Life changing cystic fibrosis drug. What role should government play?

Ubbquittious

If the government were clever they'd give some postgrad chemists a nixer synthesising this stuff in an underground bunker some place. Give them 50k a year each and threaten them with a harsh punishment if they spill the beans to anyone. Tell the CF patients a pack of lies about what pills they're getting and let some other country pay the megacorp that invented this drug.

Most of the outrageous eye-watering price of this stuff is to provide a return on investment for the megacorp and the stuff is probably not expensive to make at all

BrokenArrows

steddyeddy wrote: »

Lumacaftor/ivacaftor (brand name Orkambi) is relatively new combination drug for the treatment of cystic fibrosis. Cystic fibrosis is a disease caused by a range of mutations and deletions of the gene for a chloride transporter (CFTR) which transports chloride across membranes. If you are unable to move ions like chloride across membranes you'll also have problems regulating fluid. We have one of the highest incidences of Cf in the world with 1 in 20 people being carriers.

Ivacaftor works as a potentiator of chloride channels that are already in place and lumacaftor works by refolding protein chloride channels resulting in a higher amount of expressed chloride channels. This drug will significantly enhance the lives of CF sufferers, but we can't afford it as It costs around 160 K per patient.

I'm not having a go at the pharmaceutical company necessarily as it costs billions to get a drug to market. From the biochemical analysis that enables us to discover a drug target, to the chemistry that goes into designing a drug to fit a target and the human trials, no one can say this isn't a costly process.

Treating illnesses currently doesn't work from a strictly capitalistic view point as some rare diseases with orphan status are partly government funded as the companies wouldn't make their money back. Should governments or charities do more or should the pharm companies do more?

https://www.cfireland.ie/about-cf states that 1200 people have CF in Ireland.

at 162k per year per paitent thats 192 million per year which to be honest is insane.

Regardless of the cost of production there is no way they can justify that price. If it was a cure then maybe but as something which just makes the patients lifes better and needs a lifetime of treatment.

The average lifespan of a CF patient is 37 years. Thats 6 million over a life time to these drug companies for a single person.

ohnonotgmail

Ubbquittious wrote: »

If the government were clever they'd give some postgrad chemists a nixer synthesising this stuff in an underground bunker some place. Give them 50k a year each and threaten them with a harsh punishment if they spill the beans to anyone. Tell the CF patients a pack of lies about what pills they're getting and let some other country pay the megacorp that invented this drug.

Most of the outrageous eye-watering price of this stuff is to provide a return on investment for the megacorp and the stuff is probably not expensive to make at all

do you think they should just write off all the research costs and sell it for the manufacturing cost?

BrokenArrows

ohnonotgmail wrote: »

do you think they should just write off all the research costs and sell it for the manufacturing cost?

They should be recovering the costs over a longer period of time resulting in cheaper medicine.

Honestly organisations like the EU or the economic division of Nato should fund a single huge research centre which develops these drugs and sells to all participating nations at cost price.

A world wide collaboration on medical technology would be amazing. Never going to happen but it would be amazing.

Ubbquittious

ohnonotgmail wrote: »

do you think they should just write off all the research costs and sell it for the manufacturing cost?

Let some other country pay them. There are plenty of countries absolutely milking it off national debt payments from us and they're rolling in so much money they can well afford to pay 80,000 for a pack of pills.

IF we weren't 200bn in debt I'd say take the easy way and just buy them but when the ould kitty is empty you have to improvise

ohnonotgmail

BrokenArrows wrote: »

They should be recovering the costs over a longer period of time resulting in cheaper medicine.

Honestly organisations like the EU or the economic division of Nato should fund a single huge research centre which develops these drugs and sells to all participating nations at cost price.

A world wide collaboration on medical technology would be amazing. Never going to happen but it would be amazing.

They have a limited period of time to recover those costs. once their patent expires anybody can make the drug.

BrokenArrows

ohnonotgmail wrote: »

They have a limited period of time to recover those costs. once their patent expires anybody can make the drug.

Ya but the patent length is 20 years. Assuming 8 years from invention through to public sale that gives them 12 years to recover costs.

12 years at these prices put total earnings at 13 billion (assuming only 1% of CF patients take the product) when the cost of development for the average new drug is 2.5 billion.

ectoraige

BrokenArrows wrote: »

https://www.cfireland.ie/about-cf states that 1200 people have CF in Ireland.

at 162k per year per paitent thats 192 million per year which to be honest is insane.

Regardless of the cost of production there is no way they can justify that price. If it was a cure then maybe but as something which just makes the patients lifes better and needs a lifetime of treatment.

The average lifespan of a CF patient is 37 years. Thats 6 million over a life time to these drug companies for a single person.

Your annual cost estimate can be halved. Orkambi is only useful for a specific form of Cystic Fibrosis, and isn't given to children under twelve, which precludes about 50% of Irish patients. A small number of children won't survive to be able to receive Orkambi. It is during adolescence that sees the biggest spike in CF deaths, so it is hoped that we'll see fewer teenagers dying from the disease.

ohnonotgmail

Ubbquittious wrote: »

Let some other country pay them. There are plenty of countries absolutely milking it off national debt payments from us and they're rolling in so much money they can well afford to pay 80,000 for a pack of pills.

IF we weren't 200bn in debt I'd say take the easy way and just buy them but when the ould kitty is empty you have to improvise

I'm sure other countries would be more than willing to pay them on our behalf. Especially given the high rate of CF in this country.

Ubbquittious

ohnonotgmail wrote: »

I'm sure other countries would be more than willing to pay them on our behalf. Especially given the high rate of CF in this country.

We are a small broke pesky Island nation out in the atlantic somewhere and a small market.
Have some minister come out and say the stuff is too expensive, make it on the QT and be done with it.

Tis not up to us to Trump up the dosh, do the moral capitalist right thing and ensure everyone in VRTX gets their Christmas bonus.

The_Valeyard

It's easy to look at costs and say 'to expensive,' but just take a stroll through the children's hospital with babies hooked up to ventilators and wires for machines, if there was a drug that only had a 10% chance of success. You'd want to be made of steel to say no.

Your Face

Id like to see the bill for this incomparison to the daily wasting of government funding.

Kurtosis

donkeyoaty0099 wrote: »

Where does the money for that come from? And more than that, who does the manufacturing? All well and good having an excellent drug proven at small scale, but if you need 1000 kg plus a year of it who makes that for you? The capital cost of a new manufacturing plant is astronomical, and then you need staff to run it.

The money could come from existing expenditure on pharmaceuticals. Traditionally drug discovery, development, and manufacturing are all funded through the price paid for medicines at the point of delivery to the health system through the patent system. It's perfectly reasonable to look at alternatives to patents as a means of funding drug development research and innovation. There's some information on this concept of "delinkage" here. This isn't something Ireland can really take a solo run on but it is an option that could result in more affordable drug research.

As happens at the moment, there is plenty of capacity in the generic medicines manufacturing industry to manufacture off-patent drugs. A new system may just mean new drugs are effectively off-patent from the start and could be licensed to one or all manufacturers to produce.

ohnonotgmail

Kurtosis wrote: »

The money could come from existing expenditure on pharmaceuticals. Traditionally drug discovery, development, and manufacturing are all funded through the price paid for medicines at the point of delivery to the health system through the patent system. It's perfectly reasonable to look at alternatives to patents as a means of funding drug development research and innovation. There's some information on this concept of "delinkage" here. This isn't something Ireland can really take a solo run on but it is an option that could result in more affordable drug research.

As happens at the moment, there is plenty of capacity in the generic medicines manufacturing industry to manufacture off-patent drugs. A new system may just mean new drugs are effectively off-patent from the start and could be licensed to one or all manufacturers to produce.

if you are charging a licencing fee to recover the costs of R&D those fees are going to be very expensive for a drug with a small target market like CF. And how do you recover the cost of drug research that doesnt lead to a usable medicine?

Anita Blow

The_Valeyard wrote: »

It's easy to look at costs and say 'to expensive,' but just take a stroll through the children's hospital with babies hooked up to ventilators and wires for machines, if there was a drug that only had a 10% chance of success. You'd want to be made of steel to say no.

That's fair enough as someone not in the DoH to say, but the reality is that these decisions have to be made. The money doesn't grow on trees and the money dedicated to helping the relatively small number of such children, of which just 10% would benefit, would be taken away from other drugs which could benefit far more people with a higher success rate. It's the reason we have the Quality-of-life-adjusted Years system of judging whether to fund a drug, so we can view the merits of a drug dispassionately without being swayed by emotion.

The_Valeyard

Anita Blow wrote: »

That's fair enough as someone not in the DoH to say, but the reality is that these decisions have to be made. The money doesn't grow on trees and the money dedicated to helping the relatively small number of such children, of which just 10% would benefit, would be taken away from other drugs which could benefit far more people with a higher success rate. It's the reason we have the Quality-of-life-adjusted Years system of judging whether to fund a drug, so we can view the merits of a drug dispassionately without being swayed by emotion.

You make perfect sense, but sometime I think Charlie Chaplin was right when he said "We think too much and we feel too little

jackofalltrades

Boaty wrote: »

80 million out of a 14 billion euro health budget doesn't sound like much when you think about it.

And how many people could you take off the waiting lists for medical procedures by spending €80 million a year over the next 5 years?
I would consider that a significant sum.

The_Valeyard wrote: »

It's easy to look at costs and say 'to expensive,' but just take a stroll through the children's hospital with babies hooked up to ventilators and wires for machines, if there was a drug that only had a 10% chance of success. You'd want to be made of steel to say no.

But our health budget is fixed.
Saying yes to one person means saying no to another.
And when it comes to drugs that aren't good value it means saying no to a disproportionate amount of people.
How many patients will be left in a worse off position due to this deal?
That and deals like this will probably be used as a justification for the next large price medication.

The_Valeyard wrote: »

You make perfect sense, but sometime I think Charlie Chaplin was right when he said "We think too much and we feel too little

And how many health budgets did he manage?
Feelings should be removed from decisions like this as far as humanely possible.

The_Valeyard

jackofalltrades wrote: »

And how many people could you take off the waiting lists for medical procedures by spending €80 million a year over the next 5 years?
I would consider that a significant sum.

But our health budget is fixed.
Saying yes to one person means saying no to another.
And when it comes to drugs that aren't good value it means saying no to a disproportionate amount of people.
How many patients will be left in a worse off position due to this deal?
That and deals like this will probably be used as a justification for the next large price medication.

And how many health budgets did he manage?
Feelings should be removed from decisions like this as far as humanely possible.

Remove humanity from the decisions and people become monetary units to the meat grinder.

Anita Blow

The_Valeyard wrote: »

Remove humanity from the decisions and people become monetary units to the meat grinder.

I understand what you're trying to say, but it's essentially meaningless. Our budget is fixed and there's nothing we can do about that so decisions have to be made about opportunity costs.
Do you spend 80 million on a single drug available to just ~800 people, of which just a percentage will benefit? Or do you spend the 80 million on hiring 10 new consultants, a hundred new doctors/nurses/physios/OTs that can wipe out paediatric waiting lists for a number of diseases (some of which are life-limiting) for tens of thousands? Common sense suggests that you go with the option which has the greatest potential to do good for the most people.

And there's another problem with the emotive argument. It becomes a sort of Hunger Games for different disease groups. Patient advocacy groups for the more common diseases might have the budget and pool of patients to campaign heavily for investment in their disease, but why are they any more deserving than a person who has a disease that only 5 other people in the country has? Our current system judges the merits of a drug based on how effective it is and how economic it is, regardless of how "popular" a condition is.

Kurtosis

ohnonotgmail wrote: »

if you are charging a licencing fee to recover the costs of R&D those fees are going to be very expensive for a drug with a small target market like CF. And how do you recover the cost of drug research that doesnt lead to a usable medicine?

Those considerations already play into the current system, orphan drug designation for rare diseases gives pharmaceutical companies extended patents etc. to help recoup costs. Likewise the cost of failed research is covered through what we pay for successful research that results in useful medicines.

The suggestion is that rather than handing pharmaceutical companies a big of pot of money to buy medicines from them which funds the costs of all of their activities, why not try to separate the reward/payment for R&D. Right now, the companies themselves get to decide how much of the big pot to invest in R&D and what areas to pursue R&D in. They of course are going to target this at areas where they think they can most yield a profit. Some of the strategies involved in delinkage would mean governments/policy makers could decide on what priorities areas for research should be. How rare a disease is wouldn't matter to companies pursuing the R&D, they would receive funding whether it's a treatment for 100 people or 100 million, the cost of R&D doesn't really vary by disease prevalence.

Widdershins

Why can't we have some input into how our tax money's spent? Some of us might even opt to pay more if we knew it was going to a good cause!

idunno78

BrokenArrows wrote: »

steddyeddy wrote: »

Lumacaftor/ivacaftor (brand name Orkambi) is relatively new combination drug for the treatment of cystic fibrosis. Cystic fibrosis is a disease caused by a range of mutations and deletions of the gene for a chloride transporter (CFTR) which transports chloride across membranes. If you are unable to move ions like chloride across membranes you'll also have problems regulating fluid. We have one of the highest incidences of Cf in the world with 1 in 20 people being carriers.

Ivacaftor works as a potentiator of chloride channels that are already in place and lumacaftor works by refolding protein chloride channels resulting in a higher amount of expressed chloride channels. This drug will significantly enhance the lives of CF sufferers, but we can't afford it as It costs around 160 K per patient.

I'm not having a go at the pharmaceutical company necessarily as it costs billions to get a drug to market. From the biochemical analysis that enables us to discover a drug target, to the chemistry that goes into designing a drug to fit a target and the human trials, no one can say this isn't a costly process.

Treating illnesses currently doesn't work from a strictly capitalistic view point as some rare diseases with orphan status are partly government funded as the companies wouldn't make their money back. Should governments or charities do more or should the pharm companies do more?

https://www.cfireland.ie/about-cf states that 1200 people have CF in Ireland.

at 162k per year per paitent thats 192 million per year which to be honest is insane.

Regardless of the cost of production there is no way they can justify that price. If it was a cure then maybe but as something which just makes the patients lifes better and needs a lifetime of treatment.

The average lifespan of a CF patient is 37 years. Thats 6 million over a life time to these drug companies for a single person.

It's. It 1200 patients I think it's mOre like 550! And not all will take it I'd imagine for various reasons! Yea it's a lot of money it it's also people's lives! I bet if you had someone with cf in your family (maybe you do??) you would have a different opinion?

doc22

idunno78 wrote: »

It's. It 1200 patients I think it's mOre like 550! And not all will take it I'd imagine for various reasons! Yea it's a lot of money it it's also people's lives! I bet if you had someone with cf in your family (maybe you do??) you would have a different opinion?

would you remortgage your house to give a family member a single years supply?

idunno78

Doc22, I'm sure my parents would if it meant I'd live longer

Capt'n Midnight

steddyeddy wrote: »

I'm not having a go at the pharmaceutical company necessarily as it costs billions to get a drug to market. From the biochemical analysis that enables us to discover a drug target, to the chemistry that goes into designing a drug to fit a target and the human trials, no one can say this isn't a costly process.

Treating illnesses currently doesn't work from a strictly capitalistic view point as some rare diseases with orphan status are partly government funded as the companies wouldn't make their money back. Should governments or charities do more or should the pharm companies do more?

There is no brand loyalty. Once the patents expire health services will switch to generics unless the costs plummet. Health services and drug companies know this.

It's blackmail pure and simple. Big Pharma's MO is to make money. You could nearly think of them as a marketing company with a sideline in producing a product to market.

The R&D costs are way less than the marketing and shareholder dividends. In many cases the education of the researchers was state subsidised. In most cases it's the public purse paying for these drugs. The take in a lot of public money.


If we cut out the marketing and profit taking we could shrink costs. One option would be to pay companies to develop drugs but they don't keep the patent and they have to share all the research too.

The REAL cost of giving Big Pharma a licence to print money is
http://www.insightcrime.org/news-briefs/counterfeit-drugs-kill-1-million-annually-interpol

World police agency Interpol says more than one million people die each year from counterfeit drugs, highlighting one of the fastest-growing and most lucrative income sources for global organized crime networks.

According to the International Criminal Policing Association (Interpol), up to 30 percent of drugs sold worldwide are counterfeit

gordongekko

Hadn't read the whole thread but reduce the dole from anyone over 5 years on the dole by €10 a week and use this money to pay for the drug

idunno78

Or lower all the TDs etc wages that would be a lot of money!! Wouldn't just pay for this but lots of other things!

It won't suit a lot of people. They will either be to sick to try it, will make them sick but I think we should be given the option to try something that might make a world of difference! Hopefully other drugs will come of be back of this that are cheaper (and better hopefully!!). That will make everyone happy!

Ubbquittious

Apparently there are already counterfeits being made.. now of course the megacorp will try to convince you it's laced with cyanide but that's not necessarily the case.

Sure maybe we can all pitch in a tenner to buy a year's supply of the stuff from one of those counterfeit suppliers that you see in your spam email box, pay to have it tested and distribute it underground railroad style.