Cure ID App Lets Clinicians Report Novel Uses of Existing Drugs
fda.gov
fda.gov
I wonder if this is being used for other drugs. My curiosity stems from personal experience: I had Migraine headaches from age 17 to about 35. I was put on ancient seizure medication that's common prescribed for Bipolar[0] because this doctor had three other patients that it nearly eliminated Migraine from. It was fall, a time when I'd get about one a week. After five days of taking it, I had my first Migraine ... if you could call it that -- I could only identify that it was a Migraine by the aura; the pain was about 10% what I'm used to).
Searching through the web, I found a forum that was filled with Migraine sufferers. Sure enough, there were a handful of people who swore by it. There were also a handful of people who it didn't work for. Looking at the more official sources, there was no indication that this drug could have any effect on Migraine; they listed all of the other off-label uses[1], but Migraine was not among them.
This medication had been in the news several times (and on the front page, here[2]) over the last few years and a year ago (or so), I looked it up on the "official sources", again. It now indicated that it was prescribed for Migraine.
It made me wonder ... how are things like that figured out/communicated down-stream? Is it entirely informally amongst doctors? I went to four different specialists before I found one who suggested this drug -- and he did so in a "half-hearted manner" not truly expecting it would work. It'd be nice if this was centrally tracked/managed as it might surface both "new uses for old drugs" and "new problems with old drugs."
[0] Which I do not have.
[1] It's rarely, if ever, prescribed for what it was originally approved for.
[2] It's Depakote, I'm not being cagey for nefarious purposes, I just didn't want this to be a drug advertisement.
So in cases like valproate for migraine, those drugs usually stay off-label long term. That makes them unattractive for doctors to prescribe, since they assume more liability if something goes wrong, and insurance companies are less likely to cover the drugs.
The way information about off-label uses gets communicated is basically the same way as elsewhere in science: through scientific papers, conferences, textbooks, and the like. A doctor who discovers that their migraine patient does well on valproate might publish this as a case report. If there are many such reports, someone might do a randomized trial. Once there are many randomized trials, meta-analyses can be performed, and eventually, the information will end up in textbooks and medical school curricula.
It takes a certain amount of agency for a doctor to stay up to date on the latest research; while medical boards tend to have certification requirements for CME (continuing medical education), these are usually quite lax, and a truly lazy doctor can probably just live their years without really learning anything new.
At the same time, reading all new research that comes out is basically impossible, and even just trying to do this would risk always following the latest fads that later evidence might show to, in fact, be harmful. Every doctor needs to find their own middle ground here, and you as a patient must find a doctor that suits your own preferences.
I don't know. Maybe it's not a viable business model, but there are certainly weirder things that make money.
This is precisely why I think a gov't run research effort is the right way to handle this. You don't want to grant a new patent on the existing drug just because of new use of it. That would prevent the generics being used for the original purpose. So if the bigPharma company that created it for original purposes does not want to spend money researching alternate uses, then allow the gov't direct the research in the name of keeping its citizens healthy.
Unfortunately, that doesn't really stand up as a great solution with using the current administration as an example of what happens when you get anti-science mindsets in charge of things. It also helps to have a gov't that is empathetic for its citizens which does not always happen.
But care must be taken to avoid the tulip subsidies problem [1]: the barriers to drug approval are regulatory, not natural. If a drug regulatory agency sets high requirements for approval and another governmental agency must do expensive work to satisfy these requirements, there is a risk that costs go out of control without any real benefit.
Instead, my favoured solution would be to significantly relax regulatory requirements in cases like this where a drug has already been proven to be safe both in the original approval trials and in years of real-world use, such that when trials and meta-analyses exist that suffice to convince the medical establishment of a drug's safety and efficacy for a new indication, these would also be enough for the drug to gain approval for that indication.
[1] https://slatestarcodex.com/2015/06/06/against-tulip-subsidie...
Real world, sometimes a doctor will think up a novel use of the effect of a drug. And sometimes they'll throw stuff at the wall and see what sticks. When you don't have a good answer this can be sane medical practice. But there should be a requirement of reporting it to something like this database.
Now wave that cadaceus around my head another time, Morpheus
Also highly recommend the book.
Turns out, a drug developed by Gilead Sciences (GS-441524) which is the active metabolite of remdesivir, is remarkably effective at treating the disease. It's a nucleoside analog, so it essentially is a slightly modified adenosine molecule which disrupts the virus RNA replication process.
Within 12 hours of the first dose our cat went from dying to making a full turnaround and complete recovery following treatment. For us it was truly a miracle drug, and it only gained awareness recently due to the COVID-19 pandemic [1]. It still isn't technically "approved" for this use case, but is prescribed off-label by vets as of 2024 once the FDA relaxed its position [2]. Folks needed to seek it out on the black market (and still do in some areas).
[1] https://www.theatlantic.com/science/archive/2020/05/remdesiv...
[2] https://www.fda.gov/animal-veterinary/cvm-updates/fda-announ...
During COVID, I actually wrote a small sample app that pulls info from this DB: https://www.covidcureid.com/
And gave a talk on it: https://learn.microsoft.com/en-us/shows/azure-serverless-con...
Otherwise, my code to extract the data is here: https://github.com/CharlieDigital/covidcureid/blob/main/serv...
Doctors already have case studies which they (at least should) publish anytime they get someone who for whatever reason doesn't respond to standard treatment, or has something unknown. However it is hard for the next doctor to find any that might be relevant.
There's not much wrong with the system so long as it's used as a place to get ideas for clinical studies or absolute last-ditch attempts at saving lives. On occasion (like the Milwaukee Protocol for rabies), those last-ditch attempts do work.
It's when people pretend there's no real investigation to be done that we get problems.
But by the time it became apparent, the correction didn't spread nearly as fast nor wide as the original information.
The problem is, most people lack the scientific literacy to understand how science works, and many newspapers have long since gutted their expert journalists.