news.
Aberzombie- I don't think anybody does not think that having a new effective treatment for cystic fibrosis is not great. It is possible it may not work out as well as hoped.
You really are not in control of what other people can say you know.
On drug companies- They often buy the rights to research originally done by academics, pay for the approval process and then pocket most of the profits. And research funded by drug companies into their own products is the opposite of independent of course.
Powerful industries are in a position to influence the political process that affects their own profits and do so in spades. For instance, US politicians under the Bush presidency were very insistent that Australia should pay more for the drugs of US companies.
And drug companies often behave shamefully in the third world. They are anything but a pure force for good.
And to keep Aberzombie happy- it is awesome that there is a treatment for CF.
Except that the number is going to be much higher, since my assumed manufacturing cost is $100,000 per year per patient. If we assume a much more reasonable cost of $5,000 to $10,000 (which I bet is still very high) per year, that adds another $2 billion to the profit of the drug.
It also ignores the fact that a second drug was developed off this one. Those numbers were for Kalydeco. The one approved this week is Orkambi, which has a similar cost, but 20-30 times as many patients, though the price is slightly lower.
Orkambi isn't really a standalone drug. It's kalydeco combined with lumacaftor. The turn around time from kalydeco to orkambi was less than 3 years.
Recently a 12% share in both drugs was sold for $3.3 billion. That means that that company (Royalty Pharma, a company that exists solely to buy the intellectual property rights of drugs) thinks the two drugs will make at least $28 billion in profit.
Lastly, I can't find a breakdown of Vertex's R&D budget. I know they've received $100 million from the CFF (with $50 million more pledged), but I have no idea how much they've gotten from other charities or federal or state governments. How much work they've used from universities. Right now we know that CFF accounts for 3% of their R&D budget so far.
What Irontruth is stating is something I am particularly concerned about with these new drugs as well.
It's great news, but unless you are a millionare...how many can actually afford these drugs?
A LOT of lifetime needed drugs are starting to not be covered by many of the Health Insurers due to the ACA...supposedly there's some sort of loophole that makes it so that they can not cover them.
I've seen it happen with diabetics (the insurers will cover the worst drug instead of what is new or the better items on the market in both instances which would indicate they wouldn't cover this new drug for CF either) and with epilepsy.
I think the big thing to see, is how much is this going to cost someone in Canada, or Germany, or Russia?
For some reason, the pharmeceauticals make ALL their MAJOR profits in the US and so everyone else get's it cheaper.
If it costs a quarter of a million to an American, and so insurers won't cover it, so a majority of those who suffer CF can't even get it anyways (meaning, great news, but basically nothing that affects CF in the US as the drug is basically unobtainable at that cost)...but only costs ten to twenty thousand (or less) everywhere else...
Doesn't that strike a bell to anyone that something is drastically wrong with that price?
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The costs will come down.
As for the ACA tangent, I've had just the opposite experience. One drug I'm on was covered by the drug company for free, and then when I changed health providers, once the doctor provided preauthorization they were more than willing to cover the costs.
My mother had similar results with a $5,000 a month pill that is supposed to be the newest and coolest thing.
There are programs for some people, who qualify, to get on these meds. There are tons of hoops, but people play the lottery with worse odds.
Yikes Irontruth. What do you do for a living?
Nothing related to this.
Hey, I've got another great idea. If you want to discuss the potential evils of pharmaceutical companies, start another thread.Is your goal purely to have a thread with 50 consecutive posts repeating "that's great"? That's not really a discussion.Well, there's your first mistake, cupcake - thinking I actually want a discussion instead of just popping up a thread that celebrates something good for some friends of mine. "That's great!" is an awesome response. "That's f*##ing awesome" is an even better response.
Watch, I'll show you how easy it is.....
I think it's awesome that the drug exists.
I'm going to save the "f!%$ing awesome" for when your friends are actually given access to the drug.
This is extremely similar to a drug released in 2012. There were three people in Arkansas who the earlier drug would have helped, but were denied by Medicaid, mostly because the state didn't want to pay for extremely high treatments.
I think it would be the opposite of "f!+%ing awesome" if your friends were denied access to this drug for a similar reason.
The costs will come down.
As for the ACA tangent, I've had just the opposite experience. One drug I'm on was covered by the drug company for free, and then when I changed health providers, once the doctor provided preauthorization they were more than willing to cover the costs.
My mother had similar results with a $5,000 a month pill that is supposed to be the newest and coolest thing.
There are programs for some people, who qualify, to get on these meds. There are tons of hoops, but people play the lottery with worse odds.
some good news.
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You really are not in control of what other people can say you know.
True. However, that does not stop me from hoping (in vain apparently), that they would have the courtesy they so vigorously demand from others to keep politics and other graar out of it.
Then again, these are the Paizo messageboards. I shouldn't have expected anything less.
Isn't "This treatment's expensive, yo" relevant to the topic?
"Here is a thing, agree with me" is not a topic of discussion.
Customer Service Manager
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Isn't "This treatment's expensive, yo" relevant to the topic?
"Here is a thing, agree with me" is not a topic of discussion.
Its totally relevant. Feel free to discuss that. The issue lies in turning it from "This [specific drug] is/will be expensive," to a general debate on the morality of profit making drug companies.
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When I was playing more than I am now and able to go to conventions, I used to play with a friend of a friend. Even with CF, his attitude never wavered. He was funny when the situation called for it, with a wicked sense of humor, and a most serious opponent across the table when I GM'ed against him. Sadly, he lost his battle several years ago, shy of his 30th birthday. He is missed.
I hope Vertex is the first step in the cure for CF.
I don't like to jump on any bandwagons, either pro or con, but I do own some stock in some pharmaceutical companies.
What many people don't understand is that these companies can spend 100 million dollars each developing 10 drugs a year, and usually only 1 of them pans out.
Then they have to pay to have it tested by the FDA to be approved, which may still reject it. That is a billion dollars that went for nothing.
Please do not hate them for trying to make money back to cover the costs of developing these important drugs that help save lives.
Is this thing just better or somehow revolutionary?
Is this thing just better or somehow revolutionary?
My understanding is revolutionary.