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.
Big medical systems will post their clinical trails quarterly or so. If you have a big medical system near you you can sign up for emails. I imagine universities would have a mailing list too, if they regularly did clinical trials.
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.
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).
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.
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 isn't completely true at least in the UK. It is simply that the manufacturer is no longer responsible legally. The GMC allow prescibing of unlicensed meds. However the change needs to be made to the pathway. So many issues in the NHS has been due to pathway problems.
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.
fascinating! I'm sure there's quite a bit that can be learned through appropriate research - pathways to solve problems that haven't been thought of before
the thing is while something is better than nothing, new drug development is critical
there is absolutely no cure for certain types of long-covid and me-cfs right now
no repurposing any drug is going to cure it, they've tried everything after six years
it will take a decade to have anything even in the pipeline and won't emerge from the USA because all medical and science research investment by the government has been destroyed by Russell Vought and Heritage Foundation
JAK-STAT inhibitors will be a big treatment, not a cure, but they cost thousands per month in the USA because generics aren't allowed
The prices of drugs in the USA are especially high. This is interesting
because the USA claims to pursue a maximum capitalistic society - but if
this were the case, you'd have competition in a free market. But you don't
have that. You have a cartel (or rather more than one).
A pure capitalistic society works on assumptions that are not real. People
are often cheaters. This would have to be taken into account. But when you
have an orange Al Capone in charge, it is pillage day. Even before the orange
King you had heavily overcharged prices in the health care system. You need
to realise that you have a mafia in charge that does not want to change this
system. Why kill the cow that you can milk for free?
> This is interesting because the USA claims to pursue a maximum capitalistic society
No it doesn't. This is silly.
Drug prices in the US are high for non-generic drugs because patent law gives the patent holder an artificial government-granted monopoly, which is blatantly not "pure" or "maximum capitalistic".
Generic drugs - where the free market does apply - in the US are as cheap or cheaper than in other countries. See [0]:
U.S. prices for brand-name originator drugs were 422 percent of prices in
comparison countries, while U.S. unbranded generics, which we found account for 90
percent of U.S. prescription volume, were on average cheaper at 67 percent of
prices in comparison countries, where on average only 41 percent of prescription
volume is for unbranded generics.
Look at the development of price and quality of something that is outside the regulated medical system, like eg Lasik, and everything within that system. Its like night and day.
If we had proper competition and price discovery, things would be much better.
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.
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.
How do people needing (and willing to risk) treatment hear about repurposing studies?
Big medical systems will post their clinical trails quarterly or so. If you have a big medical system near you you can sign up for emails. I imagine universities would have a mailing list too, if they regularly did clinical trials.
You can also search here: https://clinicaltrials.gov/
I was a clinical trial participant once and it was a positive experience for me.
I think in the same way they participate in other clinical trials.
Communities (on-line or off-line) of people who share your disorder, and maybe health care professionals who see many patients with your disorder.
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.
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).
The business is already built given the drug in question is already being manufactured and available on the market.
Can confirm. LDN user 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.
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 isn't completely true at least in the UK. It is simply that the manufacturer is no longer responsible legally. The GMC allow prescibing of unlicensed meds. However the change needs to be made to the pathway. So many issues in the NHS has been due to pathway problems.
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.
> 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
fascinating! I'm sure there's quite a bit that can be learned through appropriate research - pathways to solve problems that haven't been thought of before
the thing is while something is better than nothing, new drug development is critical
there is absolutely no cure for certain types of long-covid and me-cfs right now
no repurposing any drug is going to cure it, they've tried everything after six years
it will take a decade to have anything even in the pipeline and won't emerge from the USA because all medical and science research investment by the government has been destroyed by Russell Vought and Heritage Foundation
JAK-STAT inhibitors will be a big treatment, not a cure, but they cost thousands per month in the USA because generics aren't allowed
The prices of drugs in the USA are especially high. This is interesting because the USA claims to pursue a maximum capitalistic society - but if this were the case, you'd have competition in a free market. But you don't have that. You have a cartel (or rather more than one).
A pure capitalistic society works on assumptions that are not real. People are often cheaters. This would have to be taken into account. But when you have an orange Al Capone in charge, it is pillage day. Even before the orange King you had heavily overcharged prices in the health care system. You need to realise that you have a mafia in charge that does not want to change this system. Why kill the cow that you can milk for free?
> This is interesting because the USA claims to pursue a maximum capitalistic society
No it doesn't. This is silly.
Drug prices in the US are high for non-generic drugs because patent law gives the patent holder an artificial government-granted monopoly, which is blatantly not "pure" or "maximum capitalistic".
Generic drugs - where the free market does apply - in the US are as cheap or cheaper than in other countries. See [0]:
[0] https://pmc.ncbi.nlm.nih.gov/articles/PMC11147645/The US is not a capitalist society, it is a liberal society. Capitalism is a consequence of that but it isn't the reason.
Look at the development of price and quality of something that is outside the regulated medical system, like eg Lasik, and everything within that system. Its like night and day.
If we had proper competition and price discovery, things would be much better.
On the other hand, dental work, especially anything above basic filings, are prohibitively expensive. I'd say Lasik is the exception, not the rule.