Orphan Drugs: An Introduction
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- เผยแพร่เมื่อ 7 มิ.ย. 2024
- In the United States, the median price for an orphan drug is about $100,000 per year, twenty times the price of the median non-orphan drug. Given the staggering cost, you'd think we'd have a solid handle on whether that money is well-spent. But the truth is that we don't.
Friend of the show Nick Bagley, a professor of law at the University of Michigan wrote a series for our blog on orphan drugs, and it was so good, we're bringing it to you here. For the next four weeks, orphan drugs are the topic of Healthcare Triage.
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ORPHAN DRUG - a pharmaceutical drug that remains commercially undeveloped because of limited potential for profitability.
ftfy, Great Video btw.
Rio Weber Thankyou for this comment! It is explained eventually within the vid, but I'd already come looking for answers
I know right, I did the same thing!
Rio Weber THANK YOU. LIKE GEE, can't expect everyone is familiar. Especially people outside US!
Kalydeco has saved my brother's life, but if he loses insurance through the ACA, he can't get the medicine. For us, the astronomical price and the pre-existing condition stipulation in the ACA aren't just policy abstracts; they're the red line between my brother's life and death.
gymnasticsrat94 The pre-existing conditions protection kept me from having health insurance for years before the ACA. I have a rare immunodeficiency called CVID, the medication for which would be so far outside my ability to pay for it without health insurance. I would need a six figure salary for the medication alone without insurance. I know full well that no insurance company would touch me if pre-existing protections are removed by the current administration. And I would be dead inside of a year without the treatment.
Sara Anne Miller A dear friend of mine also has CVID. I hope you get all the care you need to manage such a complex illness and don't become a pawn in these insane political games.
gymnasticsrat94 Thank you so much and I wish the same for your friend. ❤
Did you ever consider starting a gofundme page or something? Seriously, the ACA hurts millions of people. We can help you without hurting millions of others in the process.
The ACA has forced me to pay a fine that I don't want to pay for not having health insurance, so what about me? Don't I get to be considered in the law?
Did you ever stop to consider how the ACA hurts others? Selfish.
Diana Peña are you fucking kidding me? I would need approximately $130,000 per year to keep me alive and an additional $50,000 a year for health coverage and living expenses. Please find a go fund me that is going to give me around $3 million so that I can see my 60s.
If you believe that a go fund me would work, then I'm sure that you have $3 million to give me. Are you going to contribute to a go fund me for a person like myself? I hope you realize that there are approximately 6500 people in the United States with my disease. If you feel so inclined, please find $780 million to donate in order to keep people with CVID alive for one year.
Oh, you don't like those numbers? Now you see why healthcare is important.
I have a family friend who uses an orphan drug that cost $12,000 a month. Luckily there are organizations that help pay for the drug but they still have to pay $1000 a month just for the drug.
You brought up a point I can never seem to get people to understand. People on orphan drugs, like myself, don't really have a choice. For me it's pay this cost or don't hold down a job or go to school or even do basic everyday tasks. For others it's more extreme, life or death. It's easy to talk about being "responsible for your healthcare " when you don't know someone with no options. I did not chose the body I was born in and it is not an option to not have my medication; that's not a life.
Thanks Healthcare Triage Team. 👍👍👍
This additional episode on the Orphan drug policy was very informative.
It's nice to view both the cost and benefits of this policy especially within the context of its original intent.
Thanks for making videos on those of us with rare diseases!
An interesting topic that I've somehow never heard about before, I'm glad to be learning about this!
Thank you very much
Thanks super clear!
"I'm going to lower drug costs" - President Trump.
Yeah right.
I mean, if he successfully reduced the regulations surrounding creating news drugs, made it easier to get generic alternatives approved, and reduced how long patent protections keep people from producing new and generic drugs (all of which he could theoretically do) it would help quite a lot for the prices of all drugs. There are quite a few regulations, many of them unreasonable, that can be changed to help drop drug prices. Whether he will actually do that or not, however, is basically a toss up at this point. To busy bombing sovereign nations.
Get govt out of the way, and prices go down. It's pretty fucking simple. If dems had their way with HIV meds, millions of more people would be dead now.
Also, right now, approval process at the FDA are fast by historical and international standards
Chas, did you even watch the video? The profit motive is the reason they exist. If you reduce patents, they'll stop researching things that aren't profitable.
taibhsear71 they became unprofitable through government regulation and again profitable through more regulation. instead of layering protective regulation we should roll back more, making it easier and cheaper to develop these drugs. there's an argument to be made that protective patents are necessary, but there are countless other regulations that can be rolled back to further spur innovation.
100 000 USD/year!? That's more than my families yearly income. With both parents working, AND 2 young adults working entry jobs in the service industry. WTF?
Another series! I really took a lot a way from the opioids expose.
Rather than a fixed period of time for exclusivity, what if it were tied to revenue? Say, change the law so that instead of seven years, it is until the company has made enough to cover the cost of research plus a percentage as profits. This gives the manufacturers two options: Either price gouge the hell out of people but lose their exclusivity, or set reasonable prices to extend that period and, assuming they set the prices at a competitive point, make generic drugs financially unnecessary.
How can I get official numbers about the cost of orphan drugs for patients in the US and if possible around the world?
Revenue of a billion dollars/year sounds like a lot of money. But that revenue must not only cover the pharmaceutical company's research, development, marketing, and manufacturing costs for the successful drug - it must also cover all the projects that did not yield an efficacious drug (call them "failed orphan drugs"). And all that revenue comes years after those costs are incurred because of the delay imposed by the FDA approval process.
I know its not related to this subject but I would be interest on your thoughts on the NOS report about diary intake, or more specifically calcium intake and its relationship to osteoporosis. Add to this are studies like this which says "Meta-analysis showed a significant 15 % reduced risk of total fracture... sand a 30 % reduced risk of hip fractures" link.springer.com/article/10.1007/s00198-015-3386-5
I was wondering if you could explain when there seems to be a disagreement between such sources and the ones you cited on your diary video?
I'm feeling a very strong slant to this.
With the limited information I now know on this topic, I'd say we are in this weird period between when the drugs were developed and when their exclusivity period on them ends. Without having specific data on it, I would theorize that there were more orphan drugs developed and launched thanks to this Act by Congress, than there would have been without. I would also theorize that after each drug reaches the end of their 7-year exclusivity window, we could see "generic" competition bring those prices down drastically. How much they would actually come down is yet to be seen. The end result could then be that we got more drugs for people with diseases that would not have had them, and though they may cost a fortune at first, as time went on, they become more "feasible" financially.
TL;DR Making progress, still sucks, but less than before, should get better "soon"
I want information about who put wot really into my prescription drugs
I have a question. Is it true that pearl cream is good for your skin? It's something they have traditionally used in China, which makes me wonder about its effectiveness.
Avalon: The Dragon's Den As clinically effective as grounded deer horn for erectile performance.
Yeah, that's what I was afraid of
it's interesting to look at one act and argue for it's pros and cons. As useful as this might be tho, i'd say it's rather narrow in its outlook--there are MANY more important market forces (regulatory and unregulatory) accounting for big pharm not wanting to dip its stick in the orphan business.
Unfortunately. . .so long as the FDA and big government is around, shit's only going to get worse = /
A system based on monopoly creating monopolies with no check in place to make sure there is the prevention of excessive monopoly profits is always going to lead to this problem.
The math on this is super interesting. If we make the assumption that all orphan drugs that get developed will end up developed in the same order either way, then as long as the orphan drug act produced ANY increase in drug production, it will be worth it. Let's say it only produces 1 extra drug a year. Without the act, let's assume that 10 drugs a year are produced and protected under normal patent laws (for what, 3 years? I can't find it anywhere). After 7 years, you have 40 drugs under patent and 30 open to competition. With the orphan drug act, let's assume 11 are produced per year. After 7 years, you have 77 drugs under patent and 0 open to competition. However, increase the scale to 50 years. After 50 years, no orphan act results in 30 drugs under patent and 470 open to competition. The orphan drug act results in 77 drugs under patent and 473 drugs open to competition, so it ends up on top eventually with just a 10% difference. And from the chart you presented in this video, it seems like the difference is significantly higher.
Who take care orphan children religion charity or the government charity?
There's definitely something wrong when Crestor can qualify for orphan drug status.
They should be some system to get medical patents into the public domain quickly. Charities are one way but I'm doubtful they'll be able to raise enough money to development all the drugs we want, especially for diseases with bad pr. A government agency of some sort, funded by a tax or taxes that go straight to it so it has more independence and some sort of the governing body that has representatives from patients/their families, medical professionals and researchers be give A LOT of money and told to cure/make vaccines for some diseases already. Finally the government can offer 100 mil+ prizes to anybody that can make something that cures/prevents a disease after which it becomes public domain.
I've been saying that patent laws need reform to strike a better balance between innovation and consumer cost.
Big Pharma's lobby has bought off most of Congress, so passing any reforms would be difficult because it will cut into their profits and abuse of current laws. Maybe move away from simple set time based patents to price control structures that limit markup but allow a company to recoup R&D costs and a set profit over as much time as is needed.
Get rid of patent monopolies and switch to a licensing system to encourage multiple companies to produce a drug and increase competition. Companies who wanted to produce a drug would have to pay an equal share of the R&D costs, so the first licensee would give 50%, plus maybe a little more to acknowledge the risk of drug failures, a third company would pay a third split between the first two companies, and so on. Plus they would pay a licensing to the developer until they reached a profit cap where the drug would become generic. The whole point is to reduce a companies risk, while guaranteeing enough of a profit to encourage innovation and encouraging competition.
Since drugs can be replaced by newer drugs, maybe allow a royalty markup that is payed to companies whose drugs are replaced to cover their R&D costs and a profit up to their cap. Reduces a companies risk to encourage innovation.
Maybe the way the government offers bonuses for cost saving measures, they could offer a bonus for new drugs based on a percentage of how much their drug saves the government in treatment costs.
I'm sure there are giant holes in my ideas but the current unstructured, set time monopoly is resulting in a lack of innovation and affordable treatments for consumers.
I hear that increasing competition actually causes prices to rise for medical services, contrary to what one would expect. This is partially described by the Sticky Ceiling Phenomenon. I wonder how that interacts with the other forces in this domain.
That's not correct at all. More competition lowers prices.
Hence my saying that it was "contrary to expectation". I can scrounge up some citations if you want. It is a really weird phenomenon and I do not entirely understand why it is happening, but I guess that it is. The basic pressure is the fact that healthcare is not like other commodities - as an existential necessity, people will pay literally anything for it; so, as more competition enters the arena, there are more chances to charge more, and people will still accept these options; then the rest of the competition raises their own prices in order to match this new maximum until the market can no longer support price increases - and there, everybody stays. Anyway, that is my understanding of it. But it really is happening across the board, as far as I can tell.
Sure, but I just don't see this theory conforming to reality. Where we see the ability to compete (which usually isn't the case in hospitals in the US), we see lower prices. Since most healthcare isn't emergency in nature, people do have the ability to shop around, which puts downward pressure on prices when competition is allowed.
It probably has conditions and a scope of applicability, you are right. And my understanding of the mechanism which induces this phenomenon may be wrong - it is quite probable, in fact. But, if the researchers are to be believed, then the fact that this (id est: the common event of competition driving up prices in certain contexts) does in fact happen; it is not a theory but an empirical fact. It is true; it happens. Our explanations for which it begs might simply be guesswork (or maybe not), but it definitely is a real phenomenon.
The median for a non-orphan drug is $5,000.00 a year!? That's absurd.
Keep in mind, median is offset by the fact that some medications, like cancer meds, cost more, but many medications cost as little as 12 bucks a month. That's why mean should be looked at too, especially when you consider volume of the lower cost medications being purchased, like antidepressants (maybe 1k a year on the most extreme end), Sleeping medications, maybe $20 a month, Insulin, and many others.
So how much does it cost to develop and bring an orphan drug to the market, considering all the failed candidates?
Regular drugs cost about 800 million total for 10-15 years of development. Orphan drugs should not really be much more than maybe 20% higher for R&D. The problem with these is the return on investment for the firms. More than half of the candidates end up failing for any king of drug anyway
Buehehe a single, successful drug, if it passes FDA review. But most companies spend years developing drugs that may not go anywhere, get rejected, or have unintended side effects after the purchase, leading to an FDA recall and millions in lawsuits.
The price of drugs is not just to cover the R&D of that specific drug, it's to also cover all the listed expenses. Plus loans taken out and expensive facilities.
Good point Aleksandr. Healthcare Triage should take that into consideration when saying drug prices are too high. Sure they are expensive if you're sick, but they're also super expensive to bring to market
Maybe we can mind control the pharmaceutical executives so we can lower the prices.... heh heh heh 😆😆😆
If you half the orphan drug period, the prices will just double. Not solution
I thought this was about drugs for orphans.
save the orphans!
They are on them for the rest of their life, yes, but they are only protected from generics for 7 years, so this only effects them for a maximum of 7 years....
Not necessarily. The orphan drug I have been on for years is also a controlled substance, and only available through one manufacturer and one pharmacy. It used to be available over the counter. I have watched the price rise over the years, and it is really surprising. There are assistance programs that pay for it if insurance won't, but I have no idea why it went from around $2k-$3k in 2007 to over $7k now for a month's supply.
That might be, but as the video makes clear, the orphan drug law of 1983 only lasts 7 years, so any effect after that has nothing to do with the law its self.
It's not unheard of for a company to modify the drug slightly and claim a new patent. If it's in a new delivery method it could qualify for a new patent. The 7 yrs is now 27 yrs. It has to be worthwhile for another company to make a generic and bring an anti-trust suit against original company.
And, my medication is a controlled substance, so the rules for it might be governed more by its status as that rather than being an orphan drug.
I believe there is a loophole though. Companies can get the orphan drug status for the same drug multiple times. All they have to do is prove that the drug they already developed years ago can be used to treat another rare disease and they get orphan drug status all over again. Basically, they can drag out a series of back-to-back 7 year monopolies on the same drug just by finding new applications for it. And it's great that they're doing the trials to find out if it works, and that definitely does take money that companies need to be reasonably certain of recouping later. But I don't think they should be rewarded with the same incentives for finding new applications of an existing drug as they do for developing a totally new drug.
Orphan drugs are a real shame but it’s all based on an economic reality.
personally I'd rather pay exorbitant prices for 7 years than be dead
scy1192 you could bower a lot of money buy some Gold and move to Mexico
define orphan drug
it seems that orphan drugs are an example of market failure. it's just not profitable for a free market capitalist business to spend the money to develop drugs for niche markets. the government should buy up a smaller pharma company and task them with developing orphan drugs and leave the for-profit businesses to make high blood pressure pills et al.
Limit orphan drug prices to $50,000 per year. Almost all orphan drugs will still be produced, but it will cut unnecessarily high prices brought on by inelastic demand.
Is this really a bad thing? You showed the graph about how many have been approved, then you complained about the prices for the rest of the video. There is NO innovation without profit.
why do i have to give my drugs to those damn greedy orphans?!
I've been saying that patent laws need reform to strike a better balance between innovation and consumer cost.
Big Pharma's lobby has bought off most of Congress, so passing any reforms would be difficult because it will cut into their profits and abuse of current laws. Maybe move away from simple set time based patents to price control structures that limit markup but allow a company to recoup R&D costs and a set profit over as much time as is needed.
Get rid of patent monopolies and switch to a licensing system to encourage multiple companies to produce a drug and increase competition. Companies who wanted to produce a drug would have to pay an equal share of the R&D costs, so the first licensee would give 50%, plus maybe a little more to acknowledge the risk of drug failures, a third company would pay a third split between the first two companies, and so on. Plus they would pay a licensing to the developer until they reached a profit cap where the drug would become generic. The whole point is to reduce a companies risk, while guaranteeing enough of a profit to encourage innovation and encouraging competition.
Since drugs can be replaced by newer drugs, maybe allow a royalty markup that is payed to companies whose drugs are replaced to cover their R&D costs and a profit up to their cap. Reduces a companies risk to encourage innovation.
Maybe the way the government offers bonuses for cost saving measures, they could offer a bonus for new drugs based on a percentage of how much their drug saves the government in treatment costs.
I'm sure there are giant holes in my ideas but the current unstructured, set time monopoly is resulting in a lack of innovation and affordable treatments for consumers.