> 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

Have you looked into UniQure / Clearpoint Neuro ?

Exciting stuff, if it gets FDA approved.

The Price of Remission - https://news.ycombinator.com/item?id=43925396 - May 2025 (93 comments)

There tens of millions of people being treated off-label.

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.

The article points out that it is typically after patent expiry that the universities and hospitals start looking at repurposing.

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?

Specialists keep up with the latest research in their domains and talk with other specialists in their field. They usually know about these things before their patients discover them spontaneously.

You can read a lot of published papers on PubMed by searching for a condition or drug. You should be aware that there are a lot of papers published that say disease A can be treated with drug or supplement B that fail to replicate if anyone else tries it. It takes some practice to recognize when a treatment represents a real trend as opposed to a single quack doctor looking for a status boosting publication with some questionable claims.

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.

> 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.

> This is interesting because the USA claims to pursue a maximum capitalistic society

I don’t know why you think this. The US is not a maximally capitalist society. The reason drug prices are so high is due to regulations restriction who can manufacture them due to government-granted temporary monopolies through patent law.

If the US was maximally capitalist it would be a free for all with no patent protection.

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.

The US is not a capitalist society, it is a liberal society. Capitalism is a consequence of that but it isn't the reason.