Hospitals and universities repurposing drugs at lower cost
kcl.ac.uk326 points by giuliomagnifico a day ago
326 points by giuliomagnifico a day ago
I have a little insight here from working with ophthalmologists.
When the article mentions "using a cancer drug to treat a leading cause of blindness" it's talking about using Bevacizumab (brand name Avastin) to treat macular degeneration.
Avastin and Lucentis are molecularly the same drug. The difference is that Avastin isn't packaged to be injected into eyeballs, and Lucentis is. Also, Avastin costs about $50/dose, and Lucentis costs around $1,500/dose.
There are compounding pharmacies that will repackage Avastin into a format that can be injected into eyeballs.
However, many ophthalmologists are hesitatnt to use Avastin because that process introduces the potential for contamination. Not that it happens frequently, but every ophthalmologist I know lives in fear of introducing an infection into their patient's eyeballs. And what level of risk is worth saving $1,450 per dose on an injection? 1/100? 1/1000?
I know at least one ophthalmologist who would use Avastin, but would culture a sample from every batch she got from the compounding pharmacy, and had at least one batch that cultured bacteria.
Assumedly, if she hadn't, every patient she injected with that batch would have been at risk of contracting endophthalmitis, and endophthalmitis is no joke. Not infrequently, it can mean losing vision.
I know several of that ophthalmologist's colleagues who had been using compounded Avastin to try and save their patients money, stopped after that incident.
I don't really have a conclusion beyond "drug pricing is insane and the issue is complex for providers."
Compounding pharmacies can get injectibles wrong in a deadly way. Many families are still waiting for justice after the MECC pharmacy caused the deaths of 64 people:
https://en.wikipedia.org/wiki/New_England_Compounding_Center...
This note in that WP link is wild:
"On July 7, 2021, Barry Cadden's original 9-year sentence was increased to 14.5 years. An appeals court court decision required the trial judge to consider patients, and not only hospitals, as victims of the crime."
The law is always more complex than a layman like me thinks, questions of standing, etc. But I would have simply assumed that the patients hurt and killed would naturally be counted among the victims and not just the organisations left out-of-pocket.
> And what level of risk is worth saving $1,450 per dose on an injection? 1/100? 1/1000?
I've have plenty of friends that would struggle to afford that. I have friends that buy black market drugs because they can't get/afford a prescription. I don't know if I would make the same choice but I can I can understand why some people do.
The reason people get endophthalmitis is rarely due to a contaminated batch, but it certainly happens. But frankly it’s unsustainable to have all patients on branded drug, it would be too costly for patients and payers. Not to mention it is slightly unethical in the US due to drug rebates incentivizing branded drug use.
Endophthalmitis is bad, but we can treat it if caught promptly. Patient education, informed consent, good hygiene practices, and easy access to their ophthalmologist can make a tremendous difference.
Source: I do these injections for a living.
> But frankly it’s unsustainable to have all patients on branded drug, it would be too costly for patients and payers.
This is incorrect, the Canadian healthcare system negotiates drug costing at a per province rather than per patient manner and has managed to negotiate down drugs pretty significantly especially in Ontario. A national drug plan doesn't currently exist but it's likely that drug and dental coverage is going to be a goal in the near term.
In the US there is also a complex drug reimbursement program run by most manufacturers to offer rebates to patients in the form of trial cards or direct refunds that does help widen accessibility but those programs are generally limited to patients on private insurance due to the nature of incentives and, well, greed.
I think it's very fair to say "Within this current system brand name drugs are a ridiculous ask for most patients due to the availability of generic alternatives" but the system itself is deeply broken. Generics are sometimes whitelabeled versions of the same product but often what are considered inactive ingredients may be modified significantly from brand name versions leading to issues, especially when it comes to neurological drugs, of drastically different effects to patients. The brand name vs. generic problem is a lot more complex than most people give it credit for and while patients should always prefer generics when they're similarly functional there are very notable scenarios where they are not equivalent for treatment. This comment shouldn't be read in direct opposition to the full comment above as this drug is outside my wheelhouse but rather as a comment on generics in general.
Source: I work in a company that analyzes drug pricing and, more generally, comparative insurance reimbursement for a living.
The higher manufacturer price of the eyeball-safe formulation is clearly justified, then, i.m.o. Also, is it the doctor's responsibility to assume risk to save the patient money?
Assuming the compounding process is inherently more at risk of contamination. It also gets complicated when the extreme prices are involved: what risk of infection balances out with the risk that the patient will not be treated due to cost or will suffer whatever negative consequences due to bearing the cost? One in two? Probably not. One in a billion? Probably.
Considering that the doctor was able to test the batches and verify contamination, it seems like this is an important step that was skipped by the pharmacy.
How does that justify a 30 fold increase?
If an individual Dr can get a batch checked while saving the patient money, it obviously isn't justified.
They have similar targeting but are not the same molecule.
My quick searching is showing that bevacizumab is the full antibody, but lucentis (ranibizumab) is just the fab. So, very similar, but indeed not the same thing. I would generally expect them to have the same effect, but biology is full of surprises.
How does a lowly pharmacy transform a drug that is not for eye injection into one that is?
They are not transforming the drug just the form. For example, elderly patients may have a hard time swallowing pills, for some medicines (not all) a compounding pharmacy can turn the pill into a lollipop. They can also turn things into like a cream or lotion.
/use to write code for a small independent pharmacy that had a couple compounding labs
Just went down a chatgpt rabbit hole because I was also curious - seems it’s literally physical repackaging, no modification of the drug itself- as another commenter pointed out it’s not quite the same molecule but they have the same effect? Someone correct me if I’m wrong
> How does a lowly pharmacy transform a drug that is not for eye injection into one that is?
New research probably discovered new applications for their product. Investors agree to diversify. Company developed a system to inject it. The system was approved by government agency at charge of this, and they give the green light to put it in the market.
This is totally normal. See Ozempic history.
The price of a treatment reflects also the collateral risks and probability to be sued for the physician and the company. The problem is not that it cost 1000 dollars, the real problem is that US government should be subsiding at least a part of this cost. Tax money is collected exactly for cases like this. The problem is that they are instead burning 14 millions to paint a pool in "American Idiot Green" dye and nobody says, this bill must be wrong. What they used to paint this? titanium?
The US government already was subsidizing the cost: https://www.nei.nih.gov/research-and-training/research-news/...
It subsidizing the cost of developing many drugs. The question is whether their pricing reflects that.
I have been a supporter of Cures Within Reach, a nonprofit that focuses on repurposing drugs, especially for rare diseases. https://www.cureswithinreach.org
They have funded some important repurposed-drug studies for Huntingtons Disease, which runs in my family. For a disease like this, it's never going to make sense for major pharmaceutical companies to invest the effort to develop entirely new drugs, but by repurposing existing drugs, it gives people living with rare diseases a chance to ease symptoms.
> For a disease like [Huntingtons], it's never going to make sense for major pharmaceutical companies to invest the effort to develop entirely new drugs
This is ... not correct.
Roche, Regeneron, and Novartis all have novel HD drugs under development in tandem with smaller labs (Ionis, Alnylam, and PTC respectively), and then smaller labs like uniQure and Wave Life Sciences do too. Novartis have already dropped $1bn on the partnership with a committed $2b more. In addition, there are a bunch of incentive schemes for diseases like HD: both the FDA and EMA have offered orphan-drug designation to therapies for HD, the FDA does expedited programmes and can offer RMAT designation for drugs like AMT-130.
With some luck (which is always in short supply for HD treatments, sadly), people with the disease might be able to get a single-injection treatment in the next 12 months[0].
0: https://en.hdbuzz.net/the-other-shoe-drops-uniqure-shares-pl...
I'm on my cell phone and I couldn't figure out what diseases can be treated on the site. I'd say it's donor focused and not patient focused
Not exactly what you asked for, but this page lists their impact: https://www.cureswithinreach.org/about-us-repurposing-resear...
Have you looked into UniQure / Clearpoint Neuro ?
Exciting stuff, if it gets FDA approved.
I’m currently on Spravato, which is fully emblematic of how broken the incentives in the US healthcare system are.
Spravato is esketamine - a modified version of ketamine. Ketamine is made up of mirror image molecules and esketamine is the right-handed molecule. They did this because ketamine is off-patent so they needed to modify it in order to patent it, however there is evidence that esketamine is a less effective treatment than ketamine.
It’s very cheap for me but my insurance company pays about $17k a month for this treatment. Ketamine would be a more effective treatment that would be super cheap for them, but they don’t do it because it’s not FDA-approved. So they’re paying a fortune for a less effective treatment.
It would be in the insurance companies’ interests to band together to fund the research so they can save huge amounts of money in the long term but they do not do this.
> It would be in the insurance companies’ interests to band together to fund the research so they can save huge amounts of money in the long term but they do not do this.
Insurance companies do not want cheaper care.
In the US, insurance companies must spend 80% of premiums on care. So if you pay $1k/mo, they have to pay out at least $800/mo in care. (Not to you specifically, but averaged out across all subscribers.)
This is a cap on their potential profits. They always have to pay out 80% of premiums for care, so how do they make more money?
Well, imagine care is twice as expensive. Instead of paying $800, they have to pay $1600. That sounds worse, but, instead of $200/mo, they now $400/mo for themselves!
So, no, paradoxically, it is not in the interest of people paying for the treatment to save money. Quite the opposite.
It's worse than this. Their cap is 80% of the Insurer's profit. Not the Insurer's Parent company. So often the parent company will own the Insurer as a subsidiary and own the Pharmacy, Hospital, Healthcare etc under the parent company.
This way as costs go up, it's really just bypassing the 80%. Because the hospital can charge the insurance subsidary X amount, and then the hospital profits to the parent company.
There needs to be a law in the US that health insurance organizations cannot be owned by anyone who owns a healthcare provider. Nor can the insurance company own healthcare providers.
We've allows the Ma Bell of healthcare to exist.
I tried to explain to people in The Netherlands this exact problem when they were thinking about switching from single-player to a commercial insurance company model.
Insurance companies always have an incentive to make healthcare services expensive. They have even more incentive to make healthcare expensive and do deals on the backside where their suppliers give them a kickback. Even better if said kickback comes via a side door.
So we went for the commercial insurance companies. It took them 4 years before they change the drug choice from decentralised (Pharmacies deal with drugs companies, received small discounts which funded a good quality of care) to centralised (Insurance deal with drug companies, receives kickbacks) and the Pharmacies funding was drastically cut leading to worse quality of care and more drugs being used. Double-whammy because in the previous system the Pharmacy did medication reviews which almost always result in a reduction in drugs (the thing with drugs is that quite a lot of them are given to reduce side effects of other drugs, the original drug gets cancelled but the side effect reducer gets forgotten and just continues ad infinitum).
Health insurance has been super inflationary since then when controlled for quality of care.
> Insurance companies always have an incentive to make healthcare services expensive.
No, it's when their profits are capped by regulation that they have this incentive.
Insurance of other types absolutely seeks to reduce claims payouts.
> So, no, paradoxically, it is not in the interest of people paying for the treatment to save money. Quite the opposite.
I'll assume they're on company insurance. Which is often "self-insured" in that the company actually foots the bill as opposed to the insurance company.
Why don't corporations just drop insurance companies that decide to not allow cheaper medicines?
UNH is so big because its customer Apple has its own pool. Apple deducts $24k/y for your healthcare. Healthy 29 year old male doesn't use anything. UNH denies the claims anyway. It gives that money back to Apple, which doesn't give it to you.
The 80% rule has a lot of loopholes. It doesn't apply to employer funded plans. There's a reason UNH is so big!
Employer funded plans are not all the same. Large entities with a lot of money (like universities, big firms) self-insure. Thus the insurance company in those cases is simply managing all the administrative sides of insurance while the plan owner is the actual insurer of risk.
There’s an article about how a Wall St employee’s expensive care came up in C-suite meetings, as a real world consequence of this
Large swaths of US insurance are underwritten by employers, with the insurance companies acting as contracted administrators. Employers don't have the incentives you list.
Great, just work a well paying megacorp white collar job and you'll get slightly less fucked on healthcare.
What about the other 90% of the country?
You could get angry about anything I say, I don't really care, you do you.
I guess Medicare and Medicaid also don't have the incentives. Nor does Tri-care or the VA. My employer self insures as far as I know, and we are a few hundred people. Maybe 90% is a bit off?
I'm not angry about what you say, I'm angry at the parasites that have built this system, and the cynical bastards that work their ass off to perpetuate it.
>Insurance companies do not want cheaper care.
Why is there a continuous stream of healthcare providers threatening to or becoming out of network for various managed care organizations because they cannot come to an agreement on healthcare prices?
I suspect it's because the insurance companies are not in control. The idea would be to drive up costs when they profit on both sides (example: United and OptumRx) and push down reimbursements elsewhere.
We baked the snake charmer problem into the law?
Good lord.
> snake charmer problem
Perhaps you mean the "cobra effect" [0]? I think that's an apocryphal story about killing snakes, rather than using them in a performance.
[0] https://en.wikipedia.org/wiki/Perverse_incentive#Historicity...
What’s so interesting about this that I just learned after googling about Spravato that ketamine is a treatment for serious depression. Why? That’s fascinating! Is it the dissociative effects?
A significant effect is through opioid receptor activation, as demonstrated by Williams et al. <https://doi.org/10.1038/s41380-019-0503-4> by blocking the receptor with naltrexone, which attenuated the antisuicidality effects.
The Huberman Lab episode Ketamine: Benefits and Risks for Depression, PTSD & Neuroplasticity <https://www.hubermanlab.com/episode/ketamine-benefits-and-ri...> and the referenced journal articles (including the above) have a lot of detail on it.
Blame the FDA on that one.
The FDA's policy for the last couple of decades is that mixtures of mirror images will not get FDA approval unless there is a strong rationale for it.
Racemic mixture of ketamine was approved decades ago. If you want a new indication for ketamine, you will need to get approval for a single mirror image, as the FDA won't approve the old drug.
They did this because there are numerous examples of racemic mixture drugs having increased side effects from the less active mirror image, so the FDA decided no more racemic approvals.
My guess is your insurer would be happy to pay for the old version, but your doctor is heavily incentivized to use the new version as the markup on the drug is much, much higher.
Thalidomide being one of the more notable ones (though apparently neither chirality is truly safe)
It's worth noting that thalidomide is still approved, but only for leprosy and certain types of cancer.
>but your doctor is heavily incentivized to use the new version as the markup on the drug is much, much higher.
I don't think it is normal/usual for doctors to receive any benefits or profits from which drugs they prescribe. Other than golf outings with the drug company reps? Is there something I'm missing?
For drugs administered in office, doctors can markup the cost and insurance pays it. Even Medicare pays 4.3% extra.
For oncologists, it’s most of the revenue for their office since consults pay very little.
The money made on administering $5 worth of ketamine is far less than a $1500 bottle of eskatamine.
The NHS in the UK refuses to cover it in part because of the absurd cost.
> Ketamine is made up of mirror image molecules and esketamine is the right-handed molecule.
Esketamine is their cutesy way of saying the word s-ketamine. The s- comes from the Latin word "sinister" which means this is the left-handed enantiomer, not the right-handed one.
It is stupidly expensive, given how generic ketamine itself is. In our case, sleep apnea treatment proved to be a much better option than that drug, as it was just hiding an underlying condition and the treatments only last for maybe a week or two anyway.
I think there have been some people using ketamine off-label, but I don't know much about that. It does need to be tightly controlled because it can cause breakthrough psychosis in some patients. They try to screen those out, but that's not as effective as one might hope given my experience of seeing that fail. And it that was very nearly a fatal mistake.
Related, one of my all time favorite articles: https://www.propublica.org/article/revlimid-price-cancer-cel...
I tried health-tech twice. Opted out both times as there are too many misaligned incentives and useless rent-takers supported by Congress and other entrenched players.
At this point maybe the best solution is door to door doctors using AI.
This was so hurtful and blood boiling to read. How is stuff like this allowed to happen?
The Price of Remission - https://news.ycombinator.com/item?id=43925396 - May 2025 (93 comments)
This was a good read, thanks for sharing.
I feel like the best way to fight this, if I was the government, would be to stop enforcing laws preventing patients and even doctors or hospitals from importing the overpriced pharmaceuticals from overseas generics manufacturers. Like the article points out, each pill costs no more than $0.25 to make. The goal wouldn't necessarily be to get everyone using overseas suppliers but to put downward pressure on the price gougers in the country.
Happy to help. The laws you refer to exist to juice pharma industry profits. Same reason it has been so hard to change the law to allow Medicare to negotiate drug prices. The price gouging is by design for line go up.
Changing the law will take time (election cycles). If you cannot leave the US for a developed country that won't squeeze to extract from you while you're trying to meet your healthcare needs, importing your drugs that can be imported without US approval is your only path in the short term, or potentially traveling to obtain them and bring them back (roughly two-thirds of the entire U.S. population, ~200M people, live within 100 miles of a U.S. land or coastal border).
(not legal advice, i am just an internet rando)
universal health care can be offered even in 3rd world countries but they're really smart about keeping costs low. they drastically cut the costs of medical service by just offering one or two generic drugs bbought in bulk from china and india rather than all those custom expensive drugs that do the same thing. it does the same thing but at a much lower cost
Such studies are great but there is no regulatory pathway to extend the use of existing drugs for new indications of use without the consent of the manufacturer (or becoming a manufacturer yourself).
This means such studies can give more clarity on which off-label use is beneficial but it can't be an officially allowed usage.
There tens of millions of people being treated off-label.
Can confirm. LDN user here.
I checked this thread for an expansion of that initialism, which I didn't recognize. It likely stands for "low-dose naltrexone." https://ldnresearchtrust.org/what-is-low-dose-naltrexone-ldn
"a prescription medication used primarily to treat alcohol use disorder and opioid use disorder" (in addition to the off-label uses discussed here)
Also a LDN user, that drug saved my life. I bought it from a shady dude online, the initial effect was so strong that I thought they shipped me meth instead - that wore off after a week but the lifting of the brain fog persisted.
It didn’t have that sort of immediate, intense effect for me (though yours isn’t the only account like this that I’ve seen) but I’m still happy with the outcome; it dropped my average 1-to-10 pain score by about two points at three months.
It is a rare side effect, I helped a bunch of people (~50) get LDN, primarily for LongCovid, and only one other had a similar ‘too much energy’ effect and both of us have multiple TNXB SNPs and a very similar rare personality profile. I think the brain is so starved for dopamine at that point that it becomes hyper sensitive. I do miss the extra high energy but I also know that isn’t maintainable and I try to err on the side of caution.
I’ve had ME/CFS my whole life but the third covid vaccine shot sent me to new lows, to the point LDN just wasn’t cutting it anymore. These days I take a combo of modafinil in the morning and amitryptiline at night. And low dose ozempic has been super helpful as well. I was researching GLP1As prior to the current craze because I was worried about hyper sensitivity so I waited for more data before trying, I started at 1/100th the normal starting dose and still got temporary gastroparesis. These days I take a more regular dose of 1mg/wk but it also seems that my body has largely normalized as the hypersensitivities have worn off. Probably a good sign that I’ve successfully addressed actual deficiencies.
The doctors are assuming liability or let the patient sign waivers, which I guess is fine. But the lack of a pathway prevents a lot of commercial possibilities. For instance it is almost impossible to build a business around supporting off-label use cases (for instance selling necessary accessories).
I have some difficult-to-treat medical issues and about 1/3 of the handful of pills I take every day are off-label. I think when you get into less common medical concerns off-label use becomes much, much more frequent.
E.g. gabapentin is an anti-seizure medication that has been found to reduce neuropathic pain as well. It has shot up to the 5th most prescribed medication in the last decade as it has replaced long-term opioid use for new chronic neuropathic pain patients. This is 100% off-label and is prescribed by everyone from NPs all the way up to neurosurgeons for this purpose.
I’ve never been asked to sign a waiver and I can’t imagine that for-profit hospitals would allow their doctors to prescribe off-label medications willy-nilly if they represented big liability. (I don’t know this for sure, this is just what my experience implies.)
> I’ve never been asked to sign a waiver
That presumably has to do with the risk profile of the medications you've taken. If there was a high risk of devastating side effects and it was off label presumably the prescriber wouldn't be willing to take on the liability.
Do you know that for a fact or are you just guessing?
I would wager if those waivers actually did anything they'd boilerplate a generic version of it in the legal mumbo jumbo you have to sign even if you come in to have the doctor look at a stubbed toe.
Yup, I've seen this sort of off label prescription at least 3 times.
A lot of drugs require almost no evidence (especially if they are relatively cheap or common) to be given to the insurance company when they are prescribed. And if you are willing to pay for it, you can always pay out of pocket if insurance is being a pain.
Off label prescribing is extremely common.
This isn’t a new or novel concept. Doctors manage patients with off-label prescriptions all day long.
To get a drug approved you have to go through regulatory approval for a particular use. If the fuck-fuck game is you can just off-label for whatever but only if first approved by the FDA to solve some other problem, why not just bypass the bullshit and get straight to the point of approving the drug without any approved use? Oh yes, because that assures the barriers to entry are arbitrarily higher, and Pfizer and other ilk can insulate themselves from competition. Thanks FDA!
Like a lot of the systems we have today, it grew from good intentions. We need to figure out a way to refresh our systems without throwing them away. The current system does provide new drugs and benefit. The question is is it limiting, and are the tradeoff of those limits worth while.
In other words it takes hard work, with politically risky outcomes and an upside that becomes invisible as far as political careers go. We need to figure out how to refresh our systems in an environment where that type of thing just stumbles along. Maybe a couple of politicians elected who have lost out because of the status quo, but that want to improve it not throw it away. Maybe working with them to motivate them to take it on as a pet project and move things.
If we're just letting doctors yolo* off-label, i have no problem with that, but that functionally relegates FDA protections to testing risk profile at some particular dose regimen, with a bunch of extra bullshit tacked on that is highly inefficient process for off-label use even if partially applicable.
It's pretty clear if you're allowing off-label use you can just gate dose-risk profile at one gate and then just add additional gates for on-label uses if you want to approve one.
Of course the issue is the FDA regulator gets the hammer and damned if anything goes wrong but nothing good happens to them if they approve something. So they have no real incentive to approve anything except ideological satisfaction to whatever extent it lives within their mind, plus whatever revolving door benefits industry is offering them.
Probably best to reward the FDA employees for approving good drugs so that the prisoners' dilemma doesn't always fall back to erring on the side of denials, and being easier on them for taking some risk that approves bad drugs to the point they're willing to take some risk to let potentially good drugs through. The regulator doesn't get to see all the bad they cause by not approving drugs because that's invisible, and that's the unintended consequence of their activities and something they're not really held to account for.
* --------- re: below due to throttling -------
>Doctors aren't yoloing it.
If you take offense, call it whatever the hell you want, I was using it in the context of using a drug for a non-approved off-label use. You're making no legal distinction, it's not an FDA approved use and telling the FDA you have some evidence elsewhere but you won't be going through the process to approve it doesn't mean dick to the approval process anymore than my disapproved use of the world "yolo" to refer to same does. If the best my counterparties have is disapproving of "yolo" I rest my case.
Doctors aren't yoloing it.
What's happening is further research on the medicines are being done which show medicinal benefits for off label use. In many cases, it's researchers seeing a drug has a known side effect and saying "Hmm, I wonder if that side effect can counter this symptom".
What's lacking is the final FDA approval for the drugs being used for these off label treatments. That's because these drugs have to go through all the same tests that are required to get FDA approval in the first place.
IMO, for already approved drugs there should be a faster secondary route to getting these drugs approved for current off label usages. Ideally, it'd be something the FDA itself spearheads in partnership with the likes of the NIH.
Problem with FDA is that they try the "one size fits all" what often leads to blocking something that works onlu for a group of patients.
Problem with doctors preascribing against FDA is that it can lead to problems or abuse.
Om an unrelated note, FDA could have been bribed to ban something same way a doctor cpuld have been bribed to prescribe it.
The business is already built given the drug in question is already being manufactured and available on the market.
> For instance it is almost impossible to build a business around supporting off-label use cases (for instance selling necessary accessories).
There is no need for a business. Off label use is hacking around the existing commercial and regulatory system. The innovation to be had is fixing said commercial and regulatory system, not another business. Make cheap drugs or medical supplies, distribute them to those in need with as little margin and risk to the patient as possible. This is a utility system masquerading as a market. More duct tape by way of new businesses is suboptimal.
The article points out that it is typically after patent expiry that the universities and hospitals start looking at repurposing.
The patents aren't hindering off-label use. The hinder commercial exploitation by others.
Yes, but that isn't relevant to what happens after the type of trials described in the article, which tends to happen when the patents are no longer relevant. As the article points out, while the drugs are patented, the manufacturers themselves are generally very interested in repurposing because it broadens their market. The article is discussing trials that mostly happens once the patents have expired.
This is all untrue so far as I understand.
A research group can file for a new drug application (or abbreviated new drug application) for an existing drug. There is no mandate that an NDA sponsor be a manufacturer or the existing manufacturer.
Only the entity holding an approved NDA can file a supplemental NDA but that’s not the only path.
The real reason no one files for off label use is that there’s high cost and little to no practical benefit to doing so.
On paper you are right. You can file an NDA. But in reality you can't prove that the product on the market (the drug you are adding an NDA to) is sufficiently under your control to enable you to claim that it is safe and effective. Only a manufacturer can control the product. If you want to piggyback on top of an existing product in the market, you have to become a manufacturer or sign a contract with an existing one.
You are absolutely right about the incentives. There are absolutely none except to help humanity which is why only hospitals and universities are doing these off label studies.
That seems wild. Do you have a citation to back that up? And in what country/countries?
I'm in the US, and there are a couple of wrinkles to this: insurance typically won't cover off label use, so patients end up paying the full cost of the medication, and if there is a big enough market, I believe companies can patent the new use even if the patents for the original use have run out.
Doctors here are allowed to prescribe them though.
> insurance typically won't cover off label use
I’m in the US. This is not true.
Insurance will have prior authorization rules for certain drugs that are expensive that require the doctor to submit documentation of the condition, but in most cases the common medication is simply covered if prescribed. The insurance company does not receive documentation of every condition for every prescription to determine if the prescription is on label or off label.
Insurance companies can and do also support some off label treatments that are commonly used under their prior auth requirements.
I don’t know why there are so many comments in this thread making confident assertions that off label prescribing or insurance or so uncommon. This happens all day long at doctors offices and pharmacies.
Yeah I definitely misspoke on that one. I believe they aren't required to cover off label use, so it's up to them whether they do or not.
> I believe companies can patent the new use even if the patents for the original use have run out.
This is not true
> insurance typically won't cover off label use
Generally not true but it can be the case, especially for expensive medications
Yes new uses of existing drugs can be patented:
https://www.drugpatentwatch.com/blog/patenting-new-uses-for-...
This is clearly made by an LLM and thus not a credible resource.
From:
https://pmc.ncbi.nlm.nih.gov/articles/PMC9336118/
"COM claims can be difficult to gain for repurposed compounds, as the patentee must somehow differentiate their patent claims over what is in the public domain and present data that the drug is a credible candidate for the new indication [41, 42]."
citing
Do you like this source better?
https://synapse.patsnap.com/blog/what-are-the-types-of-pharm...?
It’s somebody’s random blog instead of three published, peer reviewed articles. I’m not sure what you expect to hear
It's a matter of law, not science. Ask a patent attorney.
If you like, here is a paper from the Vanderbilt law review: https://scholarship.law.vanderbilt.edu/cgi/viewcontent.cgi?a...