bit of a topic in canada as of late. covering drug costs for rare genetic disorders and diseases.
to give a quick example, Fabry's disease. a build up of fat in the kidneys and heart due to a missing enzyme. as of today, costs to treat this are $250,000cnd a year.
on a sad note this treatment has been deemed to expensive for the benifits it provides. some provinces are covering it, some are not. the feds are currently working on a research protocol to deliver these drugs through clinical trials. in turn saving pharmaceutical companies money by not having to go through traditional($$) methods of testing and approval. hence making the drugs cheaper.
anyways i am curious to how this works in the states. if you have health insurance are all/any drugs covered? or does the government provide money for these types of treatments?
expensive drugs for rare diseases
expensive drugs for rare diseases
[size=75]i never meant to give you mushrooms girl[/size]
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[xeno]Julios
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Depends on the scarcity of the starting materials, length and yields of reaction sequence, synthetic complexity and a lot of other variables.[xeno]Julios wrote:
well does it cost a lot of money to actually produce these drugs independent of the actual R&D?
But I'm afraid even if they can knock the tablets out for 1 cent a pop you can't just discount the cost of the 10 years of R&D it takes to get it to market. As my boss once said, even if it only cost a few cents to make each tablet the first one off the production line will cost you a billion dollars. :icon26:
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[xeno]Julios
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right no i completely understand that. I'm just imagining a curious situation:SIK wrote: But I'm afraid even if they can knock the tablets out for 1 cent a pop you can't just discount the cost of the 10 years of R&D it takes to get it to market. As my boss once said, even if it only cost a few cents to make each tablet the first one off the production line will cost you a billion dollars. :icon26:
company spends a few billion dollars developing a drug for an extremely rare disease.
Only a fraction of those who actually have the disease will be able to afford it.
First: what's the business logic in developing such a drug, when hardly anyone will buy it?
Second: Once you made the blunder (assuming it is a blunder - maybe i'm missing something here), why play dog in the manger)?
Wonder how much it would cost to buy the rights to a drug like that and distribute the drugs at a more affordable rate. Would be nice to see a gov't do that.
btw nice to see you around again - you still at AstraZeneca?
Hi Jules, still around just don't post as much as I used to (not that I was ever a prolific contributor!) :icon25:
As for the economics of drug development they are (rightly or wrongly) taken into account right from the first decision as to what disease targets to go after. When you're talking about a billion dollars and 10 years of development I can assure you there are no 'blunders', no company ever gets a drug to market accidentally! Be cause of the astronomical cost of drug development we rely on the few dozen 'blockbuster' drugs to subsidise the development and marketing of drugs which can be used in less viable and smaller markets. I remember seeing a report that something like 70% of all marketed drugs effectively lose money, they never sell enough to recover their development costs.
The pharma industry is going through the ringer at the minute (has been doing for the past few years) with a lot of backlash (media and political), and I'm the first to admit not all is unwarranted. As for the cost of developing diseases where development costs outweigh the commercial payout, these still go ahead but the costs tend to be shared more across the industrial/governmental divide (well that's the hope!) or across the company globally (the income from a single blockbuster can amount to tens of billions and a good percentage of that is ploughed back into more research). AZ has set up an R&D site in India dedicated solely to discovering new treatments for 'third world' diseases (currently working on TB). Most pharma companies also look at outlicensing (and in-licensing!) compounds to and from each other which lie outside there own portfolio or area of expertise. So if one finds a drug candidate in an area where they have no experience or desire to expand they will sell the rights for it to another company to develop further.
The cost and availability of medicines is a global issue and needs a global solution. No-one wants to pay high prices for drugs, but some countries have to pay more so that others can get the medicines they need for less.
It's a huge business with so many variables, issues and moral conflicts it does your head in if you think about it too much. Thankfully the weighty problems of the global market aren't in my remit, I'm happy to stay in the lab and do my research. :icon25:
As for the economics of drug development they are (rightly or wrongly) taken into account right from the first decision as to what disease targets to go after. When you're talking about a billion dollars and 10 years of development I can assure you there are no 'blunders', no company ever gets a drug to market accidentally! Be cause of the astronomical cost of drug development we rely on the few dozen 'blockbuster' drugs to subsidise the development and marketing of drugs which can be used in less viable and smaller markets. I remember seeing a report that something like 70% of all marketed drugs effectively lose money, they never sell enough to recover their development costs.
The pharma industry is going through the ringer at the minute (has been doing for the past few years) with a lot of backlash (media and political), and I'm the first to admit not all is unwarranted. As for the cost of developing diseases where development costs outweigh the commercial payout, these still go ahead but the costs tend to be shared more across the industrial/governmental divide (well that's the hope!) or across the company globally (the income from a single blockbuster can amount to tens of billions and a good percentage of that is ploughed back into more research). AZ has set up an R&D site in India dedicated solely to discovering new treatments for 'third world' diseases (currently working on TB). Most pharma companies also look at outlicensing (and in-licensing!) compounds to and from each other which lie outside there own portfolio or area of expertise. So if one finds a drug candidate in an area where they have no experience or desire to expand they will sell the rights for it to another company to develop further.
The cost and availability of medicines is a global issue and needs a global solution. No-one wants to pay high prices for drugs, but some countries have to pay more so that others can get the medicines they need for less.
It's a huge business with so many variables, issues and moral conflicts it does your head in if you think about it too much. Thankfully the weighty problems of the global market aren't in my remit, I'm happy to stay in the lab and do my research. :icon25:
Jules, remember that even large pharm companies get research money from the gov't. When a company is interested in staying competetive, they need to try churning out as many treatments as they can. Typically, the big money will go to widespread diseases, so profit loss isn't always a problem, but even large companies can stand to gain from research into rare diseases when it's not all their own money at stake.
For everything else, rely on academia, where the bulk of expensive bootstrap research is not for profit or cures. Pharm companies will often pick up this sort of research after much of the big money has alredy been spent by someone else.
For everything else, rely on academia, where the bulk of expensive bootstrap research is not for profit or cures. Pharm companies will often pick up this sort of research after much of the big money has alredy been spent by someone else.
I'm not aware of anything like that in the States, no.
It's really hard to answer your question without specific drug names, due to the complexity of insurance policies.
When people find out they have a disease here, they may have to do weeks worth of research to find out if they can find a way to be sure their condition will be covered by insurance. Even then sometimes you need an attorney after all is said and done.
I believe R&D for drugs like you mention is done by private parties like universities or independent labs - but sometimes with government grants or subsidies, and almost always with tax incentives.
At that point, what Jules mentioned comes into play... If there aren't enough people to sell the drugs to, the R&D centers can't make a profit. Therefore, if the center is purely a business, they will not undertake the work. If the center is a university, or other center geared toward public wellbeing, then the government will see that they cannot make a profit, and they will not get government grants (unless prominent decision makers are sympathetic to the institution for whatever reason, but that's not for this discussion obviously). Often they will do the R&D anyway if there is a great need for it.
Either way, the drugs wind up being extremely expensive, and it's hard to know whether your insurance will cover it, cover part of it, or refuse coverage. It becomes a tangle of clauses like pre-existing conditions etc.
If you are not covered, there are sometimes other independent organizations that will help you, like the Sara Cannon Foundation.
It's really hard to answer your question without specific drug names, due to the complexity of insurance policies.
When people find out they have a disease here, they may have to do weeks worth of research to find out if they can find a way to be sure their condition will be covered by insurance. Even then sometimes you need an attorney after all is said and done.
I believe R&D for drugs like you mention is done by private parties like universities or independent labs - but sometimes with government grants or subsidies, and almost always with tax incentives.
At that point, what Jules mentioned comes into play... If there aren't enough people to sell the drugs to, the R&D centers can't make a profit. Therefore, if the center is purely a business, they will not undertake the work. If the center is a university, or other center geared toward public wellbeing, then the government will see that they cannot make a profit, and they will not get government grants (unless prominent decision makers are sympathetic to the institution for whatever reason, but that's not for this discussion obviously). Often they will do the R&D anyway if there is a great need for it.
Either way, the drugs wind up being extremely expensive, and it's hard to know whether your insurance will cover it, cover part of it, or refuse coverage. It becomes a tangle of clauses like pre-existing conditions etc.
If you are not covered, there are sometimes other independent organizations that will help you, like the Sara Cannon Foundation.
There are experimental drugs for rare childhood cancers that are showing tremendous promise, but due to the rarity of the disease RandD and been completely stopped and they won't be pushed past the current phase of clinical trials they are in. Just watched a special a few months ago about a situation like this - the doc participating in the clinical trials was brokenhearted because the drug was working so effectively for this kid, yet she was going to have to tell the family that they weren't going to have any more of it soon.
It's a real tough issue.
It's a real tough issue.
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[xeno]Julios
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thx for the insights guys.
I was just thinking of a situation where a company knew in advance that the disease is so rare that very few people who had the disease would actually pay for it.
I still don't see the (business) logic of developing such a drug. Maybe one of you has already answered this question, but I don't see it.
Or is this situation not one that ever occurs.
I was just thinking of a situation where a company knew in advance that the disease is so rare that very few people who had the disease would actually pay for it.
I still don't see the (business) logic of developing such a drug. Maybe one of you has already answered this question, but I don't see it.
Or is this situation not one that ever occurs.
Not exactly for rare diseases, but the prices on some of the meds get pretty high.shadd_ wrote:good stuff there SIK. still interested in how thngs work in the usa. do the federal or state governments run any kind of compassionate programs to pay for these unaffordable drugs?
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Sorry Jules, been at work inventing new drugs...[xeno]Julios wrote:I was just thinking of a situation where a company knew in advance that the disease is so rare that very few people who had the disease would actually pay for it.
Like I said earlier, if there was no market the drug would never get made. Nothing gets on to the market by accident, when you're dealing with 10 years, thousands of people and billions of dollars there generally are no 'blunders'. The project would probably have never been given the go ahead to even start. To put it in perspective it's like BMW 'accidentally' creating and marketing the M3. BMW manager's don't suddenly wake up one morning and think "Oh look, we've spent years and hundreds of millions of dollars developing a new car! How the fuck did that happen?!?" (OK, bad analogy, but you get the idea)
R&D projects in pharma are followed, monitored and checked constantly throughout their lifetime, from initiation through research, through development and on to the market (and beyond) and at every critical step there is a 'tollgate' where the project is evaluated to see if it meets certain criteria before it is allowed to continue. Those criteria can be anything from biological tractability, novelty, chemical viability, market potential, competitor situation, timescale, safety, toxicology issues, you name it. I'd estimate 95-97% of projects die before making it to the market for one reason or another (generally tox/safety or PK/PD issues). Roughly one molecule out of every 20,000 we make ends up on the market. The attrition rates are enormous. I can assure you if something ends up on the market it was a hell of a long fight to get it to that stage!
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MidnightQ4
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No. Basically how it works is you sign up for medical insurance, through your work typically, and then you get a substantial discount on meds that way. But only the ones that are covered by the plan, which typically the very expensive or rare ones are not covered.shadd_ wrote:good stuff there SIK. still interested in how thngs work in the usa. do the federal or state governments run any kind of compassionate programs to pay for these unaffordable drugs?