7 ms·

Orphan Drugs

64 points by thread_id 8y ago

DennisAleynikov 8y ago
this is an awesome look into the economics of curing diseases that cannot benefit from economies of scale. Its unfortunate the rare diseases end up having such expensive cures but on the other hand people can develop cures for disease that otherwise would be uncurable. interesting double edged sword
riffraff 8y ago
This is very interesting but it lacks a key piece of information: how is this situation handled in other countries?
- monocasa 8y ago
  Quite a few countries aggressively nationalize orphan drug production facilities.
  - refurb 8y ago
    Such as? Europe has their own version of orphan drug incentives, as does Canada. I’ve never heard of a country nationalizing orphan drug production facilities.
    - monocasa 8y ago
      India for one.
- ekianjo 8y ago
  In Japan orphan drugs get priority reviews and patients don't pay anything (i.e. government/social security pays 100% for the rare disease). Companies who produce such drugs have almost carte blanche to make any price they want, since "nobody" is directly paying in the end.
  - lovemenot 8y ago
    >> Companies who produce such drugs have almost carte blanche to make any price they want That's not really right since it's not open competition. MHLW sets a price for orphan drugs, which all companies must follow. In practice, the Ministry tends to use global prices as baseline in its negotiations with orphan drug suppliers, through their trade group representatives.
plmu 8y ago
Another sign that health care and commerce do not mix. In some countries, there are plans to "regulate", in this case to mandate distributors to keep 4 months of supply. I doubt if that will work. Instead, like the UK NHS, where the healthcare itself is non-commercial and fair to everyone, the R&D + production of drugs could also be organized much better: Do the R&D via universities, that should receive grants to develop into directions that are socially wanted, instead of developing more of the same (like high-bloodpressure drugs) just because of commercial interests. States can collaborate and organize e.g. very expensive high engergy physics R&D (such as CERN) or fusion energy. Why would the same system not work for drugs R&D? The production itself, completely patent free, can be granted according to demand to commercial companies. These just produce what health care systems order. The states/systems pay a fair price allowing the companies to have a decent profit margin to remain viable, but the current profit margins in the pharma sector are much too high and immoral, since there are people dying because they cannot affort the necessary drugs. I think this sector has proven more than once, that it is not capable of taking the very high responsbility, but is driven by greed that leads to immoral actions.
- refurb 8y ago
  Considering most drugs fail to get to market, I’d offer that the profit margins aren’t all that good if you start from scratch. Saying profit margins are too high is easy if you just count the winners.
  - Retric 8y ago
    Marketing is a larger cost than Reseach for major drug companies.
    - chimeracoder 8y ago
      > Marketing is a larger cost than Reseach for major drug companies. People on HN love to point this out, as if it somehow implies that companies are wasting money. Marketing is how companies secure their revenues from the drugs that do make it to market. Without marketing, they'd have even less money to spend on R&D. (And yes, marketing takes place in countries besides the US. Marketing is more than just consumer advertising).
ekianjo 8y ago
> one rare disease drug came out with a price tag of $150,000 a year — a lot of money. If there are just 100 patients out there that's not a lot of money for the company in the end. Cancer drugs are way more common and yet remain expensive, too.
- hrktb 8y ago
  For orphaned drugs, companies have a monopoly status on it and clients litteraly can’t forgo buying, and will do whatever research is needed to find the product. There is no nudging or bribing the doctor or whatever is needed to take an edge. Considering it’s long argued that one of the main cost of a drug is marketing [0], for any drug where it’s significantly more than 100 people we shoudn’t get these crazy prices. [0] http://naturalsociety.com/research-development-new-drugs-not-paying-off-6321/ http://naturalsociety.com/research-development-new-drugs-not...
  - ekianjo 8y ago
    You are missing a major point. Lots of people who have rare disease are not even aware they have them, or not even diagnosed because their local doctor has never seen it before and mistook it for something else. So yes, you still need "marketing" to spread awareness for rare diseases, because they are, well, rare.
    - techbio 8y ago
      Yet the CDC is effective at spreading awareness of common diseases. Why do this via drug marketing?
- DoreenMichele 8y ago
  Cystic fibrosis is a "rare disease". A quick search gives me this blurb: Very rare (Fewer than 20,000 cases per year in US) My recollection is that I have heard that there are 30k CF patients in the US. I've also heard long time members of the CF community snort at that figure and say "They've been saying the same thing for decades while the list of alleles known to cause it grows longer." It's genetic and incurable. So you need treatment for the rest of your life once they ID you. I've seen articles that suggest that standard conventional treatment is anywhere from $100k to $250k annually. I've also seen articles that suggest it's called lot less than that get discussed on CF lists by people going "Where did they get their numbers? That sounds too low. My expenses are x, y, z." https://www.wsj.com/articles/vertex-gets-fda-ok-for-cystic-fibrosis-drug-1435861189 https://www.wsj.com/articles/vertex-gets-fda-ok-for-cystic-f... Vertex $259,000 Cystic Fibrosis Drug Gets FDA Approval Orkambi treats most common form of the lung disease Vertex already sells Kalydeco, which treats a different genetic type of cystic fibrosis and has an annual price of $311,000 per patient in the U.S. I know of a teenager on Okambi in another country. So the market for Okambi is not limited to the thousands of people in the US who have CF. Granted, you have to get separate approval in different countries and that's a genuine burden.
based2 8y ago
https://www.eurordis.org/en https://www.eurordis.org/en
DoreenMichele 8y ago
Excellent article. I had no idea that Quincy was involved in this legislation being passed. I would like to see more research into diet and lifestyle based approaches to dealing with "orphan" diseases. To my mind, that's a possible path out of this trap. You have to eat anyway. Food chemistry can have substantial impact on the body, especially if you have some anomaly where the body processes certain molecules differently. I manage my condition with diet and lifestyle. I get attacked and dismissed as a deluded nutter suffering Munchausen for trying to talk about that online. But I don't know what the hell else to do. The mods on HN have been really great about the situation. I've been thrown off of other forums or told that people violating forum rules to attack me are fine and I'm the problem and I just need to shut up. I try to not push it too much because I don't want to end up locked up in an insane asylum and dead because of it, like Semmelweis.* Though, in my case, dying would most likely be due to being denied the control over my diet and physical environment that makes it possible for me to be drug free and relatively healthy rather than being beaten by the guards and ending up with sepsis. You probably can't make the kind of money from researching and promoting dietary and lifestyle approaches to such conditions that you can from orphan drugs. I don't personally know how to monetize it at all. When I had affiliate links on my health blog for a particular brand of salt I got attacked for that and told I could not be trusted, I was merely trying to get rich off the suffering of sick people. Having a form of the condition myself hasn't served to give me any credibility. In the minds of most people, the real definition of CF is what my CF specialist once told me: "People like you don't get well." Since I'm getting well, clearly, I'm a liar who doesn't really have CF. I don't know how to find a path forward. Maybe the answer is someday I say "Fuck this noise" and stop trying to share what I know and just focus on something that pays my damn bills. But, for now, that idea still rankles. * https://en.m.wikipedia.org/wiki/Ignaz_Semmelweis https://en.m.wikipedia.org/wiki/Ignaz_Semmelweis
- jnovek 8y ago
  To me, it's not beyond reason that cycstic fibrosis would respond to diet and lifestyle, and is an understudied phenomenon. I see potential rational caveats, and my guess is that these caveats create some jerky knees among other people who are also suffering (and I'm sure, as a sufferer, you know how emotional the experience dismissal is). A few hypotheses: - CF responds to diet and lifestyle in some poeple but not others. - Time spent with the disease and disease progression is a factor on how well it can be managed without medication. - The above things you mentioned (e.g. you actually have another mimicking condition). If it's something like that, I think you are still operating on good faith and you shouldn't be blown off by other CF sufferers for it... but people are irrational. Don't confuse this with denial, by the way! It's simply a rational possibility. - The exact *features* of diet and exercise that affect CF aren't well studied/understood and your regiment randomly hits on them. This reminds me of people who feel better eating gluten free that don't have celiac -- eating gluten free is a diet that happens to incidentally eliminate a lot of other food allergens other than gluten. That is, you have a false solution to the problem with real solution hiding in it. In any case, I've been on the "haters" side re: diet and lifestyle preaching so I can empathize there, too. In particular with chronic migraine, there is a granola crowd who has barely suffered an ailment in their life who suggest that "perhaps a better diet and yoga will help". I've reached a point where I want to slap them sometimes; one of my migraine prodrome symtoms is irraional anger and anxiety, perhaps I will someday :-P Personally, I wish you the best of luck. I suspect it's statistically likely that you'll depend on pharmacutical intervention someday, hopefully, you can delay that is long as possible. I am by no means anti-pharmacutical, but I know from experience that maintaining a long-term drug regiment has it's own problems. Fuck chronic illness, raise your hand if you're tired of telling people that your illness won't go away, it can only be "managed". Feel as well as you can.
matt_the_bass 8y ago
This discussion is Great! Clearly this is a difficult topic that lots of people feel is important. I find it interesting that there is so little consensus in the comments. To me that is a really interesting question and discussion. I wish I had a good answer.

64 points by thread_id 8y ago

this is an awesome look into the economics of curing diseases that cannot benefit from economies of scale. Its unfortunate the rare diseases end up having such expensive cures but on the other hand people can develop cures for disease that otherwise would be uncurable. interesting double edged sword

riffraff 8y ago

This is very interesting but it lacks a key piece of information: how is this situation handled in other countries?

monocasa 8y ago
Quite a few countries aggressively nationalize orphan drug production facilities.
- refurb 8y ago
  Such as? Europe has their own version of orphan drug incentives, as does Canada. I’ve never heard of a country nationalizing orphan drug production facilities.
  - monocasa 8y ago
    India for one.
ekianjo 8y ago
In Japan orphan drugs get priority reviews and patients don't pay anything (i.e. government/social security pays 100% for the rare disease). Companies who produce such drugs have almost carte blanche to make any price they want, since "nobody" is directly paying in the end.
- lovemenot 8y ago
  >> Companies who produce such drugs have almost carte blanche to make any price they want That's not really right since it's not open competition. MHLW sets a price for orphan drugs, which all companies must follow. In practice, the Ministry tends to use global prices as baseline in its negotiations with orphan drug suppliers, through their trade group representatives.

plmu 8y ago

Another sign that health care and commerce do not mix. In some countries, there are plans to "regulate", in this case to mandate distributors to keep 4 months of supply. I doubt if that will work. Instead, like the UK NHS, where the healthcare itself is non-commercial and fair to everyone, the R&D + production of drugs could also be organized much better: Do the R&D via universities, that should receive grants to develop into directions that are socially wanted, instead of developing more of the same (like high-bloodpressure drugs) just because of commercial interests. States can collaborate and organize e.g. very expensive high engergy physics R&D (such as CERN) or fusion energy. Why would the same system not work for drugs R&D? The production itself, completely patent free, can be granted according to demand to commercial companies. These just produce what health care systems order. The states/systems pay a fair price allowing the companies to have a decent profit margin to remain viable, but the current profit margins in the pharma sector are much too high and immoral, since there are people dying because they cannot affort the necessary drugs. I think this sector has proven more than once, that it is not capable of taking the very high responsbility, but is driven by greed that leads to immoral actions.

refurb 8y ago
Considering most drugs fail to get to market, I’d offer that the profit margins aren’t all that good if you start from scratch. Saying profit margins are too high is easy if you just count the winners.
- Retric 8y ago
  Marketing is a larger cost than Reseach for major drug companies.
  - chimeracoder 8y ago
    > Marketing is a larger cost than Reseach for major drug companies. People on HN love to point this out, as if it somehow implies that companies are wasting money. Marketing is how companies secure their revenues from the drugs that do make it to market. Without marketing, they'd have even less money to spend on R&D. (And yes, marketing takes place in countries besides the US. Marketing is more than just consumer advertising).

ekianjo 8y ago

> one rare disease drug came out with a price tag of $150,000 a year — a lot of money. If there are just 100 patients out there that's not a lot of money for the company in the end. Cancer drugs are way more common and yet remain expensive, too.

hrktb 8y ago
For orphaned drugs, companies have a monopoly status on it and clients litteraly can’t forgo buying, and will do whatever research is needed to find the product. There is no nudging or bribing the doctor or whatever is needed to take an edge. Considering it’s long argued that one of the main cost of a drug is marketing [0], for any drug where it’s significantly more than 100 people we shoudn’t get these crazy prices. [0] http://naturalsociety.com/research-development-new-drugs-not-paying-off-6321/ http://naturalsociety.com/research-development-new-drugs-not...
- ekianjo 8y ago
  You are missing a major point. Lots of people who have rare disease are not even aware they have them, or not even diagnosed because their local doctor has never seen it before and mistook it for something else. So yes, you still need "marketing" to spread awareness for rare diseases, because they are, well, rare.
  - techbio 8y ago
    Yet the CDC is effective at spreading awareness of common diseases. Why do this via drug marketing?
DoreenMichele 8y ago
Cystic fibrosis is a "rare disease". A quick search gives me this blurb: Very rare (Fewer than 20,000 cases per year in US) My recollection is that I have heard that there are 30k CF patients in the US. I've also heard long time members of the CF community snort at that figure and say "They've been saying the same thing for decades while the list of alleles known to cause it grows longer." It's genetic and incurable. So you need treatment for the rest of your life once they ID you. I've seen articles that suggest that standard conventional treatment is anywhere from $100k to $250k annually. I've also seen articles that suggest it's called lot less than that get discussed on CF lists by people going "Where did they get their numbers? That sounds too low. My expenses are x, y, z." https://www.wsj.com/articles/vertex-gets-fda-ok-for-cystic-fibrosis-drug-1435861189 https://www.wsj.com/articles/vertex-gets-fda-ok-for-cystic-f... Vertex $259,000 Cystic Fibrosis Drug Gets FDA Approval Orkambi treats most common form of the lung disease Vertex already sells Kalydeco, which treats a different genetic type of cystic fibrosis and has an annual price of $311,000 per patient in the U.S. I know of a teenager on Okambi in another country. So the market for Okambi is not limited to the thousands of people in the US who have CF. Granted, you have to get separate approval in different countries and that's a genuine burden.

based2 8y ago

https://www.eurordis.org/en https://www.eurordis.org/en

DoreenMichele 8y ago

Excellent article. I had no idea that Quincy was involved in this legislation being passed. I would like to see more research into diet and lifestyle based approaches to dealing with "orphan" diseases. To my mind, that's a possible path out of this trap. You have to eat anyway. Food chemistry can have substantial impact on the body, especially if you have some anomaly where the body processes certain molecules differently. I manage my condition with diet and lifestyle. I get attacked and dismissed as a deluded nutter suffering Munchausen for trying to talk about that online. But I don't know what the hell else to do. The mods on HN have been really great about the situation. I've been thrown off of other forums or told that people violating forum rules to attack me are fine and I'm the problem and I just need to shut up. I try to not push it too much because I don't want to end up locked up in an insane asylum and dead because of it, like Semmelweis.* Though, in my case, dying would most likely be due to being denied the control over my diet and physical environment that makes it possible for me to be drug free and relatively healthy rather than being beaten by the guards and ending up with sepsis. You probably can't make the kind of money from researching and promoting dietary and lifestyle approaches to such conditions that you can from orphan drugs. I don't personally know how to monetize it at all. When I had affiliate links on my health blog for a particular brand of salt I got attacked for that and told I could not be trusted, I was merely trying to get rich off the suffering of sick people. Having a form of the condition myself hasn't served to give me any credibility. In the minds of most people, the real definition of CF is what my CF specialist once told me: "People like you don't get well." Since I'm getting well, clearly, I'm a liar who doesn't really have CF. I don't know how to find a path forward. Maybe the answer is someday I say "Fuck this noise" and stop trying to share what I know and just focus on something that pays my damn bills. But, for now, that idea still rankles. * https://en.m.wikipedia.org/wiki/Ignaz_Semmelweis https://en.m.wikipedia.org/wiki/Ignaz_Semmelweis

jnovek 8y ago
To me, it's not beyond reason that cycstic fibrosis would respond to diet and lifestyle, and is an understudied phenomenon. I see potential rational caveats, and my guess is that these caveats create some jerky knees among other people who are also suffering (and I'm sure, as a sufferer, you know how emotional the experience dismissal is). A few hypotheses: - CF responds to diet and lifestyle in some poeple but not others. - Time spent with the disease and disease progression is a factor on how well it can be managed without medication. - The above things you mentioned (e.g. you actually have another mimicking condition). If it's something like that, I think you are still operating on good faith and you shouldn't be blown off by other CF sufferers for it... but people are irrational. Don't confuse this with denial, by the way! It's simply a rational possibility. - The exact *features* of diet and exercise that affect CF aren't well studied/understood and your regiment randomly hits on them. This reminds me of people who feel better eating gluten free that don't have celiac -- eating gluten free is a diet that happens to incidentally eliminate a lot of other food allergens other than gluten. That is, you have a false solution to the problem with real solution hiding in it. In any case, I've been on the "haters" side re: diet and lifestyle preaching so I can empathize there, too. In particular with chronic migraine, there is a granola crowd who has barely suffered an ailment in their life who suggest that "perhaps a better diet and yoga will help". I've reached a point where I want to slap them sometimes; one of my migraine prodrome symtoms is irraional anger and anxiety, perhaps I will someday :-P Personally, I wish you the best of luck. I suspect it's statistically likely that you'll depend on pharmacutical intervention someday, hopefully, you can delay that is long as possible. I am by no means anti-pharmacutical, but I know from experience that maintaining a long-term drug regiment has it's own problems. Fuck chronic illness, raise your hand if you're tired of telling people that your illness won't go away, it can only be "managed". Feel as well as you can.

matt_the_bass 8y ago

This discussion is Great! Clearly this is a difficult topic that lots of people feel is important. I find it interesting that there is so little consensus in the comments. To me that is a really interesting question and discussion. I wish I had a good answer.