Comment by estearum
9 months ago
The catch is that there are thousands of promising therapies in animal models/pre-human testing. A very very tiny fraction of them will ever make it to market for a variety of both good and not-good reasons.
9 months ago
The catch is that there are thousands of promising therapies in animal models/pre-human testing. A very very tiny fraction of them will ever make it to market for a variety of both good and not-good reasons.
What's the difference between a good and not-good reason to not go to market?
Prevalence of the disease - if it only impacts 1/100 million, going to be hard to ever find sufficient patient population to test and recoup your investment.
Existing quality of treatments - if there are already efficacious drugs on the market - how sure are you that this new therapy will be best in class? Only being as good as the status quo is not an ideal competitive position. Conversely, if there is an unmet need because a disease is so lethal/debilitating, regulatory agencies can give latitude in approvals.
Likelihood patient compliance - if it is the most effective drug in the world, but requires intravenous infusion six times a day - nobody is going to adhere to that. GLP drugs are effective, but there is a needle-phobia that is preventing patients getting on board with the idea. Which is why there is an arms race for the first company to develop an oral version.
Toxicity - all chemicals are poisonous. Yet some have a lower therapeutic window than others. If you drug does what it should, but if you take 2x as much and it gives you a heart arrhythmia that is going to be a tough approval for anything but the most deadly conditions.
Also the ethically in blind studies, can't just let some random patients die just to prove that your drug works.
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I guess I'm not being clear. Are these examples of "good" or "bad" reasons to not go to market? I assume "good"? If so, what would be an example of "bad"? Or vice versa if your meaning was opposite.
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Rybelsus exists already, so there's that
Good reasons:
* Most drug candidates just don't work
* Even among the drug candidates that do, figuring how to safely deliver them to their target is very hard (looks similar to "just doesn't work")
Bad reasons:
* It's too expensive to prove that a drug works
* It's too difficult to differentiate the patients for whom a drug works and the patients for whom it does not
* It is very hard to predict recruitment and to actually recruit patients for clinical trials
* There aren't enough people with the disorder who are also rich enough to afford treatment to justify development
> It's too expensive to prove that a drug works
I'm not sure that is necessary a bad reason. You need to factor in a lot of concerns to determine what "too expensive" means.
But if you are going to spend billions of dollars to develop a drug that only treats about 2 people a year it is likely too expensive even if it is 100% effective. That money would be better spent on treatments that have wider applicability.
Of course this is not simple to measure. Costs aren't known upfront and the research may end up proving invaluable to more widely applicable treatments.
So it is a judgment call and not necessarily a bad reason.
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There is also the ole', drug works 20% of the time and kills the patient 80% of the time.
A lot of potential treatments are too easily available and can't be patented. If a big pharma company can't make massive profit from it, they won't bother bringing it to market. Consider that a not-good reason.
Other treatments may eventually prove to have too many serious negative side effects. That's a good reason to abandon them.
> A lot of potential treatments are too easily available and can't be patented.
This isn’t really an obstacle, at least not as much as it’s made out to be.
There are numerous examples of drugs being brought to market at high prices despite having been generic compounds. Even old drugs can be brought back at $1000/month or more at different doses or delivery mechanisms.
One example: Doxepin is an old antidepressant that is extremely cheap. It was recently re-certified for sleep at lower doses and reintroduced at low doses at a much higher price, despite being “off patent”.
This happens all the time. The drug companies aren’t actually abandoning usable treatments due to patent issues as much as journalists have claimed. If they couldn’t, for some reason, find a way to charge for it they could still use it as a basis for finding an improved relayed compound with more targeted effects, better pharmacokinetics, etc.
They’re not just dropping promising treatments anywhere if there’s a market for them.
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Why would a China or India care if it were a viable treatment? Unless a country wants to use their population as lab rats, it takes money and scientists to actually confirm a treatment is safe and effective.
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Wonder if some form of FOSS approach would work as an alternative development model for pharma?
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Kind of like open source software.
In the early 2010s I had a couple friends working at a biotech startup with a moonshot cancer cure. They had amazing results in animal testing, and had raised just enough funding to do a first human trial on a terminal patient.
That patient was hit and killed by a car two days after finishing their treatment.
Along with the CEO's stubborn refusal to give up any more equity in the company, they went under, and the world will never know if that treatment worked.
From what I saw, there are very few experimental therapies that claim 100% efficacy, no side effect, no patent on complex engineering process to produce the drug.
I agree with GP that it is very notable.
I mean if it works on humans, which is not a stretch, colorectal cancer is done. It's huge.
Yes there are few that claim 100% efficacy and no side effect at this stage, but there are far, far, far fewer who make it to human availability.
I wouldn't describe it working in humans as "a stretch" per se. I'm not identifying a specific reason it shouldn't work in humans. I'm just saying that's true of thousands and thousands of really great looking treatments (per year!) that, nonetheless, end up not working in humans, or not being convincing enough to even warrant putting them in humans once.
If it is the case that there are such treatments (easy to produce, 100% efficacy, no side effect) that cure fairly common deadly disease such as colorectal cancer in mice, and that never make it to human trial, there is something seriously broken about medical research...
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