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

steddyeddy

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?

s4uv3

I'd need to see the exact figures from the pharma company before I could make a judgement.

Fair enough it costs billions in R&D for these drugs, but what would be the profit margins? Yearly figures? All that has to be taken into account.

We (our government), spend a right hape of money on some proper eejiting and messing every year, so we should be able to afford to pay for these lifesaving drugs, but not if the company involved is making a killing on profits.

SEPT 23 1989

160 K per patient per year?

seamus

Healthcare always comes down to a question of finances. There is no way to say "money is no object" without also opening the doors for all sorts of spending.

Ultimately it's a question of effectiveness. If a drug is highly effective for a large proportion of those diagnosed, then we should be prepared to pay out the money for it. If it's only been proven to be effective in 1% of cases, or only offers a very limited improvement for the majority of cases, then it may not be worth funding.

There should be no question really of "orphan status" illnesses. If someone is the only sufferer in Ireland of a rare illness, and a drug exists with 99% effectiveness that costs €500k a pop, then we should buy it.
If 300 people have an illness and a drug with a 0.5% effectiveness costs €10k a pop, then we'd be stupid to pay for it.

ohnonotgmail

s4uv3 wrote: »

I'd need to see the exact figures from the pharma company before I could make a judgement.

Fair enough it costs billions in R&D for these drugs, but what would be the profit margins? Yearly figures? All that has to be taken into account.

We (our government), spend a right hape of money on some proper eejiting and messing every year, so we should be able to afford to pay for these lifesaving drugs, but not if the company involved is making a killing on profits.

You also have to take into account the money spent on R&D for drugs that dont work. The pharma companies have to recoup those costs from the profits of successful drugs.

s4uv3

SEPT 23 1989 wrote: »

160 K per patient per year?

Yup

steddyeddy

SEPT 23 1989 wrote: »

160 K per patient per year?

Yes, pretty but might be about right for a combination drug.

Gatling

How much does the government spend on methadone ,
But won't pay for a life changing drug for CF

ScumLord

ohnonotgmail wrote: »

You also have to take into account the money spent on R&D for drugs that dont work. The pharma companies have to recoup those costs from the profits of successful drugs.

The pharma companies also have to take into account all the other drugs we buy off them.

Really the government should be going in with a good overview of what they think the drugs are costing and working out some inclusive price. The pharma company is likely making good profit on other drugs, maybe offer some exclusivity to the market if they give big reductions on rare meds like these.

Ireland is a consumer group and we should be acting like one.

steddyeddy

Gatling wrote: »

How much does the government spend on methadone ,
But won't pay for a life changing drug for CF

Considering methadone was synthesized during WW2 and heavily pre and proscribed it would be significantly less.

ED E

People don't like to hear "price on a life" but healthcare is a beancounting exercise. Simple as.

The HSE have a finite budget. This isn't disputed. So spending more on x means less for y and z.

What does an ambulance driver under the NAS cost per year? Lets say its 40k inc pension etc. So you want to take 4 drivers (two ambulances) off the road for EACH patient? That doesn't help the national death stats.

VRTX won't give us 30k pppa. They can't, because then france and Germany and the UK will want the same. We may have the highest occurrence but the absolute number is still way lower, we're small fry.

Its tough for current patients but this is something that'll be offered way down the line to future patients. Politics be gone, there's very clear maths here.

mahoganygas

ohnonotgmail wrote:

You also have to take into account the money spent on R&D for drugs that dont work. The pharma companies have to recoup those costs from the profits of successful drugs.

We also must ensure we don't scare off investors. The best pharma companies in the world average a return of about 11% per year in the long term.

If we cap profits on pharma companies at say 7% then investors will move their money to better paying assets, and overnight the pharma industry will shrink. That means upcoming cancer drugs, trials, R&D gets shelved and sick people are worse off.

Interfering in the pharma market should not be a knee jerk reaction.

*I say this as somebody who demonstrated at the Dail for Orkambi.

It's 30k / year for a drug recently released that would help my girlfriend a lot. The only drug released for this disease in 60 years. It's just a fact of life that's not easy to swallow but it's that way for the same reason that governments sell rights to search for oil etc. The public would never accept the loss but always want the results.

steddyeddy

ED E wrote: »

People don't like to hear "price on a life" but healthcare is a beancounting exercise. Simple as.

The HSE have a finite budget. This isn't disputed. So spending more on x means less for y and z.

What does an ambulance driver under the NAS cost per year? Lets say its 40k inc pension etc. So you want to take 4 drivers (two ambulances) off the road for EACH patient? That doesn't help the national death stats.

VRTX won't give us 30k pppa. They can't, because then france and Germany and the UK will want the same. We may have the highest occurrence but the absolute number is still way lower, we're small fry.

Its tough for current patients but this is something that'll be offered way down the line to future patients. Politics be gone, there's very clear maths here.

I think people who look at it as a bean counting exercise are often clueless to how health care is funded. The disease that's funded the most isn't always the one that gives the most return. Look at the money pumped into depression and mental illness research and tell me we're getting value for money.

Varik

ScumLord wrote: »

The pharma companies also have to take into account all the other drugs we buy off them.

Really the government should be going in with a good overview of what they think the drugs are costing and working out some inclusive price. The pharma company is likely making good profit on other drugs, maybe offer some exclusivity to the market if they give big reductions on rare meds like these.

Ireland is a consumer group and we should be acting like one.

Going off memory from College, but only a 1/3 pay for their own research and of that 1/3 only a tiny amount will be "blockbusters" who can pay for everything else.

You also have to take into account the limited time available to them, Pharma keeps thing a old fashioned secret as long as it can because as soon as they patent it the timer starts and they need to get through testing and clinical trials. There might not be much of that ~20 years patent when they finally make it to market.

ScumLord wrote: »

The pharma company is likely making good profit on other drugs, maybe offer some exclusivity to the market if they give big reductions on rare meds like these.

Most of those making the good profit are the patent ones which already have an exclusivity (a patent is a state sanctioned monopoly). Extending patents is done, and has seen a lot of drug in the US extended far beyond their normal expiration date.

steddyeddy

ScumLord wrote: »

The pharma companies also have to take into account all the other drugs we buy off them.

Really the government should be going in with a good overview of what they think the drugs are costing and working out some inclusive price. The pharma company is likely making good profit on other drugs, maybe offer some exclusivity to the market if they give big reductions on rare meds like these.

Ireland is a consumer group and we should be acting like one.

Cystic fibrosis isn't rare really. Ireland has the highest prevalence of CF so maybe a deal can be made on that basis.

Kurtosis

The price of Orkambi does not reflect the benefits it provides on average to CF patients who take it. Most other drugs, procedures, and services in other disease areas provide more patient benefits at the same cost. This is the big issue, the health gains if Orkambi was funded at the current price would be at the expense of other patients elsewhere in the health system.

Vertex have to either reduce the price to a level that matches the overall benefits it provides or agree to some sort of risk-sharing or outcomes-based pricing system so that they only charge for patients for whom the treatment is effective.

steddyeddy

penguin88 wrote: »

The price of Orkambi does not reflect the benefits it provides on average to CF patients who take it. Most other drugs, procedures, and services in other disease areas provide more patient benefits at the same cost. This is the big issue, the health gains if Orkambi was funded at the current price would be at the expense of other patients elsewhere in the health system.

Vertex have to either reduce the price to a level that matches the overall benefits it provides or agree to some sort of risk-sharing or outcomes-based pricing system so that they only charge for patients for whom the treatment is effective.

Results from the 2 clinical trials of Orkambi show that the number of exacerbations requiring hospital treatment was reduced by 39%. This has implications outside of the main symtoms of CF, for instance reduced cost to the taxpayer from less hospitalisations and reduced risk of hospital infections.

While I agree the price doesn't currently justify it, I'm not so sure you can more benefits with several drugs.

steddyeddy

s4uv3 wrote: »

I'd need to see the exact figures from the pharma company before I could make a judgement.

Fair enough it costs billions in R&D for these drugs, but what would be the profit margins? Yearly figures? All that has to be taken into account.

We (our government), spend a right hape of money on some proper eejiting and messing every year, so we should be able to afford to pay for these lifesaving drugs, but not if the company involved is making a killing on profits.

Considering the HSE seems to be one long academic study in waste they should be very careful about what they say they can't afford.

Kurtosis

steddyeddy wrote: »

Results from the 2 clinical trials of Orkambi show that the number of exacerbations requiring hospital treatment was reduced by 39%. This has implications outside of the main symtoms of CF, for instance reduced cost to the taxpayer from less hospitalisations and reduced risk of hospital infections.

While I agree the price doesn't currently justify it, I'm not so sure you can more benefits with several drugs.

Reduced healthcare expenditure for managing exacerbations was taken into account in the economic evaluation that was carried out: http://www.ncpe.ie/wp-content/uploads/2015/12/Website-summary-orkambi.pdf. This indicated for every quality-adjusted life year (which is a measure of both quality and quantity of life) gained as a result of Orkambi, the cost would be ~€370K. Generally, the limit for new drugs to be deemed cost effective is €45K per quality-adjusted life year. Orkambi isn't even in that ballpark, hence why I said that at the current price, the same funding would do a lot more good elsewhere in the health system.

steddyeddy

penguin88 wrote: »

Reduced healthcare expenditure for managing exacerbations was taken into account in the economic evaluation that was carried out: http://www.ncpe.ie/wp-content/uploads/2015/12/Website-summary-orkambi.pdf. This indicated for every quality-adjusted life year (which is a measure of both quality and quantity of life) gained as a result of Orkambi, the cost would be ~€370K. Generally, the limit for new drugs to be deemed cost effective is €45K per quality-adjusted life year. Orkambi isn't even in that ballpark, hence why I said that at the current price, the same funding would do a lot more good elsewhere in the health system.

I agree with the funding but the pharmacodynamics should offer more of a health benefit than multi-drug therapy. Don't forget that this is multi-drug therapy.

Gatling

steddyeddy wrote: »

Considering methadone was synthesized during WW2 and heavily pre and proscribed it would be significantly less.

It's currently costing €20 million per year supplying methadone to junkies but yet the government can't fund life savings drugs for people with a multitude of illness and diseases

steddyeddy

Gatling wrote: »

It's currently costing €20 million per year supplying methadone to junkies but yet the government can't fund life savings drugs for people with a multitude of illness and diseases

So you're not in favor of funding disease related to self medication? Say goodbye to funding for diabetes type 2, certain types of lung and liver cancer related to substance abuse.

ED E

Gatling wrote: »

It's currently costing €20 million per year supplying methadone to junkies but yet the government can't fund life savings drugs for people with a multitude of illness and diseases

You cant use absolute figures, it has to be relative to the treated cohort.

Kurtosis

steddyeddy wrote: »

I agree with the funding but the pharmacodynamics should offer more of a health benefit than multi-drug therapy. Don't forget that this is multi-drug therapy.

I'm not sure I understand your comment. I know Orkambi is a combination of two drugs in a single tablet. The trial evidence shows it is effective at improving lung function and reducing exacerbations, the problem is it is not cost-effective, the fact it is a multi-drug treatment does not change that.

steddyeddy

penguin88 wrote: »

I'm not sure I understand your comment. I know Orkambi is a combination of two drugs in a single tablet. The trial evidence shows it is effective at improving lung function and reducing exacerbations, the problem is it is not cost-effective, the fact it is a multi-drug treatment does not change that.

I'm agreeing with you that it's not cost effective in the slightest. From your earlier posts I gathered you were implying that the same effect as Orkambi can be achieved with multiple treatments. I'm not sure how true that is.

Kurtosis

steddyeddy wrote: »

I'm agreeing with you that it's not cost effective in the slightest. From your earlier posts I gathered you were implying that the same effect as Orkambi can be achieved with multiple treatments. I'm not sure how true that is.

Oh sorry, what I was saying was that most treatments in other disease areas (i.e. not CF) are more cost-effective, and spending the same amount of money on these would provide greater health benefits overall to society. If Orkambi is funded at the current cost, these extra health benefits would be sacrificed, which is the reality of opportunity of opportunity costs when making funding decisions in healthcare.

steddyeddy

penguin88 wrote: »

Oh sorry, what I was saying was that most treatments in other disease areas (i.e. not CF) are more cost-effective, and spending the same amount of money on these would provide greater health benefits overall to society. If Orkambi is funded at the current cost, these extra health benefits would be sacrificed, which is the reality of opportunity of opportunity costs when making funding decisions in healthcare.

Sorry I misunderstood your post! Yes it's a pity but that's the way it works. Personally I think some lobby groups need to understand this.

Boaty

At a cost of nearly 400million over 5 years , this is a massive deal for Vertex, increasing their turnover by 5%.
80 million out of a 14 billion euro health budget doesn't sound like much when you think about it.

pickarooney

The medical research should all be funded by an international NPO and the drugs should be free or at least affordable.

zl1whqvjs75cdy

pickarooney wrote: »

The medical research should all be funded by an international NPO and the drugs should be free or at least affordable.

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.